ALS, also known as Lou Gehrig’s Disease or Motor Neuron Disease, is a chronic progressive neurodegenerative disease that affects people worldwide, with approximately 20,000 in the U.S. alone. The majority of ALS cases (~90%) are sporadic. However, ~10% of ALS cases are caused by genetic mutations.
Several genes have been discovered that are causative for ALS, including the gene encoding the sigma-1 receptor (S1R).
In patients with ALS, motor neurons in the brain and spinal cord that convey messages to the muscles degenerate, affecting the brain’s ability to communicate with muscles.
This leads to muscle wasting and progressive paralysis. Patients rapidly lose their ability to walk, speak, eat and breathe and become fully dependent on their caretakers.
ALS is a devastating disease, for patients, their families and caretakers.
Pridopidine for ALS
Pridopidine is currently being assessed in the first-ever Platform trial for the treatment of ALS.
The Healey ALS Platform Trial aims to accelerate the development of effective treatments for people living with ALS, by testing and evaluating multiple treatments simultaneously. The trial is designed to share placebo groups thus decreasing the number of placebo participants to be recruited and hastening the conduct of the trial.
Pridopidine is a promising drug candidate for the treatment of ALS for several reasons:
Pridopidine activates sigma 1 receptor and improves motor neurons survival and muscle function in animal models of ALS.
Recent data show that some individuals with decreased or loss of function in the sigma 1 receptor develop ALS.
Pridopidine is an oral drug with an established safety profile.
Pridopidine demonstrated potential clinical efficacy in maintaining total functional capacity in HD patients. Many aspects of functional capacity are shared between HD and ALS.
These findings highlight the therapeutic potential of pridopidine for treating ALS. In addition, Prilenia holds Orphan Drug Designation for pridopidine in ALS in both the U.S. and Europe.
For all these reasons, pridopidine was selected by the Sean M. Healey & AMG Center for ALS at Mass General Hospital as one of the top drug candidates to be included in the HEALEY Platform trial.
Pridopidine was chosen by an independent review committee out of 30 competing investigational treatments. The trial is currently active in the United States and has completed enrollment.
Learn more about pridopidine for ALS: