CK0803 Treg Therapy Seems Safe for ALS, Trial Set to Treat Second Cohort


The positive DSMB recommendation came after no participants were found to experience any serious adverse events from the therapy.

Cellenkos is moving forward with treating the second cohort of patients with amyotrophic lateral sclerosis (ALS) in its phase 1 REGALS trial (NCT05695521) of CK0803 neurotrophic T regulatory (Treg) cell therapy after being given a positive recommendation by the Data Safety Monitoring Board (DSMB).1

"We are very excited and encouraged by this development," Tara Sadeghi, Chief Operating Officer, Cellenkos, said in a statement.1 "This recommendation from the DSMB is an important step in bringing this potential disease modifying therapy to the patients and takes us closer to our ultimate goal of curing ALS."

The positive DSMB recommendation came after no participants were found to experience any serious adverse events from the therapy. The REGALS trial will continue to enroll in its safety run-in study before proceeding to a phase 1b, randomized, double blind, placebo control trial of CK0803 that will access 4 weekly infusions followed by 5 monthly infusions in participants with ALS.

CK0803 is a novel, allogeneic, neurotrophic, clinical-grade umbilical cord blood-derived cell therapy that consists of Treg cells with a high cell surface expression of CD11a, that leverage CXCR3/CXCL10 axis to engage with the inflamed microglia. It is developed using Cellenkos' proprietary CRANE® technology, to generate disease-specific products. Cell products do not need to be HLA or ABO-matched to the recipient and multiple doses of CK0803 can be manufactured from a single umbilical cord blood unit and cryopreserved until ready for use.

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"We are excited to participate in this important study of this novel allogeneic Treg cell product in patients with ALS," principal investigator Neil Shneider, MD, PhD, associate professor, neurology and director, Eleanor and Lou Gehrig ALS Center, Columbia, said in an earlier statement."Promising data from six ALS patients previously treated with allogeneic cord blood Treg cells through an expanded access program at our Center supports the clinical development of CK0803, and we are grateful to Cellenkos for their commitment to applying their technology to ALS".

REGALS stands for Regulatory T Cells for Amyotrophic Lateral Sclerosis and is a multicenter study being conducted across Columbia University, New York; Michael E. DeBakey VA Medical Center, Houston and Baylor College of Medicine, Houston. The study is primarily aiming to establish safety and tolerability of multiple doses of CK0803 in patients with ALS. The study will also assess the primary endpoint of combined assessment of function and survival (CAFS) based on survival time and change in the ALS Functional Rating Scale-Revised (ALSFRS-R) score, and a secondary endpoint of longitudinal measurement of neurofilament light chain levels in serum and cerebrospinal fluid (CSF) in order to evaluate preliminary efficacy.

"The dosing of the first patient marks an exciting milestone for our company. The initiation of this study of CK0803 is an important achievement that brings us closer to delivering a potential new treatment which may have a life-changing impact for ALS," Sadeghi said when the first patient was dosed in May 2023.2 "ALS is a devastating disease with no cure, and we believe that CK0803 has the potential to provide a much-needed treatment option for patients. We are enthusiastic to move forward with this trial and to further explore the potential of CK0803 in ALS and other neurodegenerative diseases."

1. Cellenkos Approved to Proceed with CK0803 Neurotrophic T regulatory Cell therapy to Treat Second Cohort in ALS Trial. News release. February 19, 2024.
2. Cellenkos Announces First Patient Dosed with CK0803 Cell Therapy for Treatment of Amyotrophic Lateral Sclerosis. News release. Cellenkos. May 2, 2023.
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