Inclusion Body Myositis Treatment with Celution Processed Adipose Derived Regenerative Cells (P8-11.005)
Document Type
Abstract
Publication Title
Neurology
Abstract
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.
DOI
10.1212/WNL.0000000000205737
Publication Date
4-9-2024
ISSN
1526-632X
Recommended Citation
Heim A, Soder R, Bhavsar D, Russo A, Agbas A, Kosa E, Pasnoor M, Jawdat O, Varon M, Farmakidis C, Chandrashekhar S, Dimachkie MM. Inclusion Body Myositis Treatment with Celution Processed Adipose Derived Regenerative Cells (P8-11.005). Neurology. 2024; 102(17 S1). doi: 10.1212/WNL.0000000000205737.
