Assessing Efficacy of Fibroblast Cell Therapy in MS: Hamid Khoja, PhD


The chief scientific officer of FibroBiologics discussed efficacy findings from a phase 1/2 trial.

This content originally appeared on our sister site, NeurologyLive.

“Lately, there have been a lot of publications showing the promise of a staggered approach, not only in terms of monitoring safety, but [also in terms of] a lot of the efficacy endpoints. It gives you more freedom to be able to monitor and change some of the study aspects.”

FibroBiologics is assessing fibroblast cell technology for the potential treatment of patients with multiple sclerosis (MS). The company's cell therapy candidate, CYMS101, was found to be safe for all 5 patients enrolled in an early safety study, with the treatment also appearing to be efficacious, albeit in a smaller patient sample. The trial assessed 5 neurological testing assays that are typically used in clinical trials for MS, namely the Paced Auditory Serial Addition Test (PASAT), the 9-Hole Peg Test (9-HPT), the Timed 25-Foot Walk test (T25-FW), the Expanded Disability Status Scale (EDSS), and a gadolinium MRI to look at brain lesions.

Noting that the results were observed in a limited number of adults, the chief scientific officer of FibroBiologics, Hamid Khoja, PhD, told NeurologyLive® that results showed patients improved in the PASAT, noting a significant improvement in addition of mental capacity within the 16-week period. General improvement as also seen in the 9-HPT and the T25-FW, he said, and there was no improvement or degradation in EDSS scores, and no new lesions observed on patients’ MRIs.

Pending FDA approval of an investigational new drug application planned to be filed in Q3 of 2022, Khoja noted they hope to begin patient enrollment in a phase 1/2 clinical trial in 2023. He further spoke to the benefit of the staggered clinical trial, which will allow investigators to better understand if the monitoring period needs to be extended. By staggering repeated dosage and dose concentration, this approach also provides the ability to modify study aspects.

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