Category Archives: Gene Therapy Trials

HOME Gene Therapy Challenges in Gene Therapy? Gene therapy is not a new field; it has been evolving for decades. Despite the best efforts of researchers around the world, however, gene therapy has seen only limited success. Why? Gene therapy poses one of the greatest technical challenges in modern medicine. It is very hard to introduce new genes into cells of the body and keep them working. And there are financial concerns: Can a company profit from developing a gene therapy to treat a rare disorder? If not, who will develop and pay for these life-saving treatments? Let's look at some of the main challenges in gene therapy. For some disorders, gene therapy will work only if we can deliver a normal gene to a large number of cellssay several millionin a tissue. And they have to the correct cells, in the correct tissue. Once the gene reaches its destination, it must be activated, or turned on, to make the protein it encodes. And once it's turned on, it must remain on; cells have a habit of shutting down genes that are too active or exhibiting other unusual behaviors. Introducing changes into the wrong cells Targeting a gene to the … Continue reading →

Gene therapy carries the promise of cures for many diseases and for types of medical treatment that didn't seem possible until recently. With its potential to eliminate and prevent hereditary diseases such as cystic fibrosis and hemophilia and its use as a possible cure for heart disease, AIDS, and cancer, gene therapy is a potential medical miracle-worker. But what about gene therapy for children? There's a fair amount of risk involved, so thus far only seriously ill kids or those with illnesses that can't be cured by standard medical treatments have been involved in clinical trials using gene therapy. As those studies continue, gene therapy may soon offer hope for children with serious illnesses that don't respond to conventional therapies. Our genes help make us unique. Inherited from our parents, they go far in determining our physical traits like eye color and the color and texture of our hair. They also determine things like whether babies will be male or female, the amount of oxygen blood can carry, and the likelihood of getting certain diseases. Genes are composed of strands of a molecule called DNA and are located in single file within the chromosomes. The genetic message is encoded by … Continue reading →

Please choose from the following list of questions for information about gene therapy, an experimental technique that uses genetic material to treat or prevent disease. On this page: Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patients cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including: Replacing a mutated gene that causes disease with a healthy copy of the gene. Inactivating, or knocking out, a mutated gene that is functioning improperly. Introducing a new gene into the body to help fight a disease. Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently only being tested for the treatment of diseases that have no other cures. MedlinePlus from the National Library of Medicine offers a list of links to information about genes and gene therapy. Educational resources related to gene therapy are … Continue reading →

Prof Krystof Bankiewicz. Overview of AADC Deficiency - AAV2-hAADC Gene Therapy Trials. Prof Krystof Bankiewicz of University of California, San Francisco, USA presents an overview of AADC Deficiency and AAV2-hAADC Gene Therapy Trials. He describes the technique and background... By: aadcresearch … Continue reading →

A new variation of gene therapy raises hopes for a safe and effective long-term treatment for X-linked severe combined immunodeficiency syndrome (SCID-X1), a life-threatening heritable disorder. The research was produced by a collaborative research team from Dana-Farber/Boston Children's Cancer and Blood Disorders Center, along with other institutions participating in an international clinical trial that involved boys from the United States and France. SCID-X1, dubbed bubble boy disease after a patient who lived for 12 years in a sterile bubble, is a rare genetic disorder that hinders the ability of individuals to combat infections. Because the disease is carried in an X-chromosome recessive pattern, the disorder occurs almost only in males. The resulting mutations inactivate a gene called IL-2 receptor gamma (IL2RG), severely weakening immune system functions. Left untreated, individuals who inherit the disorder usually die within a year. Previous gene therapy trials conducted in Europe over a decade ago promised dramatic progress, until a quarter of patients developed leukemia about two to five years following treatment. Scientists found that the previously used vectorthe device for transporting the correct gene in therapyinadvertently activated oncogenes, which can cause cancer. In this new study, the vector in use is a self-inactivating gammaretrovirus, which … Continue reading →

