Patient Dosed With Mesenchymal Stem Cell-Derived Therapy for Osteoarthritis


Reported efficacy data included a WOMAC score reduction at 6 months following a single dose.

The first patient has been dosed in Cells for Cells’ (C4C) phase 1 clinical trial (ExoOA-1; NCT05060107) of XO-101, an exosome therapy derived from mesenchymal stem cells (MSC) for the treatment of osteoarthritis (OA).1 Data from the study were presented at the International Society for Cell & Gene Therapy (ISCT) annual meeting in San Francisco and the International Society for Extracellular Vesicles (ISEV) meeting in Lyon, France.

Reported efficacy data included a Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) score reduction at 6 months following a single dose of XO-101. Earlier preclinical data also indicated potential for cartilage reduction and increases in bone mineral density, which C4C reported is now being corroborated in the first patients dosed in the clinical trial.

"Milestones like these are great boosters for pushing our clinical program forward. The lack of any observed adverse effect in this first-in-human trial reinforces the safety profile of C4C's MSC-derived exosomes and their potential as disease-modifying agents," said Maroun Khoury, PhD, chief scientific officer, C4C, said in a statement.1 

The clinical trial is the first to study the safety and efficacy of an exosome therapy for joint disease.

The single-arm trial will seek to enroll 10 patients aged 30 to 70 years with mild to moderate symptomatic OA. Patients with bilaterial symptomatic knee OA, local infection, neoplasia, a joint replacement, recent use of local steroids, or a BMI greater than 30 will be excluded from the study.

Patients will receive a single dose (3-5x1011 particles per dose) of exosomes derived from allogeneic MSC via intra-articular knee injection. The primary end point is the occurrence of adverse reactions within the 12 months after treatment. Secondary end points include measures of the incidence of injection-related pain according to the visual analog scale (VAS) after the first week of treatment and the incidence of injection-related synovitis measured according to the effusion grading scale of the knee joint after the first week of treatment. Additional secondary end points will include pain reduction measured by the change in VAS score at 52 weeks, disability reduction measured by the change in the WOMAC subscale related to function (C) at 52 weeks, and percentage of responders according to the OMERACT-OARSI Criteria Index Response at 52 weeks.

Based on the trial’s early promise, C4C has decided to launch a new US-based company.

"The first-patient milestone positioned C4C ́s exosome therapy at the forefront of the field, and, in order to capitalize upon that achievement, we decided that forming a new venture was the best route for clinical and financial success,” Maximiliano Kunze, associate director of business development, C4C, added to the statement.1 “The NewCo will seek to raise US$ 18 million to finance the XO-101's Phase IB and II studies in the US and the R&D and Phase I for XO-101+, its drug-loaded allogeneic exosome-based therapy."

CELLS for CELLS treats the first-ever patient with an exosome-produced therapy for osteoarthritis. News release. CELLS for CELLS. July 18, 2022. 
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