A Polish-American team, in collaboration with the Dystrogen Therapeutics laboratory, has just published the results of the administration to patients suffering from Duchenne muscular dystrophy (DMD) of a novel cell therapy product, DT-DEC01. The product consists of chimeric cells expressing dystrophin derived from the fusion of two myoblasts, one from a healthy donor and the other from the DMD patient subsequently treated:
- Three participants aged between six and fifteen (one had lost the ability to walk) and with different types of mutations were included in the study.
- They received a single intraosseous injection of the treatment.
- Six months after the injection, the two participants able to walk improved their walking test scores (6MWT) and their functional performance (NSAA).
- All three participants showed improvements in muscle strength and fatigue resistance correlated with recorded electromyography (EMG) data.
- No treatment-related adverse effects were observed up to 14 months after administration.
Longer-term follow-up of these three participants and others included in the study will enable these positive results to be confirmed.
