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Greenwich couples' legacy lives on in ACGT

Recently, there was a gathering of scientists at the Hyatt Regency in Greenwich. They were there to discuss their latest findings in cancer research — especially in the use of gene therapy in cancer treatment. There was tangible excitement in the room. Among those in attendance was Dr. Carl June of the University of Pennsylvania, who had, using gene therapy, actually eliminated all signs of cancer in two patients he was treating. Also there was Dr. Hui Hu, who works in the same city as June, at the Wistar Institute, and who is having similar success treating mice with cancer with his gene therapy, which he wants to apply to human patients.

The reason for this writing, however, is another reason for the gathering — the celebration of the 10th anniversary of the Alliance for Cancer Gene Therapy (ACGT), a Greenwich non-profit that has helped fund these scientists and others like them; and the legacy of Ed Netter, of Greenwich, co-founder of ACGT, who died last year, just short of Dr. Carl June’s news of his gene therapy treatment.

Barbara Netter, Ed Netter’s widow and cofounder of ACGT, was feeling the excitement in the scientific meeting held before the gala dinner honoring her late husband and ACGT.

“We have so much energy now with Carl June,” she said. “We’re receiving a lot more applications for grants. There’s a lot of collaboration and partnering happening.”

It was the death of the Netters’ daughter-in-law from breast cancer that inspired Ed and Barbara Netter to create ACGT 10 years ago as a vehicle to raise money for research into cancer gene therapy.

“The vision that Ed had is a new way to really get the science into clinical trials that will show the (gene therapy) concept at work,” said Barbara Netter.

Last August, Dr. Carl June reported to the world the success of his clinical trial, supported by ACGT, in which he genetically modified the T-cells of three patients with chronic lymphocytic leukemia to target and kill their tumors. Two of the three patients remain cancer free, with the third patient’s cancer significantly reduced.

June’s treatment, he said, was still only in the first phase. But he’s now adding more patients. “We’ve taken in our first pediatric patient — a 6-year-old girl with leukemia. She was infused today,” June said.

This is the first of 400 patients June wants to treat within the next year or two.

“The gene therapy approach is fundamentally different from current therapies,” said June, “and these ideas need to move to human treatment.”

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Greenwich couples' legacy lives on in ACGT

Greenwich Cancer Research Group Raises $1.4M

Barbara Netter, co-founder of the Alliance for Cancer Gene Therapy, receives the first-ever ACGT Partnership Award from Savio Woo, chairman of the Alliance’s scientific council and professor and founding chair of the Department of Gene and Cell Medicine at Mt. Sinai School of Medicine.

Photo credit: Courtesy of Elaine Ubia

GREENWICH, Conn. The Greenwich-based Alliance for Cancer Gene Therapy celebrated its 10th anniversary of funding cancer gene therapy research with a gala benefit last month.

Thirty of the nations top cancer research scientists and more than 450 Alliance supporters gathered on April 19 to mark the progress made in the field. The event raised more than $1.4 million, all of which will go directly to funding new grants and clinical trails.

The alliance also honored the life of its visionary co-founder, Edward Netter, who died last year, by presenting the first ever ACGT Partnership award to Netter’s wife, Barbara, who co-founded the alliance with her husband.

Ed epitomized the innovative thinker and problem solver, and he felt it important to examine the status quo, Barbara Netter said in a prepared statement. He had the perseverance to pursue a better way. Our progress, our recent breakthrough, is proving to be a fitting tribute and legacy to Ed, who worked tirelessly and passionately to this end. Ed Netter was a key figure in accelerating research and bringing us to this point.

Prior to the evenings festivities many of the nations leading cancer researchers held a scientific symposium, where they discussed the alliances progress as a leader in the field, as well as how to bridge the gap between the lab and bringing science to patients.

Robert Bazell, the Emmy award-winning chief science and health correspondent for NBC News, was the keynote speaker. Dr. Dennis Clegg, professor, Department of Molecular, Cellular and Developmental Biology, University of California, Santa Barbara, California, was the featured scientist at the dinner.

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Greenwich Cancer Research Group Raises $1.4M

Scientists may have found the key to a longer life

Scientists in Spain may have found the answer to increasing one’s lifespan.

