Cryopreserved allogeneic Treg infusions associated with slowed disease progression and decreased plasma neurofilament level in ALS
HOUSTON, April 22, 2025 -- New findings published today in NEJM Evidence reveal promising clinical outcome for Cellenkos Inc.'s cryopreserved, allogeneic, T regulatory (Treg) cells derived from umbilical cord blood, for the treatment of Amyotrophic Lateral Sclerosis (ALS)—a neurodegenerative disease with limited therapeutic options.
The study reports that patients receiving multiple intravenous infusions of this "off-the-shelf" Treg therapy showed a measurable slowdown in disease progression. Prior to therapy, participants' ALS Functional Rating Scale-Revised (ALSFRS-R) scores declined at an average rate of −1.66 points per month. During treatment, that rate slowed significantly to −0.41 points per month, with a modest post-treatment decline of −0.60 points per month—suggesting sustained benefit. Clinical improvements were accompanied by reductions in plasma neurofilament levels, a biomarker correlated with neuronal injury.
The Treg therapy was administered in an outpatient setting via peripheral IV, with each patient receiving a standardized fixed dose of 100 million cells. The regimen included weekly infusions for the first month, followed by monthly doses for six months, with optional continued dosing based on physician discretion. The therapy required no lymphodepletion, immunosuppression, interleukin-2, or HLA matching, and patients were discharged the same day. Importantly, no dose-limiting toxicities were reported.
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), received a median of 11 infusions (range: 6–22), and all were alive at the time of last follow-up. Among participants with sufficient follow-up data (n=4), the median follow-up duration was 18 months.
"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. These findings offer a compelling proof of concept and lay the foundation for our next-generation neurotropic Tregs, CK0803."
CK0803 Tregs, now in clinical trials, are modified to overexpress CD11a and CXCR3, improving their ability to migrate to inflamed brain regions, specifically, inflamed microglia — potentially enabling direct targeting of ALS-related pathology.
These findings offer renewed hope for patients, families, and researchers working toward more accessible, non-invasive, and effective treatments for ALS.
About Amyotrophic Lateral Sclerosis (ALS)
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. Currently, there is no cure.
About Cellenkos®, Inc.
Cellenkos is a clinical-stage biotech company, advancing allogeneic, off-the-shelf, cord blood-derived T regulatory cell therapies for rare inflammatory and autoimmune diseases. Its pipeline includes:
- CK0801 for Aplastic Anemia
- CK0802 for Acute Respiratory Distress Syndrome
- CK0803 for ALS
- CK0804 for Myelofibrosis
Using its proprietary CRANE® platform, Cellenkos tailors 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.
Contact
bd@cellenkosinc.com
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