Gene therapy to improve vision

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Marie McCullough, Inquirer Staff Writer Posted: Monday, March 25, 2013, 3:01 AM

Aaron and Christal Walker live in dread that their daughter will get sick, and in dread that she won't.

Six days ago, Avrey Walker, 9, of Redmond, Ore., became the seventh child to receive an experimental gene therapy for leukemia at Children's Hospital of Philadelphia.

She will soon suffer several days of fevers, nausea, headaches, maybe worse - if the therapy works as it should, marshalling her immune T cells to fight her cancer.

Four of the first five children to undergo treatment and get lab results are cancer-free, according to their families and doctors.

"The doctors said it would take seven to 10 days" for the flulike reaction to begin, Avrey's father said last weekend. "So we're just waiting and watching intently."

The medical and human drama of the T-cell therapy, developed at the University of Pennsylvania, is unfolding in ways the defy the staid traditions of scientific research. On Monday, the New England Journal of Medicine fast-tracked online publication of a paper about Children's first two pediatric patients. But those results - and more - have been out for months, released by the researchers at a conference, or by the families.

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Parents and Children's Hospital researchers await results on an experimental leukemia gene therapy

Topics: babies, dna, ivf

THE possibility that babies could be born with the DNA of three different people is a step closer, after a consultation showed that most Britons would be happy to see the law changed to allow a radical form of gene therapy.

The IVF therapy would help women in danger of passing on mitochondrial disease to their babies - a potentially fatal metabolic disorder.

An exhaustive survey of public attitudes to the replacement of an affected mother's mitochondria - the tiny "power packs" of cells - with those from an egg donor has found widespread support for the technique.

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"We've found that there is broad support for permitting mitochondria replacement to give families at risk of mitochondrial disease the chance of having a healthy child," said Professor Lisa Jardine, the chair of the Human Fertilisation and Embryology Authority (HFEA).

"Although some people have concerns about the safety of these techniques, we found that they trust the experts and the regulator to know when it is appropriate to make them available."

But David King, director of Human Genetics Alert, criticised the HFEA for ignoring the potential risks associated with the technique, which has had only limited testing on laboratory animals and is not medically practised anywhere in the world.

"These techniques go far beyond anything existing in both invasiveness to the embryo and complexity, so it's not surprising they pose serious health risks to the child - risks that the HFEA refuses properly to address," Dr King said.

Mitochondrial replacement involves fusing the egg-cell nucleus of the affected mother with an egg cell from an unaffected donor.

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Public supports gene therapy on IVF embryos

By Anne W. Semmes

Greenwich resident Barbara Netter is no stranger to the extraordinary breakthroughs that gene therapy research has led to in cancer treatment. Her nonprofit foundation, the Alliance for Cancer Gene Therapy (ACGT), has funded some of the cutting-edge research that has allowed the word "cure" to become a part of the cancer conversation.

But when she's able to stand next to Emily Whitehead, a child of seven, free of cancer for nearly a year after being treated with gene therapy that came after ACGT-funded research -- the reality of what those breakthroughs can mean can be quite moving.

"It's really very exciting," says Netter. "At the time of her treatment it was such a breakthrough. She had lymphoblastic leukemia at age 5. When she was 6 she was on a ventilator. They gave her a day to live."

Netter met both Emily and her parents at a celebratory meeting held last Tuesday at the University of Pennsylvania's Perelman School of Medicine, where Dr. Carl June developed ground-breaking treatment of Emily's leukemia that involved genetically engineering her T cells to attack her cancer with a disabled AIDS virus acting as a delivery system. June treated Emily last April when she was near death. After surviving a severe reaction to the treatment, Emily emerged cancer- free.

Now, she is happy -- and healthy.

"We did provide the initial seed money to fund Dr. June's T cell research," said Netter. It was ACGT's initial support of June in 2004 and again in 2008 -- nearly $2 million worth of funding -- that helped his clinical trials get off the ground and lead to his breakthrough treatment.

Emily recently has been joined by another 7-year-old, Maddie Major, who also has been declared cancer-free after receiving June's gene therapy treatment. She is one of four other children with advanced leukemia reported to be in similar treatment.

June has had other successes, as well, with his gene therapy treatment. Eight of 10 adults with chronic leukemia he has treated are in full remission, and that treatment is now being adapted to target solid tumors: prostate, pancreatic, ovarian and breast cancers.

Also present at the UPenn meeting was Dr. Robert Vonderheide, a senior researcher at the university. Vonderheide received funding from Netter's nonprofit in 2003 when he was chosen as an ACGT Young Investigator. The Young Investigator Award funds assistant professors who are conducting independent and innovative cell and gene therapy for cancer research in their own labs. Vonderheide shared with those at Tuesday's meeting of a clinical trial for pancreatic cancer that he hopes will soon take place.

