New findings published in NEJM Evidence reveal promising clinical outcomes for a novel cell therapy approach to treating Amyotrophic Lateral Sclerosis (ALS). Cellenkos Inc.'s cryopreserved, allogeneic T regulatory (Treg) cells, derived from umbilical cord blood, demonstrated both safety and preliminary efficacy in slowing disease progression in ALS patients.
The study showed that patients receiving multiple intravenous infusions of this "off-the-shelf" Treg therapy experienced a significant reduction in their rate of disease progression. Before treatment, participants' ALS Functional Rating Scale-Revised (ALSFRS-R) scores were declining at an average rate of -1.66 points per month. During the treatment period, this rate slowed dramatically to -0.41 points per month, with a post-treatment decline of -0.60 points per month, suggesting a sustained therapeutic benefit.
Innovative Treatment Approach
Unlike conventional cell therapies that often require complex, individualized cell harvesting and hospitalization, Cellenkos' Treg therapy offers several distinct advantages. The treatment was administered in an outpatient setting via peripheral IV, with each patient receiving a standardized fixed dose of 100 million cells. The regimen consisted of weekly infusions for the first month, followed by monthly doses for six months, with optional continued dosing based on physician discretion.
Notably, the therapy required no lymphodepletion, immunosuppression, interleukin-2, or HLA matching, and patients were discharged the same day. No dose-limiting toxicities were reported throughout the study.
"These results are both encouraging and transformative for those battling ALS," said Dr. Simrit Parmar, Founder of Cellenkos and faculty member at Texas A&M University. "Unlike conventional cell therapies that require complex, individualized cell harvesting and hospitalization, our Treg therapy is cryopreserved, ready-to-use, and outpatient-based. It eliminates the need for HLA matching and preconditioning."
Study Details and Patient Outcomes
The study included six participants with a median age of 48.5 years (range: 27–66) and a median baseline ALSFRS-R score of 31.5 (range: 23–43). Participants received a median of 11 infusions (range: 6–22), and all were alive at the time of last follow-up. Among the four participants with sufficient follow-up data, the median follow-up duration was 18 months.
Beyond the clinical improvements measured by ALSFRS-R scores, the study also documented reductions in plasma neurofilament levels, a biomarker strongly correlated with neuronal injury in ALS. This biomarker data provides additional objective evidence supporting the therapy's potential neuroprotective effects.
Next-Generation Development
Building on these promising results, Cellenkos is already advancing its next-generation neurotropic Tregs, designated CK0803. These modified cells overexpress CD11a and CXCR3, which improves their ability to migrate to inflamed brain regions, specifically targeting inflamed microglia—potentially enabling more direct targeting of ALS-related pathology.
"These findings offer a compelling proof of concept and lay the foundation for our next-generation neurotropic Tregs, CK0803," Dr. Parmar explained.
ALS Treatment Landscape
ALS is a progressive neurodegenerative disease that affects nerve cells responsible for voluntary muscles. As the disease advances, individuals experience worsening muscle weakness, paralysis, and eventual respiratory failure. Most patients succumb to ALS within three to five years of diagnosis, and current therapeutic options remain limited.
The development of effective treatments for ALS has been challenging, with few approved therapies offering modest benefits. Cellenkos' approach represents a potentially significant advance in addressing the underlying inflammatory and immune components of ALS pathology.
Broader Pipeline and Platform Technology
Cellenkos is developing a broader pipeline of allogeneic, off-the-shelf, cord blood-derived T regulatory cell therapies for rare inflammatory and autoimmune diseases. Beyond CK0803 for ALS, the company's pipeline includes CK0801 for Aplastic Anemia, CK0802 for Acute Respiratory Distress Syndrome, and CK0804 for Myelofibrosis.
The company's proprietary CRANE® platform enables the development of tissue-directed Treg therapies that require no HLA or ABO matching, can be administered in outpatient settings, and are optimized for rapid, point-of-care delivery.
These latest findings offer renewed hope for patients, families, and researchers working toward more accessible, non-invasive, and effective treatments for ALS and potentially other neurodegenerative conditions with limited therapeutic options.
