Information for patients: Chemo and blood stem cell transplantation for rare autoimmune diseases

January 4, 2017

A number of media stories have recently highlighted The Ottawa Hospital’s use of chemotherapy and blood stem cell transplantation to treat rare autoimmune diseases, such as myasthenia gravis, stiff person syndrome and scleroderma. These stories have generated a lot of hope and many questions. Answers to some of the most common questions are provided below.

1. What is the treatment and how does it work?

The treatment is called immunoablation and autologous hematopoietic stem cell transplantation (IAHSCT). In appropriate patients, it can be used to reset the immune system – replacing a diseased immune system with new one.

Normally the immune system protects the body against foreign disease-causing organisms, but in people with autoimmune diseases, the immune system mistakenly attacks the body’s own organs. It is hoped that IAHSCT can reset the immune system and prevent this.

IAHSCT is a two-step procedure:

The first step begins by giving a person chemotherapy followed by a medication that moves their hematopoietic (blood) stem cells from the bone marrow into their blood. These stem cells are then collected from the blood, purified and frozen. This step takes about two weeks.

The second step begins with high doses of chemotherapy drugs which are used to eliminate the person’s diseased immune system. The stem cells collected in the first step are thawed and then transplanted back into the same person, so that they can give rise to a new immune system. There are many potential side effects from the chemotherapy and patients require specialized care, usually in hospital, for at least four to six weeks.

The treatment is similar to a procedure that has been used to treat leukemia (blood cancer) for several decades.

2. Which conditions can be treated using this procedure and how can I get more information?

The Ottawa Hospital’s Bone Marrow Stem Cell Transplantation Program has performed a small number of these transplants in patients with severe autoimmune diseases who have not responded to conventional treatments. The diseases are listed below with links to more information.

Myasthenia gravis
• Results published in JAMA Neurology
Lay summary and CTV story

Stiff person syndrome
• Results published in JAMA Neurology
Media release and Canadian Press story

Patient story and CTV story
• The Ottawa Hospital performed this procedure based upon the experience of researchers from Europe who conducted a randomized clinical trial, published in JAMA.

Multiple sclerosis
• Results published in The Lancet
Media release

3. What are the risks of the treatment?

This treatment carries a significant risk complications including a risk of death. There is no guarantee of success.

4. How can I find out if I am a candidate for this procedure?

If you are interested in this procedure, please consult with your own medical specialist. He or she will be able to advise you and, if appropriate, make a referral to a hospital with experience in this area.

The Ottawa Hospital can only accept referrals from patients with Canadian provincial health coverage. In addition, referrals must come from specialists who have expertise in treating the autoimmune disease in question, and must be accompanied by detailed records or a synopsis of the illness. Please note that this treatment is only appropriate for people with selected autoimmune diseases.

5. Where else can I find information about stem cells and experimental therapies?

• The International Society for Stem Cell Research has compiled a list of questions to ask when considering stem cell treatments.
• The National Institutes of Health in the U.S. houses a database of clinical trials, which is searchable by disease and location.

The Ottawa Hospital: Inspired by research. Driven by compassion

The Ottawa Hospital is one of Canada’s largest learning and research hospitals with over 1,100 beds, approximately 12,000 staff and an annual budget of over $1.2 billion. Our focus on research and learning helps us develop new and innovative ways to treat patients and improve care. As a multi-campus hospital, affiliated with the University of Ottawa, we deliver specialized care to the Eastern Ontario region, but our techniques and research discoveries are adopted around the world. We engage the community at all levels to support our vision for better patient care.

Media contact

Jennifer Ganton
Director, Communications and Public Relations
Ottawa Hospital Research Institute
Office: 613-798-5555 x 73325
Cell: 613-614-5253