DT-DEC01, a cell therapy intended for Duchenne muscular dystrophy (DMD) treatment, appears to be safe and effective in a clinical trial, according to a recent press release.

Chimeric cell therapy with DT-DEC01 prevents deleterious immune system responses without producing immunosuppression that compromises the overall health of the patient. Furthermore, DT-DEC01 therapy does not require viral vectors nor involve risks of genetic cell mutation, noted the press release.

DMD is a debilitating disease caused by a deficiency of dystrophin protein. The therapy uses cell engineering technology that engrafts itself in cardiac and skeletal, which then produces a fully functional dystrophin gene. Previous studies have already demonstrated that DT-DEC01 can produce significant improvement in both skeletal muscle function and cardiac muscle function.


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“When coupled with the strong and sustained dystrophin expression results in preclinical studies and encouraging safety profile seen to date, today’s results increase our confidence in DT-DEC01 and provide additional supportive evidence for this approach as we advance to the higher dose cohort into the next stage of clinical testing,” said Maria Siemionow, MD, PhD, chief scientific officer of Dystrogen Therapeutics.

The clinical trial evaluated data from only 3 clinical trial participants with different mutations who presented with no significant adverse effects. All patients achieved improvement in baseline laboratory markers after treatment.

All 3 patients showed improvement in duration and amplitude of motor unit potential measured by electromyography. Improved grip strength, 10-meter wait time, and step count also increased in the 3 patients.

Patients in this trial received a dose of 2 million dystrophin-expressing chimeric (DEC) cells per kilogram and are considered the low-dose cohort. After assessing the safety results of the low-dose cohort, the Data and Safety Monitoring Board recommended initiating the high-dose cohort that will receive 4 million DEC cells per kilogram.

“The improvements in functional measures at 1 and 3 months in participants from the low dose cohorts who received DT-DEC01 are distinctly different from what an age-matched, natural history group would predict with DMD,” Dr. Siemionow said.

Reference

Dystrogen therapeutics investigational chimeric cell therapy DT-DEC01 for the treatment of Duchene muscular dystrophy shows safety and functional improvements. News release. Dystrogen Therapeutics; April 07, 2022