The National Institutes of Health on Thursday said it will provide funding for three clinical trials of experimental ALS drugs, part of a broader push by the federal government to support development of treatments for rare neurodegenerative diseases.
One study, run through a collaboration between Columbia University and the Utah-based biotechnology company Clene Nanomedicine, has been awarded a four-year grant totaling $45.1 million. The grant will support a so-called expanded access protocol designed to test a Clene therapy that’s comprised of drinkable gold nanoparticles and meant to protect nerve cells.
Also known as compassionate use, expanded access is a regulatory method that allows patients with serious or life-threatening diseases to receive experimental treatments outside of typical clinical trials. In the case of Clene’s drug, the new study will enroll 100 participants with ALS, or amyotrophic lateral sclerosis, and develop “novel tools patient recruitment to increase access for people traditionally underrepresented in clinical research and expanded access programs.”
Another of the NIH-funded studies will be led by researchers at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital. It is set to recruit 200 ALS patients across 45 clinical sites in the U.S. and evaluate a small molecule drug from Prilenia Therapeutics that’s designed to activate a protein expressed in motor neurons.
Both Clene’s and Prilenia’s drugs have already been tested in a high-profile, first-of-its-kind “platform” trial run by the Healey Center. While neither hit the main goals of the trial, Clene and Prilenia have each identified some positive results that they say could support developing the drugs further.
The Healey Center and MassGen will also be working on the third study receiving NIH funding. Through a three-year grant, researchers there will investigate a cell therapy from Rapa Therapeutics. The expanded access protocol has a target enrollment of 40 people, and focuses on patients with reduced pulmonary function who otherwise would not be eligible for ALS trials.
The trial will contribute to the “ultimate goal of improving respiratory function and survival in this particularly high-risk ALS patient population that has essentially no other treatment options,” said Daniel Fowler, Rapa’s chief medical officer, in a statement.
The funding comes almost two years after President Joe Biden signed into law the Accelerating Access to Critical Therapies for ALS Act, known in short as ACT for ALS. Among other requirements, the act mandates the Food and Drug Administration to award grants or contracts to cover costs associated with the study of interventions meant to diagnose, prevent, mitigate, treat or cure rare nerve-destroying diseases like ALS.
Estimates hold that around 30,000 people in the U.S. have ALS, a progressive disease hallmarked by the decay and death of nerve cells. Over time, ALS patients lose the ability to perform essential tasks like walk, talk and breathe. They typically live just two to five years following diagnosis.
Until recently, the FDA had approved just two main medicines for the disease. Clinical testing had shown the drugs respectively offered modest benefits on function and survival.
But in the last year or so, two more treatment options received nods from the FDA. Amylyx Pharmaceuticals’ Relyvrio is now cleared for the broad ALS population, while Biogen’s Qalsody is specifically for the small portion of patients who have mutations in a gene called SOD1.