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Funding gene therapy research: Local nonprofit supports efforts to find a cure for cancers

Public release date: 21-Mar-2013 [ | E-mail | Share ]

Contact: Sarah Barth s.barth@elsevier.com 215-239-6087 Elsevier

Philadelphia, PA, March 21, 2013 The April issue of Translational Research examines the progress and outlook of gene therapy research, with a specific focus on the clinical applicability of gene therapy today. Research articles included in the special issue highlight current studies that, after decades of trial and error, may provide evidence for a clear path of treatment and cure for many diseases. There are more than 1,800 genetic disorders known in humans, and only a small fraction of these can be treated and even fewer cured. Some of these disorders are exceedingly rare, others more common. The approach of gene therapy however may be applicable to all.

"The thirteen articles included in this special issue of Translational Research provide critical examples of the tools and practice of gene therapy today. They all focus on clinically meaningful studies that combine patient observations with smart experiments. The authors hope these articles will facilitate conversion of individual and disease-specific insight into a collective understanding of emerging gene transfer platforms and their subsequent translation to the bedside," explained contributing author Dr. Jakub Tolar of the Stem Cell Institute and Pediatric Blood and Marrow Transplant Program at the University of Minnesota, in his introduction to the issue. "The concept of gene therapy for genetic disorders is one of the most appealing in biomedicine because it is aimed at the cause rather than the symptoms of the disease."

Each article of this issue focuses on either a specific condition or a delivery method. Article topics included are: arthritis gene therapy, immunotherapies for type 1 diabetes mellitus, immune responses in liver-directed, lentiviral gene therapy, gene therapy for retinal disease, gene therapy in cystic fibrosis, evaluating risks of insertional mutagenesis by DNA transposons in gene therapy, pluripotent stem cells and gene therapy, gene therapy for hemoglobinopathies: progress and challenge, hemophilia clinical gene therapy-brief review, gene transfer for congestive heart failure, gene therapy for the prevention of vein graft disease, gene therapy for brain tumors, oncolytic virus therapy for cancer, and T cell-based gene therapy of cancer.

With the publication of this special issue, Translational Research identifies a need for clinical trial coordination among researchers worldwide, a focused goal of a world-scale change in medical practice, and real-time data exchange and evaluation, With these elements in place the true potential of gene therapy to treat and cure disease becomes apparent.

###

Notes for Editors

The articles appear in Translational Research, Volume 160, Issue 5 (April 2013), titled "Gene Therapy for Human Disease: Clinical Advances and Challenges," published by Elsevier, now available on ScienceDirect.

Full text of the articles included in the special issue is available to credentialed journalists upon request. Contact Sarah Barth at +1 215 239 6087, s.barth@elsevier.com to obtain copies or to schedule an interview with Dr. Jeffrey Laurence, MD, Editor-in-Chief.

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' Gene Therapy for Human Disease: Clinical Advances and Challenges'

CAMBRIDGE, Mass.--(BUSINESS WIRE)--

bluebird bio, a privately-held biotechnology company focused on gene therapy, today announced the formation of a broad, global strategic collaboration with Celgene Corporation to discover, develop and commercialize novel disease-altering gene therapies in oncology. The collaboration will focus on applying gene therapy technology to genetically modify a patients own T-cells, known as chimeric antigen receptor (CAR) T-cells, to target and destroy cancer cells. The multi-year research and development collaboration has the potential to lead to the development and commercialization of multiple CAR T-cell products. Celgene has an option to license any products resulting from the collaboration after the completion of a Phase 1 clinical study for each such product. bluebird bio will be responsible for research and development activity through Phase 1 studies.

Additionally, Celgene has also entered into a separate strategic collaboration in the CAR T-cell field with the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Childrens Hospital and The Methodist Hospital, Houston, led by Malcolm Brenner, M.D., Ph.D., professor, Department of Molecular and Human Genetics and the director, Center for Cell and Gene Therapy. bluebird bio, Celgene and Dr. Brenners team will work collaboratively to advance and develop existing and new products and programs in the CAR T-cell field.

The genetic manipulation of autologous T-cells is a new frontier in oncology, one that shows early promise in emerging clinical trials, said Tom Daniel, president, research & early development at Celgene. We see strong prospects for this collaboration between Celgene, bluebird bio and Baylor College of Medicines experienced leaders in this emerging field, led by Dr. Brenner, to advance this innovative approach to intractable problems in oncology.

We believe that our recent advances in the industrialization of our gene therapy platform will drive improvements in the potency, purity, efficiency and scalability of our lentiviral gene therapy programs. These advances provide us with an opportunity to apply our platform, intellectual property and know-how to the development of additional product candidates in indications such as CAR T-cells for cancer, stated Nick Leschly, CEO of bluebird bio. Celgene is a global leader in oncology and, combined with Baylors expertise in the CAR T-cell field, we have created a great opportunity to drive innovation in a new and exciting area.

