Innovative Cell Therapy Shows Promise in Treating ALS Patients

Recent advancements in cell therapy have demonstrated significant potential in addressing neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS). Dr. Simrit Parmar, an associate professor at Texas A&M University College of Medicine and founder of biotech firm Cellenkos Inc., has developed a novel treatment approach utilizing umbilical cord blood-derived regulatory T cells (Tregs). These cells are engineered to modulate inflammation at the core of ALS and other conditions like aplastic anemia. Her therapy has been tested on numerous patients, with results indicating it is both safe and effective.

Parmar emphasizes the broader impact of her work, stating, "If successful, millions could benefit from a treatment that improves organ function systematically without significant side effects." The promising outcomes have led to a strategic partnership between Cellenkos and a leading Saudi medical center in Riyadh, marking the first US-Saudi clinical trial alliance in this area. The joint efforts aim to advance trials for aplastic anemia and graft-versus-host disease and eventually expand into ALS and cardiovascular disease research.

Her method is distinctive because it does not require matching donor to patient, can be scaled for mass manufacturing, and uses umbilical cord cells that pose fewer risks of exacerbated inflammation compared to adult-derived Tregs. Over the past year, her team has treated over 80 patients suffering from various conditions, including ALS, with notable improvements observed in some cases.

Notably, her technology has garnered attention in major medical journals, with articles published in NEJM Evidence demonstrating the treatment's safety and benefits. In April 2025, a study detailed how multiple infusions could slow or reverse disease progression in ALS patients, while a 2024 study showed prolonged blood and platelet independence in aplastic anemia patients.

Dr. Parmar aims to achieve FDA approval in the US within two years for certain diseases and continues to seek funding for larger ALS trials. Her work is recognized for its groundbreaking potential to transform treatment options for difficult-to-treat diseases.

This innovative cell therapy represents a significant step forward in regenerative medicine, offering hope for patients with currently untreatable conditions.