Objective:

The objective of this study is to assess in Inclusion Body Myositis (IBM) the safety of an autologous graft consisting of adipose-derived regenerative cells (ADRCs) derived from the Celution 800/CRS System. 

Background:

IBM is a progressive, debilitating disease leading to proximal and distal muscle weakness, most prominently in the quadriceps and finger flexors. The Celution 800/CRS System is a closed, automated system intended to digest adipose tissue to further extract, wash, and concentrate stromal stem cells and other associated progenitor cells intended for autologous reimplantation in a real-time bedside manner.

Design/Methods:

ADRCs are isolated and purified from human abdominal subcutaneous fat tissue using the Celution 800/CRS System. ADRCs with 90% mean viability are processed aseptically then suspended in lactated Ringer’s solution for injection. Nine IBM subjects are randomized 2:1 in blocks of 3 to late (Part 1) versus early (Part 2) ADRC autologous graft injections. We inject 30 million cells divided between 8 injections unilaterally: 2 sites in the flexor digitorum profundus muscle and 6 sites in the quadriceps group of muscles. We plan to follow the subjects every 3-6 months for two years after ADRC injections for safety and efficacy measures.

Results:

All 9 IBM subjects have been enrolled in the trial and have received stem cell injections via EMG-guidance. Injections were completed in late 2023 with an estimated study completion by December 2025. We are also collecting whole blood samples to assess TDP-43 biomarker profile at various timepoints in Part 1 and Part 2.

Conclusions:

While full study results are not available, stem cell injections have been well-tolerated in all 9 subjects. The study-related adverse events have been so far limited to transient mild to moderate side effects.