New Promising Neurological Treatment Involves ALS in Phase 2 Clinical Trials

Amyotrophic Lateral Sclerosis (ALS) is a neurological disease that affects 400,000 people worldwide. It is characterized by progressive loss of motor function caused by the decay of motor neurons. There are only two approved treatments for ALS, Riluzole and Edaravone, and they both have short lasting results.  

With no other treatment options available, alternative therapies need to be considered. A recently published article proves the benefits of innovative therapies. They evaluate the benefits of a drug that is being tested to treat Alzheimer's Disease, called PXT864, on ALS patients.  

This potential oral therapy drug, PXT864, designed by Pharnext prevents the loss of motor neurons and prevents toxic protein clumps from building up within the neuron. The Alzheimer drug PXT864 displayed promising results in its Phase 1 trial and is moving on to Phase 2. Because Alzheimers and ALS are closely related, Phase 2 trials are planned for ALS as well. 

The drug has a greater effect when used along with Riluzole, one of the two FDA (Food and Drug Administration) approved treatments for ALS. Also, when PXT864 is exposed to neurons from rat models it increases the survival and maturity of motor neurons.  

These discoveries and more mark PXT864 as a potential therapy for ALS in the future. These in lab results highlight the importance of using PXT864 alongside previously existing treatments to help fight life threatening diseases like ALS.