Sabrina Paganoni, MD, PhD: Collaboration in the CENTAUR Trial of AMX0035 in ALS

“This was a phenomenal example of that [collaboration in ALS]. There are many other groups that are interested in collaborative work, and…the ALS research landscape is particularly active and collaborative. This was the perfect example of that spirit of collaboration and dedication to people with ALS and their families.”

When it comes to the development of fatal diseases such as amyotrophic lateral sclerosis (ALS), there is a natural need for cooperation between the factions that make up the community. When researchers, pharmaceutical companies, awareness organizations, and others come together to improve the methods and assessment tools, the clinical development tends to improve.

For Sabrina Paganoni, MD, PhD, co-director, Neurological Clinical Research Institute, Massachusetts General Hospital, this collaboration was on display with the recent phase 2 CENTAUR trial of Amylyx Pharmaceuticals’ investigational agent AMX0035 (NCT03127514). And while the full results of the CENTAUR trial are expected to publish before the end of the year, she offered some insight to NeurologyLive in a recent interview.

Paganoni, the trial’s principal investigator, discussed the top-level findings of the sodium phenylbutyrate (PB) and tauroursodeoxycholic acid (TUDCA), combination drug, as well as shared her thoughts on the overall state of the ALS research landscape. She spoke to the need for more collaborative efforts such as this, and how combining these efforts can give studies and research a leg up.