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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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