Financial terms of the agreement include an upfront payment and up to $225 million per product in potential option fees and clinical and regulatory milestones. bluebird bio also has the right to participate in the development and commercialization of any licensed products resulting from the collaboration through a 50/50 co-development and profit share in the United States in exchange for a reduction of milestones. Royalties would also be paid in regions where there is no profit share including in the United States if bluebird bio declines to exercise their co-development and profit sharing rights.

The gene therapy products currently in clinical development at bluebird bio for the treatment of childhood cerebral adrenoleukodystrophy, beta-thalassemia and sickle cell disease are independent of this collaboration.

Cowen and Company contributed as a strategic advisor to bluebird bio on this transaction.

About CAR T-Cell Therapy

CAR T-cell therapy represents a promising, emerging approach to treating cancer. Blood is withdrawn from a patient and the T-cells are then extracted from a patient's blood. These cells are then genetically modified to recognize and attack cancer cells and then re-introduced into the patient's blood. The patients genetically modified cells are intended to bind to and kill the target cancer cells.

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bluebird bio Announces Global Strategic Collaboration with Celgene to Advance Gene Therapy in Oncology

BOSTON - A unique new cancer treatment uses gene therapy to induce a cancer-fighting immune response whose intensity can then be controlled with a pill. The combination could help tailor treatment to a patients individual response.

The treatment uses the bodys own cells or tumor cells to produce extra copies of a naturally occurring hormone-like molecule called IL-12, which regulates anticancer immune responses. Last week, Ziopharm Oncology announced a clinical study of the treatment for patients with breast cancer. The company is already testing it in patients with melanoma.

Many researchers have explored techniques that rev up the natural response the body uses to detect and attack cancerous cells (see, for example, Engineering Better Immune Cells and Priming the Body to Tackle Cancer). But controlling the killer cells of the immune system can sometimes be a challenge, as researchers found in the 1990s when cancer patients who were given IL-12 in a clinical trial died from toxic side effects.

IL-12 is a very potent [immune system regulator] and can generate a lot of side effects, says Per Basse, a physician-scientist at the University of Pittsburgh School of Medicine, who studies immune cells and their ability to fight cancer. As a clinician, I would like to be able to dial it up and down so that if it all starts to look not so good, you can stop the process, he says.

To avoid the dangerous side of the molecule, Ziopharms system is designed to control IL-12 with a combined genetic and pharmaceutical switch. A virus is injected into the tumor to deliver the gene for IL-12. The gene starts out in off mode, so it doesnt actually produce any IL-12. To activate the gene, a patient has to take a pill that delivers another molecule. The advantage is that any patient who starts to experience nasty side effects from the IL-12 can stop taking the pill. If things go awry, you have an escape valve, says Ziopharms CEO, Jonathan Lewis.

The key to the inducible system is a version of the receptor that controls molting in arthropods (insects, spiders, and crustaceans), modified so that it determines whether the IL-12 gene is on. The gene for that receptor, which is also delivered into the body by a virus, is always on, but its protein product and thus IL-12 expression is activated by the pill. Ziopharm licensed the control system from Intrexon for use in its oncology treatment.

The inducibility is a great idea, but the trick is getting something that you can get into the tumor, says Ralph Weichselbaum, a cancer researcher at the University of Chicago, who has worked on a cancer therapy induced by radiation. Currently, Ziopharm injects the gene-toting virus directly into patients tumors, but Lewis says the plan is to inject it into muscles in the future. Muscle cells are extremely good protein production factories, he says.

But even injecting the virus into a single tumor has an effect on other tumorsboth in lab animals and in humans. In animal studies, the tumor that receives the injection will at first get bigger because immune cells are accumulating in response to the IL-12. Then it will get smaller and go away, says Lewis. Tumors that received no injection will do the same thinggrow, then shrink, and then disappear. We are seeing similar things in people, says Lewis.

Eventually, the system could be used to deliver multiple genetic treatments at once, says Lewis. With one injection you could be able to control three or four [cancer-fighting] proteins in different ways.

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Cancer gene therapy activated by a pill provides personalized treatment

Patients can turn off an experimental treatment if side effects get too bad.

A unique new cancer treatment uses gene therapy to induce a cancer-fighting immune response whose intensity can then be controlled with a pill. The combination could help tailor treatment to a patients individual response.

The treatment uses the bodys own cells or tumor cells to produce extra copies of a naturally occurring hormone-like molecule called IL-12, which regulates anticancer immune responses. Last week, Ziopharm Oncology announced a clinical study of the treatment for patients with breast cancer. The company is already testing it in patients with melanoma.

Many researchers have explored techniques that rev up the natural response the body uses to detect and attack cancerous cells (see, for example, Engineering Better Immune Cells and Priming the Body to Tackle Cancer). But controlling the killer cells of the immune system can sometimes be a challenge, as researchers found in the 1990s when cancer patients who were given IL-12 in a clinical trial died from toxic side effects.

