Investigational Autologous Muscle-Derived Cell Therapy Is Safe, Effective for Fecal Incontinence: Presented at ICS

By Eric Ramos

VIRTUAL -- October 19, 2021 -- Autologous muscle-derived cell therapy was safe and reduced the number of fecal incontinence episodes and the number of days with fecal incontinence episodes in patients who have failed conservative treatments, according to interim data of an ongoing phase 1/2 study presented at the Virtual 2021 Annual Meeting of the International Continence Society (ICS).

“A single administration of the investigational product shows promise in improving symptoms and quality of life in patients with fecal incontinence,” said Manoj Raval, MD, St. Paul’s Hospital, Vancouver, British Columbia.

The product is manufactured from approximately 50 mg to 250 mg of skeletal muscle harvested from the patient’s vastus lateralis via biopsy, and delivered into the anal sphincter during a subsequent outpatient procedure.

“An outpatient skeletal muscle biopsy is done and the material obtained is then shipped to the labs at Cook Myosite in Pittsburgh, Pennsylvania,” explained Dr. Raval. “The appropriate cells are isolated, grown, and expanded, and then the product goes through rigorous quality checks before approval. It’s then frozen and sent back to the originating clinic site, where another outpatient procedure is done to inject the patient’s own autologous cells into the external sphincter, which initiates growth of new striated muscle to augment the defected sphincter and improve muscle function.”

“We hope that since this is autologous, and live tissue is incorporated into existing tissue muscle, it will be durable and long-lasting,” he added.

At the time of the interim analysis, 35 patients completed the 3-month visit, 32 completed the 6-month visit, and 24 completed the 12-month visit. Most (n = 25) patients had fecal incontinence due to obstetric injury (14 having sustained episiotomy and 13 obstetric tear). In all patients, the cell product was evenly distributed around the external anal sphincter through 8 to 12 percutaneous injections parallel to the anal canal.

There were no serious adverse events (AEs) related to the therapy. One patient reported a product-related AE of inflammation of the injection site. There were 5 AEs related to the injection procedure including injection site discoloration, injection site inflammation, injection site pain, procedural pain, and procedural dizziness. The 2 most frequent AEs related to the biopsy procedure were procedural pain and procedural contusion. All AEs related to the biopsy procedure were of low severity.

Twelve months after injection of the cell product, there was a 45% reduction in median fecal incontinence episodes compared with baseline -- from 16 episodes to 9 episodes. There was also a significant reduction in median days with fecal incontinence episodes compared with baseline -- from 13 days at baseline, to 9.5 days at 3 months, 8 days at 6 months, and 6.5 days at 12 months (P = .03).

At the 12-month follow-up, 12 patients reported a ≥50% reduction in number of fecal incontinence episodes, 8 reported a ≥75% reduction in number of episodes, and 5 patients reported zero episodes.

Symptom severity improved over the follow-up period as demonstrated by a statistically significant reduction of Cleveland Clinic Incontinence Scores compared with baseline (P

Compared with baseline manometry, length in high-pressure canal zone showed a statistically significant change at 12 months with an increase in mean length from 1.8 to 2.4 cm.

“Autologous muscle-derived cell therapy may represent a new, safe, and minimally invasive therapeutic option for patients with fecal incontinence failing conservative treatments,” said Dr. Raval. “The advantages are that it can be done in a clinic-based setting, and the recovery time and physical impact is minimal.”

He noted that a larger, randomised controlled trial will be needed to corroborate these promising findings and to further define the patient population that would benefit most from treatment.

Funding for this study was provided by Cook Myosite.

[Presentation title: A Prospective Multicenter Nonrandomized Clinical Study of Autologous Muscle Derived Cell (AMDC) Therapy for the Treatment of Fecal Incontinence. Abstract 92]

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