ANN ARBOR, Mich.--(BUSINESS WIRE)--

RetroSense Therapeutics, a gene therapy company dedicated to vision restoration, announced completion of a pre-IND meeting with the Center for Biological Evaluation and Research (CBER) division of the FDA that took place on May 22nd 2012 regarding RetroSenses lead biologic, RST-001 for vision restoration in retinal degenerative conditions.

The purpose of the meeting was to obtain CBERs guidance for the clinical path to a Biologics License Application (BLA) for RST-001 in the US, and clarity on the steps required for Investigational New Drug (IND) submission. The discussions included manufacturing criteria, the scope and design of the preclinical studies, and the scope and design of Phase I and IIa clinical trials.

Sean Ainsworth, CEO, noted,"We were quite encouraged by the FDA's feedback and comments and identified nothing that will hinder our path to the clinic. RetroSense remains on schedule to bring RST-001 to the clinic safely and expeditiously.

Peter Francis, MD, PhD, Clinical Director, led the meeting. Dr. Francis stated, It was a successful, productive interaction with the FDA. We were thankful to have representation by Foundation Fighting Blindness, who have been supportive of our novel approach to vision restoration.

We are very excited about the potential for this innovative treatment to restore vision in people who are blind from retinal degenerations, says Stephen Rose, Ph.D., chief research officer, Foundation Fighting Blindness. It is an elegant and powerful approach to overcoming devastating eye diseases.

About RetroSense Therapeutics

RetroSense Therapeutics is a biotechnology company developing a game-changing gene therapy to restore vision in patients suffering from blindness due to retinitis pigmentosa (RP) and advanced dry age-related macular degeneration (advanced dry-AMD). There are currently no FDA approved therapies to improve or restore vision in patients with these retinal degenerative conditions. RetroSense is led by a team of seasoned veterans with deep experience in taking products from the discovery stage through to the clinic. For more information about RetroSense, visit http://www.retro-sense.com/.

About Foundation Fighting Blindness

The Foundation Fighting Blindness is a national non-profit organization driving research that will lead to preventions, treatments and cures for retinitis pigmentosa, macular degeneration, Usher syndrome and the entire spectrum of retinal degenerative diseases that affect more than 10 million Americans. Since 1971, the Foundation has raised over $450 million as the leading non-governmental funder of retinal research. Breakthrough Foundation-funded studies using gene therapy have restored significant vision in children and young adults who were previously blind, paving the way for using this method to treat a variety of retinal degenerative diseases, and proving a cure is in sight. With a network of 50 chapters, the Foundation also provides support, education and resources to affected individuals and their families in communities across the country.

The rest is here:

RetroSense Therapeutics Completes pre-IND Meeting for RST-001

Public release date: 24-May-2012 [ | E-mail | Share ]

Contact: Sarah Jackson press_releases@the-jci.org Journal of Clinical Investigation

Severe combined immunodeficiency is defect in the immune system that results in a loss of the adaptive immune cells known as B cells and T cells. Mutations in several different genes can lead to the development of severe combined immunodeficiency, including mutation of the adenosine deaminase (ADA) gene. Traditional treatment options, such as enzyme replacement therapy, are of limited efficacy, but bone marrow transplant from a compatible donor leads to a better response. A recent clinical trial indicated that gene therapy to insert the correct ADA gene in the patient's own bone marrow cells can also lead to a good response.

However, patients were noted to have defects in B cell tolerance, meaning that some B cells that react to antigens from the body fail to be eliminated, leading to an autoimmune response. Dr. Eric Meffre and colleages at Yale University in New Haven, Connecticut and Alessandro Aiuti in Milan, Italy joined together to better understand why patients developed B cell tolerance problems. They found that loss of the ADA gene directly contributes to B cell tolerance problems and that these defects are mostly corrected after gene therapy. Their results point to a previously unknown role for ADA in B cell response and support the use of gene therapy as an effective treatment option for ADA-deficient severe combined immunodeficiency patients.

