In non-ambulatory individuals with Duchenne muscular dystrophy (DMD), DT-DEC01 (Dystrogen Therapeutics), an experimental dystrophin expressing chimeric (DEC) cell treatment, was found to be safe over a 24-month period in a pilot single-site, open-label research. The results further support the development of the drug as a treatment for DMD, independent of gene mutation or disease progression, when combined with advancements on functional tests.
Under the direction of Maria Siemionow, MD, PhD, DSc, professor and director of Microsurgery Research at the University of Illinois, the analysis included 24-month data on three individuals, without immunosuppression, ages 11–16, who received dosages per kilogram body weight, respectively.
Dystrogen Therapeutics’ Chimeric Cells
Treatment with the cell therapy causes no significant adverse events (AEs), donor-specific antibodies (DSA), or study-related AEs for up to 24 months, according to a presentation made at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific meeting, which took place in Dallas, Texas, from March 16–19. [Chimeric Cells]
Effects of DT-DEC01 Cell Therapy on Different Exons
- Patient 1, a 15-year-old with exon 48-50 deletion, showed improvements in echocardiography EF by 12%, arm movements by 9%, and Motor Unit Potentials (MUP) duration, both in deltoideus (53%) and biceps brachii (23%) at 18 months. In addition, this patient saw enhancements in PUL 2.0 test score by 5%, grip strength by 6%, and spirometry by 17% after 24 months since the original procedure.
- Patient 2, an 11-year-old with exon 52 deletion, demonstrated a 17% improvement in echocardiography EF at 12 months, along with a 5% increase in PUL 2.0 test score and enhancements in MUP duration, including 19% in the deltoideus and 51% in the biceps brachii, at 18 months. Furthermore, this patient experienced a 59% improvement in spirometry and a remarkable 1150% increase in arm movements at 24 months.
- Patient 3, a 16-year-old with a nonsense mutation, reported improvements at 12 months post-DT-DEC01 administration, including a 6% increase in PUL 2.0 test score, a 34% improvement in grip strength, an 11% enhancement in echocardiography EF, and increases in MUP duration, with 49% in the deltoideus and 29% in the biceps brachii.
Conclusion
In the 12-month published data, the study authors wrote that the findings “supports the potential benefits of DT-DEC01 therapy in improving cardiac, respiratory and skeletal muscle function in patients with DMD after systemic-intraosseous administration, providing hope for better outcomes and enhanced quality of life for DMD patients. Additionally, this study also highlights use of EMG as a valuable biomarker for monitoring functional changes in muscles affected by DMD after DT-DEC01 therapy.”
