This news release describes “encouraging” results of a small 5-year trial to evaluate the safety and efficacy of stem cell transplants for aggressive cases of the most common form of multiple sclerosis, called relapsing-remitting MS. Known as HALT-MS, the phase 2 trial yielded a 69 percent remission rate, which researchers say supports the development of a large randomized trial to see how it stacks up against existing treatments.
That cautious statement — that the trial justifies the performance of a larger randomized clinical trial — is a positive of the release, since it tells readers the treatment is not yet ready. The release continues to use restraint throughout
, and gives readers a good sense of the benefits, but it’s short on perspective. There’s no discussion of costs, availability, or the number of patients who might benefit, and some trial limitations were omitted. More clarity on researchers’ ties to industry was also warranted. An explanation of how this trial relates to similar research would have been helpful, as well. And while the findings were just published in the journal Neurology, they’re somewhat dated, having already been reported at a medical conference in June 2016.
There is no cure for MS and the few medications that have been approved to slow its progression and manage symptoms — such as weakness, motor impairment, chronic pain and fatigue — come with high costs and significant side effects. This trial adds to evidence that an intensive one-time treatment, high-dose immunosuppressive therapy followed by a transplant of the patient’s own stem cells, could arrest the course of this potentially devastating autoimmune disorder. Though difficult and costly, this therapy might benefit patients with aggressive disease for whom prior standard therapy did not stop progression. This news release seemed designed to make the case for a full-scale trial that could establish it as a viable option.
There’s no mention of the cost of stem cell transplants. We could find no reliable cost estimate online, but several sources including the American Cancer Society put the price in the hundreds of thousands of dollars. Transplants that use a patient’s own stem cells, known as autologous transplants, are cheaper than those that use donor cells.
The news release states:
“Five years after receiving the treatment, called high-dose immunosuppressive therapy and autologous hematopoietic cell transplant (HDIT/HCT), 69 percent of trial participants had survived without experiencing progression of disability, relapse of MS symptoms or new brain lesions….
Five years after HDIT/HCT, most trial participants remained in remission, and their MS had stabilized. In addition, some participants showed improvements, such as recovery of mobility or other physical capabilities.”
It also points out that none of the participants resumed MS drugs after their treatment.
The news release does the bare minimum here, stating:
“The treatment carries some risks, and many participants experienced the expected side effects of HDIT/HCT, such as infections. Three participants died during the study; none of the deaths were related to the study treatment.”
Still, we think more could have been said about the side effects of chemotherapy. For example, How many patients experienced them and how bad were they? And the news release does not say how long treatment and recovery takes. The process by which the deaths were categorized is not given and leads to uncertainty about whether these patients may have died as a result of the immuno-suppression of their bone marrow.
This was a tough call. While generally upbeat, the news release offers some caution when reporting that the phase 2 trial involved just 24 patients and makes it clear that a larger randomized trial is needed:
“Although further evaluation of the benefits and risks of HDIT/HCT is needed, these five-year results suggest the promise of this treatment for inducing long-term, sustained remissions of poor-prognosis relapsing-remitting MS,” said Richard Nash, M.D., of Colorado Blood Cancer Institute and Presbyterian-St. Luke’s Hospital. Dr. Nash served as principal investigator of the HALT-MS study.
“If these findings are confirmed in larger studies, HDIT/HCT may become a potential therapeutic option for patients with active relapsing-remitting MS, particularly those who do not respond to existing therapies,” said Daniel Rotrosen, M.D., director of NIAID’s Division of Allergy, Immunology and Transplantation.
Unfortunately, the release omitted some important limitations described in the study. According to the published report, six patients did not complete the 5-year follow-up period and their results were not clear, but they were not followed for the full time. This represents a significant proportion of the results (37%) leading to a great degree of imprecision in the results. There were also four patients (17%) who got worse.
This was not a controlled clinical trial, or even a prospective study of two groups, so there is significant likelihood of bias. According to the GRADE evidence evaluation process, the quality of the evidence would be low.
There’s no disease mongering. And the release provides useful context about multiple sclerosis when it states:
“MS symptoms vary widely and may include motor and speech difficulties, weakness, fatigue and chronic pain. The most common form of MS is relapsing-remitting MS, which is characterized by periods of mild or no symptoms interspersed with symptom flare-ups or relapses. Over years, the disease can worsen and shift to a progressive form.”
The release doesn’t exaggerate the symptoms, but it does give some sense of the impact this disease may have for some people.
The news release states that the research was sponsored by the National Institute of Allergy and Infections Diseases and the NIH. But it does not mention that Baxter Healthcare Corp. supplied equipment for the trial or that several study authors have financial relationships with biotechnology companies, which are listed in the study.
It’s not easy to determine which companies have financial interests in hematopoietic stem cell transplantation, but Baxter has at least one patent relating to the procedure. This makes full disclosure an important aspect of the reporting of this trial.
The news release could have said more about other treatments beyond stating that other studies “have indicated that currently available MS drugs have lower success rates,” with no specifics.
One MS researcher told Medscape Medical News last year that current therapies yield a success rate of 38% to 48%. Thirteen medications have been approved by the U.S. Food and Drug Administration (FDA) for the treatment relapsing-remitting MS. All have been shown to reduce the number of attacks and new lesions, and they may also slow disease progression, according to the National Multiple Sclerosis Society.
There’s no mention of whether this therapy is currently available, where it’s performed, or whether regulatory approval is required. Also, there’s no discussion of whether it is likely to be covered by most insurance plans. It appears that this is still purely a research therapy and would not be available outside of research settings. However, there are many physician practices that do research, usually for industry, which don’t have the same safety features as this research lab had.
This news release explains the significance of the study in supporting initiation of a randomized trial. However, the results are not entirely “new,” as the release states; they were previously reported at a medical conference in June 2016. Also, we found it interesting that the published study states its results are consistent with three similar trials, including this Canadian study, bolstering the validity of its finding. Those trials are not mentioned in the news release.
The idea of using bone marrow transplantation for medical illnesses is not new. During the 1990s bone marrow transplantation following high dose chemotherapy was touted as a cure for breast cancer. A randomized clinical trial was begun in the late ’90s but was discontinued when results from the initial studies were found to have been fraudulently reported. (Described in a book called “False Hope: Bone Marrow Transplantation for Breast Cancer” by Peter D. Jacobson and Richard A. Rettig.)
The tone of this news release is restrained, with no sensational language.