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Scientists Construct 'Off Switch' For Parkinson Therapy

Posted on: 29 August 2009, source: ScienceDaily
A common antibiotic can function as an "off switch" for a gene therapy being developed for Parkinson's disease, according to University of Florida researchers writing online in advance of September's Molecular Therapy. The discovery in rats answers an important question — how can new, therapeutic genes that have been irrevocably delivered to the human brain to treat Parkinson's be controlled if the genes unexpectedly start causing problems? Meanwhile, in a review of Parkinson treatments, the researchers say that prior experimental attempts using growth factors — naturally occurring substances that cause cells to grow and divide — to rescue dying brain cells may have failed because they occurred too late in the course of the disease. Together, the findings suggest that gene therapy to enable the brain to retain its ability to produce dopamine, a neurotransmitter that falls in critically short supply in Parkinson's patients, could be safely attempted during earlier stages of the disease with an added likelihood of success.

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Study Clears Gene Therapy in Death of Arthritis Patient

Posted on: 10 July 2009, source: MedPage Today
The death of a young patient participating in a gene-therapy trial for a rheumatoid arthritis drug was most likely the result of another arthritis drug, adalimumab (Humira), and not the novel gene therapy agent, a new study found. "The death of this young patient who was receiving multiple forms of TNF inhibitors highlights the risk of opportunistic infections in patients receiving such agents, and the importance of having a well-designed monitoring plan when a patient in a study becomes ill," Karen M. Frank, MD, of the University of Chicago Medical Center, and colleagues wrote in the July 9 issue of New England Journal of Medicine.
The patient, a 35-year-old woman with a 15-year history of rheumatoid arthritis, first presented three days after receiving a second injection on July 2, 2007, of the active gene-therapy agent, tgAAC94, a tumor necrosis factor alpha (TNF-α) antagonist. She was suffering from a range of symptoms that including fever, chills, abdominal pain, and vomiting.

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Clonal Population of Cells Detected in a Clinical Human Gene Transfer Trial Using Lentiviral Vector

Posted on: 23 June 2009, source: Recombinant DNA Advisory Committee
The National Institutes of Health Office of Biotechnology Activities (OBA) has been informed that a "relative clonal dominance" was detected during follow-up of a subject who is participating in a French human gene transfer trial being conducted for individuals with β-Thalassemia Major and Sickle Cell Anemia. The clinical trial, sponsored by Genetix France, used hematopoietic stem cells transduced by a self inactivating (SIN) HIV-1 lentiviral vector containing the gene for β-globin under the control of the β-globin promoter. The subject received the gene modified cells in June 2007.
This clonal dominance appears to result from the integration of the vector in the gene encoding for the HMGA2 protein, which is associated with both benign and malignant tumors. The clone however has been stable for five months and the subject remains in good health. In fact, although the subject required almost monthly blood transfusions during the 11 months prior to the gene transfer intervention, the subject has not since required a blood transfusion.

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ASGT Embraces Cell Therapy with Name Change

Posted on: 29 May 2009, source: ASGCT
The American Society of Gene Therapy (ASGT) will officially change its name Saturday to the American Society of Gene & Cell Therapy (ASGCT), a change intended to better reflect the intimate relationship between the two fields. “This new name more accurately reflects who we are and what we do,” said David M. Bodine, PhD, ASGT president, who led the initiative to add cell therapy to the name. “Gene therapy and cell therapy cannot be separated; each is an integral part of the other.” Added Ken Cornetta, MD, incoming ASGCT president: “Gene therapists are cell therapists. We have the same goal, which is improved patient care.” The change goes into effect at 7:45 a.m. Saturday, May 30, during the business meeting at the ASGT 12th Annual Meeting.

