As the Telethon calls for the generosity of the French during a 30-hour marathon on December 8 and 9, 2023, we invite you to discover the medical advances that donations from the French have made possible. The dream of curing certain genetic diseases is becoming a reality, thanks to the success of the first gene therapies.
When the Telethon was launched in 1987, the idea of curing certain genetic diseases was only a distant goal. Today, as the 37th edition of this fundraising event is held on December 8 and 9, 2023, the dream of bringing treatments to sick children is beginning to come true.
Since 2018, the Genethon, which is the laboratory of the AFM-Telethon association, has made groundbreaking advances in understanding and treating previously incurable rare diseases.
Today, 29 gene therapies are available worldwide, and medical research in this field is booming. Thanks to the €90.8 million raised by the Telethon in 2022, 38 clinical trials for 29 different diseases were able to be funded by the AFM-Telethon in 2023.
Since 80% of rare diseases are genetic in origin, gene therapy is the most promising avenue for halting the progression of these diseases or even curing them. It involves replacing a deficient gene by administering a piece of DNA to patients.
Among the first successes in this field, thanks in part to donations from Téléthon, it is important to know that more than 3000 babies suffering from severe spinal muscular atrophy have been treated with a gene therapy developed by Généthon. While children affected by this disease had a life expectancy of less than 2 years, they can now live normally thanks to an injection received in the first weeks of their life.
Spinal muscular atrophy is just one example of the diseases that can now be treated with gene therapies. These revolutionary therapies also make it possible to treat 'bubble babies' who lack immune defenses, as well as myotubular myopathy, another rare disease that was often fatal before the age of 2.
On the occasion of the Telethon, it is also important to remember that research on genetic diseases and rare diseases benefits other diseases and patients.
Research on gene therapy has particularly helped to better understand cellular dysfunctions. These are scientific advances that can be applied to other diseases such as cancer or neurodegenerative diseases like Parkinson's.
Gene therapy is also a promising avenue in the treatment of certain vision diseases such as Leber's hereditary optic neuropathy. An ongoing clinical trial allows for the halting of the process that leads patients to blindness within a few months. In ophthalmology, these innovative therapies are also being tested on a more common disease: AMD (age-related macular degeneration).
Even though the victories achieved in the field of gene therapy are incredible, AFM-Téléthon needs your generosity this weekend more than ever! Out of more than 7,000 existing rare diseases, 95% still have no treatment.
The challenge is huge, but new medical breakthroughs are anticipated and hoped for by researchers as well as families. Téléthon leads 200 research programs annually in the hope of developing new therapies.
Gene therapies are shaping the medicine of the future, but for now, they are very expensive. In this field, the average cost of developing a drug exceeds one billion euros. That's why Généthon is once again counting on the mobilization and generosity of donors this year.
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