Experimental MS treatment halts disease ‘in its tracks’

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Pioneer Founding member
A Seattle man is among two dozen MS patients in a promising clinical trial that found high-dose immune-suppressing drugs and stem-cell transplants may stop the progress of the disease in those who've failed usual care.

By JoNel Aleccia
Seattle Times health reporter
1/11/15

http://seattletimes.com/html/localnews/2025434042_msstemcellsxml.html


Seven years ago, Mike Kearny of Seattle was so sick and weak from multiple sclerosis, he had to use the handrail to pull himself up the stairs of his Wallingford neighborhood home.

Daily injections of powerful drugs hadn’t slowed the neurodegenerative disease; tests showed growing numbers of lesions in his brain. The once-active cyclist and climber started missing work as a cardiac catheter lab nurse, and — worse — activities with his wife, Casey Castaneda, and his boys, Owen, then 2, and Jack, 7.

“I was not doing well,” recalled Kearny, now 47, who was first diagnosed in 2006.

So when local doctors offered a gamble on an experimental treatment that aimed to use high-dose chemotherapy and stem-cell transplants to stop multiple sclerosis (MS), Kearny took the chance.

Five years later, he says joining the small-but-promising HALT-MS clinical trial led by Seattle researchers was the best bet he ever made.

“The opportunity I was given now feels like a gift,” said Kearny, who was treated in December 2009 and has seen no progress of the disease since.

“It stopped it in its tracks,” he added.

Kearny is one of two dozen patients enrolled in the five-year clinical trial that gets its catchy acronym, HALT-MS, from a long title: “High-Dose Immunosuppressive Therapy and Autologous Hematopoietic Cell Transplantation for Relapsing-Remitting Multiple Sclerosis.”

The procedures, abbreviated as HDIT and HCT, are the focus of the multicenter trial that includes experts from seven U.S. states, plus Canada and England.

A three-year interim report published recently in the journal JAMA Neurology offered positive results: Nearly 80 percent of those enrolled, including Kearny, not only survived but also showed no increase in disability, relapse of MS symptoms or new brain lesions. Few serious complications or unexpected side effects were reported.

It’s an early but encouraging finding that the combination of high-dose immune-suppressing therapy and transplantation with the patient’s own blood-producing stem cells can reboot the immune system, said Dr. George Georges, a transplant expert at the Fred Hutchinson Cancer Research Center and the University of Washington.

Previous trials had shown some benefit of using the therapy in MS patients with more advanced disease, but the new trial offered the promise of freezing symptoms before they became so debilitating.

“The question was, might it be actually more beneficial, in terms of really preventing progression of disability, by doing the transplant at the earlier stage of disease,” Georges said.

Doctors don’t claim the technique cures MS, only that it may stop future decline.

Those enrolled in the trial, including Kearny, were diagnosed with relapsing-remitting MS (RRMS), the most common form of the disease that occurs when the immune system attacks myelin, the fatty sheaths that protect nerve fibers.

MS affects about 2.3 million people worldwide. About 85 percent are initially diagnosed with RRMS compared to the progressive forms of the disease, according to the National Multiple Sclerosis Society.

The trial patients all had failed standard treatments for MS in the 18 months before they enrolled; typically they’d exhausted three different lines of care, said Dr. Richard Nash of the Colorado Blood Cancer Institute.

They received high doses of drugs to wipe out their immune systems and then doses of their own stem cells to rebuild them.

It’s a technique first pioneered for to the now-common treatment for leukemia and other cancers.

For Kearny, the actual procedures went smoothly.

The aftermath of chemotherapy was like having a very bad case of the flu, he recalled.

Because his immune system was so vulnerable after the December 2009 transplant, he couldn’t risk being exposed to germs from his kids or his wife, a third-grade teacher. So he stayed nearby with his mother, who’s also a nurse, for three months.

Once he was better, however, Kearny said he never looked back.

Like other patients in the trial, he stopped taking MS drugs that can cost upward of $70,000 a year.

He’s been back to see Dr. James D. Bowen, an MS specialist at Swedish Neuroscience Institute and the study’s lead author, every year for five years.

On Wednesday, at his latest checkup, Kearny said his MS was pronounced “inactive and nonprogressing.”

“Post-transplant, there’s a baseline I hit and it’s just stayed the same,” said Kearny, whose procedure was covered by his health insurance. Autologous transplants, or those that use a person’s own stem cells, can cost $100,000 or more, industry experts say.

Transplants that use another person’s cells can cost twice that.

In practical terms, that means Kearny is back to working 50 hours a week, skiing, climbing and spending time with his two active boys, now ages 9 and 14.

Top researchers praised the interim results, with Dr. Anthony Fauci, head of the National Institute of Allergy and Infectious Diseases calling them “promising.”

“If the findings from this study are confirmed, HDIT/HCT may become a potential therapeutic option for people with this often-debilitating disease, particularly those who have not been helped by standard treatments,” he said in a statement.

But some experts said they’d like to see more robust results. In an editorial accompanying the new study, Dr. Brian Weinshenker of the Mayo Clinic and Dr. M. Mateo Paz Soldan of the University of Utah concluded: “The jury is still out regarding the appropriateness and indications of HCT for MS.”

Patients and doctors should expect that use of such an aggressive and expensive therapy should clearly show a benefit over existing treatments, they wrote.

Kearny is among the last of the patients recruited between 2006 and 2009 to finish the five-year follow-up. Final results will be published, but Georges and other researchers are looking forward to a broader test of the technique.

“This just means there is a strong indication to do a randomized clinical trial,” Georges said. “That is always going to be the best way to evaluate: Is this really better than standard therapy?”

In the meantime, Georges cautioned that stem-cell transplants may not be the solution for the vast majority of MS patients. There are real risks to the procedure and it can only be considered for people who have exhausted all other options, he said.

In addition, patients who have other health problems plus MS may not be healthy enough to withstand the treatment.

But for patients like Kearny, who was both healthy and willing to accept the risks of transplant, the treatment changed the direction of his life.

“Once I got my claws in it, it was a super-good option for me,” he said. “Now, I feel like I could do almost anything.”

JoNel Aleccia: 206-464-2906 or jaleccia@seattletimes.com.
 
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