Patient’s Own Stem Cells Treat Rare Neurological Disorder


Stiff-Person syndrome is a rare neurological disease that, for all intents and purposes, looks like an autoimmune disease. It is characterized by muscular rigidity that tends to come and go. This rigidity occurs in the muscles of the trunks and limbs. Patients with Stiff-Person syndrome also have an enhanced sensitivity to stimuli such as noise, touch, and emotional distress, and various stimuli may cause the patient to experience painful muscle spasms that cause abnormal postures and stiffening. Stiff-Person syndrome or SPS is more common in women than in men and SPS patients often suffer from other autoimmune conditions in addition to SPS (for example, pernicious anemia, diabetes, vitiligo, and thyroiditis). Unfortunately, the precise cause of SPS is not known, but again, it looks like an autoimmune condition.

A research team at Ottawa Hospital Research Institute has made a breakthrough in the successful treatment of SPS using bone marrow stem cell transplants. The medical director at the Ottawa Hospital Research Institute, Dr. Harold L. Atkins, who is also a physician in the Blood and Bone Marrow Transplant Program at The Ottawa Hospital and an associate professor at the University of Ottawa has used bone marrow transplants to two female SPS patients into remission.

SPS can leave patients bedridden and in severe pain, but thanks to Atkins and his team, the progression of the disease in these women has ceased, allowing both women to regain their previous function and leaving them well enough to return to work and normal everyday activities.

Adkins and his group published this case study in JAMA Neurology, which is produced by the Journal of the American Medical Association. This is the first documented report that taking stem cells from a person’s own body can produce long-lasting remission of stiff person syndrome.

“We approach these cases very carefully and are always aware that there have just been a few patients treated and followed for a short time,” says Dr. Atkins. Atkins and his extracted bone marrow stem cells from each woman, and then used chemotherapy to eliminate their immune systems. Once their immune system were reliably eliminated, both women had their own stem cells returned to their bodies in order to reconstitute their immune systems. This procedure essentially gives the immune system a “do-over.”.

“By changing the immune system, one hopes to put the stiff person syndrome into remission,” adds Dr. Atkins. “Seeing these two patients return to their normal lives is really every physicians dream.”

This very procedure, which is known as an “autologous stem cell transfer” or ASCT has been used to successfully treat people who suffer from autoimmune diseases such as multiple sclerosis, scleroderma, and systemic lupus erythematosis. Atkins and his team used high-doses of chemotherapy and antibodies that specifically bind lymphocytes to rid the women’s bodies of their rogue immune cells before their immune systems were regenerated using their own stem cells. Adkins an his colleagues viewed this as a viable treatment option based strategies that had been used to treat other autoimmune diseases.

Patient 1 was diagnosed with stiff person syndrome in 2005 at age 48 after experiencing leg stiffness and several falls. After her treatment, her symptoms disappeared and she was fully mobile again six months after receiving the stem cell transplant procedure in 2009.

Patient 2 was diagnosed with stiff person syndrome in 2008 at age 30. She had stopped working and driving, and had moved back in with her parents before her stem cell transplant in 2011. Also, she has been able to return to her work and previous activities, and has not had any stiff person syndrome symptoms in more than a year.

“The results achieved by Dr. Atkins and his team through this innovative treatment show how research at The Ottawa Hospital can lead to life-changing and, even life-saving care,” says Dr. Duncan Stewart, Chief Executive Officer and Scientific Director of the Ottawa Hospital Research Institute. “Translating research into better care for patients is what we’re all about at the research institute.”

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mburatov

Professor of Biochemistry at Spring Arbor University (SAU) in Spring Arbor, MI. Have been at SAU since 1999. Author of The Stem Cell Epistles. Before that I was a postdoctoral research fellow at the University of Pennsylvania in Philadelphia, PA (1997-1999), and Sussex University, Falmer, UK (1994-1997). I studied Cell and Developmental Biology at UC Irvine (PhD 1994), and Microbiology at UC Davis (MA 1986, BS 1984).