FOR IMMEDIATE RELEASE Tuesday, March 7, 2000 Contact: FDA Press Office (301) 827-6250 NIH Press Office (301) 496-5787 New Initiatives to Protect Participants in Gene Therapy Trials FDA's clinical trials monitoring plan addresses emerging evidence that the monitoring by study sponsors of several recent gene therapy trials has been less than adequate. To buttress the rigor of the oversight, FDA will require that sponsors of gene therapy trials routinely submit their monitoring plans to the FDA. FDA will review these monitoring plans and seek modifications as warranted to improve the quality of monitoring. FDA will also perform surveillance and "for cause" inspections of clinical trials to assess whether the plans are being followed and whether monitoring has been adequate to identify and correct critical problems. The sponsors will also have to address such issues as the experience and training of the monitors and the adequacy of the monitoring in their plans. In addition, NIH and FDA will seek to enhance the conduct of gene therapy trials by convening a conference of investigators at which the appropriate monitoring practices will be discussed by the most experienced professionals in the field. Clinical trial monitoring is a powerful tool in enhancing the safety … Continue reading →

Patient Receives First-Ever Gene Therapy for Parkinson's at NewYork-Presbyterian Hospital/Weill Cornell Medical Center Historic Procedure Also Marks First In Vivo Gene Therapy in Adult Brain NEW YORK (Aug 20, 2003) Surgeons at NewYork-Presbyterian/Weill Cornell performed the world's first gene therapy for Parkinson's disease on a 55-year-old New York man on Monday, August 18. The historic surgery, which also marked the first-ever in vivo gene therapy in the brain for an adult neurological disease, was part of a Phase I clinical trial approved by the Food and Drug Administration in October 2002. The five-hour procedure was performed by Dr. Michael G. Kaplitt, Director of Stereotactic and Functional Neurosurgery at NewYork-Presbyterian Hospital and Assistant Professor of Neurological Surgery at Weill Cornell Medical College. The patient is recovering normally and is expected to return home today, just two days after the surgery was performed. "Monday's surgery represents the realization of nearly 15 years of research in this area," said Dr. Kaplitt. "The goal of our gene therapy approach is to 're-set' a specific group of cells that have become overactive in an affected part of the brain, causing the impaired movements associated with Parkinson's disease. We hope that this trial, which is the … Continue reading →

Wiley database on Gene Therapy Trials Worldwide The Journal of Gene Medicine clinical trial site presenting charts and tables showing the number of approved, ongoing or completed clinical trials worldwide. Data is available for: Continents and countries where trials are being performed; Indications addressed; Vectors used; Gene types transferred; Phases of clinical trials; Number of trial approved/initiated 1989-2007. A searchable database is also present with detailed information on individual trials. The data are compiled and are regularly updated from official agency sources (RAC, GTAC etc..), the published literature, presentations at conferences and from information kindly provided by investigators or trial sponsors themselves. Beware that information on some trials is incomplete as some countries regulatory agencies simply do not disclose any information. See also: Gene therapy clinical trials worldwide to 2007 - an update. J. Gene Med. 2007 Oct;9(10):833-842. ClinicalTrials.gov database on clinical trials performed in the US and worldwide The U.S. National Institutes of Health, through its National Library of Medicine, has developed ClinicalTrials.gov to provide patients, family members and members of the public current information about clinical research studies. The database is a registry of federally and privately supported clinical trials conducted in the United States and around the … Continue reading →

Will Jones - Training for run for Cystic Fibrosis Trust Rocky Training Sequence My name is William Jones. I'm 10 years old and have got cystic fibrosis. On May 7th i'm doing my 2nd Cardiff Bay run. Last year you guys donated an AWESOME amount of money which helped the cf trust pay for the second phase of gene therapy trials along with all the other great work they do to help me and everyone else with cf have a longer and better life. Can you do the same again? Donate via this link below http://www.justgiving.comFrom:owenhowellsViews:529 7ratingsTime:02:15More inComedy See the rest here: Will Jones - Training for run for Cystic Fibrosis Trust Rocky Training Sequence - Video … Continue reading →

Interview on BBC Breakfast with Professor Robin Ali about Gene Therapy Trials for Eye Conditions Continue reading here: Jonathan on BBC Breakfast … Continue reading →