Martine Barkats Q & A

Our next SMA scientist, Martine Barkats from the Institut de Myologie (Paris, France), is one of the pioneers of using virus-mediated SMN protein delivery as a potential therapeutic for SMA.

When and why did you first decide you wanted to be a scientist?

I initially studied Biology at university with the hope of pursuing a career in teaching. However, during my Masters degree, I took some courses in neuroscience that I found fascinating and which convinced me that a career in neuroscience research was for me. This then led to me doing a PhD in neuroscience, followed by a post-doc working on genetic therapies for diseases of the central nervous system. This type of research was cutting edge at the time (in 1994) and is still an area of great promise for many currently incurable diseases.

How did you come to work on SMA?

In 2004, I joined Genethon (Evry, France), a non-profit organization funded by the Association Française contre les Myopathies (AFM), which is dedicated to rare disease research. I chose to work on these so-called “orphan diseases” because they are often neglected by the pharmaceutical industry as they affect relatively too few patients to make money. I came to work on SMA after meeting the mother of a ten year old girl, Marie, affected by this terrible disease, and who had just lost the ability to walk. I was touched by this particularly moving case and my research experience in a similar field (amyotrophic lateral sclerosis - ­ an adult onset lower motor neuron disease) convinced me of the contribution that I could make in this area.

What would you be if you weren’t a scientist?

I would have loved a job in the humanities, or maybe something to do with music - something nothing to do with science or medicine!

If you are not in the lab, you are...

Research keeps me very busy, but when I have a few hours of free time, I enjoy singing and playing the guitar, I take care of my plants (I love gardening!), I read and watch good films, and there is nothing better than watching my son grow up to be a man.

Describe yourself in three words

Curious, combative, altruistic.

What has been the most important moment of your career so far?

The day I saw that the first SMA mouse treated by our virus-based gene therapy system survived well beyond the normal 13 day lifespan caused by reduced SMN levels... It was the very first time that these mice survived significantly without any symptoms. It was an extraordinary moment.

What is your most memorable finding relating to SMA?

The same one!

What is your favourite conference location?

Personally speaking, anywhere sunny…! But, professionally speaking, I love small and focused conferences such as those at Cold Spring Harbor Laboratories, and the SMA Trust and Jennifer Trust (now Spinal Muscular Atrophy Support UK) conference in the UK. I also really enjoy the annual conference organised by the families of SMA in the US, which is larger but exposes you to a lot of up to date information on SMA.

What is the best scientific advice you ever received?

I used to read a lot when I was a PhD student and at the beginning of my post-doc, and one day, my boss (Jacques Mallet) told me “it's good to read, but you know, there are people who read science and those who do it”. Today, I still read a lot but I read more selectively and I'm more into the action...!

If you could start your scientific career all over again, are there things you would do differently?

I would probably study medicine before doing a PhD in science.

In your opinion, what makes a good scientist?

Creativity, rigor, curiosity, and perseverance…but not only one of these qualities. I also think that it is very important for lab heads to regularly encourage and motivate their team.

Where do you see the SMA research field In the next 10 years?

We will have had a number of years of successful clinical trials, and we will have gained a comprehension understanding of the role of the SMN protein... and my hope is that we will no longer need SMA research in 10 years.