造血干细胞移植,神经脊髓炎 z-bo 2020-09-30 343

　　In a new clinical study, researchers at Northwestern University's Fernberg School of Medicine and May York Clinic reported that hematopoietic stem cell transplantation can reverse a debilitating neurological disease called neuromyelitis opticica (Neuromyelitisoptica) in patients. Within 5 years after diagnosis, half of the people are blind and lose the ability to walk. Most patients are well-maintained 5 years after hematopoietic stem cell transplantation, and can avoid drugs that cost up to $500,000 per year.

　　Optic neuromyelitis was previously classified as a rare subtype of multiple sclerosis (MS) and is now considered to be another disease. Unlike multiple sclerosis and most other autoimmune diseases, neuromyelitis optica has AQP4, a biomarker related to disease activity. After hematopoietic stem cell transplantation, there is no identifiable AQP4 in the patient’s blood. The first author of this article, Northwest Dr. Richard Bart, professor of medicine at the University's Fairberg School of Medicine and head of immunotherapy and autoimmune diseases, said: There is a clear difference between hematopoietic stem cell transplantation and drug therapy. Hematopoietic stem cell transplantation improves the patient's neurological dysfunction and quality of life. Their condition has improved and is a sign of disease. "

　　Mayo Clinic has applied for a patent for the biomarker AQP4, and is collaborating with Bart and Northwestern University on this biomarker analysis.

　　In this clinical study, twelve patients with neuromyelitis optica received hematopoietic stem cell transplantation. Five years after the transplantation, only 2 of these 12 patients relapsed and needed retreatment. This seems to be the fourth chronic disease that can be eliminated by hematopoietic stem cell transplantation.

　　The purpose of hematopoietic stem cell transplantation is to make the defective immune system function normally. Hematopoietic stem cells are the patient. Chemotherapy is extracted from the bone marrow or blood of the human body. It completely destroys the immune system, then reintroduces hematopoietic stem cells into the patient's body, and then migrates to the bone marrow in the body to reset the immune system.

　　Bart pioneered in this field. He was the first person in medical publications to propose the use of hematopoietic stem cell transplantation to treat multiple sclerosis and the first person to do so in preclinical animal models. He is the first person in the United States to use hematopoietic stem cell transplantation to treat multiple sclerosis; a patient with sexual sclerosis; Bart is also the first to use hematopoietic stem cell transplantation to treat systemic sclerosis and multiple sclerosis. We conducted a randomized clinical trial of JAMA and achieved very positive results.

　　In January 2019, Bart published a randomized clinical study on JAMA, which has the effect of delaying recurrence. We have shown that hematopoietic stem cell transplantation can reverse the neurological dysfunction in patients with sclerosis. During this period, most patients will delay or prevent further progressive sexual dysfunction, or according to a study published by Bert, hematopoietic stem cell transplantation reverses other The diseases are systemic sclerosis and chronic inflammatory demyelinating polyneuropathy.