The 69th American Academy of Neurology Annual Meeting

What Are The Unmet Needs in ALS Research?




At AAN 2017, Merit Cudkowicz, MD, MSc., Chief of Neurology, Massachusetts General Hospital explained that ALS has changed dramatically in the last decade. “There’s so much more hope and more knowledge about what might be happening,” Cudkowicz told MD Magazine. She said that there are new foundations including the ALS Finding A Cure that aims to accelerate finding a cure. Also, according to Cudkowicz, the “Ice Bucket Challenge” really made a huge difference in awareness; people across the globe now know what ALS is.
 
Cudkowicz pointed out that researchers previously only considered ALS as one illness, but new knowledge has changed that mentality and has shown experts they need to personalize treatment approaches. “It’s important to understand the disease in different groups of people.” For instance, some patients may have a lot of inflammation that might be driving their illness, so physicians would target drugs that would act against neuro-inflammation. Whereas hyperexcitability might be a cause in other patients in whom the motor neurons are fighting too much.
 
When discussing patient demographics, Cudkowicz explained that ALS doesn’t happen in children; the typical age is in the mid-50s. It can start as young as in the 20s (like the person who started the ALS bucket challenge) and can also happen to people in their 90s.
 
Regarding treatment, there are currently two FDA approved drugs: Riluzole (Sanofi) and Nuedexta (Avenir). There is also a third drug that is approved in Japan called Edaravone (Mitsubishi Tanabe Pharma) currently under review with the FDA – Cudkowicz hopes to hear results by June. Additionally, there are some drugs coming out of Europe that look positive.
 
Despite the positive news surrounding ALS research, Cudkowicz believes there are still many unmet areas. “We need to be diagnosing and treating patients much earlier.” New research has shown that on average, it takes – from the very first symptom to when someone is told he has ALS – 12 months to diagnose and treat patients. For some people, that’s a third of their illness for which they are not getting any care and treatment.  “We’re really focused on how we do we use technology, imaging, or big data to diagnose people earlier so we can give treatment.”
 
Cudkowicz concluded that experts in the field don’t know how to visualize the illness. In multiple sclerosis, for example, physicians can do the MRI and can see the illness, so they can see whether the treatment is working. “We don’t have a tool to actually see ALS, so we’ve partnered with GE healthcare to develop advanced imaging for ALS of the spinal cord and the brain, and so far the data looked pretty hopeful. That should really speed up both the diagnosis and drugs.”


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