Autologous stem cell transplantation for Stiff Man Syndrome – pro
Stiff Person Syndrome (SPS) is a rare immune-mediated disabling neurological disorder characterized by muscle spasms and high GAD antibodies. There are only a few case reports of autologous haematopoietic stem cell transplantation (auto-HSCT) as a treatment for SPS. The most recent report of 10 patients, concludes A clinical trial of autologous stem cell transplantation exists: Stem Cell Transplantation for Stiff Person Syndrome (SPS), NCT02282514. This trial uses non-myeloablative conditioning. ” Based on our encouraging results of non-myeloablative hematopoietic stem cell transplantation, for patients with multiple sclerosis and chronic inflammatory demyelinating polyneuropathy, the investigators will investigate the role of non-myeloablative hematopoietic stem cell transplantation for patients with SPS who require assistance to ambulate: “Stiff Person Syndrome (SPS) is a rare immune-mediated disabling neurological disorder characterized by muscle spasms and high GAD antibodies. There are only a few case reports of autologous haematopoietic stem cell transplantation (auto-HSCT) as a treatment for SPS.”
A more recent small study of 23 patients found some efficacy. No one died from the procedure; however, one year after transplant, a participant died from disease-related progression. Notably, 26 percent did not respond to the therapy, but 74 percent of participants responded, and 47 percent have stayed in remission for an average of 3.5 years.
Kass-Iliyya L, Snowden JA, Thorpe A, Jessop H, Chantry AD, Sarrigiannis PG, Hadjivassiliou M, Sharrack B. Autologous haematopoietic stem cell transplantation for refractory stiff-person syndrome: the UK experience. J Neurol. 2021 Jan; 268(1): 265-275.
S, Sanders et al, Autologous Stem Cell Transplantation for Stiff Person Syndrome, Two Cases From the Ottawa Blood and Marrow Transplant Program.AMA Neurol. 2014; 71; 10): 1296-1299.
Hematopoietic Stem Cell Transplantation Found Safe and Effective for Patients with Stiff Person Spectrum Disorder https://journals.lww.com/neurotodayonline/fulltext/2021/01210/hematopoietic_stem_cell_transplantation_found_safe.4.aspx
IN THE PIPELINE
MOVEMENT DISORDERS
By Ashley Lyle
January 21, 2021