IL-12 is a very potent [immune system regulator] and can generate a lot of side effects, says Per Basse, a physician-scientist at the University of Pittsburgh School of Medicine, who studies immune cells and their ability to fight cancer. As a clinician, I would like to be able to dial it up and down so that if it all starts to look not so good, you can stop the process, he says.

To avoid the dangerous side of the molecule, Ziopharms system is designed to control IL-12 with a combined genetic and pharmaceutical switch. A virus is injected into the tumor to deliver the gene for IL-12. The gene starts out in off mode, so it doesnt actually produce any IL-12. To activate the gene, a patient has to take a pill that delivers another molecule. The advantage is that any patient who starts to experience nasty side effects from the IL-12 can stop taking the pill. If things go awry, you have an escape valve, says Ziopharms CEO, Jonathan Lewis.

The key to the inducible system is a version of the receptor that controls molting in arthropods (insects, spiders, and crustaceans), modified so that it determines whether the IL-12 gene is on. The gene for that receptor, which is also delivered into the body by a virus, is always on, but its protein product and thus IL-12 expression is activated by the pill. Ziopharm licensed the control system from Intrexon for use in its oncology treatment.

The inducibility is a great idea, but the trick is getting something that you can get into the tumor, says Ralph Weichselbaum, a cancer researcher at the University of Chicago, who has worked on a cancer therapy induced by radiation. Currently, Ziopharm injects the gene-toting virus directly into patients tumors, but Lewis says the plan is to inject it into muscles in the future. Muscle cells are extremely good protein production factories, he says.

But even injecting the virus into a single tumor has an effect on other tumorsboth in lab animals and in humans. In animal studies, the tumor that receives the injection will at first get bigger because immune cells are accumulating in response to the IL-12. Then it will get smaller and go away, says Lewis. Tumors that received no injection will do the same thinggrow, then shrink, and then disappear. We are seeing similar things in people, says Lewis.

Eventually, the system could be used to deliver multiple genetic treatments at once, says Lewis. With one injection you could be able to control three or four [cancer-fighting] proteins in different ways.

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A Cancer Gene Therapy Activated by a Pill

Professor Dinggang Li, M.D. - gene therapy, Gendicine
Professor Dinggang Li md gene therapy Gendicine. Uploaded by Danni ElKher on Mar 14 2013. Itroduction of the hospitals of Phoenix Hosiptal Group Beijing Health Palace Hospital and Beijing Yanhua Phoenix Hospital with Professor Dinggang Li md in front Gene therapy also known as Gendicine has been proven to be very useful in the fight against cancer Scandinavian patients wwwkirurgirejserdk. Plu Mb 69.

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Professor Dinggang Li, M.D. - gene therapy, Gendicine - Video

By Randy Dotinga HealthDay Reporter

THURSDAY, March 14 (HealthDay News) -- In a very early sign of medical progress on the osteoarthritis front, scientists report they've used injections of modified genes to reduce the risk that mice will develop the painful, debilitating condition.

There's no way to know if the gene therapy treatment will help humans, and scientists are far from understanding the treatment's side effects and potential cost. But the findings are more than just good news for mice with creaky joints.

"This work identifies an approach that can make a difference," explained study co-author Dr. Brendan Lee, director of the Rolanette and Berdon Lawrence Bone Disease Program of Texas. "There's a great need for treating and preventing osteoarthritis."

The disease, the most common form of arthritis, appears as your joints deteriorate with aging. It often strikes the hands, knees, neck and hips, causing pain, stiffness and difficulty moving.

Seventy percent of Americans aged 55 to 70 struggle with osteoarthritis, for which there is no cure. Doctors try to treat the pain and improve the ability of patients to move, Lee said, and may turn to joint replacement surgeries in advanced cases.

In the new study, researchers examined a protein that diminishes in people with a rare joint disorder. The protein appears to be crucial to the lubrication of joints.

Researchers injected a gene related to the protein into mice and found that the rodent bodies began producing it. The mice appeared to be resistant -- but not immune -- to damage to the cartilage of joints from injury and aging, Lee said.

There are plenty of caveats.

The research is in mice, not humans; the next step is to test the approach in horses, whose joints are similar to those of people. And the gene therapy doesn't seem to do anything for damage that's already occurred.

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Gene therapy helped mice withstand osteoarthritis

DUBLIN--(BUSINESS WIRE)--

Research and Markets (http://www.researchandmarkets.com/research/2nfn6s/gene_therapy) has announced the addition of Jain PharmaBiotech's new report "Gene Therapy - Technologies, Markets and Companies" to their offering.

