What is ALS?

In 1874, Jean-Martin Charcot coined the term amyotrophic lateral sclerosis (ALS). He used this to label the neurological cause of the now well-known symptoms of the disease.

Initial symptoms of ALS include stiffing, weakening, and twitching of muscles, including those used for speaking swallowing and even breathing.

Eventually the patient loses the ability to move, speak or swallow. Death typically follows when they are unable to breathe.

ALS is a disease of a specific group of neurons in the brain. These are the motor neurons which communicate with voluntary muscles. These motor neurons allow us to move, speak, breathe and eat.

In patients with the disease, the motor neurons die. After that, without the ability to use the muscle, it will eventually atrophy.

Lou Gehrig’s Disease

In 1939, the disease was given an informal name when famous major league baseball player Lou Gehrig was diagnosed with ALS.  

Perhaps most notably it was Professor Stephen Hawking who brought ALS into the worldwide spotlight. One of the greatest minds or all time, Hawking was diagnosed in 1963.

Search for a Cure

There is currently no cure for ALS. Research has focused on treatments.

The ALS gene was identified in 1993.

In 2014 the ‘Ice Bucket Challenge’ put a focus on raising more money to find a treatment.

At long last, it appears that a successful ALS treatment may have arrived.

Amylyx Pharmaceuticals recently published results from their CENTAUR trial evaluating AMX0035. The drug trial is an investigational neuroprotective therapy. Its goal is to reduce the death and dysfunction of motor neurons.

The CENTAUR trial of AMX0035 was a 24-week, randomized, double-blind clinical trial which wrapped up at the end of 2019.

According to the results, the trial achieved its goal of showing a meaningful and significant benefits in ALS patients.

“These results represent a major milestone for the ALS community, and I am thrilled about the promise of this therapy for people with ALS.” said Sabrina Paganoni, M.D., Ph.D., principal investigator of the CENTAUR trial.

137 people participated in the trial across 25 hospitals. Those who received the drug showed a substantial decrease in the progression of the disease compared to those receiving a placebo.

Full details about the drug and the clinical trial can be read online at: https://www.amylyx.com/2020/09/02/amylyx-pharmaceuticals-announces-new-england-journal-of-medicine-publication-of-pivotal-amx0035-data-demonstrating-statistically-significant-benefit-in-people-with-als/.

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