Stem Cell Transplant For
Multiple Sclerosis (MS) is an autoimmune disease that affects the brain and spinal cord.
Myelin, which is a protective coating around nerves, enables signals to quickly be sent between nerves and the brain. In MS, the immune system attacks the myelin, which means that signals along the nerves become interrupted or distorted.
There are two main types of MS – relapsing and progressive. In relapsing MS, symptoms come and go, but in progressive MS, there is a continuous gradual worsening of symptoms.
Medications can help some forms of MS, but not cure it.
Recently, a new treatment has been developed, which can help people who have highly active relapsing MS. Stem cell transplantation (aka haematopoietic stem cell transplantation, aka HSCT).
How does stem cell transplantation (HSCT) help MS patients?
Multiple sclerosis is an autoimmune disease, and the goal of stem cell transplantation is to “reset” the immune system, by killing off the immune cells that are attacking the nervous system, and repopulating the immune system with healthy, new cells.
Who is suitable for stem cell transplantation (HSCT)?
Stem cell transplantation is very effective if you have highly relapsing MS, despite taking disease modifying therapy.
In relapsing MS, inflammation which occurs during immune attacks is visible on MRI imaging.
HSCT is not able to repair damaged nerve cells, and so isn’t a suitable treatment if you have a very progressive form of MS, or if there’s no signs of active inflammation on imaging.
It’s also important that you’re not significantly disabled by your condition, as measured by the EDSS (Expanded Disability Status Scale), and research points towards people under the age of 45 as benefitting the most.
What will happen during stem cell transplantation (HSCT)?
After a rigorous assessment by the transplantation team, you’ll be admitted to hospital.
You’ll undergo treatment that stimulates your bone marrow to make new stem cells (aka “mobilisation”), and around 10 days later, they will be drawn off from your blood stem, and then safely stored.
Next, you’ll undergo chemotherapy over the course of a few days to either partially, or totally erase your immune system. Chemotherapy comes with side effects, such as nausea, vomiting and joint pains, but you’ll be given drugs to combat these.
Next the collected stem cells are replaced into your system via a drip, and the cells get to work to regrow your immune system. During this phase, you’ll be susceptible to infections, and so you’ll be given antibiotics, and you may need blood or other transfusions to support your system.
During this time, you’ll be taken care of in an isolation room, and most patients spend around a month in hospital.
How effective is stem cell transplantation (HSCT) for MS?
Stem cell transplantation for MS is a new treatment, but already research shows that it can be an extremely effective treatment for the right individuals. Typically, people need a good 3-6 months to recover post-transplant, but they can anticipate a very worthwhile outcome from their treatment.
The MIST research trial followed up 110 people with MS, who’d had two or more relapses within the past year, whilst taking a disease modifying therapy. 55 patients were randomly assigned to continue with disease modifying therapy, whilst 55 underwent stem cell transplantation.
The researchers followed up with all the 100 participants and looked to see how many had had relapses. One year on, 1.92% of those treated with stem cell transplantation had suffered a relapse, compared with 64.3% of those who continued with disease modifying therapies.
After five years, 15.4% of the stem cell transplant patients had suffered a relapse, compared with 85.2% of those who continued with the disease modifying therapies.
The best news is, there were no reported deaths or life-threatening adverse events in the patients who had undergo stem cell transplantation.