Cellenkos' Treg Cell Therapy Proven Safe and Potentially Effective for ALS

Researchers have unveiled a significant breakthrough in the treatment of Amyotrophic Lateral Sclerosis (ALS) through the use of cryopreserved, allogeneic T regulatory (Treg) cells, as recently detailed in NEJM Evidence. The study, conducted by Cellenkos Inc., focuses on Treg cells derived from umbilical cord blood and their potential to alter the course of ALS, a debilitating neurodegenerative disease that currently has limited treatment options.

The clinical trial results indicate that patients who received multiple intravenous infusions of the Treg therapy experienced a noticeable reduction in the progression rate of ALS. Initially, participants' ALS Functional Rating Scale-Revised (ALSFRS-R) scores showed a decline rate of −1.66 points per month. However, during treatment with Treg cells, this rate decreased to −0.41 points per month, and even after concluding treatment, there was only a slight decline of −0.60 points per month. The therapy also contributed to reductions in plasma neurofilament levels, a known marker of neuronal damage, suggesting a beneficial impact on the underlying disease pathology.

The administration of this Treg therapy was executed in an outpatient setting via peripheral intravenous infusion. Each patient received a standardized dose of 100 million cells, with a treatment schedule involving weekly infusions during the first month, followed by monthly doses over a six-month period. Additional dosing was considered based on the physician's evaluation. Notably, this therapy did not necessitate lymphodepletion, immunosuppression, the use of interleukin-2, or human leukocyte antigen (HLA) matching, and patients were able to return home the same day. Importantly, no severe adverse reactions were observed.

A cohort of six patients participated in the study, with ages ranging from 27 to 66 years and a median age of 48.5 years. The median baseline ALSFRS-R score for these participants was 31.5. Over the course of the study, participants received a median of 11 infusions, with all remaining alive at the end of the follow-up period. For those with adequate follow-up data, the median duration of follow-up was 18 months.

Dr. Simrit Parmar, the founder of Cellenkos and a faculty member at Texas A&M University, commented on the significance of these findings. Dr. Parmar highlighted the potential of this Treg therapy to provide a transformative approach for ALS patients, distinguishing it from traditional cell therapies that often require complex procedures and hospital stays. The cryopreserved and ready-to-use nature of the therapy, along with its outpatient administration, represents a significant advancement in making treatment more accessible and less burdensome for patients. This study also lays the groundwork for the development of next-generation Treg therapies, such as CK0803, which are being engineered to enhance their effectiveness in targeting ALS-specific pathology.

In conclusion, these promising results offer renewed hope for ALS patients, caregivers, and researchers striving for more effective, non-invasive treatments. The success of this Treg therapy marks a significant step forward in addressing the challenges posed by ALS and improving the quality of life for those affected by the disease.