The challenges for research into leukodystrophies



Written on Friday 16 May 2014

Guy Alba and Christine Doublé, respectively President and Vice-President of the ELA Foundation’s Board of Trustees, answer ELA Info’s questions and give their views on the challenges for research into leukodystrophies.

 

 

 

 

 

Currently what are the requirements of researchers working on leukodystrophies?

Christine Doublé. Researchers need funding and to develop international collaborations. Sometimes they also need to be able to count on the cooperation of patients’ families. Leukodystrophies are rare illnesses and it’s essential to be able to pool and share the assets which help improve knowledge and progress towards the objective of comforting and improving the lives of patients and, of course, finding cures.
Guy Alba. I think above all there’s a lack of researchers interested in our subjects. And that’s obviously a limiting factor.

Medical research is evolving in leaps and bounds. But how do you consider it possible to stay in the vanguard of biomedical research into leukodystrophies?

C.D. We need to organize specific scientific days involving experts and put patients at the heart of clinical research.
G.A. I agree with Christine. But I don’t think research is progressing in leaps and bounds, particularly not in our area. The main reason for that is a lack of means and therefore of researchers. Perhaps there is also a lack of openness towards others.

In the complex field of leukodystrophies, which research fields would you judge to be of key importance?

C.D. We need to be able to identify all forms of leukodystrophies, speed up programmes of clinical tests which can slow down or stop the illness, promote and facilitate research to bring comfort to patients. All the ELA families need to be reassured that they aren’t isolated.
G.A. An association of families like ELA first has to look after the patients we are responsible for. As it’s a progressive illness, we need to prioritize research subjects which may bring benefits for our patients in the short- term while also lobbying the authorities so that they take responsibility for basic and longer-term research.

Access to new technological platforms is essential for researchers to help them keep up with the latest cutting-edge technological developments. Are you developing projects of that sort?

C.D. Currently we’re developing a technological platform at the University Hospital Centre in Nancy (north-east France). This innovative tool will associate biological samples and medical data to help improve research into leukodystrophies and gain better understanding of the disease’s natural history.
G.A. It also needs to help enable researchers identify new genes and create homogeneous groups of patients. The latter point is crucial to launch new clinical tests.

Young researchers represent the future of research. How do you imagine they can be supported so that research into leukodystrophies continues in the long term?

C.D. We need to drive and promote scientific collaborations and help enhance the visibility of quality researchers and their work.
G.A. We have to provide strong support for the teams which perform the best in our fields while maintaining high standards. In this way we can enable the selected laboratories to be better equipped, more efficient and therefore more attractive for young researchers.

How will make sure funded research projects provide applications for patients?

C.D. Researchers have to be given precise objectives and more clinical tests need to be funded. We can also promote more transparency on current research and increase exchanges between patients and researchers.
G.A. It’s the Scientific Council’s role to assess the relevance of research programmes and results and it’s up to ELA directors to take funding decisions. But the best assessment comes from the patients themselves.

What financial means are necessary to develop this scientific policy?

C.D. Money is the key. As Guy pointed out earlier, ELA can’t manage basic and long-term research alone. Also, while new technologies have accelerated research progress, they’ve also increased costs.
G.A. Obviously ELA can’t do everything. We can persuade the authorities to contribute more and why not also try to get private laboratories involved. This is already happening with gene therapy research for adrenoleukodystrophy (ALD).

How can the public help ELA achieve these goals?

C.D. We have to run more awareness-raising events where we can collect donations and also organize large-scale events for people to take part in which combine awareness-raising and collecting donations. It’s essential for these rare progressive illnesses not to be left in obscurity. It’s also a way of providing support for families to keep their hopes up.
G.A. Researchers achieving results for patients will make it easier to get the public involved. I’d like to say to them – help us to help you.