Gene therapy can be broadly defined as the transfer of defined genetic material to specific target cells of a patient for the ultimate purpose of preventing or altering a particular disease state. Genes and DNA are now being introduced without the use of vectors and various techniques are being used to modify the function of genes in vivo without gene transfer. If one adds to this the cell therapy particularly with use of genetically modified cells, the scope of gene therapy becomes much broader. Gene therapy can now combined with antisense techniques such as RNA interference (RNAi), further increasing the therapeutic applications. This report takes broad overview of gene therapy and is the most up-to-date presentation from the author on this topic built-up from a series of gene therapy report written by him during the past decade including a textbook of gene therapy and a book on gene therapy companies. This report describes the setbacks of gene therapy and renewed interest in the topic

Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.

Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.

Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2012, over 2030 clinical trials have been completed, are ongoing or have been approved worldwide.A breakdown of these trials is shown according to the areas of application.

Since the death of Jesse Gelsinger in the US following a gene therapy treatment, the FDA has further tightened the regulatory control on gene therapy. A further setback was the reports of leukemia following use of retroviral vectors in successful gene therapy for adenosine deaminase deficiency. Several clinical trials were put on hold and many have resumed now. The report also discusses the adverse effects of various vectors, safety regulations and ethical aspects of gene therapy including germline gene therapy.

The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2012-2022. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets

Profiles of 179 companies involved in developing gene therapy are presented along with 203 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade. These companies have been followed up since they were the topic of a book on gene therapy companies by the author of this report. John Wiley & Sons published the book in 2000 and from 2001 to 2003, updated versions of these companies (approximately 160 at mid-2003) were available on Wiley's web site. Since that free service was discontinued and the rights reverted to the author, this report remains the only authorized continuously updated version on gene therapy companies.

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Research and Markets: Gene Therapy : Technologies, Markets and Companies 2013 Update with Added Company Profiles

ELK GROVE VILLAGE, Ill., March 13, 2013 (GLOBE NEWSWIRE) -- Nationwide Children's Hospital (Columbus, OH) and Families of Spinal Muscular Atrophy (Elk Grove Village, IL) announce the award of a multi-million dollar cooperative agreement from the National Institute of Neurological Disorders and Stroke (NINDS) to advance a gene therapy development program for Spinal Muscular Atrophy (SMA).

This three-year multi-million dollar cooperative agreement to Brian Kaspar, PhD, principal investigator in the Center for Gene Therapy at The Research Institute at Nationwide Children's Hospital in the amount of $3,752,462, funds pre-clinical drug development up to the filing of an Investigational New Drug Application (IND) to the Food and Drug Administration (FDA). This agreement represents an innovative collaboration between Government, Advocacy and Academic groups to advance a promising new therapy for SMA.

In May 2012, Families of SMA (FSMA) announced the award of up to $750,000 to Dr. Kaspar. This ongoing award supports the preclinical development of a Central Nervous System (CNS)-delivered gene therapy for SMA. Direct CNS delivery likely allows for less virus to be used, which significantly increases the likelihood that older and larger SMA patients can be treated with gene therapy. With the funding from FSMA, Dr. Kaspar's team initiated studies to jumpstart the research prior to obtaining government and later commercial involvement. This cooperative award from the NINDS will now support advancing the program to the point of human clinical trials. The program will be evaluated using quantitative go/no-go milestones, determined by Nationwide Children's and NINDS.

SMA is an often-fatal genetic disorder resulting from the loss of both copies of the Survival Motor Neuron (SMN1) gene. This causes a chronic deficiency in the production of the SMN protein, which is essential to the proper functioning of the motor neurons in the spinal cord to the control of muscles in the limbs, neck and chest. SMA is typically marked by the deterioration of the muscles that control crawling, walking, swallowing or breathing. There are no approved therapies for the treatment of SMA. Approximately 1 in 6,000 babies born is affected. One in 40 people, or approximately 8 million in the United States, are genetic carriers of the disease.

Gene therapy is an approach to treating diseases by replacing faulty genes. In the case of SMA, the most direct approach for a gene therapy is to replace the mutated SMN1 gene. In the past, the challenge with gene therapy for SMA has been to find a way to deliver the genetic material efficiently to motor neurons. In recent years, Dr. Kaspar's group was the first to demonstrate Adeno-Associated Virus 9 (AAV9) targeted motor neurons effectively. Administration of AAV9-SMN into one day-old SMA mice resulted in increased SMN protein levels in motor neurons, correction of synaptic function, and a significant extension of life span.

"At Families of SMA we are extremely pleased that our initial investment at an early stage of this program has provided the preliminary data to leverage larger funding from the NIH. We feel this grant award is positive validation of the Families of SMA research funding and partnering strategy, as well as for this approach for gene therapy in SMA," said Jill Jarecki PhD, Research Director at Families of SMA. "The Families of SMA funding strategy for preclinical drug development is to invest seed funds to begin early-stage programs for SMA. As programs advance, we look for funding to transition from non-profit to government and commercial sources."