###

TITLE:

Defective B cell tolerance in adenosine deaminase deficiency is corrected by gene therapy

AUTHOR CONTACT:

Eric Meffre Yale University School of Medicine, New Haven, CT, USA Phone: 1-203-737-4535; Fax: 1-203-785-7903; E-mail: eric.meffre@yale.edu

ACCOMPANYING COMMENTARY

Here is the original post:

Gene therapy can correct forms of severe combined immunodeficiency

21-05-2012 17:24 More Science Videos Shirts and Stuff Follow us on Twitter Like us on Facebook Medical News Neuroscience News Biotechnology News Hosted by Contributors

Read this article:

FIRST Anti-Aging Gene Therapy (Brainstorm Ep72) - Video

Public release date: 16-May-2012 [ | E-mail | Share ]

Contact: John Pastor jdpastor@ufl.edu 352-273-5815 University of Florida

Using gene transfer techniques pioneered by University of Florida faculty, Taiwanese doctors have restored some movement in four children bedridden with a rare, life-threatening neurological disease.

The first-in-humans achievement may also be helpful for more common diseases such as Parkinson's that involve nerve cell damage caused by lack of a crucial molecule in brain tissue. The results are reported today (May 16) in the journal Science Translational Medicine.

The children in the study, who ranged in age from 4 to 6, inherited a rare disease known as aromatic L-amino acid decarboxylase deficiency, or AADC. Patients with AADC are born without an enzyme that enables the brain to produce the neurotransmitter dopamine. They generally die in early childhood.

In a phase 1 clinical trial led by Paul Wuh-Liang Hwu, M.D., of the National Taiwan University Hospital, surgeons used a delivery vehicle called an adeno-associated virus type 2 vector to transport the AADC gene into localized areas of the brains of three girls and a boy.

Before therapy, the children showed practically no spontaneous movement and their upper eyelids continually drooped. After receiving the corrective gene, the children gradually gained some head movement. Sixteen months afterward, the children's weight had increased, one patient was able to stand and the other three were able to sit up without support.

The study shows gene therapy that targets AADC deficiency is well-tolerated and leads to improved motor development and function, according to co-authors Barry Byrne, M.D., Ph.D., director of UF's Powell Gene Therapy Center, and Richard O. Snyder, Ph.D., director of UF's Center of Excellence for Regenerative Health Biotechnology. Both are members of the UF Genetics Institute.

"The children in this study have the most severe form of inherited movement disorder known, and the only treatments so far have been supportive ones," said Byrne, a pediatric cardiologist and associate chairman of the department of pediatrics in the College of Medicine. "It is gratifying to see it is possible to do something to help them, other than providing feeding tubes and keeping them safe. This absolutely opens the door to the possibility of even earlier treatment of neurological diseases by direct gene transfer, and has implications for Parkinson's disease, ALS and even cognitive diseases such as dementia when caused by gene defects."

The Powell Gene Therapy Center provided expertise to the Taiwanese physicians on treating the patients and engineering the corrective gene that spurs production of the absent AADC enzyme. UF's Center of Excellence for Regenerative Health Biotechnology manufactured the vector, packaging genetic material it received from Taiwan into virus particles that were purified, characterized and tested for sterility and stability before being shipped to the clinic for use in patients.

Continued here:

Children with rare, incurable brain disease improve after gene therapy

Delivering a missing enzyme to the brains of paralyzed children with a rare, life-threatening neurological disease restores movement and builds muscle mass.

An inherited disorder known as aromatic L-amino acid decarboxylase deficiency, or AADC, leaves patients unable to produce the neurotransmitter dopamine, leaving them nearly paralyzed until they die in early childhood. But a new gene therapy, which involves the delivery of the missing L-amino acid decarboxylase enzyme that converts the chemical precursor L-DOPA to dopamine, helped four Taiwanese children, aged 4 to 6 years, move their heads and sit up on their own, according to a study published today (May 16) in Science Translational Medicine.

The children in this study have the most severe form of inherited movement disorder known, and the only treatments so far have been supportive ones, pediatric cardiologist Barry Byrne, director of the University of Floridas Powell Gene Therapy Center, said in a press release. It is gratifying to see it is possible to do something to help them, other than providing feeding tubes and keeping them safe. This absolutely opens the door to the possibility of even earlier treatment of neurological diseases by direct gene transfer, and has implications for Parkinsons disease, ALS, and even cognitive diseases such as dementia when caused by gene defects.

