A Chicago-based center that has long operated a clinical trial program for stem cell therapies, has stopped recruiting further patients as its chief, Richard Burt, leaves for a research sabbatical.

Source The Scientist

A pioneering stem cell clinical trial program in the US is closing up shop, a surprise move for what had appeared to be a successful endeavor at testing hematopoietic stem cell transplantations (HSCT) for autoimmune and other diseases. Northwestern Medicine’s immunotherapy and autoimmune diseases program, based at Northwestern Memorial Hospital in downtown Chicago, will no longer accept new patients for clinical trials, a spokesperson for Northwestern Memorial HealthCare confirmed this week (September 5).

The center specializes in conducting hematopoietic stem cell transplantations (HSCT), whereby stem cells are intravenously infused into a patient whose bone marrow or immune system is defective due to disease. The stem cells are obtained from a patient’s own bone marrow or peripheral blood, or that of another person. Patients are typically treated there as part of clinical studies testing the use of HSCT for autoimmune diseases, such as multiple sclerosis (MS), and systemic lupus erythematosus, but sometimes also outside of trials as part of an FDA-sanctioned program known as “expanded access,” according to Science.

The reason for the center’s closure is that its chief, Richard Burt, will be taking a research sabbatical to teach the HSCT protocol he’s developed to other clinics across the US and writing a book requested by a textbook publishing company, he tells The Scientist.

Some in the field question why the program can’t continue without him. “If his program is so successful, [why] it should suddenly stop if he’s not there is a little bizarre,” remarks Mark Freedman, a neurologist who directs a research division focused on multiple sclerosis at Ottawa Hospital. Burt and Northwestern did not say why the program could not continue without him.

The news of the closure, which first surfaced on a Facebook group for patients with autoimmune diseases being treated with HSCT, follows promising results from a multiple sclerosis trial reported by Burt and his colleagues in January. The study—one of a handful worldwide that have investigated HSCT for multiple sclerosis—suggested that HSCT proved a more effective treatment than conventional drugs in a majority of patients who had suffered relapses of the disease after a first line of standard treatment.

Earlier this month, actress Selma Blair reported on Instagram that she can “walk much better” after having undergone the HSCT treatment for multiple sclerosis at the clinic, but is still recovering from the chemotherapy, which is used to deplete the body’s immune system before receiving the stem cells.

Burt’s work has attracted some concern in the past due to a letter he received in November 2016 from the US Food and Drug Administration (FDA), containing a list of violations in the conduct of clinical studies at Northwestern, including the failure to report patient deaths and adverse events experienced by patients to the FDA in a timely manner.

Burt stresses that the closure of his clinic is not linked to issues cited in the FDA letter. “In December 2016, the FDA sent a follow up letter saying our response was satisfactory and that there were no further actions or questions. The FDA found no treatment-related deaths in any of the studies,” he writes to The Scientist in an email.

During his upcoming research sabbatical, he plans on working with universities and clinics across the country to make the HSCT protocol he’s developed more widely available. “This is in my assessment . . . the best way to help the world, and humanity, and help patients to move this forward, to get the treatment more widely around the world,” he says.

Paul Knoepfler, a stem cell biologist at the University of California, Davis, has raised questions about some practices at Burt’s clinic on his blog, where he is known for sounding the alarm on unproven stem cell treatments. “Some patients have said on the Internet that they are required to pay as much as $150,000 for participation in the Northwestern MS stem cell trial or in parallel off-study experimental administration of experimental stem cells,” Knoepfler wrote in 2017.

Although asking patients to contribute financially to the cost of clinical trials would not go against FDA policies, the practice is considered “controversial” by some because it may exclude patients who can’t afford to participate, Knoepfler explains.

“Insurance usually pays for treatment, sometimes a patient pays,” Burt says in an email. This practice was a decision made by the National Institutes of Health, where he received a major grant years ago for developing HSCT for autoimmune disease. “The grant specified that patients or their insurance must pay for the treatment. . . . This is typical for all university centers in America that have done HSCT for autoimmune diseases or cancer,” he adds.

According to one report by Riverside-Brookfield Landmark, a local Illinois newspaper, an insurance company recently denied coverage for HSCT treatment at the clinic—outside of a clinical study—to a multiple sclerosis patient, “because it is considered experimental.” The family members ended up taking a second mortgage on their home to finance the procedure, according to the article.

Some researchers in the field have questioned the HSCT methods Burt used in the multiple sclerosis trial. One aspect of the study that surprised Freedman in particular was his choice of participants. The majority of those enrolled had relapsed after they went through fairly mild first-line MS treatments, such as interferons. Freedman wouldn’t consider these cases aggressive enough to warrant a treatment as high-risk as a stem cell transplant, where fatal complications can occur during the procedure, he explains. “This is an aggressive [therapy] so it has to be delivered to patients who are considered to have aggressive disease,” explains Freedman, who along with colleagues conducted a trial in 2016 investigating the use of HSCT in aggressive multiple sclerosis.

In addition, the chemotherapy dose Burt is using “is what we would consider to be inadequate to fully rid the body of the cells that are causing disease. He’s going chemo light,” Freedman says. Burt, however, considers his treatment less aggressive and potentially less toxic than those used at other centers, he writes in an email. “A lot of universities use my protocols,” he adds.

Trials investigating the use of HSCT led by Burt for Devic’s disease and systemic lupus erythematosus finished last December, around a year sooner than the estimated completion date posted on ClinicalTrials.gov. A spokesperson for Northwestern Memorial HealthCare did not clarify whether this is due to the program’s closure.

All currently enrolled patients will continue their treatment at the center, explains Christopher King, director of media relations and communications at Northwestern Memorial HealthCare, in a statement sent to The Scientist. “Northwestern Medicine is grateful for Dr. Burt’s pioneering achievements in this field and the care he has provided to his patients.” 

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