Cell therapy for patients with amyotrophic lateral sclerosis shows promise in human trials

Dr. Simrit Parmar, an associate professor in the College of Medicine at Texas A&M University and founder of the clinical-stage biotech company Cellenkos Inc. in Houston, has invented a new cell therapy that shows strong promise for the treatment of amyotrophic lateral sclerosis (ALS), aplastic anemia and other devastating diseases.

Her therapy has been evaluated in dozens of human patients and has been shown to be safe and, to date, effective.

“If we succeed, millions of lives could be improved—organically, systematically and without significant side effects,” Parmar says.

Her results are promising enough to attract a major investment and partnership between Cellenkos and the leading medical center in Riyadh, Saudi Arabia. Announced May 22, it is the first alliance for medical clinical trials between the U.S. and Saudi Arabia.

Results also have led to two recent articles in NEJM Evidence. The articles draw attention to promising potential treatments for patients with aplastic anemia and ALS.

Parmar is giving hope to patients with a newly developed use of umbilical cord blood-derived T regulatory (Treg) cell therapy. The therapy treats the inflammation at the center of a number of hard-to-treat or untreatable illnesses. Derived from umbilical cord blood (CB), these Tregs are naturally wired to resolve inflammation without becoming harmful themselves.

The three key components of Parmar’s innovation are:

• The cell therapy does not require matching an individual donor to an individual patient.
• The cell therapy can be manufactured at scale, where a single starting CB unit can generate multiple ready-to-use products that can be frozen for up to three years and thawed later for use. This solves enormous logistical challenges associated with other cell-based treatments.
• By using umbilical cord-derived cells, the therapy sidesteps the dangers posed by adult-derived Tregs, which carry an elevated risk of further inflammation.

Parmar is addressing conditions where conventional treatments have failed. Her team has treated more than 80 patients suffering from multiple conditions, including ALS, aplastic anemia, myelofibrosis and acute respiratory distress syndrome (ARDS).

In April 2025, NEJM Evidence highlighted a study of Parmar’s technology for the treatment of ALS, where multiple infusions led to improvement in patients’ function decline.

In May 2024, the journal highlighted a study of her technology in the treatment of aplastic anemia, where a single infusion of her treatment led to durable independence from patients’ blood and platelet transfusions for up to 3.5 years.

The agreement between Cellenkos and King Faisal Specialist Hospital & Research Centre in Riyadh will begin with two clinical trials in Saudi Arabia for aplastic anemia and graft-versus-host disease and will pave the way for expansion into studies of patients with ALS and cardiovascular diseases. Cellenkos will also assist the Saudi Research Center with training and education in cell and gene therapy manufacturing.

In the U.S., Parmar hopes to have regulatory approval from the FDA in about two years for treatments of certain diseases, including aplastic anemia. She also is seeking funding for larger research studies of ALS patients in the U.S.

Parmar was in the lymphoma and myeloma department at MD Anderson Cancer Center in Houston until last fall and prior to that, in the department of stem cell transplantation. She was attracted to the Texas A&M College of Medicine as part of a broader effort by the Texas A&M University System to strengthen health sciences innovation among researchers.

“Dr. Parmar is working on absolutely transformational technology,” said John Sharp, chancellor of the Texas A&M System. “We’re proud that she has brought her innovative skills and dedication to improving patients’ lives to the Texas A&M faculty.”

General (Ret.) Mark A. Welsh III, president of Texas A&M University, also recognized Parmar’s contributions.

“Texas A&M University researchers, like Dr. Parmar, are driving innovation in the most crucial areas, solving problems to build a brighter, safer world for the people of Texas, the nation and beyond,” Welsh said. “We look forward to seeing the positive impact this invention will have, and we’re incredibly proud of Dr. Parmar and her work in this space.”

Dr. Simrit Parmar, an associate professor in the College of Medicine at Texas A&M University and founder of the clinical-stage biotech company Cellenkos Inc. in Houston, has invented a new cell therapy that shows strong promise for the treatment of amyotrophic lateral sclerosis (ALS), aplastic anemia and other devastating diseases.

Her therapy has been evaluated in dozens of human patients and has been shown to be safe and, to date, effective.

“If we succeed, millions of lives could be improved—organically, systematically and without significant side effects,” Parmar says.

Her results are promising enough to attract a major investment and partnership between Cellenkos and the leading medical center in Riyadh, Saudi Arabia. Announced May 22, it is the first alliance for medical clinical trials between the U.S. and Saudi Arabia.

Results also have led to two recent articles in NEJM Evidence. The articles draw attention to promising potential treatments for patients with aplastic anemia and ALS.

Parmar is giving hope to patients with a newly developed use of umbilical cord blood-derived T regulatory (Treg) cell therapy. The therapy treats the inflammation at the center of a number of hard-to-treat or untreatable illnesses. Derived from umbilical cord blood (CB), these Tregs are naturally wired to resolve inflammation without becoming harmful themselves.

The three key components of Parmar’s innovation are:

• The cell therapy does not require matching an individual donor to an individual patient.
• The cell therapy can be manufactured at scale, where a single starting CB unit can generate multiple ready-to-use products that can be frozen for up to three years and thawed later for use. This solves enormous logistical challenges associated with other cell-based treatments.
• By using umbilical cord-derived cells, the therapy sidesteps the dangers posed by adult-derived Tregs, which carry an elevated risk of further inflammation.

Parmar is addressing conditions where conventional treatments have failed. Her team has treated more than 80 patients suffering from multiple conditions, including ALS, aplastic anemia, myelofibrosis and acute respiratory distress syndrome (ARDS).

In April 2025, NEJM Evidence highlighted a study of Parmar’s technology for the treatment of ALS, where multiple infusions led to improvement in patients’ function decline.

In May 2024, the journal highlighted a study of her technology in the treatment of aplastic anemia, where a single infusion of her treatment led to durable independence from patients’ blood and platelet transfusions for up to 3.5 years.

The agreement between Cellenkos and King Faisal Specialist Hospital & Research Centre in Riyadh will begin with two clinical trials in Saudi Arabia for aplastic anemia and graft-versus-host disease and will pave the way for expansion into studies of patients with ALS and cardiovascular diseases. Cellenkos will also assist the Saudi Research Center with training and education in cell and gene therapy manufacturing.

In the U.S., Parmar hopes to have regulatory approval from the FDA in about two years for treatments of certain diseases, including aplastic anemia. She also is seeking funding for larger research studies of ALS patients in the U.S.

Parmar was in the lymphoma and myeloma department at MD Anderson Cancer Center in Houston until last fall and prior to that, in the department of stem cell transplantation. She was attracted to the Texas A&M College of Medicine as part of a broader effort by the Texas A&M University System to strengthen health sciences innovation among researchers.

“Dr. Parmar is working on absolutely transformational technology,” said John Sharp, chancellor of the Texas A&M System. “We’re proud that she has brought her innovative skills and dedication to improving patients’ lives to the Texas A&M faculty.”

General (Ret.) Mark A. Welsh III, president of Texas A&M University, also recognized Parmar’s contributions.

“Texas A&M University researchers, like Dr. Parmar, are driving innovation in the most crucial areas, solving problems to build a brighter, safer world for the people of Texas, the nation and beyond,” Welsh said. “We look forward to seeing the positive impact this invention will have, and we’re incredibly proud of Dr. Parmar and her work in this space.”