The U.S. Food and Drug Administration (FDA) has lifted the clinical hold and cleared the investigational new drug (IND) application to assess IMS001 in the treatment of multiple sclerosis (MS), ImStem Biotechnology announced.
The company plans to start a Phase 1 study in the U.S. this year to evaluate the stem cell therapy in several types of MS, including primary progressive (PPMS), secondary progressive (SPMS), and relapsing-remitting MS (RRMS).
“As a company, we are excited to have reached this clinical stage, and to move one step closer to providing an effective treatment option for patients suffering from MS,” Michael Men, CEO of ImStem, said in a press release.
IMS001 is an investigational stem cell therapy that uses cells from donors (allogeneic) to be administered via intravenous (IV) infusion to MS patients.
Specifically, the therapy consists of human embryonic stem cells (ESC)-derived mesenchymal stem cells (hES-MSC). These cells harbor regenerative and immunomodulatory properties, meaning they can control and stabilize an overreactive immune system.
A previous study with two distinct animal models of autoimmune disease, showed that these cells expressed a high level of the immune inhibitory molecule PD-L1, and led to a durable control of harmful immune system responses.
Moreover, ESCs as a source for MSCs not only have less variability and more potency than cells derived from adult sources, but also have the advantage of being a relatively stable and unlimited supply source.
hES-MSCs also were shown, in in vitro studies, to produce low levels of pro-inflammatory molecules, which minimizes its potential inflammatory and disease-exacerbating adverse events (side effects), an advantage over other stem cell models.
In a mouse model of MS, the potential cell therapy reduced clinical symptoms and prevented neuronal demyelination (loss of myelin, the protective layer around neurons), a hallmark of MS. Importantly, these cells showed a better ability to migrate into the inflamed central nervous system (CNS) tissues, and potentially stabilize the the blood-brain-barrier (BBB).
So far, IMS001 has undergone a series of IND-enabling studies, including on its distribution in tissues, ability to proliferate, toxic effects, immune system-related toxicity, and behavior in the body (pharmacology studies), according to ImStem.
In 2017, the U.S. Patent and Trademark Office issued a patent for the investigational cell therapy and its method of production. IMS001 was developed by ImStem in collaboration with the University of Connecticut (UConn). ImStem is a spinoff company from the UConn Stem Cell Core Lab.
“As an inventor of our proprietary technology, we look forward to building a company whose foundation lies in our science based upon years of pioneering research, and we are so grateful for the continuous support from the University of Connecticut,” said Xiaofang Wang, PhD, chief technology officer of ImStem Biotechnology.
According to the company, given the mechanism of action shown in preclinical studies, IMS001 is expected to reduce relapses, disability progression, and induce disease arrest in patients with MS.
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