The following is a summary of Lessons for future clinical trials in adults with Becker muscular dystrophy: Disease progression detected by muscle magnetic resonance imaging, clinical and patient-reported outcome measures, published in the March 2024 issue of Neurology by Wel et al.

Researchers started a retrospective study to address the gap in outcome measure evaluation for adult Becker muscular dystrophy (BMD), a disease with variable progression.

They assessed muscle MRI, patient-reported outcomes (PROs), and various clinical measures (Motor Function Measurement (MFM), muscle strength, and timed-function tests) in 21 adults diagnosed with BMD at the beginning of the study and at 9 and 18 months into the follow-up period.

The results showed a significant increase in proton density fat fraction in 10 out of 17 thigh muscles after 9 months and in all thigh and lower leg muscles after 18 months. The 32-item MFM-32 scale showed a decrease of 1.3% (P=0.017), North Star Ambulatory Assessment decreased by 1.3 points (P=0.010), and the patient-reported activity limitations scale deteriorated by 0.3 logits (P=0.018) after 9 months. After 18 months, the 6-minute walk distance decreased by 28.7 meters (P=0.042), 10-meter walking test decreased by 0.1 meters per second (P=0.032), time to climb four stairs test decreased by 0.03 meters per second (P=0.028), and Biodex peak torque measurements of quadriceps decreased by 4.6 Nm (P=0.014) and hamstrings by 5.0 Nm (P=0.019). MFM-32 domain 1 had the highest sensitivity to change, with a standardized response mean of 1.15.

Investigators concluded that whole-thigh PDFF MRI was sensitive to BMD muscle fat changes and that MFM-32 was the most responsive clinical measure.

Source: onlinelibrary.wiley.com/doi/full/10.1111/ene.16282

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Insights for Future Adult BMD Clinical Trials: Detecting Disease Progression via Muscle MRI, Clinical, and PROs - Physician's Weekly

Researchers at Stanley Manne Childrens Research Institute at Ann & Robert H. Lurie Childrens Hospital of Chicago have made a major breakthrough that could lead to new treatments for neurological diseases and cancer. The team, led by Dr. Yongchao C. Ma, found that a chemical change in RNA, called RNA methylation, plays a crucial role in controlling how mitochondria work in cells.

Mitochondria are known for producing energy in cells, but they also act as a control center that regulates many important biological processes. Dr. Mas previous research has linked problems with mitochondria to the development of spinal muscular atrophy (SMA) and autism, while other researchers have connected mitochondrial issues to cancer.

Our finding establishes a critical link between RNA methylation, mitochondria and diseases that relate to mitochondrial dysfunction, which means that now we have potential for new treatments for many different disorders, said Dr. Ma, who holds the Childrens Research Fund Endowed Professorship in Neurobiology at Lurie Childrens and is Associate Professor of Pediatrics, Neurology, and Neuroscience at Northwestern University Feinberg School of Medicine.

The researchers discovered that RNA methylation controls the production of key enzymes that are part of mitochondria. They showed in both stem cells and mice that when RNA methylation was lost, it significantly altered how mitochondria functioned in stem cells and neurons.

This groundbreaking finding opens up exciting possibilities for developing new treatments that target RNA methylation to fix mitochondrial defects. Dr. Ma and his team are enthusiastic about the potential impact of their discovery.

We are very excited about this discovery and the promise of innovative treatments, which could involve developing modifiers of RNA methylation to rectify the mitochondrial defect, said Dr. Ma.

The study, published in the journal Human Molecular Genetics, is a significant contribution to the rapidly growing field of RNA methylation research. Dr. Ma and his team at Lurie Childrens are committed to continuing their work on RNA methylation in the nervous system, with the goal of gaining new insights into brain development and neurological disorders.

Lurie Childrens is a top-ranked, nonprofit childrens hospital dedicated to providing exceptional care to every child. The hospitals research is conducted through Stanley Manne Childrens Research Institute, which focuses on improving child health, transforming pediatric medicine, and ensuring healthier futures through the relentless pursuit of knowledge. Lurie Childrens also serves as the pediatric training ground for Northwestern University Feinberg School of Medicine.

With this remarkable discovery, Dr. Ma and his team have opened the door to a new era of innovative treatments for neurological diseases and cancer, offering hope to countless patients and their families.

The material in this press release comes from the originating research organization. Content may be edited for style and length. Want more? Sign up for our daily email.

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Groundbreaking Discovery Paves Way for New Treatments of Neurological Diseases and Cancer - ScienceBlog.com

A report recently published in The Lancet, using vast data from 1990 to 2021, has found that heart disease is now trailing behind neurological conditions in terms of commonality, but it remains our biggest killer.

According to the statistical analysis more than 3.4 billion people, thats 43 percent of the global population, suffered with a neurological condition in 2021, a number far more than experts had previously feared. The study was overseen by researchers at the Institute of Health Metrics and Evaluation (IHME) in Seattle, Washington, and illustrates that nervous system disorders have overtaken heart disease as the number one cause of ill health. Neurological conditions (diseases of the central and peripheral nervous systems) such as strokes and dementia, in addition to migraines and other mental health diseases has now topped the list in terms of commonality.

Researchers looked at 37 different neurological conditions to determine just how they influenced illness, disabilities, and premature deaths in more than 204 territories across the world and found that an 18% decrease in life years had been experienced collectively due to those disorders. More than 11 million people had sadly died from neurological conditions in 2021 concluded the report.

While adjustments for the populations age and overall growth could refute the findings in terms of the decrease in terms of life years, stroke still comes out as the most serious neurological ailment. Other neurological conditions follow, such as encephalopathy (a type of brain damage) and Alzheimers disease. Nerve damage suffered as a result of diabetes is also significantly hampering the population.

One reason for neurological conditions overtaking heart disease in terms of prevalence can be attributed to a recent World Health Organisation classification edit, since the WHO has recently moved stroke into the neurological grouping. But, while weve established that stroke has emerged as the most serious cognitive ailment faced today, the biggest overall killer is still cardiovascular disease.

According to the IHME, heart disease accounted for 19.8 million worldwide deaths in 2022.It is no doubt sobering to read that both heart and cognitive diseases are still very much at war with the human race, but its somewhat its reassuring to know that exercise is one of the best methods we have for improving both mental and physical health. The ultimate reason to renew that gym membership!

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Neurological Conditions Found To Be The Most Common Disease - Muscle & Fitness

WATCH TIME: 3 minutes

Welcome to this special edition of Neurology News Network. Im Marco Meglio.

According to an announcement, Amylyx Pharmaceuticals will voluntarily discontinue AMX0035 (Relyvrio) and remove it from the market in the United States and Canada based onnegative topline datafrom its phase 3 PHOENIX trial (NCT05021536) that showed AMX0035 did not meet its primary end point of change in ALS Functional Rating Scale-Revised (ALSFRS-R).1,2AlthoughAMX0035will no longer be available for new patients with amyotrophic lateral sclerosis (ALS), those currently on therapy in the US and Canada who wish to stay on treatment and consult with their cliniciancan be transitioned to a free drug program. In PHOENIX, results showed no significant difference on ALSFRS-R between AMX0035-treated and placebo-treated patients over a 48-week treatment period (P = .667).

In a newly published first-in-human trial (NCT02362438), treatment with scAAV9/JeT-GAN, an investigational gene therapy administered directly into the spinal fluid, was well tolerated and showed signs of therapeutic benefit among children with giant axonal neuropathy (GAN). Published in theNew England Journal of Medicine,the investigators observed various rates of slowed motor function decline, with nearly half of the small cohort regaining sensory nerve response. scAAV9/JeT-GAN is a self-complementary adeno-associated viral (AAV) serotype 9 vector that carries a codon-optimized humanGANtransgene with expression controlled by the minimal synthetic recombinant JeT promoter consisting of 5 elements.

According to an announcement from Eisai and Biogen, the companies have officially submitted a supplemental biologics license application (sBLA) for a new monthly intravenous (IV) maintenance dosing for lecanemab-irmb (Leqembi), its FDA-approved therapy for early-stage Alzheimer disease (AD). Those whove already completed the biweekly IV initiation phase are now eligible to receive a monthly IV does that maintains effective drug concentration to sustain the clearance of highly toxic protofibrils. Lecanemab, a humanized immunoglobulin gamma 1 monoclonal antibody directed against aggregated soluble and insoluble forms of amyloid-, was approved in July 2023 in 100 mg/mL injections for IV use. In the newly submitted sBLA, the companies included data from Study 201 (NCT01767311), a phase 2 trial, and its open-label extension (OLE), as well as the phase 3 Clarity AD trial (NCT03887455), the study that lecanemab was traditionally approved on, and its OLE.