"My research team at Nationwide Children's Hospital is excited to advance this promising cerebrospinal fluid delivery approach of AAV9-SMN to the clinic for SMA patients and we are extremely grateful to FSMA and NINDS for the support of this important work," said Dr. Kaspar, also a faculty member at The Ohio State University College of Medicine. "We stand committed to bring SMA experimental therapeutics to the clinic in the most rapid and safe manner."

"Development of therapies requires collaboration of academics, advocacy, industry, and government--no single party has the resources to do this alone. The collaboration between Dr. Brian Kaspar, Families of SMA, and the NIH is an exciting model in leveraging resources and expertise in the hope of accelerating therapy development for SMA," said Dr. John Porter, PhD, Program Director at the National Institute of Neurological Disorders and Stroke.

About Families of SMA:

Families of SMA is the world's leader focused on funding SMA research to develop a treatment and cure for the disease. The successful results and progress that the organization has delivered, from basic research to drug discovery to clinical trials, provide real hope for families and patients impacted by the disease. The charity has invested over $55 million in research and has been involved in funding half of all the ongoing novel drug programs for SMA. Families of SMA is a nonprofit 501(c)3 organization, with 31 Chapters and 90,000 members and supporters throughout the United States. The organization's work has produced major discoveries, including identification of the underlying cause and a back-up gene for the disease, which provides a clearly defined target for disease altering therapies. The organization is also dedicated to supporting SMA families through networking, information and services and to improving care for all SMA patients. http://www.curesma.org.

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Families of SMA and Nationwide Children's Announce Multi-Million Dollar Award From NINDS to Advance CNS Gene Therapy ...

By Lisa Raffensperger | March 13, 2013 1:22 pm

Most people who live to old age will suffer from arthritis. The conditions prevalence is growing alongside a graying world population.

However the only treatments at the moment address the symptoms rather than the causethe loss of cartilage in joints. Joint replacement is a last-ditch solution for some sufferers. Now a gene therapy approach has demonstrated promise in staving off arthritis in mice, opening the door to human testing.

The inspiration for the research came from studying children with a genetic form of arthritis that strikes early. These children are deficient in the gene for a protein called lubricin. Lubricin is thought to act as a lubricant between the bones in a joint.

Since a lack of lubricin caused arthritis, researchers thought perhaps additional lubricin could stave it off.

They tested this hypothesis by creating a strain of mice with an additional lubricin gene in their DNA. When these mice suffered an injury to their knees they didnt develop injury-induced arthritis. Inspection of the mices joints found that their cartilage resembled mice whod never been injured in the first place. Non-modified mice, on the other hand, had symptoms of arthritis just a month after injury.

Whats more, as the mice that made extra lubricin aged, their cartilage stayed youthful. That suggests the protein may protect against both common forms of arthritis: injury-related and age-related.

The treatment also works if the replacement genes are injected right into the joint itself, the researchers report in Science Translational Medicine today. Its delivery to human patients, then, could be similar to the injection of joint lubricants that some arthritis sufferers currently rely on.

However no gene therapies are currently approved by the FDA for human treatment, so this research will likely stay in the lab for some time yet.

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Gene Therapy Could Prevent Arthritis

Marie McCullough, Inquirer Staff Writer Posted: Sunday, March 10, 2013, 5:22 AM

Still groggy from painkillers, Maddie Major, 7, clutched her stuffed Pooh Bear and laid her head on her father's shoulder as he carried her to the hospital cafeteria.

Maddie, dad Tim, mom Robyn, and big sister Candace spent that February morning at Children's Hospital of Philadelphia, where a doctor extracted samples of the child's spinal fluid and bone marrow.

In a few days, the biopsies would reveal whether Maddie's leukemia had been wiped out by an experimental gene therapy made from her own white blood cells - crucial disease fighters called T cells.

Medical science has been trying for 40 years to harness the immune system to cure cancer, or at least turn it into a docile chronic disease. Excitement has invariably been followed by disappointment.

But after 30 months of testing in more than a dozen adults and children - patients with no conventional options left - worldwide excitement over the T cell therapy's unprecedented power continues to build. The treatment, developed at the University of Pennsylvania, has eradicated advanced blood cancers in mere weeks, and is being adapted to attack solid tumors including prostate, pancreatic, ovarian, and breast cancer.

Maddie's parents, who live in La Plata, Md., marveled that the unique therapy was a cakewalk compared with what she has been through since her diagnosis at age 3.

She has had thousands of doses of toxic chemotherapy. Head-to-toe radiation. Hundreds of blood transfusions. Life-threatening infections in her kidneys, liver, and brain. Months on life support in intensive care. An experimental cell therapy at the National Institutes of Health.

And still, "the beast," as her parents call Maddie's acute lymphoblastic leukemia, would not stay away.