In the phase I trial led by Wuh-Liang Hwu of the National Taiwan University Hospital, doctors injected an adeno-associated virus loaded up with a good copy of the AADC gene into the childrens putamen, a part of the brain where the enzyme works to make dopamine. Within 16 months of the surgery, all the patients showed increased head movements, higher weight, and were able to sit up without assistance. One patient was even able to stand. In the months that followed, the patients continued to gain weight, and scored higher on cognition and motor development tests. Their parents also said that the children slept better and had improved eye coordination and emotional stability.

Given the positive results, the doctors plan to treat eight more childrenfour in Taiwan and four in the United States, Byrne said.

AADC is not the only disease currently being targeted with gene therapy. Recent successes in treating hemophilia B, numerous cancers, and a variety of blindness disorders are stirring excitement around this once-embattled field. Look out for the feature story, Targeting DNA, in the June issue of The Scientist to learn more about the ways that gene therapy could change the face of medicine.

See the original post here:

Gene Therapy for Brain Disease

33054641 story Posted by Soulskill on Tuesday May 15, @08:17PM from the boosterspice-before-i-get-old-please dept. Grond writes "ScienceDaily reports, 'Researchers at the Spanish National Cancer Research Centre have demonstrated that the mouse lifespan can be extended by the application in adult life of a single treatment acting directly on the animal's genes. Mice treated at the age of one lived longer by 24% on average (PDF), and those treated at the age of two, by 13%. The therapy, furthermore, produced an appreciable improvement in the animals' health, delaying the onset of age-related diseases like osteoporosis and insulin resistance and achieving improved readings on aging indicators like neuromuscular coordination.' Notably, the therapy did not cause an increase in the incidence of cancer." You may like to read: Post

I can resist anything but temptation.

Working...

Read the original:

Gene Therapy Extends Mouse Lifespan

ScienceDaily (May 14, 2012) A new study consisting of inducing cells to express telomerase, the enzyme which -- metaphorically -- slows down the biological clock -- was successful. The research provides a "proof-of-principle" that this "feasible and safe" approach can effectively "improve health span."

A number of studies have shown that it is possible to lengthen the average life of individuals of many species, including mammals, by acting on specific genes. To date, however, this has meant altering the animals' genes permanently from the embryonic stage -- an approach impracticable in humans. Researchers at the Spanish National Cancer Research Centre (CNIO), led by its director Maria Blasco, have demonstrated that the mouse lifespan can be extended by the application in adult life of a single treatment acting directly on the animal's genes. And they have done so using gene therapy, a strategy never before employed to combat aging. The therapy has been found to be safe and effective in mice.

The results were recently published in the journal EMBO Molecular Medicine. The CNIO team, in collaboration with Eduard Ayuso and Fatima Bosch of the Centre of Animal Biotechnology and Gene Therapy at the Universitat Autonoma de Barcelona (UAB), treated adult (one-year-old) and aged (two-year-old) mice, with the gene therapy delivering a "rejuvenating" effect in both cases, according to the authors.

Mice treated at the age of one lived longer by 24% on average, and those treated at the age of two, by 13%. The therapy, furthermore, produced an appreciable improvement in the animals' health, delaying the onset of age-related diseases -- like osteoporosis and insulin resistance -- and achieving improved readings on aging indicators like neuromuscular coordination.

The gene therapy consisted of treating the animals with a DNA-modified virus, the viral genes having been replaced by those of the telomerase enzyme, with a key role in aging. Telomerase repairs the extreme ends or tips of chromosomes, known as telomeres, and in doing so slows the cell's and therefore the body's biological clock. When the animal is infected, the virus acts as a vehicle depositing the telomerase gene in the cells.

This study "shows that it is possible to develop a telomerase-based anti-aging gene therapy without increasing the incidence of cancer," the authors affirm. "Aged organisms accumulate damage in their DNA due to telomere shortening, [this study] finds that a gene therapy based on telomerase production can repair or delay this kind of damage," they add.

