FDA prioritizes Hope’s stem cell treatment with RMAT designation
Company says designation is 1st for an MS treatment
The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to Hope Biosciences‘ adipose-derived mesenchymal stem cells (HB-adMSCs) to treat relapsing-remitting multiple sclerosis (RRMS).
RMAT status is given to therapies with compelling proof that they may substantially improve patient outcomes compared with existing treatments. It allows closer communication with the FDA and makes HB-adMSCs eligible for accelerated approval or priority review of a future biologics license application.
The designation is a first for an MS treatment, and is “a tremendous acknowledgment of our technology’s potential,” Donna Chang, Hope’s CEO, said in a company press release.
The FDA’s decision was based on clinical data suggesting the experimental therapy may help adults with RRMS live better lives.
“We are grateful to FDA for recognizing tremendous need for development of new solutions for multiple sclerosis,” Chang said. “We remain focused on getting treatment to people in need and will move rapidly to collaborate with FDA on advancing access, including filing our Biologics License Application.”
Using stem cells to reverse damage
In multiple sclerosis (MS), the immune system mistakenly attacks the protective covering around nerve cells in the brain and spinal cord, causing damage. Treatment usually involves using disease-modifying therapies that can reduce inflammatory damage, which lowers the risk of relapses and slows the accumulation of disability caused by the disease. However, available therapies cannot repair or reverse existing damage to nerve cells.
Hope’s stem cell therapy uses a patient’s adipose-derived mesenchymal stem cells, adult stem cells collected from fat tissue. When given the right cues, these stem cells can develop into other types of cells. They also secrete signaling molecules that could help repair damage caused by the disease.
A now-completed Phase 2 clinical study (NCT05116540) enrolled 24 adults with RRMS who had mild to moderate disability and were able to walk with or without aids. They were randomly assigned to receive six infusions of 200 million stem cells each, or a placebo, over 32 weeks (about eight months), and were followed for one year.
According to the company, the study was the first in which people with MS received such high doses of pure mesenchymal stem cells repeatedly.
The main goal of the trial, which ran at a single location in the U.S., was to watch for changes in the MS Quality of Life-54 Instrument, a clinical tool that measures various aspects of health-related quality of life, including physical and mental health.
“This tool … combines general and MS-specific points related to fatigue, physical and cognitive function, sexual impairment, pain, energy, mobility, level of disability, and other considerations – all contributors to quality of life we keep front of mind, when we think about how a therapeutic might revolutionize how we approach chronic degenerative disease,” said Ridhima Vij, PhD, clinical research scientist at Hope Biosciences Research Foundation, which sponsored the study.
Results showed that treatment with HB-adMSCs resulted in significant improvements in both physical and mental health compared with a placebo.
“HBRF has been an instrumental partner, leveraging their unparalleled expertise in cell therapy as well as their proven ability to deliver compassionate care tailored to our community’s needs through pioneering, patient-centered protocols,” Chang said. “It is a privilege to collaborate with them in advancing hope for patients.”
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