During lunch in the cafeteria, Robyn Major said she was optimistic about the T cell therapy. She did not elaborate because Maddie - perked up and chowing down on pizza, spaghetti, and Fritos - knows more than a 7-year-old should about the limits of modern medicine.

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Pinning their hopes on a Penn gene therapy

Mesothelioma Law Firm Baron and Budd Repeats ADAO Platinum Sponsorship to Support Advocacy for Accelerated Breakthroughs

DALLAS (PRWEB) March 05, 2013

Currently, gene therapy is still one of the treatment strategies under investigation, but accumulating clinical data suggest that it can produce anti-tumor effects which have not been achieved by other therapies, Japanese researchers at Chiba University in Chiba, Japan, write. Mesothelioma is obviously one of the target tumors for gene therapy, and in fact several clinical studies are now in progress. Gene therapy currently remains an experimental approach for mesothelioma treatments, but the preceding clinical trials provided many points to be considered for the future application of gene therapy (BioMed Research International, Volume 2013, A Potential Therapeutic Strategy for Malignant Mesothelioma with Gene Medicine).

The National Cancer Institute (NCI) defines gene therapy as a type of medicine whereby recoded genetic material is inserted via generally harmless laboratory viruses into the bodys cells to combat cancer and other serious diseases. Its aim is to repair or reprogram faulty DNA coding. Each of the bodys cells contains a core of molecules called genes responsible for communicating to cells the instructions they need to thrive. When a carcinogen such as asbestos enters the bodys system, it sometimes causes genetic damage to DNA coding. Next, the production of certain defense proteins, vital to healthy cells, may be disrupted. When gene mutations prevent these proteins from immunizing healthy tissue, cells may begin haphazardly dividing like wildfire, finally warping into malignant tumor formations. Recoded gene copies would foreseeably inhibit or reverse mutant cancerous cell division.

Gene therapy is especially ideal for treating mesothelioma because mesothelioma develops within a closed cavity (the pleura cavity separating the inner and outer linings of the lungs) and remains localized within this cavity until it progresses to the terminal stage. Clinical trials underway in Europe and Asia corroborate the ease and safety of local intrapleural injections, emphasizing their potential to induce anti-tumor immune responses.

On March 22-24, the Ninth International Asbestos Disease Awareness Organization (ADAO) Asbestos Awareness Conference focuses on The Asbestos Crisis: New Trends in Prevention and Treatment, with a distinguished roster of keynote speakers scheduled to discuss cutting-edge medical technology such as gene therapy, as well as mesothelioma patient advocacy, prevention and a global asbestos ban. With strong support from national mesothelioma law firm Baron and Budd s second consecutive platinum sponsorship, ADAO represents one of the worlds most accredited nonprofit organizations for mesothelioma patient advocacy and asbestos awareness. Likewise, Baron and Budds mesothelioma attorneys have fought for 35 years to protect the rights of those affected by asbestos exposure.

For more on ADAO and the ninth international conference, visit http://www.asbestosdiseaseawareness.org.

If you or someone you know has been diagnosed with mesothelioma, visit Mesothelioma News at http://www.mesotheliomanews.com to learn more about your options. Mesothelioma News is a dedicated website underwritten by the national mesothelioma law firm of Baron & Budd.

About Baron & Budd, P.C.

The national mesothelioma law firm of Baron & Budd, P.C. has a more than 30-year history of Protecting Whats Right for asbestos sufferers and their families. As one of the first law firms to successfully litigate an asbestos lawsuit, Baron & Budd continues to actively represent veterans, industry workers and others who are suffering as a result of exposure to asbestos. Baron & Budd achieved the largest mesothelioma verdict ever in the state of Texas, a $55 million verdict for an asbestos sufferer and his family in El Paso, Texas. Contact Baron and Budd at 1.866.855.1229 for additional information on mesothelioma treatments, mesothelioma cancer doctors and treatment centers and mesothelioma attorneys.

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Researchers, FDA Evaluating Experimental Gene Therapy Procedures for Mesothelioma Treatment

STAMFORD, Conn.--(BUSINESS WIRE)--

The 2012 "Young Investigator" grant recipients have been announced by Alliance for Cancer Gene Therapy, Inc. (ACGT), including its first international awardee.

ACGT (www.acgtfoundation.org) is the nations only not-for-profit exclusively dedicated to cancer cell and gene therapy treatments for all types of cancer. 100% of contributions go directly to research, and fund grants with leading scientists in the U.S. and Canada. ACGT has funded 41 grants since its founding in 2001 by Barbara Netter and her late husband, Edward, to conduct and accelerate critically needed innovative research for all types of cancer.

The ACGT Young Investigator Award funds assistant professors on the tenure track who are conducting independent and innovative cell and gene therapy for cancer research in their own dedicated lab. ACGT grants are typically the first they have received, which later attract additional funding and are critical in helping Young Investigators establish their independence.