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Amylyx Pulls ALS Drug AMX0035, Gene Therapy Shows Promise in Giant Axonal Neuropathy, Eisai Submits sBLA for ... - Neurology Live

Joseph Schneider December 22, 2023

I consider myself a patient as well as a healer. I remember lying in my hospital bed after I suffered a stroke in May 2017. I could not move the right side of my body, and I thought all my hopes and dreams had gone away, including my hopes of ever practicing as a functional neurologist and DC again or being able to work with my patients. Prior to that, I suffered three concussions from various sports injuries and motor vehicle accidents. My brain went through a lot.

I am grateful to my colleagues in chiropractic functional neurology, and the knowledge I gained from extensive experience with my patients. Through it all, I have learned a lot about brain recovery from overcoming my stroke and now I am committed to helping my patients achieve optimal health without medications and/or surgeries. Now I have a vision to help other DCs learn about the future innovations in technology, and the research and breakthroughs happening today, which that will enable chiropractic functional neurologists to lead the field in brain regeneration.

At my clinic, I work to heal hurt brains at my clinic, where all the treatment plans incorporate chiropractic adjustments, functional neurology and the most cutting-edge modalities available. Those modalities include the latest technology to accelerate patient outcomes, such as oxygen therapy, multi-axis rotating chair therapy, neurofeedback, interactive noninvasive imaging studies, vibration therapy and photobiomodulation (low-level light therapy).

The most important aspect of any brain-specific program is to have an impact that changes the patients life in four critical areas: 1. work relationships, 2. recreation, 3. household chores and 4. sleep. The goal is to make life more dynamic and vibrant for the individual.,

Many patients come to my clinic after they have exhausted all other methods. My website is full of video testimonials from patients who regained their health thanks to the latest technologies the center uses for brain improvement. Brain-specific rehabilitation is a combination of functional medicine and functional neurology. All the pieces have to be put in place for maximum improvement and outcome. In addition, there is a different and very specific order for each patient. There isEach usually has a collection of symptoms, and they all need to be traced back to the systems in the body and treated holistically and synergistically.

Some patients experienced asymptomatic concussions. Perhaps the injury occurred 20 years ago, and they thought they were OK, but lately have noticed signs of dementia, brain fog and emotional challenges. These symptoms, we know, are related to the degeneration of the brain over time as a result of past trauma. The good news is we can help people at any point. We treat many patients who have struggled for years since their brain injury.

I started studying functional neurology in 1989 with Ted Carrick, DC, PhD, MS-HPEd. Carrick has been my friend, my mentor and my doctor after my stroke. In the 1990s, at the beginning of my career, I used to go to seminars with Carrick before I got board-certified in neurology. After a weekend of learning, I would go back to my practice and I would start to look at eye movements, balance, finger-pointed-at-the-nose things for looking at metric movements and dysmetria for the cerebellum. I looked carefully at my patients because Carricks big lesson was to know normal.

The brain is the master control system of the body, so as chiropractors we use spinal manipulation as a way of improving function throughout the body. Our rightful place is to continue to have our examination skills at a level in which we can look at the function of the brain and the brains interaction with the body. And looking at most of the contemporary diseases today, such as movement disorders, dystonia, visual issues, visual dysfunction, vertigo, dizziness and balance issues, chiropractors can improve their skills and understanding of the issues by observing patients and absorbing abnormal findings. Traditional chiropractic methods combined with brain-based adjustments can improve patient outcomes.

You may wonder if DCs can use their technique to change brain function. The answer isyes! Its called brain-based adjustments. There are ways of adjusting the spine that have a brain effect. If you want to, you can take your practice to the point where you start evaluating brain function through examination techniques and diagnostic technologies. By evaluating in this way, you can rate function and create a baseline for the patient. Then you can use different technologies and exercise systems to actually improve the pathways in the brain through connectivity and also take neurons from stem cells and create new neurons in the cortical areas.

Once I understood the brain was the master control system of the body, a light went off in my brain, and I knew I wanted to be a DC. I was an engineer, so people I told would ask me, Why do you want to be a chiropractor? Why not be a medical doctor? But I said, No, I want to change the master control system of the body. And thats what I did. I left my engineering career and went off to New York Chiropractic. When I look back, I realize, my love of chiropractic prompted my love of changing brains. Now every single patient that who comes to my office gets adjusted.

JOSEPH SCHNEIDER, DC, is a Board Certified Chiropractic Neurologist. He graduated from New York Chiropractic College in 1987. Schneider is in clinical practice at Hope Pain Relief in Chadds Ford, Penn.

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The key to brain-based adjustments: traditional chiropractic + functional neurology - Chiropractic Economics

The averageheadachedoesnt require a call to a neurologist or even your family doctor. But if youre experiencing frequent headaches and usingmedication for them regularly, thats a different story.

If you have a history of headaches that come once or twice a month and go away when you take an over-the-counter (OTC) medication such asacetaminophen (Tylenol) or ibuprofen (Aleve), you may not need to seek further treatment, saysSandhya Kumar, MD, a neurologist andheadache specialistat Wake Forest Baptist in Winston-Salem, North Carolina.

If youre having headaches more than four times a month, especially if they are debilitating and keeping you home from work, you should see a provider for diagnosis and medication, says Dr. Kumar.

As a general rule, for nonsevere headaches, your family doctor is a great person to start with. Approximately 7 out of 10 people talk to their primary care doctor first, according to theAmerican Headache Society.

If the recommended treatments are not working well or you have unusual symptoms, your doctor may refer you to a neurologist, who specializes in disorders of the brain and nervous system.

Possible signs that you may need to see a specialist for your headaches include:

According to headache expertPeter Goadsby, MD, PhD, a professor of neurology at the UCLA GoldbergMigraineProgram in Los Angeles, a valuable tool in diagnosis is your headache history.

A thorough history, aided by your detailed notes, can pinpoint causes, triggers, and even potential solutions. Make careful notes about your headache experiences before you go to the doctor. Include the following:

Dr. Goadsby recommends using a monthly calendar so that the pattern of headache days is clearly visible to you and your doctor.

If you are having severe or disabling headaches, dont wait a full month to call for an appointment make notes about what you recall or are experiencing and see a doctor as soon as you can.

The tests your doctor orders will depend in part on what they suspect could be causing your headaches and whether its a primary headache such as amigraineor tension headache or a secondary headache, which means that its a symptom of another health concern.

Although primary headaches can be painful and debilitating, they arent life-threatening.

Secondary headaches are much rarer and can be the sign of a serious health issue sometimes even one that requires urgent medical attention.

The process of diagnosis may include the following:

Medical HistoryYour doctor will want to know about any other health conditions you have as well as any medications, supplements, or herbal treatments you take.

Family HistoryBe prepared to provide details about any family members who have headaches or migraine at what age their headaches started and any other health diagnoses they may have. As Goadsby notes, Very often, family members wont know theyve got migraine, but they will know they are prone to headaches. Since migraine has a strong genetic component, a family history of migraine-like symptoms is an indicator that your headaches are also being caused by migraine.

Physical ExamYour doctor will examine you, paying close attention to yourhead,neck, and shoulders, which can all contribute to headache pain in various ways.

Neurological ExamA neurological exam may include tests of your vision, hearing, short-term memory, reflexes, sensation, balance, and coordination.

Blood TestsBlood tests may be ordered to rule out infection and other health conditions that have headache as a symptom.

Spinal Fluid TestThis may be necessary if your doctor suspects that your headaches are caused by certain types of infection or by bleeding in your brain.

UrinalysisA urine sample may be ordered to help rule out infection and other health conditions.

Imaging TestsComputed tomography(CT) or magnetic resonance imaging (MRI) scans may be ordered. These imaging tests can show structures in your head, neck, or elsewhere in the body that may be causing your headaches.

Neuroimaging Tests These may be done during a headache episode to get a clearer picture of what is going on during an actual headache.

Electroencephalogram (EEG)This test can show your doctor whether there are changes in brain wave activity. It can help diagnose brain tumors, seizures, head injury, and swelling in the brain.

Working closely with your family practitioner and a neurologist, if needed, will bring you closer toheadache relief.

Warning signsthat you need immediate medical attention for your headache ormigraineinclude:

RELATED:When Should You Worry About Your Headache and Seek Immediate Help?

Additional reporting byBecky Upham.

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Is It Time to See a Neurologist for Your Headaches? - Everyday Health

In 2023, the NeurologyLive staff was kept on its toes while covering clinical news and data readouts from around the world across a number of key neurology subspecialty areas. Between major study publications and FDA decisions, and from societal conference sessions and expert interviews, the team spent all year bringing the latest updates to the website's front page.

Among our key focus areas is headache and migraine, two of the most common neurological diseases worldwide. Treatments for headaches have advanced over the years; however, providing lasting and effective treatment for all headache types has proven to be difficult for all practitioners. The field has been advanced significantly by the introduction of calcitonin gene-related peptide (CGRP)-targeting inhibitors, a class of highly effective and safe agents. Whatever the reason for the attention these stories got, their place here helps provide an understanding of the themes in this field over the course of 2023.

Here, we'll highlight some of the most-read content on NeurologyLive this year. Click the buttons to read further into these stories.

