Foundation for NIH announces partnership to steward new ALS database

Key takeaways:

• The AMP ALS research team will centralize all datasets in an openly accessible portal.
• Centralization aims to provide rapid access to new or modified clinical outcome assessments.

The Foundation for the NIH has announced the launch of a public-private partnership to advance knowledge of and accelerate drug development for individuals affected by amyotrophic lateral sclerosis.

The Accelerating Medicines Partnership in Amyotrophic Lateral Sclerosis (AMP ALS), launched with roughly $60 million in funding, will combine resources and expertise of the National Institute of Neurological Disorders and Stroke (NINDS) at NIH, the FDA and Critical Path Institute, with additional stakeholders from academia, life science companies, foundations and patient-focused groups, according to a release from the FNIH.

“One major focus of the partnership is to develop diagnostic biomarkers that would help identify patients much earlier in their clinical course than is typical today,” Julie Gerberding, MD, MPH, president and CEO of FNIH, told Healio in an email. “In addition, standardized and new clinical outcome assessments will optimize the efficiency, design and analysis of future trials.”

Adding credence to the 5-year project will be input from those with lived experience of the debilitating condition, including affected individuals and families, caregivers and others at high genetic risk for developing ALS.

The initiative is a component of the collaboration included in the Accelerating Access to Critical Therapies for ALS Act, signed into law by President Joe Biden in December 2021. Multiple clinical sites across the country have joined the NIH-funded Access for All in ALS (ALL ALS) consortium, created with the express purpose of recruiting those in the ALS sphere to collect valuable clinical data and biosamples, according to the release.

The chief goal of the AMP ALS research team will be to collect and centralize both current and future ALS datasets in a cloud-based, openly accessible knowledge portal. Armed with this body of knowledge, researchers will be able to focus on identifying and validating biomarkers and therapeutic targets specific to ALS. Centralization also aims to provide rapid access to new or modified clinical outcome assessments, including those originating from patients themselves.

“Working collaboratively, through AMP ALS, the partners can advance efforts to understand what triggers ALS and discover new targets for effective treatments, as well as identify biomarkers that can be used to predict whether promising interventions have the intended effects in people,” Walter Koroshetz, MD, director of NINDS, said in the release.