What is the drug thought to do in the body?
RAPA-501 is a cell treatment which is thought to protect motor neurons from inflammation. The body produces T-cells to fight infections within the body. This can cause neurones to become inflamed. Normally, T-regulatory cells (Tregs) reduce this inflammation, however, levels of Tregs have been shown to be reduced in people with MND. This can result in higher levels of inflammation which can cause motor neurons to die. RAPA-501 aims to increase the levels of Tregs in people with MND. The treatment involves taking blood from the participant, isolating T-cells, editing them to work like Treg cells and then injecting them back into the participant.
Phase 2/3 Trial
Current status: In preparation
Primary objective: To investigate the feasibility and safety of giving people with MND the highest established safe dose of RAPA-501
The phase 2/3 trial will investigate if RAPA-501 is safe in people with MND. The study will recruit 41 participants who will be given four injections of RAPA-501 over 18 weeks and monitored for a further 30 weeks, with a follow-up call after 2 years. The study also aims to find the highest safe dosage. This is an open-label trial, meaning everyone receives the treatment. Find out more about the clinical trial here.
Previous Clinical Trials
Phase 1 trial
The Phase 1 study successfully showed RAPA-501 to be safe in a small cohort of 9 people. The open-label study offered most participants 2 doses over 42 days. The study also showed an increase in T-reg cell protective markers.
Latest News
2023
The trial gained a 3-year grant to allow expanded access to people with MND who would struggle to enter other clinical trials due to disease progression and exclusion criteria. The grant allows the treatment to be given to a broader group of people with MND. Read more here.
Last updated: 21/08/2024