An international clinical trial of a stem cell treatment for a common form of multiple sclerosis may be nearing completion, and several media outlets in the UK jumped on encouraging testimonials from some of the patient participants. The BBC and The Guardian both issued stories on the same day reflecting much the same information, including interviews with the same patients and very similar quotes. It is not clear why the story emerged at this point, as there is no indication that the trial is complete or that data from it have been analyzed. And the same patients from the very same trial were featured prominently in a similar round of stories from 2013, many of which carried “miracle” headlines. Can these glowing testimonials stand the test of time? If not, then the stories may have done a disservice to MS sufferers.
Multiple sclerosis can be a terrifying disease for patients and their families. While for some, they have a mild course for long periods without evidence of progression, for others it can lead to permanent disability. This article talks about a form that comes and goes, but the general course is one of gradual worsening that leads to eventual disability. For each episode, treatment may shorten the duration of the flare, but it doesn’t appear to alter the long-term prognosis. There is considerable research investigating new medications that can decrease the progression to permanent disability. This study refers to interest in a different form of treatment, using the patients own cells to reconstitute their immune system after having undergone chemotherapy to wipe out the immune cells that are thought to cause the MS in the first place. As described, this is a treatment used for serious conditions such as cancer. It is associated with real risks including infection, prolonged hospitalization and even death. Despite the testimonials in this piece, it is unclear whether this form of treatment will represent an advancement over current therapy or not. Only controlled studies comparing patients who get this new treatment with similar patients who are treated with current standard therapy can tell. Even if helpful in the short-term, it is unclear whether the immune system will in the future again attack the patient’s nervous system and cause new symptoms of MS. This is because the patient is given back her/his own cells, not someone’s who doesn’t have MS, and so it is possible that those cells given back will still carry the risk for future MS. For patients with this condition who are desperate for treatment that can lead to improvement, this piece may provide false hope that a new cure is right around the corner.
Low in the story, one patient is identified as having spent £30,000 (approximately $42,500) on the treatment. We’ll give credit for this nod in the direction of cost, but it would be hard for a reader to generalize from that. It isn’t clear whether this is covered by insurance or not. It also isn’t clear whether the price paid by this one patient is also the cost for the others. At the least, some context for that reference would have been helpful. We don’t think of UK patients as paying out of pocket for health care. And since this is the U.S. edition of the Guardian, the info should be tailored for a U.S. audience.
Quantification of benefits may not be possible at this point, as the “about 20 patients” in this UK branch of an international trial are still presumably embedded in the trial protocol. The story, instead, relies on personal testimonials from two patients who had received the treatment, who describe major improvements in their conditions. Note: There seems to have been at least some empirical data available. At least one earlier study, published in January 2015 in JAMA, showed that the treatment was linked to reduction in level of disability in 64% of the 123 patients treated.
It isn’t clear why this story is coming out now. If this is part of a larger trial, why are these results being shared? Presumably this isn’t a blinded trial, but it isn’t even clear if there is a control group that received standard treatment without the bone marrow transplant.
Risks are left unexplained. One source notes that haematopoietic stem cell transplant (HSCT) “is an aggressive treatment that comes with significant risks.” And one patient who received the treatment noted that “I’d rather have done 10 rounds with Mike Tyson.” But none of that illuminates the nature of the risks that someone would encounter.
This is the weakest point of the story. This is a treatment with real risks. They way it is presented gives very little insight to the reader about what these risks are.
The story is clear that a clinical trial of HSCT is under way and that the treatment is suitable for only a specific subset of MS patients. It also explains that the number of participants at the Sheffield Teaching Hospitals site is small. But the text is mum regarding the trial process (A description of the study at ClinicalTrials.gov indicates that the study is still recruiting participants and anticipates a December 2017 completion date). And there is little describing the study and whether these patients are being compared to others who haven’t had this treatment. There is no information about experience at other sites and why these investigators are sharing the results now. The article implies that while this isn’t for everyone, it highlights those patients being studied in this trial. Implicit is that these are the patients who should be treated with a bone marrow transplant. Of course, we can’t say that because we don’t know what to compare these results to. We are told of positive responses after treatment, but we also know that this form of MS involves relapses and remissions. So are these remissions due to the treatment or simply the natural history of the patient’s condition?
Relapsing remitting multiple sclerosis is a common form of the disease for which there is currently no cure. So the stakes are sufficiently high.
Funding sources are not identified, but the principal investigator for the international trial notes that “we have achieved this [the international clinical trial] without industry backing,” which is helpful. Most of the sources in the story are either doctors involved with the trial or patients participating in the trial. One potentially independent source, who heads clinical trials at the MS Society in the UK, reacts cautiously to the testimonials, reminding readers that HSCT “needs to be carried out at an accredited centre or as part of a clinical trial.”
Although It isn’t clear that anyone quoted doesn’t have a vested interest in this trial, including the head of clinical trials for the MS society, we’ll give the story the benefit of the doubt since it does at least specify no involvement from industry. The story would have been better had it commented on involvement from other vested interests.
Aside from noting that there is no cure for this type of MS, the story offers no information about existing treatments such as drugs to lessen relapses and steroid treatments to cope with flare-ups. More detail could be provided about how this form of MS is currently treated. It also isn’t clear whether the bone marrow treatment will affect long-term disability or not.
The reader must infer that, because the information comes from an ongoing clinical trial, the treatment is not yet available. And the story implies that it may be available outside of a research study: “It needs to be carried out at an accredited centre or as part of a clinical trial”. Does this mean that if I have 30,000 pounds, I can have this done to me in the UK?
A round of similar stories on patients in this same trial — many with “miracle” headlines — appeared in March 2015. It’s not clear what’s different about this story or why these results are being reported now.
We couldn’t find any news release that could have spawned this story. However, its use of almost identical sources and direct quotes to those of a BBC News story that was posted earlier on the same day leads us to question the nature of the material used by the newspaper. The BBC claimed that it had been given “exclusive access” to the patients in this clinical trial, but that does not explain the details in The Guardian. Given the troubling overlap between these stories, we’re reluctant to award a Satisfactory since the story does seem at the very least to have borrowed text either from the BBC or an unknown source news release. We’ll rate this Not Applicable, which means we’re neither crediting nor penalizing the story.