People with multiple sclerosis sometimes become desperate and will travel to other countries for experimental stem cell treatments. That’s not always a good idea.
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Faced with an incurable condition, people with multiple sclerosis will sometimes seek risky alternative treatments as their disabilities increase.
One of these experimental treatments is Hematopoietic Stem Cell Therapy (HSCT). And it comes with a high price.
HSCT involves extracting white blood cells from the bone marrow of the person with multiple sclerosis (MS). These cells are treated with chemotherapy to make them, in essence, forget they have MS.
Then, they’re reinfused back into the person in hopes of slowing down disease progression. HSCT has been studied and shows promise, but it’s not yet approved as a therapy by the U.S. Food and Drug Administration (FDA).
The promise and hope of a cure is enough for many who have MS and are desperate.
As a result, “stem cell tourism” has grown significantly in the past four years, according to researchers at The Ohio State University.
Stem cell tourism provides unproven stem cell treatments. The program often includes patients and families traveling to countries outside of the United States where the procedure’s performed.
In addition, there are more than 570 clinics currently offering these unapproved treatments in the United States.
Critics say it’s unethical, risky, usually not covered by insurance, and expensive at about $80,000 per treatment, plus travel costs.
“Doctors are taking [HSCT] as a complete process — but it’s not. It hasn’t been demonstrated effectively,” explained co-author Dr. Jaime Imitola, director of the Progressive MS clinic at The Ohio State University. “They’re selling it as a cure, which is not OK.”
Imitola discussed with Healthline this phenomenon known as “therapeutic hope.”
“When battling an incurable disease, you hope for things that help. You want a cure. It’s a natural, biological, and psychological response,” he said.
“I think cell-based therapies hold promise,” added Dr. Bruce Bebo, executive vice president of research at the National Multiple Sclerosis Society (NMSS). “But, they need to be studied in a rigorous and thorough fashion. Unfortunately, it’ll take us some time to fully appreciate the promise and risks of stem cells.”
Balancing risks and rewards
Bebo said stem cell tourism occurs because “people with MS don’t have the time to wait for the rigorous and thorough process to occur.”
He said he worries people may pursue HSCT without fully understanding the benefits and risks.
He notes that patients spend a lot of money on a treatment with no proven effectiveness. There are pain, time, and financial consequences.
“Everyone has their own tolerance for risks and benefits,” said Bebo.
He suggests people with MS look at all other interventions before taking an experimental approach.
“Consider incorporating lifestyle changes. Stopping smoking, eating a healthy diet, and exercise are all suggestions to help reduce possibility of disease progression,” he said.
“Striving to participate in a rigorous trial is a smart way to go,” added Bebo. “The NMSS provides funding and resources based on rigorous, well-controlled research. We make recommendations based on solid research.”
Supporting research
The NMSS is currently supporting 12 stem cell research projects.
The organization has supported 68 stem cell studies over the past 10 years. In addition, they collaborated with more than 70 experts to create guidelines for speeding research on stem cells.
The Tisch Multiple Sclerosis Research Center of New York is currently recruiting for an FDA-approved stem cell trial, but applicants must be part of International Multiple Sclerosis Management Practice.
“Our goal is for the trial to lead to an FDA-approved treatment for MS and one day become available to people with MS from centers all over the world,” Beth Levine, RN, clinical trials manager for Tisch, told Healthline.
“We’re hopeful that the encouraging results from our phase I stem cell clinical trial indicate the possibility of a stem cell therapy in the future, to reverse the damage caused by MS in people with established disability,” she added.
Not all trials are the same
The site ClinicalTrials.gov offers a list of clinical trials, both private and publicly funded. Not all the trials located on this site are FDA-approved.
Some are used to recruit participants for unapproved trials.
FDA Commissioner Scott Gottlieb has labeled these types of trials as “bad actors.”
“[The] FDA looks at stem cell research differently,” said Paul S. Knoepfler, a professor in the Department of Cell Biology and Human Anatomy at University of California, Davis.