Researchers at the National Cancer Research Centre in Spain used gene therapy to successfully extend the lifespan of mice by an average of 24 percent, according to a new study.

The scientists treated two groups, one-year-old and two-year-old mice, with gene therapy and found that the younger mice lived 24 percent longer while the older mice lived 13 percent longer.

“Gene therapy is typically thought of as a way to deliver genes into cells to correct genetic defects or diseases,” said Maria Blasco, director of the Spanish National Cancer Research Centre, according to QMI.

“However, if we consider that ageing is, at least in part, the consequence of defective gene function, gene therapy is also a valid strategy to delay ageing or to increase lifespan.”

Read more on GlobalPost:Red wine anti-aging properties shown in new study

The study saw researchers induce the animals’ cells to produce telomerase, an enzyme that helps to maintain the physical structure of the ends of chromosomes, reported Slate.

“Our results show that telomerase gene therapy is not only a viable anti-ageing intervention but it also has remarkably beneficial effects on health and fitness without increasing the incidence of cancer,” she said, according to TG Daily.

Zee News reported that the therapy improved the animal’s health by delaying age-related diseases and improving coordination.

The therapy even improved insulin-resistance.

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Scientists may have found the key to a longer life

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

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.

Gene Therapy Extends Mouse Lifespan

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

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Gene Therapy Extends Mouse Lifespan

Gene therapy dramatically extends mouse lifespan

Gene therapy has been used to increase the lifespan of mice by up to 24 percent – and improve their health at the same time.

Telomerase helps to maintain the physical integrity of the ends of chromosomes. And mice that received a single treatment to deliver the enzyme to different cells in the body showed drastic improvements in health, fitness and longevity, says the team.

“Gene therapy is typically thought of as a way to deliver genes into cells to correct genetic defects or diseases. However, if we consider that ageing is, at least in part, the consequence of defective gene function, gene therapy is also a valid strategy to delay ageing or to increase lifespan,” says Maria Blasco, director of the Spanish National Cancer Research Centre.

Telomeres are the caps of repetitive DNA nucleotide sequences that sit at the ends of chromosomes, and are known to play a part in ageing. As they are gradually worn down, cell stop dividing and eventuially die.

The scientists used an adeno-associated virus vector to introduce the telomerase gene into the cells of adult mice, where it added DNA back to the ends of chromosomes.

The mice that were used in the experiments typically live for approximately 150 weeks. However, one-year-old mice that received the gene therapy lived on average 24 percent longer, and the average lifespan of two-year-old mice increased by 13 percent.

“In addition to living longer, engineered mice had stronger bones, improved metabolic functions, better motor coordination and balance, as well as improved performance in object-recognition tests,” says Bruno Bernardes de Jesus, a researcher at the Spanish National Cancer Research Centre.

The findings are the first proof-of-principle that telomerase gene therapy is both feasible and potentially safe. The team now plans to investigate whether the therapy could work for animals with longer lifespans.

Human Gene Therapy highlights new advances in human retinal disease

Gene therapy strategies to prevent and treat inherited diseases of the retina that can cause blindness have progressed rapidly. Positive results in animal models of human retinal disease continue to emerge, as reported in several articles published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The articles are available free on the Human Gene Therapy website at http://www.liebertpub.com/hum.

After 20 years of promising research, testing in animal models, and initial clinical trial results of gene therapy to treat retinal dystrophies, Robin Ali, University College London Institute of Ophthalmology (UK) is “optimistic about the future prospects for retinal gene therapy” mainly because the retina has proven to be a good and accessible target for gene delivery, and scientists have developed efficient and safe viral delivery systems that can successfully introduce therapeutic genes into photoreceptor cells in the retina. The main challenge now is to increase the number of gene therapies in clinical trials and to optimize these treatments for patients, says Dr. Ali in the Commentary “Gene Therapy for Retinal Dystrophies: Twenty Years in the Making.”

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Human Gene Therapy highlights new advances in human retinal disease

Penn researchers report a gene-therapy success

For a quarter of a century, gene therapy has been stymied, largely because the patient’s immune system attacks the treatment as a suspected rogue – or because it actually does turn rogue.

