A Polish-American team working in collaboration with the laboratory Dystrogen Therapeutics has published the results of administering a new cell therapy product, DT-DEC01, to patients suffering from Duchenne muscular dystrophy (DMD). This product consists of chimeric cells expressing dystrophin derived from the fusion of two myoblasts, one from a healthy donor and the other from a DMD patient subsequently treated with these new cells:
- three participants aged between six and fifteen years (one of whom lost his ability to walk) and with different types of mutations were included in the study;
- they received a single intra-osseous injection of the treatment;
- one year after the injection, the two participants who were able to walk improved their scores on the walking test (6MWT) and their functional performance (NSAA);
- all three participants showed improvements in muscle strength and fatigue resistance correlated with recorded electromyography (EMG) data;
- pulmonary and cardiac functions, measured by spirometry and echocardiography, were improved or stabilised in all patients.
- No treatment-related adverse events were observed up to 21 months after administration.
Longer-term follow-up of these three participants and other patients included in this study will provide further efficacy results.
