Expert analysis: Treating MS with autologous hematopoietic stem cell transplant

Tanuja Chitnis, MD, senior neurologist at Brigham and Women’s Hospital and professor of neurology at Harvard Medical School and coauthor of the new National MS Guidelines, spoke with Healio about autologous hematopoietic stem cell transplant as treatment for MS.

She discussed patient groups that would most benefit from the treatments, risks and benefits and take-away messages from the new guidelines.

Which patient groups are best treated with autologous hematopoietic stem cell transplant?

[Those best treated are] relapsing, remitting patients who are under the age of 45 and who have less than 10 years of disease duration.

What are the risks and benefits of an autologous hematopoietic stem cell transplant?

Stem cell transplant is a highly effective way of reducing inflammation, including new relapses and new MRI lesions and it’s quite effective, especially for younger relapsing patients. It is a very involved procedure, which takes anywhere from around 2 weeks for the procedure. In addition, a great deal of follow-up as well as pre-screening [is required]. The concerns are, after the transplant, there is a high risk for infection and so post-transplant we have to be very careful about infections and then potentially for the next 1 to 2 years, there is a significant infection risk.

Is there more research that is needed? In what areas?

One outstanding question is: ‘what is the best combination of treatments or regimens?’ – and there's several different regimens that have been used; ... The question about who is the best candidate still needs more information and the main issue is optimizing safety.

How pervasive is the knowledge and expertise of clinicians who can perform autologous hematopoietic stem cell transplant? What does access to this type of treatment look like, whether distance, monetary or otherwise?

Clinicians need to be very familiar with this procedure to administer it. They may potentially be working with an oncology unit because oncologists use this procedure more frequently than neurologists typically do. Sometimes people are cross trained in neurology and oncology or work with an oncology unit, or have developed their own expertise as a neurologist. Conduct of autologous hematopoietic stem cell transplant really requires that deep understanding of the procedure, as well as the side effects.

Currently, there are very few centers within the U.S. who are performing stem cell transplant, and I won’t guess how many but it’s less than five. It’s a very specialized type of treatment [and] it may or may not be covered by insurance. I had a recent experience with a patient who was seeking this, and we applied to several units that were doing this and the waiting lists were too long or for some other reasons they were not able to take her. Some patients have gone overseas. ... Standardizing procedures and collecting data from any transplant to take place in a registry for MS patients will help to answer questions like we’ve been numerating. This type of work will answer questions including who responds better, is there a certain patient profile, which regimens work better, which regimens have fewer side effects. There is also a rare but still tangible risk for mortality. It was up to 10%, but it has improved significantly as people have gotten better at understanding how to give these drugs and do this procedure and now the range is 0% to 4%. But again, it depends on return area their expertise and things should improve as we move forward.

The cost is anywhere from about $100,000 to $150,000 for one procedure. But insurance generally does not cover the procedure, with rare exceptions. Comparatively, some of the disease modifying treatments this is in the range of 2 years of treatment.

What type of follow up is needed?

Monitoring their blood count and making sure that there are white blood cells, including their T-cells and B-cells are returning to normal levels. It is monitoring for any viruses and immunizations also would need to be reintroduced.

What important messages do you want other clinicians to take away from the new National MS Society Guidelines?

Stem cell transplant is an option for people who have a significant breakthrough disease and are not able to take or have failed several other MS treatments. The best patient profile, based on all the studies so far, show that it's more effective in people younger than 45 and who are relatively early in their relapsing disease within 10 years so I would not use it in a long-term progressive MS patient. It’s not a good solution there, and there is significant potential mortality or morbidity. That’s probably the most important take home message.