Thomas Voit , Scientific Director at the Institute of Myology, gives his perspective of the past year and of course what’s to be expected in 2010.

An interesting and eventful year has just ended, what is the forecast for 2010?
The year 2009 proved to be difficult from an administrative and logistical point of view, however, scientifically we have had an extraordinary year! We published over 80 articles in leading journals, we submitted 7 patents, not to mention the important discoveries with patents pending and which have not yet been published. So the first goal for 2010 will be to publish these findings when they will be patented and the second is to complete the pending patents. The third goal will be to invest more in research units: for example, with Martine Barkats' arrival at the Institute. Another example: Sonia Berrih-Aknin and her team, who work on myasthenia, are about to join U974, but we still need to find premises! We will try everything to make the most of the synergies between these teams. The beneficial effect of this proximity is already tangible and we want to continue working with this quality and pace.

What are the themes of these discoveries under patent?
They concern possible treatment strategies for Duchenne muscular dystrophy, spinal muscular atrophy, Becker muscular dystrophy, dysferlin deficient limb girdle muscular dystrophy and myotonic dystrophy. At the experimental level, these diseases are becoming increasingly accessible to molecular or pharmacological treatment. The major problem is the implementation of such therapeutic approaches because it takes time, it is very expensive and it often exceeds the capacity of a research unit. The mission of the unit is to find such leads. Afterwards, the research unit is only partially concerned with the development; other forces are necessary to progress.

And clinical trials?
There are many, for example the DELOS trial from Santhera investigating the effect of idebenone in Duchenne muscular dystrophy patients. This trial will begin in the upcoming weeks at the Institute, the physiotherapists who will carry out the force measurements are completing their training. Regarding Prosensa’s PRO51 trial in DMD, Phase III will be launched this quarter. We suggested to Prosensa that the trial will physically take place at Hospital Necker and the evaluations will be conducted at the Institute. Another example: we are in the extension phase of the PTC 124 trial, so all of our patients were switched to the high dose in an open study. The results should be available in March. But there are also negotiations for the establishment of new trials with AVI BioPharma (skipping of exon 51 with morpholinos in wheelchair-bound DMD boys), or with Acceleron (anti-myostatin antibodies in patients with DMD and FSH), amongst others.

Do you think that the results of the Telethon 2009 will affect projects developed at the Institute?
First of all I think, and this is my personal opinion, that the Telethon 2009 was a great success given the general conditions, the economic crisis and also the controversy. I am not sure if the result will really have an impact on the AIM, even if the basic support of the AFM is essential. Concerning the research units, we also have a few other sources of funding; in any event, it is up to the AFM Administrative Council to decide upon the priority of projects.

So for 2010...
The overall project of the Institute is very promising, but as always, when part of a dynamic growth, we must now find the right scientific and relational balance. I hope that we can continue in this direction, and in particular, continue to develop innovations like we did last year. Innovation requires very good heads... we have them, but they must be put in the right environment and this is where we still have a lot of work to do!
Finally, I would like to say that collaborations with the AFM-associated institutes, Genethon and I-stem, as well as with Nantes and Marseille in particular, have intensified and yielded a fair number of very positive results. Such a collaboration in complete confidence is a real pleasure.

January 2010