ACGT's Board approved two 2012 Young Investigator Awards, pending final contract approval. The award to Alexander Stegh, PhD, Assistant Professor, Neurology, at Northwestern University, Feinberg School of Medicine, will fund a research study into a potential new treatment for brain cancer. Stegh's study seeks to better understand metabolic vulnerabilities to the most common and aggressive malignant primary brain tumor in humans, glioblastoma (GBM), and aims to establish more effective methods of attacking and destroying a cancer that has been particularly treatment resistant.

Douglas Mahoney, PhD, Assistant Professor, Department of Microbiology, Immunology and Infectious Disease, at the University of Calgary, Canada, is ACGTs first international grantee. His study focuses on oncolytic virus therapy, which, unlike conventional drugs, orchestrates tumor cell death in multiple ways, simultaneously. Virus-based treatment is considered among the most promising gene therapy techniques. Dr. Mahoney and his team will work to engineer next-generation and virus combinations to break through treatment barriers. Although Dr. Mahoneys initial research was on breast cancer, the technique can be used against many cancers.

"It is discouraging to see federal cutbacks in cancer research funding," said Barbara Netter, ACGTs President. "ACGT is stepping into the breach to boost funding for promising research that can deliver cancer cell and gene therapy discoveries like the recently announced breakthrough leukemia treatments pioneered by Dr. Carl June at the University of Pennsylvania, one of ACGTs first Clinical Translation grantees."

ACGT has awarded 27 grants to Young Investigators and 14 grants to Clinical Investigators totaling $23.7 million funding innovative basic research and clinical translation. ACGTs Scientific Advisory Council comprised of 16 renowned physicians and researchers, conducts the rigorous review process. Young Investigator Grants range from $250,000 to $500,000 over a 2-3 year period. Clinical Translational Grants range from $500,000 to $1,000,000. Seventeen ACGT funded research projects have been approved for human clinical trials; 11 of which are underway. To donate, please visit http://www.acgtfoundation.org or call 203.358.8000.

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2012 "Young Investigator" Grant Recipients Announced; Funding Cutting-Edge Cell and Gene Therapy Research for Cancer

RICHMOND, Calif. (TheStreet) -- Sangamo BioSciences (SGMO) is a gene therapy company with a single product in human clinical studies and a market value of more than $500 million. The rest of the company's pipeline is still preclinical, meaning the only testing being done is in test tubes and rats.

Preclinical-stage drug companies don't typically carry market values of $500 million, which makes Sangamo's SB-728 HIV therapy -- the product in phase II studies -- really important. Sangamo won't trade at $10 per share, like it does today, if SB-728 blows up. Without any drugs in human studies, Sangamo's market cap might easily be cut in half or more, which would be appropriate, particularly for a company trying to develop something as challenging as gene therapies.

Keep this perspective in mind when you hear Sangamo executives talk about the potential for SB-728 to be a "functional cure" for HIV. Throwing the words "cure" and "HIV" into the same sentence generates serious buzz -- and rightly so -- because there is no current curative treatment for HIV. Unfortunately, the data presented on SB-728 to date, including Wednesday, do not match the hype.

See if (SGMO) is in our portfolio

It is unrealistic to believe SB-728 will ever become a "functional cure" for HIV because the scientific and regulatory bar for any drug to warrant that label is extremely high. Current HIV medicines aren't curative, but they do drop viral loads to undetectable levels and keep them there basically forever. HIV patients can take a single pill each morning and basically never have to worry about their disease getting worse. These patients will grow old and die of something else before they succumb to AIDS. That's an amazing achievement in a disease that was a certain death sentence 30 years ago.

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Sangamo's HIV Gene Therapy Is A Valuation Prop, Nothing More

OMICS Group-Genetic Syndromes amp; Gene Therapy-7412-3-118
OMICS Group is an Open Access publication model that enables the dissemination of research articles to the global community. Thus, all articles published under Open Access can be accessed by anyone.

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OMICS Group-Genetic Syndromes & Gene Therapy-7412-3-118 - Video

OMICS Group - Genetic Syndromes amp; Gene Therapy-7412-3-119
OMICS Group is an Open Access publication model that enables the dissemination of research articles to the global community. Thus, all articles published under Open Access can be accessed by anyone.

By: OMICSGroup I Articles

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OMICS Group - Genetic Syndromes & Gene Therapy-7412-3-119 - Video

18 - Gene Therapy and Cancer Prevention - Interview with Dr. Veena Rao
For additional information visit http://www.cancerquest.org In this video, Dr. Veena Rao discusses how gene therapy can lead to better cancer prevention. To learn more about cancer and watch additional interviews, please visit the CancerQuest website at http://www.cancerquest.org

By: CancerQuest

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18 - Gene Therapy and Cancer Prevention - Interview with Dr. Veena Rao - Video