Earlier this year, the FDA approved IntelGenx/Gensco'srizatriptan benzoate (Rizafilm VersaFilm) oral thin film for acute migraine treatment through the 505(b)(2) new drug application (NDA) pathway. This newly approved treatment is an orally disintegrating film formulation of the 5-HT1 receptor agonist designed to be bioequivalent to Mercks Maxalt-MLT, an orally disintegrating rizatriptan treatment.

The FDA has expanded the indication for atogepant (Qulipta; AbbVie) to include the prevention of chronic migraine in adults, adding to its existing indication for episodic migraine, according to an announcement from AbbVie.1 The approval was granted based on data from the phase 3 PROGRESS trial (NCT03855137) that showed that 60-mg atogepant resulted in a significant reduction in mean monthly migraine days (MMDs) compared with placebo across 12 weeks of treatment.

Using a cohort of medically-insured individuals in Arizona, findings from a recently conducted analysis showed specific factors associated with receiving a migraine diagnosis vs a headache diagnosis. Those in the migraine cohort tended to be middle aged, female, White race, non-Hispanic ethnicity, and have English as their primary language. All told, issues within the social determinants of health categories of family unit dynamics (OR, 1.1; 95% CI, 1.06-1.14) and income and social protection (OR, 1.13; 95% CI, 1.08-1.18) were associated with a higher odds of being in the migraine vs headache cohort.

Findings from a pilot study (NCT04437199) assessing up to 60 g/day of tricaprilin (Cerecin), an investigational ketogenic compound, suggested potential benefit in treating patients with migraine. At the end of the 3-month treatment period, some patients opted to enter the Compassionate Access Program, which provides continued access to the therapy for up to 1 year after completion of the clinical study.

According to a new update from WL Gore & Associates, also known as Gore, patients in the RELIEF clinical study (NCT04100135) have completed their multi-month enhanced screening process and have begun entering the final randomization phase. The trial, initiated in November 2022, assesses whether closing the patent foramen ovale (PFO) using the GORE CARDIOFORM Septal Occluder may provide relief for patients with migraine. The GORE CARDIOFORM Septal Occluder is a permanently implanted device indicated for the percutaneous, transcatheter closure of ostium secundum atrial septal defects (ASDs) and PFO, intended to reduce the risk of recurrent ischemic stroke.

At the 2023 International Headache Congress, held September 14-17, in Seoul, Korea, new data from the phase 2 HOPE trial (NCT05133323) highlighted the effects of Lu AG09222 (Lundbeck), a pituitary adenylate cyclase-activating polypeptide (PACAP)-targeting agent, as a potential preventive for migraine. All told, the trial met its primary end point, with significant between-group differences observed in the high dose group of treated patients over a 12-week double-blind period.

The European Commission approved AbbVies atogepant (Aquipta) for the prophylaxis of migraine in adults who have 4 or more migraine days per month, becoming the first and only calcitonin gene-related peptide (CGRP) agent indicated for prevention of both episodic and chronic migraine. The approval was based on data from 2 phase 3 studies, PROGRESS (NCT03855137) and ADVANCE (NCT02848326), in which atogepant-treated patients showed greater reduction of monthly migraine days (MMDs) than placebo.

Investigators published full findings of the phase 3 PRODROME study (NCT04492020) demonstrating ubrogepants (Ubrelvy; AbbVie) positive impact on migraine during the prodrome phase in The Lancet. At the conclusion of the trial, absence of moderate or severe headache within 24 hours after initiating treatment occurred in 46% (190 of 418) of qualifying prodrome events that had been treated with ubrogepant compared with 29% (121 of 423) of events treated with placebo (OR, 2.09; 95% CI, 1.63-2.69; P <.0001).

Newly announced topline findings from the CHALLENGE-MIG trial (NCT05127486), the first head-to-head clinical study comparing 2 medications targeting calcitonin gene-related peptide (CGRP), revealed similar efficacy between galcanezumab (Emgality; Eli Lilly) and rimegepant (Nurtec ODT; Biohaven Pharmaceuticals). Despite this, galcanezumab outperformed Rimegepant on secondary end points at the end of the 3-month trial.

The FDA has accepted Satsuma Pharmaceuticals 505(b)(2) new drug application (NDA) for its novel, investigational dihydroergotamine (DHE) nasal powder product, STS101, for the acute treatment of migraine. The agency is expected to make a decision on the therapy by January 2024. STS101 is designed to provide significant benefits vs existing acute treatments for migraine, including the combination of quick and convenient self-administration and other clinical advantages that current DHE liquid nasal spray products and injectable dosage forms lack.

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NeurologyLive Year in Review 2023: Top Stories in Headache and Migraine - Neurology Live

The NeurologyLive staff was hard at work in 2023, covering clinical news and data readouts from all over the United States and the world, across a number of key neurology subspecialty areas. From major study data and FDA decisions to medical society conference sessions and expert conversations, the team spent all year bringing the latest news and updates to the website's front page.

Among our key focus areas is movement disorders, which include a number of complex diseases that have benefitted greatly from recent advances in medical care and therapeutic development. Although major news itemssuch as first-time approvals or new guidelinesoften appear among the top pieces our team produces, sometimes smaller stories reach those heights for other reasons, such as clinical impact and interest, or concerns about other facets of care, for example. Whatever the reason for the attention these stories got, their place here helps provide an understanding of the themes in this field.

Here, we'll highlight some of the most-read content on NeurologyLive this year. Click the buttons to read further into these stories.

Exploratory findings from a phase 3 randomized, controlled trial (NCT03329508) assessing P2B001 (Pharma Two B), a low dose combination of extended-release pramipexole and rasagiline, in Parkinson disease (PD) showed efficacy that was comparable to extended-release pramipexole (Prami-ER) alone, but with reduced sleep-related and dopaminergic adverse events (AEs). Pharma Two B planned to submit a new drug application for P2B001 to the FDA in 2023.

Newly announced findings from a triple-blinded, randomized controlled trial showed that treatment with SYMBYX Neuro infrared light therapy helmet significantly improved symptoms of Parkinson disease (PD) in areas of facial expression, upper and lower limb coordination and movement, walking gait, and tremor. Using the standardized Movement Disorder Society Unified Parkinsons Disease Rating Scale-III (UPDRS-III), compared with the placebo group, those on the light therapy improved 24% to 58% over baseline across all 5 areas tested, unlike the placebo group, which demonstrated statistically valid improvement in lower limb coordination and movement only.

After showing positive results in a phase 3 clinical program, the FDA has accepted Revance Therapeutics supplemental new biologics license application (sBLA) for daxibotulinumtoxinA injection (Daxxify), as a new treatment for adults with cervical dystonia. The agency ultimately approved the therapy on August 15, 2023. DaxibotulinumtoxinA is an acetylcholine release inhibitor and neuromuscular blocking agent indicated for the temporary improvement of moderate to severe glabellar lines associated with corrugator and/or procerus muscle activity in adults. To date, the therapy has shown promising results in 2 phase 3 studies of cervical dystonia, ASPEN-1 (NCT03608397) and ASPEN-OLS (NCT03617367).

Despite years of use of gold-standard therapy levodopa, therapeutic development in Parkinson disease has advanced rapidly and expanded to numerous novel pathways and targets. MedStar Georgetown's team of Katelynn Getchell; Gonul Ozay, MD; Brian Nagle, MD; Irma Zhang, MD; Luke Lovelace; Emma Waldon, RN; Yasar Torres-Yaghi, MD; and Fernando L. Pagn, MD explore this in depth.

Topline data from the Synuclein-One Study of CND Life Sciences Syn-One Test, an -synuclein skin biopsy test used for the detection of the pathology in Parkinson disease (PD), dementia with Lewy bodies (DLB), multiple system atrophy (MSA), and pure autonomic failure (PAF), suggest that the test is sensitive and specific in said detection of phosphorylated -synuclein. As misdiagnosis remains a consistent challenge in neurodegenerative disorderssome estimates suggest a misdiagnosis rate of 30%this represents an opportunity to address this clinical obstacle.

Using prospective cohort studies of community-dwelling elders followed up to 20 years, findings published in Neurology identified specific cognitive and functional declines in patients who developed incident Parkinson disease (PD). There were important sex differences as well, as men with incident PD had a steeper decline in executive function compared with women, but only women with incident PD exhibited detectably faster prediagnostic decline in global cognition.

BIAL R&D announced the dosing of the first patient in its phase 2 clinical trial, ACTIVATE (NCT05819359), to investigate BIA 28-6156, an allosteric activator of the enzyme beta-glucocerebrosidase (GCase), as a treatment of patients with genetically-mutated Parkinson disease (PD). The trial, which includes those with a mutation in the glucocerebrosidase 1 (GBA1) gene (GBA-PD), otherwise the most common genetic risk factor of the disease, is screening patients across sites in North America and with a Europe-based trial planned to initiate in the third quarter of 2023.

