Ethical questions are crucial, but they shouldnt stall the progress of this promising branch of medicine
In late November, Reuters reported a milestone in medical history: a gene therapy drug could go on sale in Germany next year, after winning the approval of European regulators two years ago. The drug, Glybera, by a Dutch firm called UniQure currently being scrutinised by Germanys federal joint committee would be the first commercial use of gene therapy in the Western world (China has had a gene therapy drug for a specific form of cancer in the market since 2004). This marks a potential turning point in an area of medicine that has been the subject of highs and lows over more than two decades of clinical trials.
Gene therapy which can involve a number of things, including replacing a malfunctioning gene or introducing a new gene with the ability to fight a disease has been in conceptual development for far longer. Its origins could be said to go back as early as the 1920s, well before the discovery of the structure of DNA, when a British scientist, Frederick Griffith, put forward what he described as the transforming principle; he successfully converted a non-virulent strain of bacteria into a virulent one, after injecting mice with both.
From the late 1960s, when the concept of gene therapy began to involve, it took several decades for the first clinical trial to take place in 1990. A young girl in the US with a genetic defect that had left her with a severely weakened immune system was successfully injected with her own white blood cells containing a corrected form of the malfunctioning gene.
However, the boost gene therapy got following that first successful trial was soon tarnished, in the view of the public, by a tragedy in 1999; an 18-year-old American boy, who had a mild version of a liver condition, which meant his body couldnt process ammonia, died during a gene therapy treatment. This was after a massive response by his immune system to the vector or carrier used to introduce the corrected gene.
The episode raised a number of issues including that of informed consent of those participating in clinical trials as well as the fact that identifying and correcting a defective gene was far from the only challenge facing gene therapy. Selecting the appropriate vector was also vital and not without risks.
Despite predictions that gene therapy would be lastingly damaged by the tragedy, research and trials continued with many promising results for a range of conditions ranging from immune system conditions to cancer, cystic fibrosis, Parkinsons disease and hemophilia.
The renewed confidence in gene therapy is highlighted by the fact that the worlds largest pharmaceutical companies have also entered the market (earlier this week, Pfizer announced collaboration with Spark Therapeutics, a Philadelphia based company on the development of a hemophilia B treatment).
Over 1,700 approved gene therapy trials have taken place in the past two decades, estimated an article on the history of gene therapy in Gene magazine last year with many successes and a few hits. Among the latter were trials conducted in France in 2001 on Severe Combined Immunodeficiency, a condition where the immune system is so crippled that in one case it required a boy to live in a germ-free bubble. Several infants involved in the trial subsequently developed leukemia, though other clinical trials for gene therapy since have been successful.
There have been some understandable public concerns about gene therapy and its impact on the one hand it offers that tantalising potential of curing some of the most lethal conditions while on the other, tampering with genetic makeup is something that has long conjured up fears in the public imagination of genetic engineering and exacerbating discrimination against those with disabilities and disease.