'Resetting' the biological clock

Telomeres are the caps that protect the end of chromosomes, but they cannot do so indefinitely: each time the cell divides the telomeres get shorter, until they are so short that they lose all functionality. The cell, as a result, stops dividing and ages or dies. Telomerase gets around this by preventing telomeres from shortening or even rebuilding them. What it does, in essence, is stop or reset the cell's biological clock.

But in most cells the telomerase gene is only active before birth; the cells of an adult organism, with few exceptions, have no telomerase. The exceptions in question are adult stem cells and cancer cells, which divide limitlessly and are therefore immortal -- in fact several studies have shown that telomerase expression is the key to the immortality of tumour cells.

It is precisely this risk of promoting tumour development that has set back the investigation of telomerase-based anti-aging therapies.

Read more from the original source:

First gene therapy successful against aging-associated decline: Mouse lifespan extended up to 24% with a single ...

Researchers in Spain may have discovered the secret to a longer life, a new study says.

Go here to read the rest:

Gene therapy may extend life: Study

click here to continue to article cliquez ici pour lire l'article weiter zum Artikel clicca qui per visualizzare l'articolo weiter zum Artikel ir a la noticia klik hier om door te gaan naar het artikel Yazya devam etmek iin tklayn Tovbb a cikkre

See the original post:

Gene therapy dramatically extends mouse lifespan

Washington, May 15 (ANI): Scientists have successfully extended the lifespan of mice using gene therapy, a strategy never before employed to combat ageing.

View post:

Gene therapy extends mouse lifespan by 24 pc

ScienceDaily (May 11, 2012) Regenerating sensory hair cells, which produce electrical signals in response to vibrations within the inner ear, could form the basis for treating age- or trauma-related hearing loss. One way to do this could be with gene therapy that drives new sensory hair cells to grow.

Researchers at Emory University School of Medicine have shown that introducing a gene called Atoh1 into the cochleae of young mice can induce the formation of extra sensory hair cells.

Their results show the potential of a gene therapy approach, but also demonstrate its current limitations. The extra hair cells produce electrical signals like normal hair cells and connect with neurons. However, after the mice are two weeks old, which is before puberty, inducing Atoh1 has little effect. This suggests that an analogous treatment in adult humans would also not be effective by itself.

The findings were published May 9 in the Journal of Neuroscience.

"We've shown that hair cell regeneration is possible in principle," says Ping Chen, PhD, associate professor of cell biology at Emory University School of Medicine. "In this paper, we have identified which cells are capable of becoming hair cells under the influence of Atoh1, and we show that there are strong age-dependent limitations on the effects of Atoh1 by itself."

The first author of the paper, Michael Kelly, now a postdoctoral fellow at the National Institute on Deafness and Other Communication Disorders, was a graduate student in Emory's Neuroscience program.

Kelly and his coworkers engineered mice to turn on the Atoh1 gene in the inner ear in response to the antibiotic doxycycline. Previous experimenters had used a virus to introduce Atoh1 into the cochleae of animals. This approach resembles gene therapy, but has the disadvantage of being slightly different each time, Chen says. In contrast, the mice have the Atoh1 gene turned on in specific cells along the lining of the inner ear, called the cochlear epithelium, but only when fed doxycycline.

Young mice given doxycycline for two days had extra sensory hair cells, in parts of the cochlea where developing hair cells usually appear, and also additional locations (see accompanying image).

The extra hair cells could generate electrical signals, although those signals weren't as strong as mature hair cells. Also, the extra hair cells appeared to attract neuronal fibers, which suggests that those signals could connect to the rest of the nervous system.

"They can generate electrical signals, but we don't know if they can really function in the context of hearing." Chen says. "For that to happen, the hair cells' signals need to be coordinated and integrated."

See the rest here:

Gene therapy for hearing loss: Potential and limitations

The study involved painstaking molecular analysis of blood samples taken annually from the patients, who participated in separate studies begun in 2000, 2002, and 2004.