Biogen and Denali have announced that they are discontinuing a portion of the clinical development program for BIIB122 (also known as DNL151), an investigational small molecule inhibitor of LRRK2 in development for the treatment of Parkinson disease (PD). As a result of this decision, the phase 3 LIGHTHOUSE study (NCT05418673), which was initiated in September 2022, will be terminated.

New data from a first-in-human phase 1 study (NCT04802733) assessing bemdaneprocel (BlueRock Therapeutics/Bayer), an investigational cell therapy, showed that the agent met its primary objective of safety, with encouraging results on other measures of motor and nonmotor outcomes. Based on these results, the companies are planning for a phase 2 trial that is expected to begin enrolling patients in the first half of 2024.

The FDA has issued a complete response letter (CRL) to Amneal Pharmaceuticals for IPX203, an oral formulation of carbidopa/levodopa (CD/LD) extended-release capsules designed for the treatment of Parkinson disease. The reasons behind the decision were not based on efficacy or manufacturing for the agent, but rather established safety for an ingredient of the therapy. Amneal plans to work closely with the FDA to address the comments and align on the best path forward.

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NeurologyLive Year in Review 2023: Top Stories in Movement Disorders - Neurology Live

KANSAS CITY, Mo. Dr. Brian Aalbers, DO, a pediatric neurology specialist, is charged in federal court complaint with one count of attempted production of child pornography.

Court documents stated that Oct. 23, 2023, Kansas City, Missouri, police officers were sent to meet a reporting party who said he located hidden video cameras. The location of where the officers were sent and other details were blacked out in a court document.

Aalbers arrived at the scene, but did not provide a statement, according to the court document.

The person who reported the hidden video cameras contacted KCMO police about text messages he received from Aalbers.

Officers found Aalbers in a Lenexa hotel and took him to Advent Health for voluntary treatment, the document states.

The hospital security staff took a backpack from Aalbers that contained laptop computers, iPad tablets and a cell phone.

Aalbers did not have access to the devices while in the facility and asked a friend to get his backpack.

According to the court document, Aalbers asked that person to destroy the devices because there was "bad stuff" on them.

In November, a forensic examiner told investigators there over 20,000 videos found on a MacBook Pro.

The videos were from December 2020 to October 2023.

In addition, the forensic examiner "noted that approximately 10-12 additional minor victims were captured in the video recordings."

Aalbers is back in court for preliminary and detention hearings on Nov. 21 in federal court in KCMO.

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Well-regarded Johnson County pediatric neurologist charged with ... - KSHB 41 Kansas City News

In 2023, the NeurologyLive staff was a busy bunch, covering clinical news and data readouts from around the world across a number of key neurology subspecialty areas. From major study publications and FDA decisions to societal conference sessions and expert interviews, the team spent all year bringing the latest information to the website's front page.

Among our key focus areas is epilepsy and related seizure disorders, a field that features complex diseases that are often medically refractory and difficult to manage. Although major news items often appear among the top pieces our team produces, sometimes smaller stories reach those heights for other reasonsclinical impact and interest, or concerns about the small- or big-picture parts of care, for example. Whatever the reason for the attention these stories got, their place here helps provide an understanding of the themes in this field in 2023.

Here, we'll highlight some of the most-read content on NeurologyLive this year. Click the buttons to read further into these stories.

Findings from a comparative effectiveness research study showed that use of levetiracetam and lamotrigine as first-line treatments have similar efficacy on idiopathic generalized epilepsy (IGE) syndromes in females; however, levetiracetam was more effective in treating juvenile myoclonic epilepsy. Further studies are still needed to identify the most effective antiseizure medication alternative in other IGE syndromes.

The FDA has issued a warning for the use of antiseizure medicines levetiracetam (Keppra, Keppra XR, Elepsia XR, Spritam) and clobazam (Onfi, Sympazan), which can cause drug reaction with eosinophilia and systemic symptoms, known as DRESS, arare but serious adverse effect. The reaction may start as a rash but can quickly progress, resulting in injury to internal organs, the need for hospitalization, and even death. As a result, the FDA is requiring new warnings about this risk to be added to theprescribing informationand patientmedication guidesfor these medicines.

According to an announcement from Cumulus Neuroscience, the FDA has granted clearance to its novel, dry-sensor EEG headset, a user-friendly device that enables self-directed use and generates clinical-grade data for remote physician review. The Cumulus EEG device, designed for both adult and adolescent patients, is available in 4 sizes, and is easily self-applied with guidance from the Cumulus mobile app. The platform combines clinical-grade, at-home data with machine learning analytics and a large real-world database of annotated, longitudinal, matched data.

Using a large-scale cohort of electronic health records, recently published findings identified robust and clinically meaningful independent associations between incident epilepsy and both epilepsy/enzyme-inducing antiseizure medication use with incident osteoporosis. These data highlight the need for enhanced vigilance and consideration of prophylaxis for all patients with epilepsy.

At the 35th International Epilepsy Congress, held September 2-6, 2023, in Dublin, Ireland, UCB Pharma presented several posters showcasing the clinical benefits of fenfluramine (Fintepla) across multiple forms of epilepsy, including rare epileptic disorders such as Dravet syndrome (DS), Lennox-Gastaut syndrome (LGS), and CDKL5 deficiency disorder. The first presentation was a review of 13 studies assessing the impact of the therapy on generalized tonic-clonic or tonic-clonic seizures in a cohort of rare epilepsy syndromes; another abstract assessed the safety and efficacy of adult patients with DS who did not participate in the phase 3 clinical trials but enrolled in the open-label extension study de novo; and a comparative analysis of clinical trial data further highlighted fenfluramines impact on drop seizure frequency (DSF) in dose-capped patients with LGS.

Cornelia Drees, MD, senior associate consultant in the Department of Neurology at Mayo Clinic, provided insight on an early feasibility study on the clinical impact of microburst vagus nerve stimulation in patients with drug-resistant epilepsy, presented at the 2023 American Academy of Neurology (AAN) Annual Meeting, held April 22-27, in Boston, Massachusetts.

A post hoc analysis newly published in Epilepsy & Behavior on the phase 3 open-label extension (OLE) study (NCT01529034) assessing midazolam (Nayzilam; UCB), an FDA-approved nasal spray, showed that 90 minutes was the estimated median time to return to full baseline functionality (RTFBF) regardless of treatment with 1 or 2 doses among patients who experienced seizure clusters (SCs). These findings suggest that the dose of midazolam did not influence the time to RTFBF in SC episodes and further support the favorable profile of repeated intermittent use of midazolam in patients with SCs.

Using data from spontaneous and solicited reports, findings from a new analysis showed that lacosamide (Vimpat; UCB Pharma), an antiseizure medication, was safe to use during pregnancy, with most exposed pregnancies resulting in live births. Lacosamide, listed as a Pregnancy Category C medication, had no new safety concerns associated with its use in data presented at the 2023 American Epilepsy Society (AES) annual meeting, held December 1-5, in Orlando, Florida.

Data from a published retrospective analysis of adolescents and children presenting with seizures showed that midazolam is not an effective first-line therapy in prehospital settings, indicated by the nearly 40% of patients who required rescue therapy. Published in JAMA Network, the study featured 1172 children with a mean age of 5.7 years for whom a mobile intensive care unit was dispatched for an active seizure.

New post hoc data from a recently completed phase 3 trial (NCT02721069) assessing diazepam nasal spray (Valtoco; Neurelis), an FDA-approved antiseizure medication (ASM), indicated that faster time to administration was associated with shorter time to seizure cluster cessation and overall shorter seizure duration. Over 12 months, investigators also noticed a statistically significant change in SEIzure interVAL, or the time between seizure clusters, that was independent of the age of changes with concomitant ASMs.

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NeurologyLive Year in Review 2023: Top Stories in Epilepsy and Seizure Disorders - Neurology Live

In 2023, the NeurologyLive team spoke with hundreds of people and posted hundreds of hours of interview clips. The staff spoke with neurologists, investigators, advanced practice providers, physical therapists, advocates, patients, pharmacists, and industry expertsanyone involved in the process of delivering clinical care.

These conversations were had with individuals from all over the world, both virtually and in person. The team attended more than a dozen annual meetings of medical societies, each time sitting down with experts on-site to learn more about the conversations driving care and the challenges being overcome.

From those in the field of sleep medicine, we learned much this year: recent updates to restless legs syndrome care; the challenges in managing narcolepsy's secondary symptoms; CPAP's effects in other neurologic disorders; heart health associations with sleep; and more.

Here, we'll highlight the most-viewed expert interviews on NeurologyLive this year. Click the buttons to watch more of our conversations with these experts.

The chief of the Sleep Disorders Clinical Research Program at Massachusetts General Hospital provided insight on new updates to the management of restless legs syndrome, including removing dopamine agonists as first line treatments.

WATCH TIME: 8 minutes

"Dopamine agonists are not first line treatments. The reason for that is theres substantial evidence that dopamine agonists when used for restless legs syndrome are associated with an augmentation of symptoms, a worsening of the underlying disorder."