Original post:
Gene therapy makes a slow comeback
- Gene Therapy: Ethical Issues: Information from Answers.com - February 6th, 2014 [February 6th, 2014]
- Gene, immune therapy help cancer war - April 10th, 2014 [April 10th, 2014]
- Gene Therapy - Genetics Home Reference - Your guide to ... - April 10th, 2014 [April 10th, 2014]
- What is gene therapy? - Genetics Home Reference - April 10th, 2014 [April 10th, 2014]
- Gene therapy - Wikipedia, the free encyclopedia - April 10th, 2014 [April 10th, 2014]
- Gene Therapy PSA Dan Lesser Period 2 - Video - April 15th, 2014 [April 15th, 2014]
- Gene therapy basics - Video - April 15th, 2014 [April 15th, 2014]
- In vivo gene therapy - Video - April 15th, 2014 [April 15th, 2014]
- Ex vivo gene therapy - Video - April 15th, 2014 [April 15th, 2014]
- Broad Institute Gets Patent on Revolutionary Gene-Editing Method - April 17th, 2014 [April 17th, 2014]
- Gene Therapy - Video - April 17th, 2014 [April 17th, 2014]
- Mark Hamalainen: Gene Therapy - Video - April 17th, 2014 [April 17th, 2014]
- JFK - Gene Therapy for Brain Tumors - Video - April 18th, 2014 [April 18th, 2014]
- Fixing the Liver with New Gene Therapy (Brainstorm Ep178) - Video - April 18th, 2014 [April 18th, 2014]
- Gene Therapy | Buzzle.com - April 22nd, 2014 [April 22nd, 2014]
- Gene Therapy I - RCN Corporation - April 22nd, 2014 [April 22nd, 2014]
- Ricky Ashby Microbiology Podcast - Video - April 22nd, 2014 [April 22nd, 2014]
- Regrown nerves boost bionic ears - April 24th, 2014 [April 24th, 2014]
- Cochlear Implant Plus Gene Therapy Could Restore Hearing to the Deaf - April 24th, 2014 [April 24th, 2014]
- Scientists reverse memory loss in mice with Alzheimer's - April 24th, 2014 [April 24th, 2014]
- Bionic Ears Boosted by Gene Therapy and Regrown Nerves - April 24th, 2014 [April 24th, 2014]
- Study: Gene Therapy May Boost Cochlear Implants - April 24th, 2014 [April 24th, 2014]
- Gene therapy may boost hearing, study finds - April 24th, 2014 [April 24th, 2014]
- Pronounce Medical Words Gene Therapy - Video - April 24th, 2014 [April 24th, 2014]
- Gene therapy may boost power of cochlear implants, study says - April 25th, 2014 [April 25th, 2014]
- Gene therapy shows promise to help people regrow auditory nerve cells - April 25th, 2014 [April 25th, 2014]
- Cochlear Implant Also Uses Gene Therapy to Improve Hearing - April 25th, 2014 [April 25th, 2014]
- Gene Therapy May Enhance Cochlear Implants, Animal Study Finds - April 25th, 2014 [April 25th, 2014]
- Uniqure Gene Therapy Info. - Video - April 25th, 2014 [April 25th, 2014]
- Jean Bennett on gene therapy as a treatment for blindness - Video - April 25th, 2014 [April 25th, 2014]
- mice-science-AFPrelax-250414.jpg - April 26th, 2014 [April 26th, 2014]
- Cochlear implant enhances patient experience through gene therapy - April 28th, 2014 [April 28th, 2014]
- Gene therapy may help hearing - April 28th, 2014 [April 28th, 2014]
- Ethics of Gene therapy - Video - April 30th, 2014 [April 30th, 2014]
- Novel Financing For Gene Therapy Company - May 1st, 2014 [May 1st, 2014]
- Gene Therapy To Enhance Hearing - ABC Austrlia News - 2014 - - Video - May 1st, 2014 [May 1st, 2014]
- Biotechnology - GMO's & Gene Therapy - Video - May 1st, 2014 [May 1st, 2014]
- Cochlear Implant Gets an Upgrade with Gene Therapy - Video - May 4th, 2014 [May 4th, 2014]
- Gene therapy for haemophilia - Professor Amit Nathwani - Video - May 4th, 2014 [May 4th, 2014]
- Retrovirus in Gene Therapy - Video - May 4th, 2014 [May 4th, 2014]
- Regeneron, Avalanche To Develop Novel Gene Therapy Products In Ophthalmology - May 5th, 2014 [May 5th, 2014]
