Harold Atkins told Medscape News, "We are always reluctant to talk about this type of disease being cured, but these patients have all been disease free without any
maintenance therapy since the procedure, which is very encouraging," Harold Atkins is a leading researcher and an MD at University of Ottawa, Ontario, Canada,
Dr Atkins and colleagues describe their findings in a paper published online in JAMA Neurology.
"While autologous hematopoietic stem cell transplants have been successfully used in other autoimmune conditions, such as multiple sclerosis, this is the first time this treatment has been described in myasthenia gravis, to our knowledge," Dr Atkins said.
"The key point with stem cell transplant is that we are not just globally suppressing the immune system, as is the case with usual drug therapies for autoimmune condition. We are instead wiping out the malfunctioning immune system and allowing a new one to grow that is functioning properly by using the patient's own stem cells, which have not yet developed the autoimmune memory," he explained.
The procedure has been associated with remarkable results. Since the current paper was submitted, another patient has been treated, bringing the total to eight. All eight have remained completely symptom free on no further treatment, and the first patient treated is now at 13 years post-procedure.
"A Grueling Procedure"
However, despite these impressive results, this therapy is not suitable for mainstream use.
"It is a grueling procedure which carries significant risks, so will only be suitable for very severe patients who have run out of other options," Dr Atkins comments. "Most myasthenia gravis patients can be controlled on drug therapy, and this is not appropriate for them. Because the immune system is eliminated with very-high-dose chemotherapy during stem cell therapy, this brings about many toxic side effects and carries a small risk of death, so patients really do have to be having regular myasthenia crises and be refractory to all other therapies before considering this. But for some of these patients it is a promising option."
The seven patients reported in the current paper had a mean age of 44 years. They all had persistent severe or life-threatening myasthenia gravis-related symptoms despite continued use of intensive immunosuppressive therapies.
Autologous hematopoietic stem cell grafts were mobilized with cyclophosphamide and granulocyte colony-stimulating factor, collected by peripheral blood leukapheresis, and purified away from contaminating lymphocytes using CD34 immunomagnetic selection. Patients were treated with intensive conditioning chemotherapy regimens to destroy the autoreactive immune system, followed by graft reinfusion for blood and immune reconstitution >>> CONTINUE READING
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