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Moving Gene Therapy Forward With Mobile DNA

Posted on: 11 May 2009, source: ScienceDaily
Gene therapy is the introduction of genetic material into a patient's cells resulting in a cure or a therapeutic effect. In recent years, it has been shown that gene therapy is a promising technology to treat or even cure several fatal diseases for which there is no attractive alternative therapy. Gene therapy can be used for hereditary diseases, but also for other diseases that affect heart, brain and even for cancer. Indeed, recent results suggest that gene therapy can be beneficial for patients suffering from aggressive brain cancer that would otherwise be lethal.

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Analysis: Gene therapy has immense potential

Posted on: 14 April 2009, source: Times Online
After almost two decades, gene therapies have recently started to deliver on their immense promise. Gene therapies have been a part of medicine for almost two decades. Only recently have they started to deliver on their immense promise after a string of setbacks raised doubts about the safety of manipulating human DNA, centre, to treat disease.
The idea behind gene therapy is that by inserting new copies of a particular gene into human cells, it should be possible to correct defects, or to enhance beneficial biological processes. This can be done with viruses, which introduce their own genetic material into the cells they infect, and can be engineered to carry a human gene.

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Gene Therapy Gets Closer to a 'Cure'

Posted on: 3 March 2009, source: Boston.com
Two decades ago, medicine seemed on the cusp of a revolution. Doctors would soon treat diseases at their very roots, inserting "good" genes to replace patients' faulty ones. Gene therapy was a seductively straightforward idea that offered promise for treating everything from cancer to sickle cell disease.
But only now, after overcoming unexpected scientific obstacles and the high-profile death of a teenage patient, is gene therapy racking up some clear-cut successes. Promising studies are sending ripples of excitement through the field. Some researchers are daring to use the word cure.

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Ark Therapeutics Files Cerepro® Application for European Marketing Authorisation

Posted on: 8 January 2009, source: Biospace
Ark Therapeutics announced that the Marketing Authorisation Application (MAA) for Cerepro®, Ark’s novel gene-based therapy for operable malignant glioma (brain cancer) which was recently filed with the EMEA, has cleared the validation stage. The Cerepro® MAA application now commences formal review via the centralised procedure which is the standard route for all advanced therapies.
Cerepro® was originally filed for marketing approval in late 2005 and was reviewed by the EMEA with reliance on Phase II data. The review established that the technical chemistry and manufacturing controls (CMC), preclinical and environmental sections appeared acceptable, but more clinical data were needed to confirm the Phase II findings and to demonstrate the reproducibility of the results in a larger multi-centre Phase III trial.

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Proposed ASGT name change to the American Society of Gene and Cell Therapy (ASGCT)

Posted on: 27 November 2008, source: ASGT website / Molecular Therapy
A proposal to change the name of the American Society of Gene Therapy (ASGT) to the American Society of Gene and Cell Therapy (ASGCT) has recently been adopted by the 2008 Membership Committee and forwarded to the Board of Directors of ASGT. The Board has unanimously approved the proposal, which is now being forwarded to the general membership of the Society for a final vote in April of 2009. The Membership Committee’s rationale in making this forward-looking proposal is that the concept of gene therapy includes gene-modified cell therapy, and an inclusive name will empower the Society to expand its membership base and help foster further collaborative research to advance the use of genes and cells as medicine to treat disease.

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Gene Therapy For Blindness Improves Vision, Safety Study Indicates

Posted on: 8 September 2008, source: ScienceDaily
All three people who received gene therapy at the University of Florida to treat a rare, incurable form of blindness have regained some of their vision, according to a paper published online today in Human Gene Therapy.
The patients with a type of hereditary blindness called Leber congenital amaurosis type 2, volunteered to test the safety of an experimental gene-transfer technique in a phase 1 clinical research study. In this form of LCA disease, photoreceptor cells cannot respond to light because a gene called RPE65 does not properly produce a protein necessary for healthy vision. In the study, researchers used an adeno-associated virus to deliver RPE65 to a small area of the retina. Not only were there no ill effects other than routine postsurgical soreness, the subjects said the vision in their treated eyes was slightly improved in dim lighting conditions.

