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Abstract Details

Inclusion Body Myositis Treatment with Celution Processed Adipose Derived Regenerative Cells
Neuromuscular and Clinical Neurophysiology (EMG)
P8 - Poster Session 8 (5:30 PM-6:30 PM)
11-005
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.
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. 
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.
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.
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.
Authors/Disclosures

PRESENTER
No disclosure on file
No disclosure on file
No disclosure on file
No disclosure on file
No disclosure on file
No disclosure on file
Mamatha Pasnoor, MD, FAAN (University of Kansas Medical Center) Dr. Pasnoor has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx BVBA. Dr. Pasnoor has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Takeda Pharmaceuticals. Dr. Pasnoor has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen Pharmaceutical. Dr. Pasnoor has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Immunovant. Dr. Pasnoor has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Pasnoor has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for catalyst pharmaceuticals. Dr. Pasnoor has received research support from NIH.
Omar Jawdat, MD (The University of Kansas Medical Center) Dr. Jawdat has nothing to disclose.
Matthew Varon, MD (University of Kansas Medical Center) Dr. Varon has nothing to disclose.
Constantine Farmakidis, MD, FAAN Dr. Farmakidis has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Momenta. Dr. Farmakidis has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx.
Swathy Chandrashekhar, MD (University of Kansas Medical Center) Dr. Chandrashekhar has received publishing royalties from a publication relating to health care.
Mazen M. Dimachkie, MD, FAAN (University of Kansas Medical Center) Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for EcoR1 Capital, LLC. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cello Health BioConsulting, previously Defined Health. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for CSL Behring. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Octapharma. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for ArgenX. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Spark Therapeutics. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for RAF-5. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Houston NeuroCare. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Shire. Dr. Dimachkie has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Catalyst. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kezar. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for RaPharma/UCB. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for ArgenX. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi. An immediate family member of Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kezar. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Catalyst. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Takeda. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for SCHOLAR Rock. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Jenssen. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amazentis / Vandria. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Spark Therapeutics. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roivant. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for SANEM. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medlink. Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for CSL Behring. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Third Rock. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Orphazyme . Dr. Dimachkie has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Astellas. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roivant . Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dianthus. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for TACT / Treat NMD. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cabaletta Bio. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Clinical Neurological Society of America, Inc . Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ig Society, Inc. Dr. Dimachkie has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abcuro. Dr. Dimachkie has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Covance / Labcorp. The institution of Dr. Dimachkie has received research support from CSL Behring. The institution of Dr. Dimachkie has received research support from Orphazyme. The institution of Dr. Dimachkie has received research support from FDA-OOPD. The institution of Dr. Dimachkie has received research support from NIH. Dr. Dimachkie has received intellectual property interests from a discovery or technology relating to health care. Dr. Dimachkie has received publishing royalties from a publication relating to health care.