Now, University of Pennsylvania researchers have convincingly shown that they can overcome these formidable obstacles. Cells that were genetically modified to fight HIV have persisted for up to 11 years – and counting – without bad side effects in 41 patients. In two other patients, the modified cells were safe but not as durable, according to the Penn study, published last week in Science Translational Medicine.

Although the gene therapies being tested showed hints that they could be effective against the AIDS virus, the studies looked at whether the approach was feasible and safe, not whether it worked. All of the patients were also taking standard HIV drugs, called antiretrovirals.

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.

Another problem is finding precise ways to insert the modified virus and ensure that it will cause the desired genetic change. The chosen virus may put its genetic payload into a host’s DNA in a way that triggers cancer. That happened in France when 3 of 10 children who underwent gene therapy and were cured of a severe combined immune deficiency disease – better known as the “Bubble Boy disease” – later developed leukemia.

The Penn analysis showed that its therapeutic gene entered varying spots in the DNA of the T cells, which then went on to divide and produce a new generation of cells. Yet annual monitoring of the patients “has not detected any suspected or documented” disorders that might be traced to the genetic manipulation.

A Media Event on Clinical Developments in Gene and Cell Therapy

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 15, 2012, immediately preceding the 15th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) on May 16-19, 2012. The event will focus on recent clinical developments in targeted biotherapeutics for various diseases.

Members of the media will also receive complimentary full-access registration to the ASGCT 15th Annual Meeting at the Pennsylvania Convention. To complete your registration to the Media Event and the Annual Meeting, please visit the ASGCT website.

2:00 pm 2:20 pm: Gene and cell-based immunotherapy for cancer Many cancers are known to acquire the ability to suppress anti-tumor immune responses in the host. The genetically modified cells developed and used in this clinical trial are designed to reawaken immune cells that have been suppressed by the leukemia and stimulate the generation of so-called memory T cells, which can provide ongoing protection against recurrence. Although long-term effectiveness of this novel treatment is not yet known, the doctors have found that months after infusion, the new cells had multiplied and continued their seek-and-destroy mission against cancerous cells throughout the patients bodies. The new paradigm provides a tumor-attack roadmap for the treatment of other cancers including lung, myeloma, ovarian cancer and melanoma.

Carl June, MD is currently the Director of Translational Research at the Abramson Cancer Center,, an Investigator of the Abramson Family Cancer Research Institute, and a tenured Professor of Pathology and Laboratory Medicine at the Perelman School of Medicine of the University of Pennsylvania.

2:20 pm 2:30 pm: Question and answer period

2:30 pm 2:50 pm: Gene therapy for hemophilia B Hemophilia B is an X-linked bleeding disorder caused by a deficiency of blood coagulation Factor IX, and patients need to be infused with the newly developed long-acting protein concentrates twice a month. Advances in gene therapy using intravenous infusion of an AAV vector expressing human Factor IX in a clinical trial on hemophilia patients have significantly improved circulation of Factor IX levels that transformed their disease from severe hemophilia to mild hemophilia. Other strategies are also under development that may confer the ability to correct the disease permanently by gene therapy.

Katherine High, MD is the William H. Bennett Professor of Pediatrics at the University of Pennsylvania School of Medicine, an Investigator of the Howard Hughes Medical Institute and Director of the Center for Cellular and Molecular Therapeutics at the Children Hospital of Philadelphia. Dr. High has pioneered safe and effective clinical translation of genetic therapies for inherited disorders including hemophilia B, and she is a Past President of the American Society of Gene and Cell Therapy.

2:50 3:00 pm: Question and answer period

3:00 pm 3:20 pm: Gene and cell-based therapy for severe combined immunodeficiency Severe combined immunodeficiency (ADA-SCID, a.k.a. bubble boy disease) is a rare disorder of immunity and without treatment; children affected with ADA-SCID will succumb to infections within the first two years of life. Over the past years >40 patients have been treated with gene-corrected hematopoietic stem cell transplantation, which has led to the regeneration of immune cells and the reconstitution of immune function in >70% of the patients. While hematopoietic stem cell transplantation from a matched donor remains the therapy of choice for ADA-SCID, the results obtained by the current clinical trials have indicated that gene and cell therapy should be considered as the first treatment option in the absence of a matched donor.

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

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: www.curesma.org.