What does the drug do?
Lenzumestrocel (Neuronata-R® inj.) is new potential treatment developed in South Korea for sporadic ALS. The treatment contains a collection of stem cells taken from the participant, called ‘mesenchymal stem cells’ which can develop into many different cell types such as nerves and muscles. Treatment with Lenzumestrocel is expected to target several pathways that can stimulate the growth of new nerve cells and regulate the immune systems response to damaged cells.
Phase 3 Trial - ALSUMMIT
The phase 3 trial is investigating the long-term effects, safety and efficacy of Lenzumestrocel in a placebo-controlled, double-blind trial. The study has enrolled 115 participants divided into 3 groups, 2 treatment groups and a placebo group. Both treatment groups will be given 2 injections at 26-day intervals, with the second treatment group being given an additional 3 injections at 3-month intervals. All participants will be given Riluzole as a baseline throughout the study. Read more about this study here.
The trial recruited in South Korea only.
Previous Clinical Trials
Phase 1 trial
The phase 1 clinical trial tested the safety of Lenzumestrocel by injecting one dose into 8 participants with ALS. The study measured changes to the participants vital signs such as breathing ability, heart health and blood tests, showing the treatment was safe.
Phase 2 trial
The phase 2 trial compared the safety and tolerability of the drug in 37 people living with ALS with a control group of healthy individuals. Participants received the treatment twice over 2 months and were then monitored for 6 months. Some participants saw a slowing in disease progression 3 months after treatment, as measured by ALSFRS-R. The treatment was also found to be safe and well-tolerated. Find out more about the completed trial here and published results here.
Last updated: 18/04/2024