ImStem Announces First-in-Human Dosing of IMS001, a Proprietary T-MSC Stem Cell Therapy for Multiple Sclerosis, in U.S. Phase I Trial
Amstan Biotechnology Co., Ltd. recently announced that the first patient with multiple sclerosis has been dosed with IMS001, an investigational trophoblast-mesenchymal stem cell (T-MSC) therapy, in its Phase I clinical trial at the Shepherd Center in Georgia, USA. This marks the world’s first clinical trial involving systemic administration of embryonic stem cell-derived cells in humans. Previously, embryonic stem cell technology had only been applied in experimental treatments for localized neurological and ocular diseases. This breakthrough signifies that the development and application of embryonic stem cell technology have entered a more advanced stage.
Embryonic stem cells are stem cell lines derived from the blastocyst stage of embryos and established in vitro. They can be expanded indefinitely in culture and possess the potential to differentiate into nearly all tissues and organs of the body. Due to their capacity for unlimited expansion and differentiation, embryonic stem cells can be produced in a standardized manner similar to chemical drugs, thereby avoiding the limitations of source availability and inconsistent quality associated with traditional adult stem cells. This grants them significant application prospects in medicine. Compared with induced pluripotent stem (iPS) cells, embryonic stem cells have not undergone genetic engineering, offering greater advantages in terms of safety and genetic stability.
T-MSC is a specialized mesenchymal-like stem cell derived from embryonic stem cells. Utilizing Amstein’s patented two-step cell differentiation technology, T-MSCs are differentiated from single human embryonic stem cells via trophoblast intermediates. Following comprehensive preclinical safety and efficacy validation in animal models, as well as rigorous quality control studies for scalable manufacturing, the T-MSC investigational new drug (IND) has become the first human embryonic stem cell-derived mesenchymal stem cell therapy worldwide to receive FDA clinical trial authorization for the treatment of multiple sclerosis.
T-MSC, an investigational stem cell new drug, is an allogeneic off-the-shelf stem cell product that can be administered to patients via intravenous injection. Imstein plans to conduct a dose-escalation, open-label study in patients with relapsing-remitting, secondary progressive, or primary progressive multiple sclerosis who have failed prior disease-modifying therapies (DMTs), to evaluate the safety, tolerability, and exploratory efficacy of a single intravenous dose of the investigational T-MSC new drug.
“A series of T-MSC stem cell drug candidates under development by our company are all based on our research achievements over the past decade,” said Dr. Wang Xiaofang, Chief Technology Officer of Amstan and inventor of the T-MSC technology. “We are pleased to see the clinical trials proceeding smoothly, which undoubtedly represents a critical milestone. The clinical value of mesenchymal stem cells has gained broad consensus within the industry. Based on results from earlier animal studies and preclinical trials, T-MSC stem cells demonstrate notable advantages in consistency, efficacy, and scalable manufacturing, possessing inherent druggability. This characteristic is crucial for the widespread application of T-MSC novel drugs in various autoimmune diseases, including multiple sclerosis.”
Dr. Jeffery Cohen, a globally renowned expert in multiple sclerosis, Director of the Department of Neurology at Cleveland Clinic’s Mellon Medical Center, and a specialist in stem cell therapy for neurological disorders, has expressed great hope for the investigational new drug based on T-MSCs. “As a scientific advisor to Amstein, I am thrilled to see the smooth progress of this clinical trial. We sincerely hope to identify a medication that can effectively treat, or even cure, multiple sclerosis.”
IMS001 is an allogeneic mesenchymal stem cell product derived from a human embryonic stem cell line. Preclinical studies have demonstrated IMS001’s immunomodulatory reparative capacity and its ability to cross the blood-brain barrier (BBB). These inherent properties may confer significant advantages in the treatment of neurological disorders, autoimmune diseases, and rare diseases, thereby addressing higher unmet needs in disease management. Consequently, these characteristics may enable more effective reduction of disease relapse and sustained progression, as well as improved therapeutic outcomes in conditions such as multiple sclerosis.