The pediatric neurologist and sleep medicine specialist at Geisinger Medical Center provided commentary on the current unmet needs for patients with narcolepsy, including improvements in treatment options.

WATCH TIME: 3 minutes

"The reality is the disease is characterized, at a minimum, by a pentad of symptoms, which is excessive daytime sleepiness, cataplexy, sleep-related hallucinations, sleep paralysis, and disturbed nocturnal sleep.

The associate professor in the department of neurology and neurosurgery at McGill University discussed results from a study on the long-term use of continuous positive airway pressure treatment among patients with multiple sclerosis and sleep apnea presented at MSMilan 2023.

WATCH TIME: 5 minutes

"Our study indicates that CPAP treatment in patients with MS and sleep apnea is associated with a reduction in fatigue and an improvement in physical quality of life, offering potential benefits for long-term symptom management. Clinicians should consider exploring sleep apnea as a factor contributing to fatigue and poor sleep quality in patients with MS, as adequate treatment may lead to noticeable symptom improvement."

The medical director of SleepMed in South Carolina discussed the need for more overall awareness of poor sleep and the risk factors associated with worsened heart health.

WATCH TIME: 3 minutes

"Its critical to get the word out. We need to understand whats happening biologically, in terms of sleep homeostasis, sleep wake processor, and how thats controlled. What are the set points of heart rate and blood pressure? How are they modified? [We need to] Get the message out."

The cofounder and chief product development officer of Zevra Therapeutics talked about the phase 1 clinical trial of KP1077 for narcolepsy and potentially using it to treat other conditions..

WATCH TIME: 5 minutes

The biggest thing with this study is it is will help us inform study designs for future research. We are looking for the appropriate dosing regimen [of KP1077] and what will work best for patients with narcolepsy.

The senior vice president of medical and clinical affairs for Avadel Pharmaceuticals provided commentary on recently published research supporting once-nightly sodium oxybate (Lumryz) in narcolepsy regardless of the subtype.

WATCH TIME: 5 minutes

"Ensuring that clinicians are having conversations with patients with narcolepsy routinely, and asking about the more subtle presentation of cataplexy, is important. Many patients have their diagnosis changed from NT2 to NT1."

The associate professor, department of medicine, division of neurology, Institute of Medical Science, University of Toronto, talked about the importance of establishing normal values for sleep studies, particularly the multiple sleep latency test, to help with effectively diagnosing sleep disorders.

WATCH TIME: 5 minutes

The purpose of this study was to perform a larger and pretty comprehensive meta-analysis on the mean sleep latency derived from the MSLT (Multiple Sleep Latency Test). We also wanted to look at the impact of things like age, sex, body mass index, other sleep metrics. In addition, we wanted to investigate different methodological variables, such as sleep onset definitions, and sleep study features, as well other markers preceding the sleep study and see if that did affect the mean sleep latency on the MSLT that was performed.

The duo from Indiana University School of Medicine discussed the ongoing research initiatives to better understand sleep disorders among pediatrics, and ways to improve approaches like cognitive behavioral therapy.

WATCH TIME: 4 minutes

"There is a tendency in medicine and in society to focus on the need to eliminate screens and/or to assume that screens are causing the insomnia. This can be very minimizing to kids with insomnia because it implies that theres a simple solution and that a lifestyle factor is causing the insomnia."

The professor of neurology at UMass Chan School of Medicine discussed the various impacts Daylight Savings Time has on sleep quality and overall health in children and adolescents.

WATCH TIME: 3 minutes

"But what a lot of people don't realize is the long-term effects during the 8-month period on Daylight Saving Time. We may blame it on other things, but what we know is that those hour later sunrises and sunsets are associated with about a 10% increase rate of cancer, at least a 10% increase risk of obesity, and increased risk of heart disease."

The sleep epidemiologist and assistant professor at the Rollins School of Public Health at Emory University discussed the multi-level effort needed to improve sleep issues seen in individuals most impacted by social determinants of health.

WATCH TIME: 3 minutes

"The other piece is, how can we modify some of our recommendations to fit disadvantaged communities. For example, we say to sleep in a dark, quiet room, but we know that everyone cant do that because of safety issues. Adjusting the recommendation to say, put a light on in a hallway or somewhere else in the house."

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NeurologyLive Year in Review 2023: Most Watched Interviews in Sleep Disorders - Neurology Live

A Type of Belly Fat May Be Linked to Increased Risk of Developing Alzheimers NBC News November 20 -Joel Salinas, MD, MBA, clinical assistant professor, Department of Neurology, the Pearl I. Barlow Center for Memory Evaluation and Treatment

Avoid Flus, Colds, More This Holiday Season with These Health Tips TODAY Show November 17 -Natalie E. Azar, MD, clinical associate professor, Department of Medicine, Division of Rheumatology

Can You Eat Enough at Thanksgiving to Make Your Stomach Explode? Technically, Yes New York Post November 17 -Sophie M. Balzora, MD, clinical professor, Department of Medicine, Division of Gastroenterology and Inflammatory Bowel Disease

Diabetic Eye Disease Treatment Methods WBNG-TV November 17 -Yasha S. Modi, MD, associate professor, Department of Ophthalmology

Nobodys Talking About This Hidden Threat in Generative AI Fast Company November 17 -NYU Langone Health

How Much Does Medical School Cost and Can I Afford It? Business Insider November 17 -NYU Langone Health

Philips Inks Multi-Year Health Technology Deal with NYU Langone Health Mass Device November 17 -Nader Mherabi, executive vice president, vice dean, and chief digital and information officer

Surgeons That Dont Fit the Culture Can Increase Diversity in Spine, Orthopedic Care Beckers Spine Review November 17 -Qusai Hammouri, MD, clinical assistant professor, Department of Orthopedic Surgery

Inside a Long COVID Clinics Fight to Meet Crushing Patient Needs (Free log-in required.) Medscape November 17 -Jonathan H. Whiteson, MD, associate professor, Departments of Rehabilitation Medicine, and Medicine, the Leon H. Charney Division of Cardiology, vice chair, Rusk Rehabilitation Clinical Operations

Genetic Information May Predict Recurrence in Lung Cancer Cure Today November 17 -Igor Dolgalev, PhD, assistant professor, Department of Medicine

Surgeons at NYU Langone Perform First-Ever Eye and Partial-Face Transplant Washington Square News November 17 -Eduardo D. Rodriguez, MD, DDS, the Helen L. Kimmel Professor of Reconstructive Plastic Surgery, chair, the Hansjrg Wyss Department of Plastic Surgery -Vaidehi S. Dedania, MD, associate professor, Department of Ophthalmology -Steven L. Galetta, MD, the Philip K. Moskowitz, MD Professor and Chair of Neurology, Department of Neurology, professor, Department of Ophthalmology

Man Receives the First Eye Transplant Plus a New Face. Its a Step Toward One Day Restoring Sight Cherokee Phoenix November 17 -Eduardo D. Rodriguez, MD, DDS, the Helen L. Kimmel Professor of Reconstructive Plastic Surgery, chair, the Hansjrg Wyss Department of Plastic Surgery -Vaidehi S. Dedania, MD, associate professor, Department of Ophthalmology -Steven L. Galetta, MD, the Philip K. Moskowitz, MD Professor and Chair of Neurology, Department of Neurology, professor, Department of Ophthalmology

The Main Symptoms of Mold Exposure, According to Doctors Mens Health November 17 -Lorna E. Thorpe, PhD, MPH, professor, Department of Population Health

Sharon Osbourne Reveals She Weighs Less Than 100 lbs. After Ozempic. Why Dropping Too Much Weight Is a Legitimate Concern. Yahoo! Life November 17 -Priya Jaisinghani, MD, clinical assistant professor, Department of Medicine, Division of Endocrinology, Diabetes, & Metabolism

Cancer Deaths Among Kids and Teens Fell 24% Over Past Two Decades, CDC Reports: Better Treatments FOX News November 19 -Marc K. Siegel, MD, clinical professor, Department of Medicine, Division of General Internal Medicine

At Least 20 Potentially Infectious Agents in Illegal Underground Lab, According to CDC FOX News November 17 -Marc K. Siegel, MD, clinical professor, Department of Medicine, Division of General Internal Medicine

Sam Altman Sought Funding for New AI Ventures Before OpenAI Ouster: Report FOX News November 19 -Marc K. Siegel, MD, clinical professor, Department of Medicine, Division of General Internal Medicine

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NYU Langone Health in the NewsMonday, November 20, 2023 - NYU Langone Health

WEDNESDAY, Nov. 22, 2023 (HealthDay News) -- Ten hours of acoustic stimulation improves postconcussive symptoms, but linking tones to brain electrical activity does not reduce symptoms more than random tones, according to a study published online Nov. 22 in the Annals of Clinical and Translational Neurology.

