FDA Greenlights Stem Cells Trial Aiming to Improve Quality of Life
The U.S. Food and Drug Administration (FDA) has given the green light to a Phase 2 clinical trial that will assess the efficacy of using adult mesenchymal stem cells (MSCs) to treat the symptoms of mild to moderate multiple sclerosis (MS).
The non-profit Hope Biosciences Stem Cell Research Foundation (HBSCRF), which received the FDA authorization, believes such stem cell treatments may work to improve the quality of life of people with the neurodegenerative disease.
“Current treatments for MS involve suppressing the immune system and often come with unwanted side effects,” Donna Chang, founder of HBSCRF, said in a press release.
“We hypothesize that the stem cells, given in high, repeated doses, will be able to regulate the immune system so that the body stops attacking itself,” Chang said. “Degeneration must stop in order for regeneration to be possible.”
MSCs are a type of adult stem cell found in bone marrow, fat, and skin, among other tissues. These stem cells can mature into a variety of other cell types.
Because of their ability to suppress excessive immune responses — which often lead to chronic inflammation — protect the health of nerve cells, and regenerate some cells and tissues, stem cells have attracted attention as a potential therapy in immune and neurological disorders.
Researchers have had some success in using MSCs to delay MS progression, although none have yet become approved therapies.
In the proposed trial, 24 individuals will receive six MSC infusions over 32 weeks, or about eight months. Each infusion will consist of approximately 200 million cells taken from a patient’s own fat tissue — also referred to as an autologous stem cell transplant.
This amounts to approximately 1.2 billion MSCs per participant over the course of the study.
According to the HBSCRF, the organization is the only one capable of administering pure fat-derived mesenchymal stem cells in these high quantities over repeat treatments.
“While this is our first study for multiple sclerosis, our focus has been on chronic neurodegenerative diseases for some time,” Chang said.
“We have also been studying autoimmune diseases, and so have a deeper understanding as to how the body responds to repeated doses of mesenchymal stem cells,” she added.
The chronic neurodegenerative and autoimmune disorders that HBSCRF is studying include lupus, traumatic brain injury, Alzheimer’s disease, and Parkinson’s disease. Launched in March 2020, the organization to date has received FDA authorization for 22 clinical studies across 12 disease areas.
In its first year, the HBSCRF reported that it administered nearly 100 billion cells to participants in its studies, all at no cost to patients.
The foundation also is investigating the use of MSCs in treating COVID-19.