"We were astonished that we could detect the modified cells for so long. It's a relatively small number of patients, but more than 500 years of patient data," said University of Pennsylvania pathologist Bruce Levine, a leader of the research. "But it's difficult to separate with certainty the effectiveness of this treatment from the antiretrovirals."

Gene therapy harnesses the insidious ability of viruses to slip their DNA into the cells they infect. By neutralizing a virus and then using it as a "vector" to insert DNA that is helpful rather than harmful, gene therapy can theoretically treat ailments ranging from arthritis to infections and cancer.

Levine, his Penn colleague Carl June, and their team have tested a variety of ways to outwit HIV with gene therapy. Their approach has focused on T cells, which are the big guns of the immune system but also the cells that HIV infects. The researchers took some of the patients' T cells and inserted a gene that makes them better at recognizing and killing HIV-infected cells. Then these super-T cells were multiplied using growth-stimulation technology and put back into the patient.

Over the years, many other research groups have tried using modified T cells, but the patient's immune system perceived them as invaders and wiped them out, sometimes within hours.

Read this article:

Penn researchers report a gene-therapy success

Tales of biotechnology advances are unlikely to raise a lump in the throat or bring a tear to the eye on their own. Thats what makes Ricki Lewiss recent talk at Lexington Community Education so clever and engrossing. Lewis wrapped a lecture on human genetics and gene therapy in the genuinely moving stories of children whose lives were altered -- some for good, some for bad -- since the field was born in the early 1990s.

As in her book, The Forever Fix: Gene Therapy and the Boy Who Saved It, Lewis talked about the children impacted by rare diseases and the scientists who labored to get closer to a forever fix, a cure that would repair problems at the genetic level.

Lewis is the author of the science textbook Human Genetics: Concepts and Applications, now in its 10th edition and used worldwide, Discovery: Windows on the Life Sciences, and the novel Stem Cell Symphony, about using rock music to activate stem cells in the brain to cure disease. She holds a Ph.D in genetics from Indiana University, and is currently a genetic counselor and teaches at the Alden March Bioethics Institute of Albany Medical College.

She began the lecture with the story of 8-year-old Corey Haas, who in 2008, only four days after undergoing gene therapy to cure his hereditary blindness, screamed in pain when visible sunlight impacts his eyes for the first time.

Lewis uses Coreys story, and how he came to be one of the early beneficiaries of gene therapy, to tell the story of the birth of that miraculous treatment.

Corey was in the right place and the right time, she said. His blindness was treatable but the treatment was experimental.

Gene therapy involves the placement of a gene that a person cant make in their own body into virus cell which is implanted in the body. In Coreys case, his eyes were unable to use Vitamin A, which is necessary for the eye to take in and process light. A virus delivered DNA wrapped in a protein to correct faulty instructions in the affected body part, Lewis said.

Lewis also discussed the story of Jesse Gelsinger, a teenager whose death during gene therapy trials nearly brought the experimental treatment to a close while it was still in its infancy. While Gelsingers story was a tragic failure, Lewis said that scientists were committed to learning from their errors and moving the field further.

One of the most famous children Lewis discussed was David Vetter the so-called Bubble Boy of the 1970s, depicted in the film The Boy in the Plastic Bubble, starring John Travolta. Vetter died of an immunological disease in 1984 at age 30, but the condition is now treatable through gene therapy.

By treating rare diseases such as Vetters, Lewis explained that researchers will learn what they need to treat more common diseases in the future.

Link:

Lewis speaks on gene therapy at Lexington Community Education event

The American Society of Gene & Cell Therapy is pleased to honor Dr. David Williams, MD, as the recipient of the Outstanding Achievement Award at the Society’s 15th Annual Meeting on Saturday, May 19th.Milwaukee, WI (PRWEB) May 04, 2012 Pioneering researcher recognized for scientific achievements in gene and cell therapyThe American Society of Gene & Cell Therapy is pleased to honor Dr. David ...

