Stem cell transplant and clinical trials for autoimmune diseases

Colorado Blood Cancer Institute is a FACT-accredited hematologic transplant and cellular therapy center with extensive experience in stem cell transplantation and immune-based therapies. In select cases, this expertise is applied to the treatment of severe autoimmune diseases through autologous hematopoietic stem cell transplantation (AHSCT) and investigational cellular therapies, including CAR-T cell therapy, offered through clinical trials.

Patients are evaluated and treated in close collaboration with referring neurologists, rheumatologists, and pulmonologists.

Make an appointment

If you would like to learn more about our treatments options or be screened for our clinical trials, contact Cat Booher at (303) 577-6480.

Autologous stem cell transplant (AHSCT) for MS, SSc and rare autoimmune neurologic disorders

Active-progressive or relapsing-pemitting Multiple sclerosis (RRMS)

Multiple sclerosis (MS) is a chronic autoimmune disease in which the immune system attacks the protective covering of nerve fibers in the brain and spinal cord, leading to inflammation and damage within the central nervous system. Symptoms may include fatigue, sensory changes, visual disturbances, and progressive disability.

With decades of experience, our team offers myeloablative AHSCT for select patients according to the recommendations published by the National MS Society in 2020 and the American Society of Blood and Marrow Transplantation in 2019. This therapy is available to patients with substantial MS disease activity despite treatment with best available medications, known as disease modifying therapies (DMTS).

Research has shown that patients with active, inflammatory MS early in their disease course are the most likely to benefit from AHSCT. Age, time since diagnosis, recent disease activity (MRI changes or worsening disability), and level of disability will all be considered when screening a patient for eligibility. Patients who have not had disease activity in the past 3 years, who have not tried disease modifying therapy, or who have other conditions that would put them at significant risk during transplant will not be eligible for AHSCT. Evaluation and decision making occur in close collaboration with a neurologist.

Systemic sclerosis (SSc, Scleroderma)

Systemic sclerosis (SSc), sometimes called scleroderma, is a systemic autoimmune disease that can affect the skin and internal organs, including the lungs, heart, gastrointestinal tract, and kidneys. It is distinct from localized forms of scleroderma, which do not involve internal organ disease.

Our team offers AHSCT to select patients with systemic sclerosis and severe, progressive disease despite standard immunosuppressive therapy.

Patients most likely to benefit from AHSCT are those with early, active disease and evidence of progression, including skin involvement and/or internal organ disease such as interstitial lung disease. Eligibility is determined on an individual basis, considering disease duration, rate of progression, organ involvement, prior therapies and overall medical fitness for transplant. Localized scleroderma is not treated with AHSCT.

Rare autoimmune neurologic disorders

Autologous hematopoietic stem cell transplantation (AHSCT) may also be considered for select patients with severe autoimmune disorders affecting the central or peripheral nervous system. These conditions are uncommon and treatment decisions are made on an individual basis in close collaboration with the patient's neurologist and other specialists.

Conditions evaluated at our center include:

  • Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
  • Stiff-Person Spectrum Disorder (SPSD)
  • Myasthenia Gravis
  • Primary Angiitis of the CNS (PACNS)
  • Rasmussen Encephalitis
  • Immune-Mediated Peripheral Neuropathies (e.g., anti-Hu [ANNA-1], anti-GM1, anti-MAG, antiganglioside, antisulfatide)
  • Autoimmune Cerebellar Ataxia
  • Autoimmune Encephalitis (including paraneoplastic and antibody-mediated subtypes)
  • Late-Onset Ataxia with Neuropathy (also known as Gait Ataxia with Late Age Onset - Polyneuropathy (GALOP))
  • Lambert–Eaton Myasthenic Syndrome (LEMS)
  • HTLV-1–Associated Myelopathy / Tropical Spastic Paraparesis (HAM/TSP)
  • Opsoclonus-Myoclonus Syndrome (OMS), including anti-Ri–associated disease
  • Neuromyelitis Optica Spectrum Disorder (NMOSD)
  • Other rare autoimmune central or peripheral nervous system disorders

Determining eligibility

Because these disorders are rare and often complex, eligibility for AHSCT is determined on an individual basis. Factors considered include:

  • Disease severity and progression
  • Response to prior therapies
  • Overall medical fitness for transplant
  • Potential risks and expected benefits
  • Recommendations from the patient's treating neurologist and multidisciplinary team
Insurance coverage

Insurance coverage for autoimmune disease is diagnosis and plan specific. Approval for AHSCT is not guaranteed, particularly for rare autoimmune neurologic disorders, and some insurers may consider treatment investigational. Our team works with patients, referring physicians, and the insurance company to obtain approval. This may take time and, rarely, it can be unsuccessful.

Autoimmune clinical trials at CBCI

Studies in development

  • Allogeneic cellular therapy for primary progressive MS
  • AHSCT for interstitial lung disease

Availability is dependent on regulatory approval and study activation timelines.