Could a stem cell transplant stop the immune system from attacking the brain?

NCT ID NCT00716066

Ongoing Disease control Sponsor: Fred Hutchinson Cancer Center Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 2 trial tests whether high-dose chemotherapy followed by a stem cell transplant can stop the immune system from attacking the nervous system in people with autoimmune neurologic diseases like multiple sclerosis and stiff person syndrome. The treatment aims to weaken the faulty immune response and then rebuild it with the patient's own stem cells. Researchers are monitoring side effects and how well the disease responds in 53 participants who have not improved with standard therapies.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: High-dose chemotherapy (carmustine, etoposide, cytarabine, melphalan) plus antithymocyte globulin followed by stem cell transplant
What this could lead to: If it works, this could offer a way to stop the immune system from attacking the nervous system in people with severe autoimmune neurologic diseases that haven't responded to other treatments.
What could go wrong: This is an early phase 2 trial with only 53 participants, so results may not apply to everyone. The high-dose chemotherapy has serious risks, including severe organ toxicity and death.

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Conditions

The condition(s) this trial relates to.

autoimmune disease autoimmune disorder of the nervous system central nervous system vasculitis cerebellar degeneration chronic inflammatory demyelinating polyradiculoneuropathy Lambert-Eaton myasthenic syndrome myasthenia gravis neuromyelitis optica opsoclonus-myoclonus syndrome Rasmussen subacute encephalitis stiff-person syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

AUTOIMMUNE DISEASE AUTOIMMUNE NERVOUS SYSTEM DISORDER AUTOLOGOUS TRANSPLANT AUTOIMMUNE CENTRAL NERVOUS SYSTEM VASCULITIS CEREBELLAR DEGENERATION CHRONIC INFLAMMATORY DEMYELINATING POLYNEUROPATHY CIDP TRANSPLANT HCT FOR NEUROLOGIC AUTOIMMUNE DISORDERS LAMBERT-EATON MYASTHENIC SYNDROME MS STEM CELL TRANSPLANT MULTIPLE SCLEROSIS STEM CELL TRANSPLANT MULTIPLE SCLEROSIS TRANSPLANT MYASTHENIA GRAVIS MYASTHENIA GRAVIS TRANSPLANT NEUROLOGIC AUTOIMMUNE DISEASE NEUROMYELITIS OPTICA OPSOCLONUS MYOCLONUS SYNDROME RASMUSSEN SUBACUTE ENCEPHALITIS STIFF PERSON SYNDROME

Contacts and locations

Locations

Colorado Blood Cancer Institute

Denver, Colorado, 80218, United States
Fred Hutch/University of Washington Cancer Consortium

Seattle, Washington, 98109, United States
Swedish Medical Center-First Hill

Seattle, Washington, 98122-4307, United States

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