Read the rest here:

Generational Achievements in Gene and Cell Therapy Honored at ASGCT 15th Annual Meeting

ELK GROVE VILLAGE, Ill., May 2, 2012 (GLOBE NEWSWIRE) -- Families of SMA is pleased to announce the award of up to $750,000 for an important new grant to Dr. Brian Kaspar at Nationwide Children's Hospital. This award will support preclinical development of a CNS-delivered Gene Therapy for Spinal Muscular Atrophy. With funding from FSMA, Dr. Kaspar's team will initiate the studies needed for an Investigational New Drug (IND) application for this therapy to the Food and Drug Administration (FDA).

"Families of SMA is excited to be awarding new goal-directed drug discovery funding for this gene therapy program. This work follows up on a 2010 grant from FSMA to test the age-dependence in primates of this gene therapy. The new funding will allow us to accomplish several key goals simultaneously", says Jill Jarecki, PhD, FSMA Research Director. "First, it will allow us to advance this very promising new therapy for SMA towards human clinical trials. Second, it will allow FSMA to fund multiple SMA drug programs concurrently, which have different approaches. Doing this will increase our community's chances of successfully finding a treatment for SMA."

"This is extremely important funding from FSMA to allow us to collect additional pre-clinical data for a CNS delivered AAV gene delivery into the cerebrospinal fluid, which will be important information to present to the FDA. It also jump starts research prior to obtaining government and commercial involvement which we are actively pursuing. We are quite hopeful for a positive funding decision on a recent NIH proposal for co-funding of this project with FSMA." Brian Kaspar, PhD, Associate Professor, Principal Investigator The Research Institute at Nationwide Children's Hospital, The Ohio State University.

The overall project goals are: 1) to optimize the dosing regimen for CNS-delivered SMA gene therapy; 2) to conduct the GLP toxicology, immune response, and bio-distribution experiments required by the FDA; 3) to prepare and hold a pre-IND meeting with the FDA; 4) to submit an IND to the FDA to begin human clinical trials; and 5) to produce clinical grade material for human studies. The overall timeline for this work is expected to be three years.

This Program was chosen for funding by the FSMA Translational Advisory Committee (TAC), after reviewing multiple potential new drug programs. Every drug program carries risk of encountering hurdles at each of the stages described above. Therefore, a project specific Steering Committee has been put in place, which is comprised of experts in both gene therapy and in SMA biology, with representatives from academia and industry.

This committee will help manage the project, ensuring it progresses in an efficient and well-run manner. In addition, project funding will be awarded upon meeting predetermined milestones, decided on by the Steering Committee.

"I am incredibly excited by FSMA's decision to support Dr. Kaspar and his team in this very important project. As a pharmaceutical scientist who works every day in drug discovery and development, I am encouraged by the quality of the science and the fact that it aims to address SMA treatment from a different vantage point from other programs in the SMA drug pipeline. This is only the first step, but it's a critically important step toward assessing whether gene therapy is a viable approach in SMA. Time will tell but I, for one, am incredibly hopeful and look forward to working with FSMA to facilitate the efforts of Dr. Kaspar and his team. I should add that as a parent of an SMA child, I am always looking for a medical breakthrough that could the transform the lives of SMA patients." Timothy P. Reilly, PhD, DABT Director, Drug Safety Evaluation, Bristol-Myers Squibb. TAC Member.

A major goal at FSMA has been to build the SMA drug pipeline, and we have been investing in drug research since 2000 towards this goal. Even with our community's current progress in adding programs to the SMA drug pipeline and advancing programs to start clinical trials, FSMA believes it is critical to do more. Statistics show that only 10% of all drugs initiating human clinical trials ultimately receive FDA approval. The new funding announced here by FSMA for this preclinical drug program will help achieve this goal. FSMA has been involved in funding half of all the ongoing novel drug programs for SMA.

About Families of Spinal Muscular Atrophy

Families of SMA funds and directs the leading SMA research programs 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 $50 million in research and 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 85,000 members and supporters throughout the United States, and is dedicated to creating a treatment and cure by: funding and advancing a comprehensive research program; supporting SMA families through networking, information and services; improving care for all SMA patients; educating healthcare professionals and the public about SMA; enlisting government support for SMA; embracing all touched by SMA in a caring community. For more information: http://www.curesma.org.