Wesley R. Cole, Ph.D., from the University of North Carolina in Chapel Hill, and colleagues randomly assigned 106 military service members, veterans, or their spouses with persistent symptoms (Neurobehavioral Symptom Inventory [NSI] Score 23) after mild traumatic brain injury three months to 10 years ago to receive 10 sessions of engineered tones linked to brainwaves (LB; intervention) or random engineered tones not linked to brainwaves (NL; sham control).

The researchers observed a reduction in NSI from 41.0 at baseline to 27.2 after, among all study participants, with gains largely sustained at three and six months (31.2 and 28.4, respectively). No significant differences were seen between the LB and NL groups. Patterns were similar for the posttraumatic stress disorder checklist for the Diagnostic and Statistical Manual of Mental Disorders fifth edition and for depression symptom severity. No difference was seen in heart rate variability between the groups.

"The results of this study do not suggest that in a primarily active duty group with postconcussive symptoms, listening to acoustic stimulation based on one's own brain electrical activity reduces symptoms, or improves brain function or heart rate variability, more than randomly generated, computer engineered acoustic stimulation," the authors write.

One author disclosed being employed by Brain State Technologies.

Abstract/Full Text

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Acoustic Stimulation Reduces Postconcussive Symptoms - HealthDay

By: Lisette Hilton | November 21, 2023 | 5 min. read| Share

University of Miami Miller School of Medicine researchers have developed a protocol to reach the gait-promoting center of the brain for patients with this type of walking difficulty.

Doris Gamelin was years into her Parkinsons disease diagnosis when she began to experience a strange symptom.

Id be walking and everything would be OK. Then Id come to a door frame or a change in flooring, like from a wooden to a tile floor, and I would freeze, Gamelin said. I couldnt walk. And Id fall. It happened quite frequently.

Gamelin broke her hip, arm and chipped a tooth because of those falls.

The 76-year-old said she was desperate for answers. She found them at the University of Miami School of Medicine, where researchers are pioneering a new use of deep brain stimulation to relieve Parkinsons patients of this debilitating symptom.

Gamelins symptoms, called freezing of gait, occur in some with Parkinsons, according to Corneliu Luca, M.D., Ph.D., associate professor of clinicalneurology, co-director of the Movement Disorders Fellowship, and director of the Deep Brain StimulationProgram at the Miller School.

Its the inability to initiate walking. Your feet get stuck to the ground and when they try to make a step, they cant, Dr. Luca said. This is very difficult to treat and many in neurology dont know what to do about it.

But laboratory research by Brian R. Noga, Ph.D., research professor at the Miller Schools Miami Project to Cure Paralysis, suggested deep brain stimulation had a place in treating patients like Gamelin. Dr. Noga and colleagues found that they could use deep brain stimulation to target the gait-promoting center in the mid-brain to improve their ability to walk.

Jonathan R. Jagid, M.D., professor of clinical neurological surgery, neurology, orthopedics and rehabilitation at the University of Miami Health System, The Miami Project and UM/Jackson Memorial Hospital, has since helped to develop an algorithm to reach that specific part of the brain in humans. He also has performed deep brain stimulation for freezing of gait in three people, including Gamelin.

Gamelin said the surgery, from which she went home the next day, gave her many months of walking free of the dangerous freezes. And she didnt fall. Gamelins Parkinsons has deteriorated in other ways in the last few months, but she said she doesnt regret having the surgery.

Before the surgery, they had me walk around a circle and it took me 22 steps. After surgery it took me five steps, Gamelin said. Done by experienced surgeons like those at the University of Miami, the surgery does what its supposed to do.

Dr. Jagid said he believes the Miller School is one of the few facilities in the U.S. targeting this area of the brain stem with deep brain stimulation, using a directional lead.

Other attempts have not used new technology, Dr. Jagid said. Directional leads steer currents, which gives an advantage when targeting this area of the brain stem.

Results, according to Dr. Luca, have been spectacular.

People who have an inability to walk, as soon as you turn on the electricity you are able to make steps, turn around, etc., Dr. Luca said.

Freezing of gait is a unique symptom complex of Parkinsons. A subset of patients who have dopamine-resistant freezing of gait.

That means that their Parkinsons medication helps with a lot of the symptoms, but it does not help with their significant and profound freezing of gait, Dr. Jagid said. Before this, you really couldnt do much for dopamine-resistant freezing of gait because the commonly used medication for Parkinsons was not working for that particular symptom in that particular subset of patients.

Deep brain stimulation has long been used to address other types of debilitating neurological symptoms, from tremors to slow movement and stiffness.

The standard approach to deep brain stimulation, however, will not help freezing of gait.

The difficulty with this particular area of the brain stem is that the target that were trying to put this electrode into is not visible on imaging. We at the university have developed a protocol that allows us to try and hone in on where this small nucleus is in the brain stem, Dr. Jagid said. The way we do it is by using fiber tracking to get a particular type of image that allows us to see and target some of the deep circuitry of the brain.

Dr. Jagid said he and Dr. Luca continue to accrue patients who are candidates for the surgery to better determine consistency of results and the procedures safety.

If the safety record is proven and we see a few more patients with the same kind of results, the next step would be to submit grants for funding and then potentially carry this onto a larger study, Dr. Jagid said.

While standard deep brain stimulation has about 25 years of safety and effectiveness data and is FDA-approved, there is still much to learn about this specific approach and indication.

We are following these patients over time. We dont have long-term data but, so far, the results are lasting. That is the hope that its more than a temporary solution, Dr. Luca said.

Tags: Deep Brain Stimulation Program, Department of Neurology, Dr. Corneliu Luca, Dr. Jonathan Jagid, Miami Project to Cure Paralysis, Parkinson's disease

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Deep Brain Stimulation for Freezing of Gait in Parkinson's Disease ... - University of Miami

Newly published in the journal of JCI insight, findings from a study showed that stimulation in a specific area of the amygdala brain region produced a persistent loss of breathing that continued following the end of a seizure among patients with epilepsy.1 These findings provide new insights into the mechanisms that underlie the probable cause of sudden unexpected death in epilepsy (SUDEP) which may help uncover preventative treatments and identify those most at risk of it.2

In the study, investigators observed that seizures that originated in the amygdala caused postictal apnea and identified a distinct region in the amygdala where electrical stimulation was sufficient to reproduce prolonged breathing loss continuing well after stimulation. Among 20 participants, 5 patients developed postictal apnea following the stimulation of the amygdala region. According to the authors, this suggested that some individuals with uncontrolled seizures may be more prone to the condition than other patients with epilepsy.1

"The importance of our latest work is immense. As you may know, the majority of SUDEP cases are due to loss of breathing that occurs after a seizure ends (postictally)," senior author Brian Dlouhy, MD, associate professor of neurosurgery and pediatrics at the University of Iowa, told NeurologyLive. We have now identified a focal site in the amygdala that not only causes ictal apnea but also persistent and prolonged postictal apnea following amygdala seizures. We also identify novel connections between this site and the brainstem critical for chemosensation using novel intraoperative experiments and electrical stimulation concurrent with blood-oxygen-level-dependent (BOLD) functional MRI (fMRI)."

READ MORE: Intermittent Use of Midazolam Shows Continued Favorable Profile in Seizure Clusters

Investigators enrolled 12 adults and 8 children with epilepsy that could not be managed with medications, and who also were undergoing intracranial electroencephalography (iEEG) to control their seizures. Seizures were induced in the participants by the researchers using direct electrical stimulation under medical supervision to assess forebrain control of breathing and apnea. The authors then used a technique combining electrical stimulation with fMRI to recognize any connections between the amygdala site and the brainstem region that could be critical for sensing changes in blood CO2 levels and breathing control.

These new findings are a critical step in developing our understanding of what causes SUDEP and in the development of ways in which to identify those individuals at highest risk and ways to prevent SUDEP, Vicky Whittemore, PhD, program director at the National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health, which contributed funding for the study, said in a statement.2

In the patients who experienced postictal apnea with the stimulation, the persistent apnea was resistant to rising CO2 levels and air hunger failed to occur. Based on this finding, the authors suggested that postictal apnea impaired CO2 chemosensitivity among the patients. Additionally, investigators observed that amygdala stimulation altered BOLD activity in the pons and medulla and in the ventral insula when combining electrical stimulation with functional MRI. Overall, the authors noted that additional research is required to confirm these results on the role of the amygdala in breathing suppression and its involvement in SUDEP.3

"We have identified a focal site in the amygdala that is critical for postictal apnea and possibly SUDEP. We may be able to identify patients at highest risk of SUDEP and we may now have a potential therapeutic target to prevent SUDEP," Dlouhy, also a member of the Iowa Neuroscience Institute, told. "With this work along with some upcoming and potentially groundbreaking discoveries we will be submitting soon for publication, we believe we are at the precipice for understanding amygdala's role in all of breathing, SUDEP, and interoception/fear/anxiety, and possibly a future clinical trialfor SUDEP in epilepsy patients at the highest risk of SUDEP."