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Introgen Submits ADVEXIN® Regulatory Applications in the U.S. and Europe

Posted on: 1 July 2008, source: FierceBiotech
Introgen Therapeutics, Inc. submitted a Biologics License Application (BLA) to the FDA requesting marketing approval for ADVEXIN p53 therapy to treat recurrent, refractory head and neck cancer. Simultaneously, Gendux Molecular Limited, an Introgen subsidiary, submitted a Marketing Authorization Application to the EMEA for the same indication. ADVEXIN represents the first of a new class of tumor suppressor cancer therapy and is the first of its kind to be submitted for regulatory approval in the United States and Europe.

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Gene Therapy Improves Vision In Nearly Blind Patients

Posted on: 28 April 2008, source: ScienceDaily
In a clinical trial at The Children's Hospital of Philadelphia, researchers from The University of Pennsylvania have used gene therapy to safely restore vision in three young adults with a rare form of congenital blindness. Although the patients have not achieved normal eyesight, the preliminary results set the stage for further studies of an innovative treatment for this and possibly other retinal diseases.
An international team led by The University of Pennsylvania, The Children's Hospital of Philadelphia, the Second University of Naples and the Telethon Institute of Genetics and Medicine (both in Italy), and several other American institutions reported their findings in an online article in the New England Journal of Medicine.

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Boy develops leukemia after gene therapy in UK

Posted on: 18 December 2007, source: Reuters
A three-year-old "bubble boy" undergoing pioneering gene therapy in London has developed leukemia, marking another setback for the experimental treatment. Doctors at Great Ormond Street Hospital said on Tuesday the boy had been successfully treated for SCID-X1, or x-linked severe combined immunodeficiency, often known as "baby in the bubble syndrome", but had developed leukemia two years later. The news is a blow to the treatment program at the London hospital, which has a worldwide reputation from treating sick children.

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FDA lifts ban on trial after investigating death

Posted on: 27 November 2007, source: Nature News
A clinical trial of gene therapy in which a woman died this summer is due to recommence, thanks to a decision from the US Food and Drug Administration (FDA) announced yesterday. The experimental therapy for rheumatoid arthritis was stopped after one of the participants, 36-year-old Jolee Mohr, died in July. Autopsy findings presented in September showed that Mohr died of a massive fungal infection complicated by major internal bleeding, and that the gene therapy itself may not have been to blame (see: Study Clears Gene Therapy in Death of Arthritis Patient). Independently of the gene therapy, Mohr was taking several drugs for her arthritis that suppress the immune system, which is a risk factor for this type of infection.

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FDA Statement on Gene Therapy Clinical Trial

Posted on: 26 July 2007, source: FDA
On July 24, 2007 the U.S. Food and Drug Administration (FDA) was informed by Targeted Genetics Corporation of Seattle about the death of a patient who received an investigational gene therapy product in a clinical trial for the treatment of active inflammatory arthritis. FDA's condolences go to the patient's family. FDA is providing this preliminary information in recognition of the public's interest in these types of new therapies. Targeted Genetics notified FDA earlier that a patient in its trial experienced a serious adverse event. Even though the cause of the illness wasn't known, and is still uncertain, the agency immediately placed the trial on clinical hold--meaning no further product can be administered and no new patients can be enrolled. The product that was being studied uses a particle called a vector that is designed to deliver treatment genes to target cells.