View post:

Families of SMA Awards New Funding to Advance a CNS Delivered Gene Therapy for Spinal Muscular Atrophy

By Robert Bazell, Chief science and medical correspondent, NBC News

One step closer to a cure for AIDS that is the implication of results out Wednesday from from several leading research centers.

It should be noted that many people involved in AIDS research, including several who carried out the latest research, avoid the c word. Their goal is to allow people infected with HIV to live without daily doses of the medications that usually keep the virus under control-- at a large financial cost --and a risk of side effects.

The latest work, published in Science Translational Medicine, details 43 HIV-infected volunteers who had experimental genes inserted into their disease-fighting white blood cells 11 years ago. All patients are doing fine. After more than a decade with this gene therapy, there are no side effects. In almost every one the inserted genes are still working properly.

While these experiments were never intended to treat or cure anything, they lay the groundwork for gene therapy that could have a substantial impact on HIV disease.

A cure for AIDS became an obvious goal as soon as the disease was discovered 30 years ago. But it became the dirty four letter word, as Jon Cohen of Science magazine put it, after some spectacular failures. Soon after the powerful cocktail of anti-AIDS drugs came on the market in 1996, some scientists speculated they could use the drugs to knock out all the infection in the body. But that idea crashed as repeated experiments showed that pockets of infected cells hid in various parts of the body, emerging quickly as soon as the drugs were withdrawn.

HIV has infected some 50 million people in the world and none has been cured -- except perhaps Timothy Ray Brown.

It was the case of Brown, also known as the Berlin patient, that energized the new search for a cure.Infected with HIV, Brown was dying not of that disease, but of leukemia. His only hope was a bone marrow transplant first killing, then replacing all the cells in his body that make blood cells with those from a donor. Browns doctor Gero Htter was not an AIDS specialist, but he knew that about 1 percent of people of European decent have a mutation in a receptor called CCR5 on certain white blood cells that make HIV infection very difficult.So the doctor sought a donor with that mutation.

The transplant took place in 2007. In 2010 Htter published his results. Not only had the transplant eliminated Browns leukemia, he no longer needed to take his HIV medications and the most sophisticated tests find no trace of HIV in his body.

A transplant with a serious risk of death, costing more than $250,000, will not be a treatment for a disease contained by medications.But the case raised the possibility that modifying the white blood cells with gene therapy might do the trick. Several experiments are underway in both animals and humans and more are planned.

Read more here:

AIDS gene therapy safe -- is it a "cure"?

Academic and industry leaders in gene and cell therapy will be featured at a Media Event in Philadelphia, PA on May 15, 2012, immediately preceding the 15th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) on May 16-19, 2012.Milwaukee, WI (PRWEB) May 02, 2012 Academic and industry leaders in gene and cell therapy will be featured at a Media Event in Philadelphia, PA on May ...

Go here to read the rest:

A Media Event on Clinical Developments in Gene and Cell Therapy

PLoS Biology

HIV particles assemble at the surface of a white blood cell called a macrophage.

Millions of people around the world are living with HIV, thanks to drug regimens that suppress the virus. Now there's a new push to eliminate HIV from patients' bodies altogether. That would be a true cure.

We're not there yet. But a report in Science Translational Medicine is an encouraging signpost that scientists may be headed in the right direction.

Forty-three patients got immune cells designed to attack and kill cells infected with HIV. As long as 16 years later, these genetically engineered T cells are still circulating in their bloodstreams. And there's been no sign the gene therapy caused any cancers, or is likely to.

That may seem like a modest victory. After all, there's no evidence yet that the gene therapy did what it's supposed to eliminate the reservoir of HIV hiding in the patients' cells, waiting to emerge as soon as patients stop taking their antiviral drugs.

But to scientists in HIV and gene therapy research, it's a highly encouraging indicator. "We're not hitting a home run. This is a single," AIDS researcher Pablo Tebas of the University of Pennsylvania tells Shots.

"It looks like if you do this, it's going to be safe because we have not seen any toxicity in 16 years," he says. "And two, the genetically modified cells are still circulating. They perpetuate. Those are two important things this study is telling us."

