Clinical translation
From model system to registered study
What it took to move a differentiation protocol from the bench into a regulated clinical study — the preclinical package, the regulatory path, and what remains open.
IMS001
The first cell product derived from the T-MSC platform.
NCT04956744
Registered on ClinicalTrials.gov, sponsored by ImStem Biotechnology.
Phase 1
Listed enrolment of 30 participants with multiple sclerosis.
Aug 2021
Study start date of 31 August 2021 as listed on the registry.
Evidence base
The preclinical package
An Investigational New Drug application for a cell therapy requires evidence that goes well beyond an efficacy signal in a disease model. For IMS001 the laboratory completed studies of biodistribution, engraftment, tumorigenicity, toxicology and immunogenicity, with EAE selected as the principal efficacy model. The IND-enabling toxicology study was conducted with Charles River Laboratories.
Tracing where cells go after infusion is itself a research question. Preclinical safety evaluation and tracing of human mesenchymal stromal cell spheroids following intravenous injection into cynomolgus monkeys was published in Biomaterials, 2022, 289:121759.
Regulatory path
In March 2020, IMS001 received FDA Investigational New Drug clearance for the study of multiple sclerosis. To ImStem's knowledge this was the first allogeneic, human embryonic stem cell-derived, intravenously administered mesenchymal stem cell investigational product cleared by the FDA for study in multiple sclerosis patients. The first patient was screened for the Phase 1 study in the third quarter of 2021. IMS001 is administered intravenously and is designed not to require donor matching.
Status. The registry lists NCT04956744 as active and not recruiting. Trial status changes; the registry entry is the authoritative source. Nothing on this page should be read as a claim of demonstrated clinical efficacy — a Phase 1 study is designed principally to evaluate safety and tolerability.
What the trial does not yet answer
- How long infused cells persist in humans, and whether persistence tracks with any biological effect.
- Whether the immunomodulatory profile measured in vitro and in animals reproduces in patients.
- How dose, dosing interval and route should be optimised.
- The relative contribution of whole cells versus secreted extracellular vesicles — an active laboratory question.
Patients & clinicians
Where to find authoritative trial information
This site is a research resource. For trial eligibility, sites and current recruitment status, consult the registry entry directly, or speak with your neurologist.