What does the drug do?
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 neurons to become inflamed. Normally, T-regulatory cells (T-reg) will then reduce this inflammation, however, levels of T-reg cells have been found to be reduced in people with MND. This can result in higher levels of inflammation which can cause motor neurons to die. RAPA-501 is a cell treatment which aims to increase the levels of T-reg cells into people with MND. The treatment involves taking blood from the participant, isolating T-cells, editing them to work like T-reg cells and then injecting them back into the participant.
Phase 2/3 Trial
Current status: Open to recruitment in the US only
RAPA-501 is an open-label phase 2/3 trial being carried out by Rapa Therapeutics in the US. 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 aims to determine if RAPA-501 is safe to use and to find the highest safe dosage. Find out more about the clinical trial here and the expanded access open-label extension 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: 18/04/2024