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Study Reveals Association Between Amygdala Region and ... - Neurology Live

After the initial data was presented earlier this year, investigators have published full findings of the phase 3 PRODROME study (NCT04492020) demonstrating ubrogepants (Ubrelvy; AbbVie) positive impact on migraine during the prodrome phase in The Lancet.

Led by David W. Dodick, MD, professor of neurology at Mayo Clinic Scottsdale, PRODROME was the first large placebo-controlled trial evaluating the efficacy of an acute treatment administered during the prodrome. At the conclusion of the trial, absence of moderate or severe headache within 24 hours after initiating treatment occurred in 46% (190 of 418) of qualifying prodrome events that had been treated with ubrogepant compared with 29% (121 of 423) of events treated with placebo (OR, 2.09; 95% CI, 1.63-2.69; P <.0001).

"As a neurologist, I have many patients who can describe the premonitory, or prodrome, symptoms of their migraine attacks, and previously we have not had adequate data for treatment options during this earliest phase, Peter J. Goadsby, MD, PhD, FRS, neurologist and professor at Kings College London, said in a statement.1 "These new data speak directly to a gap in migraine treatment and the option to use ubrogepant."

Conducted between April 2020 and April 2022, 518 participants aged 18-75 years with at least 1-year history of migraine with or without aura were randomly assigned to double-blind crossover treatment. Patients were split 1:1 to either placebo to treat the first qualifying prodrome event and ubrogepant 100 mg to treat the second qualifying prodrome event or to receive ubrogepant 100 mg to treat the first qualifying prodrome event and placebo to treat the second. Those who gave interventions and assessed outcomes were masked to group assignment during the study.

READ MORE: Nerivio Neuromodulation Therapy Demonstrates Continued Efficacy, Safety At 1 Year

The safety population included 480 participants and the modified intent-to-treat (mITT) population included 477 participants, most of which were female (88%). Patients underwent a 60-day screening period followed by the 60-day double-blind portion. In total, 85% (n = 438) of patients the total sample completed the trial, with failure to treat 2 qualifying prodrome events (10%) within 60 days as the most common reason for discontinuation.

Within 48 hours after initiating treatment, the absence of moderate or severe headache was achieved in 41% (159 of 391) of qualifying prodrome events treated with ubrogepant 100 mg vs 25% (100 of 407) of qualifying prodrome events that were treated with placebo (OR, 2.13; 95% CI, 1.63-2.78; P <.0001). During 24 hours after treatment, more participants had no disability, able to function normally, status after a prodrome event with ubrogepant 100 mg than on placebo (OR, 1.66; 95% CI, 1.40-1.96; P <.0001).

"Migraine impacts nearly 40 million Americans and is a highly debilitating disease that can cause people to miss work, and time with friends and family. For patients who are able to identify prodromal symptoms, the ability to treat a migraine attack before the headache phase creates an opportunity to stop migraine attacks before they become fully debilitating," Dawn Carlson, vice president of Neuroscience Development at AbbVie, said in a statement.1 "These data published inThe Lancetdemonstrate the important role of UBRELVY in treating migraine attacks early and reducing the overall burden of a migraine attack."

Between the 2 groups, adverse events (AEs) occurring 48 hours after study-drug administration were found in 17% (77 of 456) and 12% (55 of 462) of qualifying prodrome events for those treated with ubrogepant and placebo, respectively. In the study, nausea (ubrogepant: 5%; placebo: 3%), fatigue (3% vs 2%), dizziness (2% vs 3%), and somnolence (2% vs 11%) were reported as the most common AEs observed.

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Phase 3 PRODROME Study Highlights Ubrogepant's Impact on ... - Neurology Live

TUESDAY, Dec. 26, 2023 (HealthDay News) -- Are you necessarily at higher risk of Alzheimer's disease just because you're 80, and not 75? New research shows it's more complex than that.

The findings suggest that it's the pace of buildup in the brain of Alzheimer's-linked amyloid protein plaques that matters most, not age.

Our findings are consistent with studies showing that the amyloid accumulation in the brain takes decades to develop," said study lead author Dr. Oscar Lopez, a professor of neurology at the University of Pittsburgh.

His team's findings were published Dec. 22 in the journal Neurology.

Neuroscientists have long known that the slow but steady accumulation of amyloid-beta protein plaques within brain tissue is a hallmark of Alzheimer's disease, although whether it actually causes the illness is still debated.

Rates of dementia do rise with advancing age, but is age alone the key factor?

To find out, Lopez' team examined amyloid buildup in the brains of 94 people who were 85 at the time they enrolled in the study. All were tracked for 11 years or until they died, and all received two PET scans of their brains during that time.

The researchers compared levels of amyloid buildup seen in those scans to those seen in scans from a younger group of patients (in their 60s) observed in a prior Australian trial.

As expected, amyloid plaque buildup rose over time, regardless of how much of the protein had infiltrated a participant's brain at the time they joined the Pittsburgh study.

Plaques seemed to accumulate faster among people in their 80s, however, compared to people in their late 60s', Lopez' team reported.

None of the elderly people in Lopez' trial who developed dementia were without some plaque buildup in their brains, confirming its key role in the disease.

Most importantly, when brain plaque buildup began seemed key to how soon dementia set in.

For example, people who were already displaying amyloid buildup in their PET scans at age 80 (when they enrolled in the study) developed dementia two years earlier than folks without such early buildup, the Pittsburgh team found.

Finally, the long-term links between amyloid beta buildup and other brain health indicators was more strongly linked to dementia than just the short-term growth of plaque on its own, Lopez' group added.

That's consistent with other studies, which found that amyloid buildup "takes decades to develop, and occurs in the context of other brain pathologies," Lopez said in a university news release.

Lopez, who also directs Pitts Alzheimer's Disease Research Center, said that "understanding of the timing of the presence of these pathologies will be critical for the implementation of future primary prevention therapies.

More information

Find out more about Alzheimer's disease and the brain at the Alzheimer's Association.

SOURCE: University of Pittsburgh, news release, Dec. 22, 2023

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Brain Plaques Point to Who'll Need Alzheimer's Treatment Most - HealthDay

Note: Each year, we partner with Dr. Amy Shecks students at the North Carolina School of Science and Math to profile some unsung heroes of the Duke research community. This is the second of eight posts.

Meet a star in the realm of academic medicine Dr. Kyle Todd Mitchell!

A man who wears many hats a neurologist with a passion for clinical care, an adventurous researcher, and an Assistant Professor of Neurology at Duke Mitchell finds satisfaction in the variety of work, which keeps him driven and up to date in all the different areas.

Dr. Mitchells educational journey is marked by excellence, including a fellowship at the University of California San Francisco School of Medicine, a Neurology Residency at Washington University School of Medicine, and an M.D. from the Medical College of Georgia. Beyond his professional accolades, he leads an active life, enjoying running, hiking, and family travels for rejuvenation.

Dr. Mitchells fascination with neurology ignited during his exposure to the field in medical school and residency. It was a transformative moment when he witnessed a patient struggling with symptoms experience a sudden and remarkable improvement through deep brain stimulation. This therapy involves the implantation of a small electrode in the brain, offering targeted stimulation to control symptoms and bringing relief to individuals grappling with the challenges of Parkinsons Disease.

You dont see that often in medicine, almost like a light switch, things get better and that really hooked me, he said. The mystery and complexity of the brain further captivated him. Everything comes in as a bit of a mystery, I liked the challenge of how the brain is so complex that you can never master it.

Dr. Mitchells research is on improving deep brain stimulation to alleviate the symptoms of Parkinsons disease, the second most prevalent neurodegenerative disorder, which entails a progressive cognitive decline with no cure. Current medications exhibit fluctuations, leading to tremors and stiffness as they wear off. Deep brain stimulation (DBS), FDA-approved for over 20 years, provides a promising alternative.

Dr. Mitchells work involves creating adaptive algorithms that allow the device to activate when needed and deactivate so it is almost like a thermostat. He envisions a future where biomarkers recorded from stimulators could predict specific neural patterns associated with Parkinsons symptoms, triggering the device accordingly. Dr. Mitchell is optimistic, stating that the technology is very investigational but very promising.

A key aspect of Dr. Mitchells work is its interdisciplinary nature, involving engineers, neurosurgeons, and fellow neurologists. Each member of the team brings a unique expertise to the table, contributing to the collaborative effort required for success. Dr. Mitchell emphasizes, None of us can do this on our own.

Acknowledging the challenges they face, especially when dealing with human subjects, Dr. Mitchell underscores the importance of ensuring research has a high potential for success. However, the most rewarding aspect, according to him, is being able to improve the quality of life for patients and their families affected by debilitating diseases.

Dr. Mitchell has a mindset of constant improvement, emphasizing the improvement of current technologies and pushing the boundaries of innovation.

Its never just one clinical trial we are always thinking how we can do this better, he says.

