Neurological Autoimmune Disorders
Autoimmune diseases can take several different forms, and even within the same disease type, the progression and severity of symptoms can vary greatly from patient to patient. In some patients, symptoms will develop rapidly, and they may fail conventional therapy. These patients may be eligible for high-dose chemotherapy and autologous hematopoietic stem cell transplantation (AHCT) as part of a clinical trial or as standard therapy.
Here are some of the most common autoimmune conditions we treat:
Multiple Sclerosis (MS) is an autoimmune disease where the immune system attacks and eventually destroys the nerves in the brain and spinal cord. Most people with MS have an initial immune attack, followed by a period of stability. As time goes on, they might have episodes of new and worsening symptoms with failure of conventional therapy.
MS includes these subtypes:
- Clinically Isolated Syndrome (CIS)
- Relapsing-Remitting MS (RRMS)
- Secondary progressive MS (SPMS)
- Primary Progressive MS (PPMS)
MS Symptoms: MS can cause loss of vision, weakness or incoordination, loss or changes in sensation, problems thinking or remembering things, urinary incontinence and other disabilities. While there are more than a dozen approved medicines to treat relapsing-remitting forms of MS, some people do not respond to treatment, which is why doctors are actively researching other new drugs and treatments, and doing clinical trials including the BEAT-MS study.
Recommendations for Relapsing/Remitting MS: Dr. Richard Nash helped to write a position statement released in 2019 by the American Society for Transplantation and Cellular Therapy (ASTCT). In it, MS neurologists and transplant physicians reviewed recent studies and trials and recommended high-dose chemotherapy and AHCT (stem cell transplant) for the treatment of active relapsing forms of MS in patients who have failed previous conventional therapy, to prevent further clinical relapses, brain lesions, and worsening disability. The report stated that “Based on the available evidence, the ASTCT recommends that treatment-refractory relapsing MS with high risk of future disability be considered a ‘standard of care, clinical evidence available’ indication for AHCT.”
Please click on the links below for further published studies:
- ASTCT position statement: https://www.bbmt.org/article/S1083-8791(19)30139-9/fulltext
- HALT- MS outcomes: https://n.neurology.org/content/88/9/842
- JAMA: https://jamanetwork.com/journals/jama/fullarticle/2720728
CBCI offers stem cell transplant for patient who meet criteria both on and off clinical trials. Please view the criteria for stem cell transplant for MS.
We will also be opening a clinical trial for Primary Progressive MS in 2020. This will involve allogeneic transplant (getting stem cells from a donor). More information will be posted as it becomes available.
Coming Soon: BEAT-MS: The BEAT-MS clinical trial is a study conducted by the Immune Tolerance Network and supported by the NIH (NIAID). We will begin enrolling eligible patients for this new randomized, multi-center clinical trial as soon as it opens (anticipated late 2019/early 2020). The BEAT-MS trial will compare the best available therapy for MS to high-dose chemotherapy and autologous hematopoietic stem cell transplantation. When the study opens, this information will be updated.
Stiff Person Syndrome (SPS)
Also called Stiff Man syndrome, stiff person syndrome is a rare condition affecting the nervous system, primarily the brain and spinal cord. The syndrome is progressive and painful and affects twice as many women as men. The symptoms may include extreme muscle stiffness, rigidity and spasms both the trunk and limbs that make it extremely hard to move around. Most people with SPS experience symptoms between age 30 and 60 and eventually become disabled.
Myasthenia gravis is a chronic autoimmune neuromuscular disease characterized by muscle weakness in the arms and legs as well as the muscles used for breathing. Oftentimes muscles used to control eye and eyelid movement, facial expressions, talking, chewing and swallowing are also affected. The muscle weakness is worse after doing a lot of activity and usually improves with rest. Along with weakness in the arms, hands, fingers, leg and neck, symptoms can include drooping of one or both eyelids, changes in facial expression, blurred or even double vision, shortness of breath, impaired speech and trouble swallowing.
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Chronic Inflammatory Demyelinating Polyneuropathy, or CIDP, is a neurological disorder that targets the nerves in your body. It's caused by nerves and nerve roots that get inflamed. While symptoms can vary from person to person, the most common ones include feeling tired, slow reflexes, and weak arms and legs. There also might be numbness and pain. Symptoms must persist for at least eight weeks for CIDP to be considered. CIDP is most common in older adults. Men are diagnosed with it more often than women.
We treat other rare autoimmune central or peripheral nervous system disorders, including:
- Primary CNS Vasculitis
- Rasmussen's Encephalitis
- Autoimmune Peripheral Neuropathy
- Autoimmune Cerebellar Degeneration
- Gait Ataxia with Late Age Onset Polyneuropathy (GALOP)
- Lambert Eaton Myasthenic Syndrome
- HTLV-1- Associated Myelopathy (HAM)/ Tropical Spastic Paraparesis (TSP)
- Opsoclonus/ Myoclonus (Anti-Ri)
- Neuromyelitis Optica
Promising research still leaves open questions.