NIH Reporter #5U01NS136020-03

Intermediate-Size Expanded Access Trial of Autologous Hybrid TREG/Th2 Cell Therapy (RAPA-501) of Amyotrophic Lateral Sclerosis

This award funds an Expanded Access Program to administer RAPA-501 autologous T cell therapy to high-risk ALS patients, aiming to assess safety, feasibility, and efficacy signals, using AI methods for treatment effect estimation.

Description

The primary purpose of this Expanded Access Program (EAP) is to administer the novel RAPA-501 autologous T cell therapy to high-risk individuals with ALS (pwALS). The goal is to establish the feasibility and safety of this intervention and to obtain signals of efficacy. The project aims to deepen understanding of the neuroinflammatory component of ALS progression and its potential mitigation through RAPA-501 TREG/Th2 cell therapy. It will incorporate AI-based comparator cohort methods to more accurately estimate treatment effects and contribute to global efforts to include exploratory comparator arms in ALS clinical trials lacking placebo controls.

View original record