Could a stem cell transplant stop the immune system from attacking the brain?
NCT ID NCT00716066
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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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53 people
The number who actually took part.
- Start date
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Jun 2008
- Expected to finish
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Jan 2030
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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Up to 71 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Patients with an autoimmune disorder of the central or peripheral nervous system will be eligible; this will include: * Primary Central Nervous System (CNS) vasculitis * Rasmussen's encephalitis * Autoimmune peripheral neuropathy (anti-Hu \[Anna-1\], anti-GM1 \[GD1b\], anti-MAG, anti-ganglioside, anti-sulfatide) * Autoimmune cerebellar degeneration * Gait Ataxia with Late age Onset Polyneuropathy (GALOP) * Stiff Person Syndrome * Chronic Inflammatory Demyelinating Polyneuropathy * Myasthenia Gravis * Lambert-Eaton myasthenic syndrome * Human T-cell lymphotropic virus (HTLV)-1-associated myelopathy (HAM) / tropical spastic paraparesis (TSP) * Opsoclonus/myoclonus (anti-Ri) * Neuromyelitis optica * Multiple sclerosis * Other central or peripheral nervous system autoimmune diseases as approved by study neurologists and the Fred Hutchinson Cancer Research Center (FHCRC) faculty at Patient Care Conference (PCC) * Patients must satisfy the criteria for a diagnosis of one of the severe neurological autoimmune disorders outlined * Patients age =\< 70 years * Evidence of disease activity as outlined (e.g. gadolinium enhancement on magnetic resonance imaging of the brain or clinical progression) * Patients must have failed at least 2 lines of standard therapy as outlined for the specific diseases * DONOR: Sibling of any patient enrolled on this protocol proven by ABO typing, human leukocyte antigen (HLA) typing and variable number tandem repeat (VNTR) analysis to be syngeneic with the patient (e.g. identical twin) * DONOR: Willing to undergo multiple apheresis procedures (except donors \< 12 years who will undergo bone marrow harvests) Exclusion Criteria: * Age \>= 71 years * Pregnancy or expressed plans to become pregnant within 1 year of the procedure * Patients who are serologically positive for human immunodeficiency virus (HIV) * Patients with pulmonary, cardiac, hepatic or renal impairment that would limit their ability to receive cytoreductive therapy and compromise their survival; this should include patients with any of the following: * Severe pulmonary dysfunction associated with a carbon monoxide diffusing capacity (DLCO) (corrected for hemoglobin) \< 60%, or requires supplemental oxygen; patients who are unable to perform pulmonary function test (because of underlying disease) will be excluded if the oxygen saturation is \< 92% on room air * Uncontrolled malignant arrhythmias, or clinical evidence of congestive heart failure (New York class III-IV) or ejection fraction \< 50% * Renal disease with estimated glomerular filtration rate (GFR) by creatinine clearance or iothalamate clearance \< 50 ml/min/1.73 m\^2 body surface area * Serum glutamate pyruvate transaminase (SGPT)/aspartate aminotransferase (AST) \> 3 times normal or direct bilirubin greater than 2.5 mg/dL on two repeated tests * Active uncontrolled infection * Demonstrated lack of compliance with prior medical care * Patients whose life expectancy is limited by illness other than their neurological condition * Patients with evidence of myelodysplasia * Active malignancy (excluding localized squamous cell or basal cell carcinoma of the skin) * DONOR: Inadequate documentation that donor and recipient are syngeneic * DONOR: Donors who do not fulfill criteria as apheresis donors as established by institutional guidelines * DONOR: Concordant for autoimmune neurological disease(s) as determined by neurological evaluation
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Colorado Blood Cancer Institute
Denver, Colorado, 80218, United States
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Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, 98109, United States
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Swedish Medical Center-First Hill
Seattle, Washington, 98122-4307, United States
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