His recent research reported that the FDA cracked down on two unapproved clinics, filing suit in May against California Stem Cell Treatment Center and U.S. Stem Cell, Inc.
The FDA has also issued two warning letters during Gottlieb’s term.
“By comparison, for example, in 2018, the FDA seemed to have issued 40 warning letters, primarily to e-cigarette makers,” Knoepfler told Healthline.
The FDA then created a process called Regenerative Medicine Advanced Therapy to speed up the testing of a large number of therapies.
The result of a quicker review process and little authority could lead to new risks as the FDA promotes weaker trials with less supporting data.
“Unapproved procedures take away from legitimate research,” Knoepfler said.
Knoepfler, a stem cell biologist and cancer survivor, offers a guide to better understanding the experimental HSCT treatment on his blog, TheNiche.
“I realize there’s desperate need to understand why someone would roll the dice,” Knoepfler said. “But it’s to everyone’s benefit to understand risks with the benefits. Timely reporting during a trial is essential.”
In November 2017, Dr. Richard Burt at the Northwestern University Feinberg School of Medicine, was issued a warning from the FDA for numerous violations.
FDA officials said two of the four deaths that occurred during one of Burt’s HSCT trials were not reported in a timely manner. Corrective actions were by taken by Burt within a month, according to a close out letter from FDA.
Patients who will take the risk
In a 2009 benefit-risk analysis of MS patients, most of the 651 patients surveyed indicated they were willing to risk their life in order to receive better health results.
Nadine Shapiro took that risk and hasn’t looked back.
“I couldn’t walk five years ago,” Shapiro told Healthline. “I now no longer use a wheelchair, walker, or cane.”
Receiving an MS diagnosis in 2001, Nadine ran out of options 14 years later.
Her disease modifying treatment (DMT) was no longer working. The medications for her symptoms weren’t effective. Her fatigue and cognitive problems forced her into retirement.
Having used chemotherapy before, she had no problems signing up to be an HSCT participant in 2014 at Northwestern.
Shapiro said she was told on her first visit, “You will walk again.”
She was fast-tracked through the system and her insurance covered everything.
“I had no money,” she said. “My only option was insurance.”
Today, Shapiro is on a fraction of her symptom-treating medications and is taking no DMTs.
The treatment didn’t help her cognitive issues, but her Expanded Disability Status Score improved from 6.5 to 3.5. Shapiro is physically functional once again.
“If I hadn’t had [HSCT], I’d still be in the nursing home. It was perfect for me, a lifesaver.” Shapiro is 40 years old.
Different systems, different countries
Unproven treatments are growing in popularity around the world.
“Each country’s different,” said Hamza Malik, whose sister is recuperating from her second stem cell treatment. “Russia doesn’t include family members. Mexico does, but moves you from hospital to apartment.”
Malik’s sister, Sanaa, lives in Dubai, United Arab Emirates.
Since her MS diagnosis, she’s steadily declined. There’s no access to DMTs in her country. She needs physical therapy, but there are only two therapists in the entire country. And their cost is more than 200 percent of that in the United States.
After studying at Johns Hopkins University, Malik became a U.S. citizen. His sister lives on the other side of the world. Sanaa has tried everything by adjusting modifiable risk factors, diet, and yoga. So far, nothing has worked.
In 2011, the family pooled $40,000 to try an experimental stem cell treatment in Germany.
“It was a scam,” Malik told Healthline.
Today, Sanaa is in her room in Dubai, sleeping.
“She’s very weak,” said Malik. “Her immune system is being rebuilt.”
Sanaa expects to be in this condition for about a month and then might see some changes.
“Her chances for change are small but worth it,” said Malik.
Her most recent treatment took place in Russia and cost about $70,000.
Malik started an online fitness store, TheFitState.com, to raise money for his sister’s treatments.
“Pray for Sanaa,” Malik asked. “Please, pray for Sanaa.”
Editor’s note: Caroline Craven is a patient expert living with MS. Her award-winning blog is GirlwithMS.com, and she can be found on Twitter.
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