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MS treatment: study shows chemo and stem cell transplantation can reverse the disease



When Amanda Loy became 28, her multiple sclerosis came to the point that she could no longer work full time. Her hands and legs felt numb all the time, the bladder always felt full and she had to rely on a stick to walk for more than 10 minutes. After giving birth to a son a year later, in 2008 the symptoms worsened. It was about then that she decided to leave her home in Anchorage, Alaska, to Chicago to ask for a new treatment she'd heard about at a Seattle hospital.

Almost a dozen years on, Loy is now back to work full time as a teacher of the radiology program at the University of Alaska. She runs the half marathon and plays football with her son, who is 1

0. She no longer takes MS medication. Her only lingering symptom is some mild nerve pain from time to time.

"It sounds so dramatic, but [the treatment] gave me my life back," she said.

The treatment is an experimental chemotherapy and hematopoietic stem cell transplant offered in the first randomized trial comparing the results of patients receiving treatment to patients taking standard MS medication.

The results of the trial appeared Tuesday in the journal JAMA and they are impressive. Among the 55 patients in the control group who took medication so the disease worsened. But for the 55 (including Loy) who received the chemo and stem cell transplant, only three were worse. The rest so improve their quality of life and disability.

It is the best evidence to date that treatment works for patients with what is called "relapsing remitting" MS. "I think it will change MS's natural history," said Northwestern University's stem cell researcher Richard Burt, the study's lead author and Loy's physician. "When you use it in the right group of patients with MS, you get these really pleasing results."

Doctors still do not know if the therapy will work for people with other forms of MS. They do not know whether the risk of a transplant (including death) will always outweigh the benefits. It is rare that turnarounds this dramatic for patients with MS are rare – and worth examining.

Physicians who run the experiment do not expect such dramatic improvements in patients treated with stem cell treatment. 2.3 million people worldwide, especially women in more temperate climates such as Canada and the northern United States.

Instead of protecting the body from foreign invaders, in patients with MS, the immune system turns on the host, especially destroying the myelin, a protective coating around the nerve fibers of the brain and spinal cord.

Finally, these attacks can seriously damage and destroy the nerves and myelin, interfere with communication between the brain and the body, and lead to symptoms such as numbness, difficulty walking and even blindness.

But not all patients' symptoms manifest themselves in the same way. People with "relapsing remitting" MS – or 85 percent of patients with the disease – experience it in passports and starts: their symptoms occur for a few days or weeks followed by weeks, months or even years of remission.

For most patients with this version of the disease, these periods of remission become less over time and eventually disappear and move them to a second phase of the disease known as "secondary progressive MS."

Loy and the other patients in the trial had relapsing remitting MS. Loy had tried medicine and nothing worked.

Burt told her the best she could hope for from stem cell transplantation that her symptoms would not worsen. "I don't really expect all these improvements. I went into it and thought," If at least I don't get worse from this point, it'll be okay, "she said." So all the improvements have been quite unexpected, but one nice surprise, that's for sure. "


Amanda Loy with her son during her treatment.
Courtesy of Amanda Loy

How stem cells changed Loy's immune system

] The road to Loy's stem cell transplant was not straightforward. found out about the approach a few years after her diagnosis and contacted a hospital in Seattle to ask for another similar study in 2010. "They did not take new patients but they told me there is this guy in Chicago I could look in, "she said.

That guy was Burt in Northwestern, then went to meet in 2010 and found out she was qualified for her study, but she was randomly assigned to the control group – and first she didn't get the treatment.

But within two years worsened her MS symptoms. Burt asked her to come back to Chicago to assess her disability and establish that she could transfer to the treatment group. In 2012, she traveled to Chicago for a transplant.

The treatment involved four main steps: The doctors first put her through a short treatment of chemotherapy to stimulate the production of hematopoietic stem cells, which are the cells responsible for the regeneration of the immune system. They then hung up Loy on a machine that cycled through her blood to collect the stem cells that were frozen. Another higher dose of chemo eradicated Loy's immune system.

"I had trouble with the chemo," remembers Loy. "It made me really, really sick and pretty miserable – feeling – nausea, vomiting."

Then, the stem cells were thawed and transplanted back into Loy, like blood transfusion. She was so tired of the chemo that she napped through the transfusion.

But then something amazing happened: Right after getting the transplant, Loy saw that her MS-related bladder symptoms had gone away. And by one year all her other symptoms – fatigue, heat intolerance, numbness, nerve pain – were improved or dissolved.

The only side effect from the treatment, she said, was temporary menopause under the chemo – but it also went away quickly and had no effect on fertility. Common side effects in the other patients in the study's transplant group were infections (such as colds and urinary tract infections) and electrolyte disturbances. No one died as a result of the transplant.

Not all MS patients will be eligible for this treatment

The study, which gave Loy the chance to reverse MS was impressive – an international, multi-center, blinded, randomized trial conducted at four centers: in Chicago at Sheffield Teaching Hospital NHS Foundation Trust & University of Sheffield in England, at the University of Uppsala in Sweden and at the University of São Paulo in Brazil. Patients were enrolled at each of these hospitals between 2005 and 2016 and followed up for at least one year to see if their illness had emerged.

The study is based on decades of basic research on MS, stem cells and the immune system, including many years of experience in treating patients with blood and bone marrow cancer such as leukemia and lymphoma. It is also based on previous studies investigating this approach to treating MS, such as a rigorous 2016 study from Canada, where 70 percent of the 24 patients receiving chemotherapy and stem cell bone marrow transplantation experienced the progression of their disease arrested. or reversed. Recognized medical specialists at the time called to study a "miracle."


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