It is moving towards a multi says Bruno Dubois

In announcing Tuesday the discovery "of a new method for the treatment of Alzheimer's disease", Etienne-Emile Baulieu has taking the risk to back a part of the scientific community, full of many associations of sick disappointed by an "announcement effect". The team of the Inserm he leads has in fact opened a trail where everything remains to be done. This conceptual breakthrough targets a form of tau protein, naturally present in nerve cells. In some circumstances, this molecule detaches the media on which it circulates within neurons (microtubules). Leaving these biological rails, it produces aggregates "in the form of Bush" that cause asphyxia of neurons.

While recognizing that scientists "do not like to make temporal predictions", the French specialist of steroid hormones believes that this solution, currently at the stage of testing in vitro, "could enter into clinical trials in two or three years". To achieve this goal, he relies on Pierre Bergé, who has pledged to support the project for at least five years. "It must be between 1 and 2 million euros per year for three years". "This will go fast because the molecule that we use is part of a well known family," said Etienne-Emile Baulieu. But specialists of the neurodegenerative diseases do not share this point of view. According to Françoise Forette, Director of the Foundation France Alzheimer's, "the calendar is a little optimistic." For Bruno Dubois, expert of these pathologies INSERM, "it is a new path yet little studied". In other words: caution.

Optimistic schedule

Currently, about 70 molecules are in development in the laboratories of industry (phases 2 and 3 only). The majority of these drug candidates is the other identified cause of Alzheimer's disease: amyloid plaques formed between neurons. Among the rare molecules in development targeting tau is yet "Rember", of the firm Singapore Taurx Therapeutics. This aggregation inhibitor is currently in phase 2 (efficacy trials). As a general rule, it takes a minimum of ten years between the time where a new molecule leaves the bench of academic research to obtain the authorization issued by the agencies of the drug (FDA, Emea, Afssaps) market.

The team of the Inserm has indeed highlighted the protective effect of a natural protein: FKBP52. "She clings to the pathogenic form of tau and inhibits its action." "The relationship between these two proteins in the brain will also measure the risk of an individual factor to develop Alzheimer's disease", said Etienne-Emile Baulieu. Now is to find another (available in the existing pharmacopoeia) molecule as a pacemaker for the FKBP52. "We already have three or four ideas in view," said Etienne-Emile Baulieu. Tests in animals should continue at the Pitié-Salpêtrière hospital, in Paris. Other side of the coin, the pharmaceutical industry is rarely drawn by rights free natural molecules that do not allow to build an industrial strategy, because they are not patentable.

In theory, this "anti-tau" approach could find applications in many diseases. "Several neurodegenerative diseases have in common the tau protein abnormalities", explains Professor Baulieu. In the meantime, the portfolio of drugs against the my remains limited to 4 treatments whose effectiveness is seen as "moderate" by specialists. In the next two years, the therapeutic arsenal should grow two or three new (see illustration). For many experts, truly effective treatment should be available in less than ten years. "This is probably not a single drug." "It is moving towards a multi", says Bruno Dubois.