The first patient was dosed in a phase 1 clinical trial of testing the safety, tolerability, and efficacy of dystrophin expressing chimeric cells DT-DEC01 treatment for Duchenne muscular dystrophy (DMD).

“We are very pleased to begin dosing patients in our pilot study,” Kris Siemionow, MD, PhD, founder and chief executive officer of Dystrogen Therapeutics said in a press release.

“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.”


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The patient who was dosed was a boy aged 6 years who has a deletion in exons 3 to 12 in the DMD gene. He tolerated the procedure well and showed no side effects and no signs of inflammation, infection, antibody formation, or other adverse effects on organs.

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There was a nonclinically significant improvement in functional outcomes. His daily activity levels also significantly improved according to a subjective parent-reported assessment, as well as his quality of motion and performance according to a subjective therapist-reported assessment.

The trial aims to enroll a total of 10 boys with DMD, aged 5 to 18 years, to investigate the effect of 3 escalating doses of the treatment using these measures: range of motion, muscle strength, heart muscle function, the Performance of Upper Limb, the 6-Minute Walk Distance, and the NorthStar Ambulatory Assessment.

DT-DEC01 is a chimeric cell therapy that is based on the fusion of allogeneic human myoblasts with autologous human myoblasts from patients with DMD. These cells maintain the ability to make functional dystrophin protein.

It is hoped that they will increase the normal myoblast pool and reduce inflammation and induce fibrotic tissue replacement. This could improve muscle strength and function in patients with DMD. 

Reference

Dystrogen Therapeutics announces first in man dosing of novel chimeric cell therapy for Duchenne muscular dystrophy and reports 6-week clinical outcomes. News release. Dystrogen Therapeutics; January 4, 2022.