How rare models suggest new treatment strategies

July 22, 2014 by Fiona Dunlevy
How rare models suggest new treatment strategies

Elucidating the many possible causes of a rare auto-immune disease called Myasthenia Gravis could help point towards possible treatment for such disease.

Myasthenia Gravis (MG) is a rare auto-immune disease—whereby patients' immune systems attack their own bodies— arising from a breakdown in communications between the nervous and muscular systems. The mistakenly makes antibodies that block signals in , where nerve impulses are translated into physical actions. An EU-funded project is investigating the causes of MG and ways to combat the disease. Here, Sonia Aknin-Berrih, project coordinator, who is also director of research in immunology leading a team on MG at INSERM, in Paris, France, talks to about her search for new treatments for MG.

How are patients with MG affected?

Muscles affected include those connected to the patients' eyes; they can no longer open their eyes correctly because the eye muscles are closed. MG also affects the legs and arms and patients experience fatigue. Sometimes the disease is even more severe when the respiratory muscles are affected and patients often need to go to hospital. Patients do not know when this respiratory crisis is going to hit and it makes them very anxious. MG affects their ability to do simple things like housework and many of them cannot work or lead a normal life.

What treatments exist already and what approaches are you taking develop new MG therapies?

The video will load shortly

There are treatments available to treat symptoms, but most have serious side effects. And because it is a chronic diseases, treatments have to be used over and over again, so there is a need to find better therapies.

As scientists, we try to better understand MG by finding and targeting the early events of the disease. We think an infection could be an early event in MG, but we do not know when that happens, which makes it is difficult to treat. We also see dysregulation of the immune system at different levels, so a therapeutic molecule that could act on the different problematic cells could be a very effective treatment.

Another treatment idea is to use stem cells that could target all the different cells of the immune system, which is more efficient than targeting only one kind. An alternative is to push immune system defence cells, known as T-regulatory cells, to be more numerous and effective. These cells would normally down regulate over-active immunity but they are defective in MG patients. There are also some molecular options using monoclonal antibodies, and we have described many .

Does the need for new MG therapies push you to be more innovative?

Yes, for sure. We are very much in contact with the patients' associations. We always keep in mind that they are waiting for new treatments. We have a responsibility—they count on us to find new therapies—so yes that makes us try to be more innovative. We are not only doing basic research, we also have contact with the patients. This helps us develop all kinds of models and test any hypothesis for potential treatment that looks interesting.

How soon could you have a treatment in clinical trials?

We have very good relevant models for testing potential treatments. Also, the molecules that we would like to use are already on the market. So if they are efficient in our model there is no reason not to try them on patients. I think that the first clinical trial could be done in less than 3 years. My research lab is in a very big hospital in Paris and we have a lot of collaboration with clinicians. Therefore, a clinical trial would be easy to organise because we already have access to over 1,000 MG .

I think we are in a good position and it should work, but you never know. Even if we meet all the conditions to have promising results, we have to test it to be sure.

Explore further: Network of experts join forces to fight myasthenia gravis

More information:

Related Stories

Network of experts join forces to fight myasthenia gravis

July 22, 2014

Collaboration between research groups is key in tackling rare diseases such as auto-immune disease myasthenia gravis (MG). Indeed, the rarity of the disease means that it can be difficult to collect enough samples of blood ...

Auto-immune disease: The viral route is confirmed

December 19, 2012

Why would our immune system turn against our own cells? This is the question that the combined Inserm/CNRS/ Pierre and Marie Curie University/Association Institut de Myologie have strived to answer in their "Therapies for ...

FDA approves generic version of celebrex

June 3, 2014

(HealthDay)—The U.S. Food and Drug Administration on Friday gave its approval to the first generic version of the pain reliever Celebrex (celecoxib).

Secukinumab effective in moderate-to-severe psoriasis

July 11, 2014

(HealthDay)—For patients with moderate-to-severe plaque psoriasis, the fully human anti-interleukin-17A monoclonal antibody, secukinumab, is effective, according to research published online July 9 in the New England Journal ...

Recommended for you

Bioelectricity new weapon to fight dangerous infection

May 26, 2017

Changing the natural electrical signaling that exists in cells outside the nervous system can improve resistance to life-threatening bacterial infections, according to new research from Tufts University biologists. The researchers ...


Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.