Neurology: clinical trials have shown that hematopoietic stem cell transplantation is reversible for optic neuromyelitis

October 15, 2019 / Biovalley BIOON / - -- in a new clinical study, researchers from Northwestern University's Feinberg School of medicine and mayo clinic report that hematopoietic stem cell transplantation can reverse a debilitating neurological disease called neuromyelitis Optica's disease causes half of the patients to be blind and lose the ability to walk within five years after diagnosis Most patients remain well after five years of hematopoietic stem cell transplantation, and can avoid drug treatment that costs up to $500000 a year The related research results were recently published in the Journal of Neurology The title of the paper is "automatic nonmyloablative thermopoietic stem cell transformation for neuromelitis optica" The picture is from cc0 public domain Neuromyelitis optica, previously classified as a rare subtype of multiple sclerosis (MS), is now considered another disease Unlike multiple sclerosis and most other autoimmune diseases, optic neuromyelitis has biomarker AQP4, which is related to disease activity After hematopoietic stem cell transplantation, patients no longer have recognizable AQP4 in their blood "None of the previous treatments resulted in the continued disappearance of AQP4 or the patient's freedom from treatment," said Dr Richard Burt, lead author of the paper and professor of medicine at Feinberg School of medicine at Northwestern University and head of immunotherapy and autoimmune diseases "There is a clear difference between hematopoietic stem cell transplantation and drug therapy," Burt said Hematopoietic stem cell transplantation improved the neurological dysfunction and quality of life They got better, and the disease marker disappeared for as long as five years after the transplant Mayo Clinic has a patent for biomarker AQP4, which was analyzed in collaboration with Burt and Northwestern University In this clinical study, 12 patients with optic neuromyelitis received hematopoietic stem cell transplantation Five years after transplantation, only two of the 12 patients had relapses and had to be re treated This seems to be the fourth chronic disease that can be reversed by hematopoietic stem cell transplantation The goal of hematopoietic stem cell transplantation is to make the defective immune system work normally Hematopoietic stem cells are taken from a patient's bone marrow or blood, and then their immune system is completely destroyed by chemotherapy Next, they reintroduce their hematopoietic stem cells into the patient's body, where they migrate to the bone marrow, allowing their immune system to reset Burt pioneered this area He was the first person to propose hematopoietic stem cell transplantation for multiple sclerosis in medical publications, the first to do so in preclinical animal models, and the first in the United States to use hematopoietic stem cell transplantation for multiple sclerosis patients Burt also conducted the first clinical trials of randomized hematopoietic stem cell transplantation for systemic and multiple sclerosis, and achieved very positive results In January 2019, Burt published a randomized clinical study on JAMA, which showed that in patients with relapsing remitting multiple sclerosis, hematopoietic stem cells In more than five years, most patients delayed or prevented further sexual dysfunction, or showed signs of new disease activity According to the study published by Burt, other diseases reversed by HSCT are systemic sclerosis and chronic inflammatory demyelinating polyneuropathy (biooon Com) reference: 1 Richard K Burt et al Automatic nonmyeloablative thermopoietic stem cell transformation for neuromelitis optica Neurology, 2019, doi:10.1212/WNL.0000000000008394 2.Richard K Burt et al Effect of Nonmyeloablative Hematopoietic Stem Cell Transplantation vs Continued Disease-Modifying Therapy on Disease Progression in Patients With Relapsing-Remitting Multiple Sclerosis JAMA, 2019, doi:10.1001/jama.2018.18743 3.Stem cell transplant reverses disabling MS-like disease https://medicalxpress.com/news/2019-10-stem-cell-transplant-reverses-disabling.html