The pursuit of excellence is not without its challenges, particularly when attempting to improve on already effective technologies. Dr. Mitchell juggles his hats of being an educator, caregiver, and researcher daily. So let us tip our own hats and be inspired by Dr. Mitchells unwavering dedication to positively impact the lives of those affected by neurological disorders.

Guest post by Amy Lei, North Carolina School of Science and Math, Class of 2025.

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Pioneering New Treatments in Deep Brain Stimulation for Parkinson's Disease - Research Blog - Duke University

A five-year project funded by a $3.9 million National Institutes of Health (NIH) research grant will study how team-based palliative care might lead to better health services and ease disparities in Parkinsons disease and disease-related dementia care.

Palliative care focuses on improving the quality of life for individuals with serious illnesses, aiming to address the physical, emotional, and social needs of patients and their families.

But in practice, it can be excessive with inappropriate treatments, of low value, and unequal in the care given patients by their socioeconomic or ethnic status, a project co-investigator noted.

The grant was awarded to UTHealth Houston, the University of Pennsylvania, and other institutions, and includes a $1.9 million subcontract given to Adriana Prez, PhD, a professor in the department of biostatistics and data science with UTHealths School of Public Health in Austin, Texas.

Prez, a project co-investigator and biostatistician, will determine the scope and drivers of unequal and limited value for Parkinsons patients.

The strength of our proposed research is the focus on health equity with the use of Medicare claims which would provide new knowledge that could be used to reduce disparities and improve end-of-life care for vulnerable sub-groups, she said in a university press release.

Parkinsons is caused by the progressive loss of nerve cells in the brain responsible for making dopamine, a chemical these cells use to communicate with one another. Their loss leads to the hallmark motor symptoms of the disease.

But Parkinsons patients also experience nonmotor disease symptoms that include dementia, mood disorders such as depression and anxiety, sleep disturbances, and problems in the autonomic nervous system responsible for involuntary processes like digestion and blood pressure. These also can significantly impact a persons overall well-being and quality of life.

Dementia is reported to develop in about one-third of Parkinsons patients, and like the difficulties with movement and balance that patients experience, it is due to the accumulation in the brain of toxic clumps of the alpha-synuclein protein, called Lewy bodies.

While dementia and other nonmotor symptoms can be given less attention and insufficient treatment, they are the leading causes of hospitalization and institutionalization among patients with these diseases in the U.S., UTHealth noted in the release.

According to Prez, current care models provide many people living with Parkinsons disease or related dementia with excessive services of little value, and are unequal across palliative domains.

Previous research also indicates that women and patients of Hispanic, Asian, or African American ethnicity along with factors guiding healthcare systems and policies are not given needed information and resources.

End-of-life care for people with Parkinsons and related dementia is often substandard and fraught with disparities, Prez said.

To address this, the scientists are conducting a large-scale trial of team-based, neurology-led palliative care in 33 of the 65 academic Movement Disorders Centers across the U.S., a model previously found to be effective at a few of them. The study will compare Medicare data at the involved centers with data collected at the 32 centers not part of this trial.

This model is based on increasing advance care planning, and decreasing acute care for non-motor symptoms, low-value end-of-life care, prescribing of potentially inappropriate medications, and sociodemographic disparities, Prez said.

The grants principal investigator is Allison Wills, MD, an associate professor in the department of neurology at the University of Pennsylvania. Other co-investigators include Benzi Kluger, MD, a professor of neurology at the University of Rochester Medical Center; and Jean Kutner, MD, a professor of internal medicine at the University of Colorados Anschutz Medical Campus.

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Study may improve palliative care for Parkinson's, related dementia - Parkinson's News Today

What once would have been thought of as science fiction has become science fact.

Researchers at UT Southwestern Medical Center in Dallas have developed a device that can isolate blood flow to the brain, keeping it alive and functioning independent from the rest of the body for several hours. The research could lead to advances in understanding how the brain controls functions in the body.

This novel method enables research that focuses on the brain independent of the body, allowing us to answer physiological questions in a way that has never been done, Juan Pascual, M.D., Ph.D., professor of neurology, pediatrics, and physiology, and in the Eugene McDermott Center for Human Growth and Development at UT Southwestern, said in a statement.

Pascual is a member of the Peter ODonnell Jr. Brain Institute at UTSW and holds the Ed and Sue Rose Distinguished Professorship in Neurology and The Once Upon a Time Foundation Professorship in Pediatric Neurologic Diseases.

UTSW said the device was tested using a pig brain model and was described in Scientific Reports.

It could lead to new ways to study the human brain without influence from other bodily functions, UTSW said. Also, it could inform the design of machines for cardiopulmonary bypass that better replicate natural blood flow to the brain.

UTSW said these findings build on earlier research by Pascual and his colleagues.

The brain is master control for a variety of processes, such as regulating heart rate, breathing, and sleep and wake cycles, among other processes. Its function is affected by factors that originate in the body, such as blood sugar, blood pressure, and oxygenation.

Until now, Pascual said there has been no way to separate the brain from the body to study these influences.

UTSW said that in an animal model using anesthesia, researchers redirected the brains blood supply through a pump that maintained or adjusted a range of variables, including blood pressure, volume, temperature, oxygenation, and nutrients.

They discovered that brain activity and other measurements had minimal to no changes over a five-hour period, UTSW said.

Isolating the brain, researchers are able to manipulate inputs to this organ to study how they change brain function without the bodys influence.

Pascual said, for example, the team already has used this system to better understand the effects of hypoglycemia (low blood sugar) in the absence of other factors. Although scientists can induce hypoglycemia by restricting food intake in lab animals or dosing them with insulin, the body can partially compensate for either of these scenarios by altering metabolism, in turn, altering the brain.

UTSW said that in contrast, the new device permits researchers to alter the glucose content directly in blood pumped to the brain.

Cardiopulmonary bypass devices replicate some functions of the heart and lungs, delivering a continuous flow of oxygenated blood throughout the body.

The new device delivers blood using a pulsative flow, much like the human heart, a difference that may prevent brain-related side effects sometimes caused by cardiopulmonary bypass machines, UTSW said.

Pascual said this device has been patented to test its effectiveness for this indication.

Other UTSW researchers who contributed to this study include co-first authors Muhammed Shariff, visiting junior researcher in neurology, and Aksharkumar Dobariya, M.S., graduate student researcher in biomedical engineering; Bret Evers, M.D., Ph.D., assistant professor of pathology and ophthalmology; Ulrike Hoffmann, M.D., Ph.D., assistant professor of anesthesiology and pain management and neurological surgery; Vikram Jakkamsetti, Ph.D., instructor of neurology; Michael Jessen, M.D., professor and chair of cardiovascular and thoracic surgery; Bruce Mickey, M.D., professor emeritus of neurological surgery; Matthias Peltz, M.D., professor of cardiovascular and thoracic surgery and surgical director of cardiac transplant; Cameron Longfellow, perfusionist; Debra Douglass, chief perfusionist; Levi Good, Ph.D., visiting senior researcher in neurology; Gauri Kathote, data scientist in neurology; Gus Angulo, research technician in neurology; Qian Ma, M.D., Ph.D., senior research scientist in neurology; Ronnie Brown, senior research associate in neurological surgery; Misha Dunbar, DVM, senior staff veterinarian; John Shelton, lab manager; and Sourav Patnaik, senior biomedical engineering specialist.

One of the nations top academic medical centers, UT Southwestern integrates pioneering biomedical research with exceptional clinical care and education.

Its faculty members have received six Nobel Prizes and include 26 members of the National Academy of Sciences, 20 members of the National Academy of Medicine, and 14 Howard Hughes Medical Institute Investigators.

The full-time faculty of more than 3,100 is responsible for groundbreaking medical advances and is committed to translating science-driven research quickly to new clinical treatments, UTSW said. Its physicians provide care in more than 80 specialties to more than 120,000 hospitalized patients, more than 360,000 emergency room cases, and oversee nearly 5 million outpatient visits a year.

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Lyda Hill Philanthropies and TAMEST, the Texas Academy of Medicine, Engineering, Science, and Technology, have launched the Hill Prizes. The new program will provide $2.5 million in funding to support the research of Texas scientists.

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Oak Lawn Place will provide "high-quality affordable rental housing" for residents 55 and over, with "a further goal of creating a supportive environment for members of the LGBTQ community and individuals affected by HIV in Dallas," Resource Center has said.

Saturday's event is one of many marking this year's 150th anniversary of Dallas' historic Deep Ellum neighborhood. Saturday's event will begin just down the street from the center beneath an overpass, where attendees will experience the Centers outdoor installation, Invisible Deep Ellum, followed by a live music performance at the new center by the Light Crust Doughboys.

The Fort Worth Local Development Corp. approved $500,000 over two years to create the Fort Worth Entrepreneurship Center. The center will be operated by The DEC Network in the city's Near Southside neighborhood, according to the Fort Worth Report.

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UTSW Researcher Develop Device the Keeps Brain Alive ... - dallasinnovates.com