- Gene Therapy Used to Preserve Sight in Patients - May 5th, 2014 [May 5th, 2014]
- SCID gene therapy class - Video - May 6th, 2014 [May 6th, 2014]
- Gene Therapy using SMaRT - Video - May 6th, 2014 [May 6th, 2014]
- gene therapy video - Video - May 9th, 2014 [May 9th, 2014]
- Chronic Granulomatous Disease (CGD) - Video - May 12th, 2014 [May 12th, 2014]
- Gene Therapy (Breast Cancer) - Video - May 12th, 2014 [May 12th, 2014]
- Suicide gene therapy - Video - May 12th, 2014 [May 12th, 2014]
- Employment of gene therapy in depression (psychiatric disorder) - Video - May 12th, 2014 [May 12th, 2014]
- Gene therapy for blindness (2012) - Video - May 12th, 2014 [May 12th, 2014]
- AGRC1020 Video Presentation Gene Therapy restores colour vision in monkeys - Video - May 12th, 2014 [May 12th, 2014]
- gene therapy~3 - Video - May 12th, 2014 [May 12th, 2014]
- Gene Delivery System (In-vivo and Ex-vivo) HD - Video - May 12th, 2014 [May 12th, 2014]
- Spark Therapeutics to open headquarters in West Philadelphia - May 14th, 2014 [May 14th, 2014]
- Gene Therapy - Nature - May 14th, 2014 [May 14th, 2014]
- Gene therapy for RTT syndrome benefits and drawbacks - Video - May 16th, 2014 [May 16th, 2014]
- Gene Therapy (Using Biolistic gene gun for gene delivery) - Video - May 16th, 2014 [May 16th, 2014]
- Germline Gene Therapy - Video - May 16th, 2014 [May 16th, 2014]
- Gene Therapy Biology Project for Swavely - Video - May 16th, 2014 [May 16th, 2014]
- Gene therapy extends survival in an animal model of spinal muscular atrophy - May 22nd, 2014 [May 22nd, 2014]
- Novel RNAi therapy silences mutated Huntington's disease gene and reduces symptoms - May 22nd, 2014 [May 22nd, 2014]
- Global Gene Therapy Market: Analysis, Size, Share, Growth, Trends and Forecast 2013 - 2019 - May 22nd, 2014 [May 22nd, 2014]
- Gene therapy hits blood cancers hard - May 22nd, 2014 [May 22nd, 2014]
- Spark Therapeutics late-stage gene therapy for inherited blindness takes a $73M step forward - May 31st, 2014 [May 31st, 2014]
- Genome Editing to Reverse Bubble Boy Syndrome - May 31st, 2014 [May 31st, 2014]
- Genetic Researching: Gene Therapy - Video - May 31st, 2014 [May 31st, 2014]
- BEYOND THE BUBBLE: 10 Years of Gene Therapy - Video - May 31st, 2014 [May 31st, 2014]
- Gene Therapy Combined with IMRT Found to Reduce the Rate of Positive Prostate Biopsy After Treatment for Intermediate ... - June 4th, 2014 [June 4th, 2014]
- Gene therapy combined with IMRT found to reduce recurrence for select prostate cancer patients - June 4th, 2014 [June 4th, 2014]
- Gene therapy combined with IMRT reduces rate of positive prostate biopsy after treatment for intermediate-risk ... - June 4th, 2014 [June 4th, 2014]
- Bluebird, Agios: Preview of Next Week's Important Pipeline Updates - June 12th, 2014 [June 12th, 2014]
- Attack of the B - Team EP8 - Drowning for Science! - Video - June 12th, 2014 [June 12th, 2014]
- 3rd International Conference and Exhibition on Cell & Gene Therapy - Video - June 14th, 2014 [June 14th, 2014]
- Blood Disorder Patients Respond Strongly to Bluebird Bio Gene Therapy - June 15th, 2014 [June 15th, 2014]
- Gene Therapy Partnering Terms and Agreements - Video - June 15th, 2014 [June 15th, 2014]
- Gene Therapy - GMAT Free - GFSC120 - Video - June 18th, 2014 [June 18th, 2014]
- New Gene Therapy Rapidly Helps Patients With Rare Blood Disorder - Video - June 18th, 2014 [June 18th, 2014]
- Isis Could Get a $2 Billion Gain From Heart Attack Trials - June 22nd, 2014 [June 22nd, 2014]
- Gene Therapy for Beta Thalassemia - Video - June 22nd, 2014 [June 22nd, 2014]
- A level. R.7. Cystic fibrosis and gene therapy (Ms Cooper) - Video - June 24th, 2014 [June 24th, 2014]