#United States #Arlington #Clinical Trials #ALS Association #ALS Therapies

ALS Association's Commitment to ALS Research

In an exciting development for the ALS community, the ALS Association has committed $2 million to support early-stage clinical trials for two promising therapies aimed at treating amyotrophic lateral sclerosis (ALS). These funds were awarded through the prestigious Hoffman ALS Clinical Trial Awards Program, which is dedicated to accelerating the development of effective new treatments for this debilitating disease.

The announcement made on December 4, 2025, highlights the pivotal role these early-phase trials play in advancing research. These trials will not only focus on safety and dosing but also aim to uncover critical biomarkers that could signal the effectiveness of novel therapeutic approaches. According to Kuldip Dave, Ph.D., the senior vice president of research at the ALS Association, early-stage funding is essential as it acts as a catalyst for the research and development engine. “The knowledge gained from these initial trials is crucial in propelling drug development forward,” he explained. “This allows for more informed choices in larger efficacy studies and reduces risk for potential investors.”

This year’s awardees were selected from a highly competitive international pool of applicants. Each application presented a compelling case supported by rigorous preclinical data, a detailed biosink program, and a strategic clinical development plan. Among the notable recipients:

1. Mayo Clinic Jacksonville: Led by Jaimin Shah, M.D., a phase 1 trial will investigate the investigational therapy PAS-004, developed in collaboration with Pasithea Therapeutics Corp. This treatment targets MEK, an enzyme implicated in inflammation and nerve damage associated with ALS. The trial will include 12 participants diagnosed with ALS, assessing the safety, tolerability, and optimal dosing of PAS-004. Additionally, researchers will measure biomarkers to detect any early signs of therapeutic effects. Dr. Shah noted, “While early-phase studies cannot definitively prove a drug’s efficacy in slowing ALS, they provide vital insights into the drug's potential therapeutic value.”

2. Molefy Pharma SL: Co-founder and Scientific Director Ana Martínez, Ph.D., will spearhead testing for the investigational therapy AP-2, which aims to restore the balance of the protein TDP-43 that, when malfunctioning, contributes to ALS in approximately 97% of patients. The phase 1 study will establish the highest safe dosage of AP-2 and gauge any side effects, marking a significant step towards clinical development of this promising drug.
“This first-in-human trial is a critical initial step towards working on ALS patients and advancing the development of new TDP-43-modifying therapeutics,” Dr. Martínez stated.

Clinical trials represent the most reliable and expedient method to translate cutting-edge laboratory discoveries into effective treatments. The ALS Association is committed to ensuring that the funding of such early-stage trials leads to the swift availability of promising therapies. Dr. Dave accentuated the urgency of the efforts: “People living with ALS urgently need new treatments. By investing in early clinical testing, we are dedicated to bringing effective therapies closer to those who need them most.”

The ALS community has long faced challenges with limited treatment options, resulting in a significant unmet need for innovative therapies that can address both functional deficits and disease progression. The funding from the Hoffman ALS Clinical Trial Awards underscores the commitment to bettering lives of those affected by ALS and fosters hope for future advancements in treatment.

About the ALS Association

The ALS Association stands as the largest organization focused on ALS globally, providing crucial funding and resources for research, patient support, and advocacy efforts. Their mission centers on enhancing the lives of those diagnosed with ALS while relentlessly pursuing new treatments and a cure.

To learn more about the ALS Association and the ongoing initiatives, please visit www.als.org.