Tebas is not an author of the study, but he works with the Penn researchers who did the work. They were unavailable for comment.

Previous attempts at this kind of gene therapy, called adoptive T cell transfer, have been plagued by cancers that can arise when the genes introduced into engineered cells insert themselves next to growth-promoting genes. In other cases the engineered cells have died out before they have a sustained positive effect.

Read the original:

A Step Forward For Gene Therapy To Treat HIV

By Elizabeth Lopatto - Wed May 02 18:00:00 GMT 2012

HIV patients given gene therapy more than a decade ago are healthy and the altered DNA they received remains stable in their bodies, according to a study that scientists say proves the treatment may safely be tested as a way to attack other illnesses.

All except two of 43 people treated with genetically- altered versions of their own infection-fighting T cells were healthy as many as 11 years later, according to the research reported today in the journal Science Translational Medicine.

Previous uses of gene therapy in experiments have suggested that leukemia caused by the viruses that transfer the genes to the cells might be a risk. Todays finding allays that concern, enabling researchers to move beyond immediately life-threatening illnesses, such as HIV and cancer, said Bruce Levine, a study author and researcher at the University of Pennsylvania.

We turned those cells into heat-seeking missiles directed against HIV-infected cells, said Levine, head of the Clinical Cell and Vaccine Production Facility at the Philadelphia universitys Perelman School of Medicine. What really surprised us was when we got those samples, not only could we detect the gene-modified cells but they appeared to be present at relatively stable levels.

Because the therapy has been found to work safely over a significant period of time, the designer cells should be considered a platform technology that can also be used by scientists researching other diseases besides HIV, Levine in a telephone interview.

Each patient in the study cited today received at least one transfusion of their own immune cells between 1998 and 2005. The T cells were designed to look for an HIV protein and kill any infected cells they encountered, before the virus has a chance to mature.

Its like a controlled burn, Levine said.

The U.S. Food and Drug Administration required the patients be followed for 15 years to see if any late-developing side- effects, such as cancer, might arise from the therapy. The patients were followed every year after their initial dose.

No gene therapy has been approved by the FDA. The field almost halted in 1999, when 18-year-old Jesse Gelsinger died within hours of being injected. Earlier, in a French trial, two of 10 patients acquired leukemia following gene therapy for Bubble Boy disease, or severe combined immunodeficiency.

See the article here:

Gene Therapy Safe in Decade-Long HIV Study That May Widen Use

By Randy Dotinga HealthDay Reporter

WEDNESDAY, May 2 (HealthDay News) -- New research shows that gene therapy can have long-lasting effects on the immune cells of HIV patients -- a promising sign -- even though the specific treatment being studied did not eradicate the virus.

This approach is one of several gene therapy strategies that are being investigated by scientists as possible ways to keep the AIDS virus from spreading in the blood.

In this case, "people were treated by gene therapy and nothing bad happened. It was safe," said study co-author Frederic Bushman, a professor of microbiology at the University of Pennsylvania.

In addition, he said, the treated immune cells managed to remain around for about a decade. "The general picture that emerges about genetic alterations to human immune cells is that they can persist for a long time if you do it right."

The study appears in the May 2 issue of Science Translational Medicine.

Researchers have long been exploring gene therapy -- in which cells in the body are genetically modified -- as a possible treatment for infection with HIV, the virus that causes AIDS. The idea is that the therapy would offer a permanent alternative to costly medications that come with potentially disabling side effects.

"Just think about what an HIV patient has to do: take drugs every day for the rest of his life, and the minute he stops taking them, the virus starts coming back," said John Rossi, chair of the department of molecular and cellular biology at the Beckman Research Institute of the City of Hope, in Duarte, Calif. He was not associated with the new research.

The study looks at 43 HIV-positive patients. Between 1998 and 2002, researchers removed blood from the patients, genetically modified it, and injected it back into them.

The plan was to program immune cells known as T cells to kill HIV cells.

Visit link:

Gene therapy for HIV safe, but effectiveness still unclear