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First Oncolytic Viral Therapy Approved for Head and Neck Cancer

Posted on: 27 November 2005, source: Business Wire
Shanghai Sunway Biotech Co. Ltd. announced today that the Chinese State Food and Drug Administration (SFDA) has approved H101, an oncolytic adenovirus, to be used in combination with chemotherapy as a treatment for patients with late stage refractory Nasopharyngeal cancer, a type of head and neck cancer prevalent in China. This marks the first oncolytic viral therapy approved by any regulatory agency in the world.
H101 is a modified adenovirus, a type of common cold virus found in most people. The deletion of an E1B-55kd segment in the virus results in its ability to selectively replicate in and kill tumor cells, while leaving normal cells unaffected. The therapy has a very good safety profile -- for most patients the main side effect is fever. The SFDA approval is based on a multi-center, randomized parallel-group study comparing 5-fluorouracil and cisplatin-based chemotherapy with and without H101. The H101 study group demonstrated a 27% increase in the number of patients who had complete or partial tumor size reduction compared to the control group.

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Third complication case in X-SCID gene therapy clinical trial

Posted on:24 January 2005, source: Afssaps
In May 2004, the French Health Product Safety Agency (Afssaps) authorised the restart of the gene therapy clinical trial conducted by Prof. Alain Fischer and Marina Cavazzana-Calvo, in Necker-Enfants-Malades hospital in Paris. The clinical trial is aimed at assessing efficacy of a gene therapy approach in the treatment of X-linked severe combined Immuno-deficiency (X-SCID), an inherited genetic disease.
This clinical trial, which included 11 patients, was put on hold in October 2002, after a first notification of a complication in one of the patients had been observed, consisting in an uncontrolled T-lymphocyte proliferation. The same complication has been reported for a second patient at the end of 2002. The hold was maintained until analysis and identification of the mechanism(s) responsible. One of the patients died last October, the other is progressively recovering. The clinical trial has been authorised to resume after the investigators proposed several protocol modifications aimed at reducing the risk of insertional oncogenesis. Since the restart of the clinical trial, one new patient has been treated. On January 18th, 2005, a new complication was notified to Afssaps. It concerns a third child who was 9 months old when receiving the treatment in April 2002.

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Cancer gene therapy is first to be approved in China

Posted on: 28 November 2003, source: New Scientist
For the first time, a gene therapy-based treatment has been given the go-ahead by regulatory authorities. China's medicines authority approved the cancer therapy after it achieved promising results in a clinical trial. The treatment, called Gendicine, will be launched commercially in January by SiBiono GeneTech of Shenzhen, Guangdong province. The results of the trial will be published in December in China's national medical journal (see also 'Gendicine'), says Zhaohui Peng, the company's founder and head, and he plans to translate the paper into English to submit to an international journal. Gendicine's approval was announced over a month ago but has gone largely unnoticed outside China.

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New cancer case halts US gene therapy trials

Posted on: 15 January 2003, source: New Scientist
Nearly 30 US gene therapy trials were halted on Tuesday following the announcement that a second child in a pioneering French gene therapy trial has developed leukaemia following the treatment. The French trial is testing a treatment for "bubble boy" disease, or X-SCID (X-chromosome-linked Severe Combined Immunodeficiency). The initial results of the trial were hailed one of the first great successes for gene therapy. But the trial was halted in October 2002 following the first diagnosis of leukaemia in one of the boys. Three similar US gene therapy trials were suspended at the same time. A similar trial in the UK was not halted, as British doctors argued that without the treatment many of patients would certainly die.

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'Miracle' gene therapy trial halted

Posted on: 3 October 2002, source: New Scientist
A "miracle" gene therapy treatment for children suffering from the fatal "bubble boy" disease has been halted in France, after one of the patients developed leukaemia as a direct consequence of the treatment. However, British doctors argue that without the treatment many of the patients are certain to die, and say a similar trial in UK will continue. Boys with X-SCID (Severe Combined Immunodeficiency) have a faulty copy of a gene on their X chromosome that makes an immune protein called interleukin-2. As a result, they have no resistance to infection and die unless treated. In 2000, a team led by Alain Fischer at Necker Hospital, Paris, carried out the first gene therapy treatment, which replaced the faulty gene. It was one of only a handful of successful gene therapy trials in people. In April 2002, the mother of a Welsh boy treated at Great Ormond Street hospital in London described his progress as "nothing short of a miracle".

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