First Participant Dosed of Novel Chimeric Cell Therapy for Duchenne Muscular Dystrophy 

The first patient has been treated in a phase 1 clinical study of dystrophin-expressing chimeric cells (DEC)(DT-DEC01; Dystrogen Therapeutics, Miami, FL) for the treatment of Duchenne muscular dystrophy (DMD).

The first participant, age 6 years, received an infusion of DEC, tolerated the procedure well, and was discharged home. At day 7, day 28, and week 6 follow up, no adverse events from treatment occurred. Subjective parent-reported assessment of post-treatment daily activity level and subjective therapist assessment of quality of motion, as well as patient performance, demonstrated a significant improvement compared with baseline. 

Screening and enrollment of 10 participants, to be treated with 1 of 3 doses, is expected to continue over the course of the next 12 months in the European Union. Clinical improvement will be measured with NorthStar Ambulatory Assessment (NSAA), 6MWD, Muscle strength by myometry, range of motion (ROM), performance of upper limb (PUL and 5-grade Lovett scale), and cardiac muscle evaluation by ECHO.

"We are very pleased to begin dosing patients in our pilot study. This is the first time we will assess our chimeric cell therapy in Duchenne patients, which will provide us not only with safety data but could also give us an early look into the potential efficacy of this treatment in possibly halting disease progression and even partly restoring functional loss in boys with muscular dystrophy", said Kris Siemionow, MD, PhD founder and chief executive officer of Dystrogen Therapeutics.

DEC is a chimeric cell therapy, expressing CD56, that expressed high levels of dystrophin and resulted in functional improvements in animal models of DMD. DEC therapy does not require immunosuppression and is not associated with any genetic manipulation, making DEC a potentially universal therapy for all people with DMD.

Currently, laboratory data demonstrated no signs of inflammation, infection, antibody formation, or other adverse systemic effects.