The 23eédition of the Telethon which begins today follows a week of controversy about the legitimacy of this event to remove "both" of money for a class of patients "if large." The criticism is not new, but the trial to support is poorly educated, because the search strategy and development of the French Association against myopathies just always focussed to avoid through which he is charged. And the spectacular results that it comes in several of its research programs illustrate the principles.
"In the first place, the AFM, if it represents the health of its members interests, do not hesitate to make manna from the Telethon of other"forgotten"suffering from rare diseases, or even not at all rare, by funding research programs whose applications are broad", observes Serge Braun, Scientific Director of the AFM. This is the case for example of the work of the team of Marc Peschanski, who managed to reconstruct the whole of a skin from human embryonic stem cells. Announced on November 20, the first will allow patients with genetic diseases affecting the skin, but also to burn or people suffering from ulcer disease related to diabetes to be relieved and more quickly. For more than two decades already, doctors used in cell culture, to rebuild the epidermis destroyed from a small sample of skin from the patient, but the process was long (several weeks). They have now at the outset of the surfaces of the skin necessary.
A lock released
Another principle of AFM research strategy: create or improve technology tools that will be used to its patients, but many also others. Another dramatic announcement of the month of November, the success of a gene therapy trial that led to stop the evolution of a fatal neurodegenerative disease, the adrenoleucodistrophy in two young patients, is a good example.
"After years of disappointment, it seems that one of the major technological locks that made this ineffective approach has been unblocked", says Patrick Aubourg, co-author of the first. It is indeed, development by Saint-Vincent-de-Paul and teams of the Genethon of a new vector derived from a virus of the family of AIDS led to transfer sufficient functional copies of the faulty gene and achieve these results. "But this vector will be able to be used in other hereditary diseases, handled so far by marrow transplant, notes Patrick Aubourg." And, more generally, it is the gene therapy approach all confused applications which will benefit from a renewed interest.
Open a new path of research for the understanding of a disease or of a physiological mechanism, even if then the relay for wider developments, is also among the principles of the AFM. "Clinical trial started in November 2008, at the hospital la Timone in Marseille to test the combination of two molecules for the treatment of the progeria, fits in this context", explains Serge Braun. The progeria is a very rare disease (25 cases in Europe) which translates into an accelerated ageing as early as the age of twelve to twenty-four months and a 13-year life expectancy. The genetic origin of the disease has been elucidated and also identified a dozen other hereditary illnesses related directly or indirectly to the same mutations that the clinical trial will be instructive. The OM team also showed that patients with AIDS treated with antiretroviral accumulation of toxic proteins similar to those characterizing the progeria and now prepares a clinical trial to try to preserve the secondary effects of triple AIDS patients.
Technological Paris
Finally, the mechanisms involved in the progeria have strong similarities with the normal mechanisms of aging. The progeria can thus play the role of "disease model" for all those who work on ageing, the great "disease" of developed countries.
"Finally, said Serge Braun, the AFM does not hesitate to take risks by helping some emerging technologies to their evidence.Do not rely on the pharmaceutical industry to fund bearing technologies as hopes and uncertainties than exons jumping or genomic surgery because the meganuclease.
The distribution of the budget of research of the AFM (EUR 60.5 million in 2009), which is assigned to height 50 gene therapy and 6.7 of Cellular therapy, reflects the magnitude of the bet is made. Only patients together in an association are capable of taking informed of such risks.
He tells the genuine tragedy of Sand CreekYou want better control over public spending And that someone is me was it custom to saythey have finally been simply reprimandedFor the French Edition winter is far from the fallWe are asked to vote for a still virtual territorial AdviserOnly 13 of them received a stock or free shares this year heremille et un imponderables disrupt its daysTwentyfive years twentyfive million deathsBercy is very firm on the merits but does not exclude some arrangements
