Jennifer Molson couldn’t feel anything from her chest down. Her partner, Aaron, had to bathe and dress her, and cut her food.
“I remember making a bowl of cereal, putting it on my walker, and dropping it on the floor,” Jennifer says. “I just sat on the floor and cried.”
Just four years earlier, in 2000, she’d been diagnosed with an early, aggressive form of multiple sclerosis, which had already relapsed. Switching to a new, higher-dose medication brought no relief.
So, when a neurologist at the Ottawa, Canada, hospital where Molson was getting treatment suggested she join a clinical trial, she was interested.
The trial was exploring whether a stem cell transplant, using stem cells from her own bone marrow, could get her MS under control.
“They were trying to stop my disease activity,” Molson says.
The procedure is called an autologous hematopoietic stem cell transplantation, or aHSCT. You may have heard of it as a bone marrow transplant. Here’s how it works.
- First, you get treatment to prompt your bone marrow to release blood stem cells into the bloodstream.
- Next, those blood stem cells are gathered and frozen in a lab.
- Then you get high-dose chemotherapy to eliminate your malfunctioning immune system.
- Then you get a transplant of hematopoietic stem cells, which are found in the blood and bone marrow. These are “autologous” stem cells, meaning that they come from your own body.
The goal is to restore more normal immune function, according to Jeffrey Cohen MD, director of the Experimental Therapeutics Program at the Mellen Center for Multiple Sclerosis Treatment and Research at the Cleveland Clinic.
Weighing the Risks
This type of stem cell transplantation for MS is considered experimental in the U.S. It can work well but does have serious risks and involves spending weeks in a hospital. Along with side effects like nausea, hair loss, and infertility that are common with chemotherapy, there’s a small chance of fatal complications.
New research shows that an autologous hematopoietic stem cell transplant, or aHSCT, can stop inflammation in the brain and can stabilize or improve functioning in relapsing-remitting MS. Other studies found that people treated with aHSCT were less likely to have their MS worsen or relapse than those treated with disease-modifying drugs.
At the Cleveland Clinic, Cohen is leading a National Institutes of Health-funded clinical trial called BEAT-MS to show that the procedure, which costs upwards of $150,000 and is rarely covered by insurance, is a safe, cost-effective approach to treating MS. He aims to finish recruiting patients to the trial this year.
Cohen says that people with MS who get stem cell transplants might get “potent disease control benefits” that last up to 10 years with no need for more medication. But he notes that it’s not the right treatment for everyone with MS.
“It’s a risky procedure,” he says. “We select it for patients that need it but avoid it in patients that can benefit from other therapies.”
Molson knew the risks were high. But she felt like she was out of options. “I’d tried everything else,” she says. “This was my last hope.”
Slow Improvements
Molson got the stem cell transplant in May 2002. She was only the fifth person in Canada to have the procedure.
As part of the treatment, Molson spent a month in the hospital and then returned daily for blood draws to find out if she needed blood transfusions. Her side effects ranged from severe nausea to bladder and kidney infections. The chemotherapy also put her into menopause at age 27.
In the months after the stem cell transplant, Molson started noticing subtle changes. She could go grocery shopping without extreme fatigue and walk to the mailbox without using a cane for balance. Three years after the stem cell transplant, she started driving again and returned to work.
“It wasn't like I got a stem cell transplant and started running down the hall,” she says. That’s not how it worked. “It was these gradual milestone achievements, these little steps. That was when doctors started to realize that something cool was going on, that they were starting to see recovery in patients.”
For Molson, little steps led to big leaps. She went from using a wheelchair and walker to swimming, kayaking, and downhill skiing.
“I was doing things that I never, ever in a million years thought I would ever be able to do again,” she says.
Lasting Results, Lingering Questions
As part of the research study, Molson had an MRI every six months for 10 years. Her final MRI, which was in 2012, showed no new disease activity. She hasn’t taken any disease-modifying drugs since the stem cell transplant and has had more time in lasting remission longer than when her disease was active.
Although Molson had life-changing results from the stem cell transplant, the treatment is not a one-size-fits-all approach for everyone living with MS. There are still a lot of unanswered questions, Cohen says. And he advises against seeking treatment from commercial stem cell clinics.
Molson is also careful when talking to others about stem cell transplants for treating MS.
“I can't say enough about it; it gave me my life back,” she says. “But the treatments that are available now, compared to when I had my transplant, are so much better and different and, like my neurologists said, ‘Why would you want to use a nuclear bomb when you don't have to?’ It’s not for everybody.”
New Hope in CAR T Therapy for MS
Advances in stem cell therapies for MS have led to the start of a new approach: chimeric antigen receptor (CAR) T-cell therapies. Cohen explains that the procedure involves collecting T cells, engineering them to carry a lab-made CAR receptor, and infusing them back into the patient to attack and destroy the disease-causing immune cells.
“It’s very early days,” he says. “But results have been impressive, not only in MS, but in other autoimmune conditions.”
Compared to aHSCT, which involves up to eight weeks of hospitalization, CAR T-cell therapies require fewer than two weeks in the hospital and could eventually be offered on an outpatient basis. Cohen calls it an “exciting development” and potential new treatment for MS. But again, it’s not standard care as it’s still considered experimental.
