The story from STAT, an affiliate of Boston Globe Media, reports on the first patient to have a drug delivered into her brain facilitated by ultrasound waves, which work to open up the blood brain barrier. The procedure was carried out only to evaluate safety, with no therapeutic goal for the patient involved. A brain cancer patient, she had her tumor removed after the ultrasound procedure.
This is interesting science and the story does a great job of explaining what happened in this innovative procedure and why it’s potentially important. But our review raises a number of questions about the way the story was reported and whether it could have been more balanced. Costs aren’t mentioned and potential harms are glossed over. And despite the fact that many experts are quoted, there is no one to offer a restraining hand against the story’s overt enthusiasm. This was, after all, only a single patient whose results were presented at a news conference and haven’t been published or peer reviewed.
Bottom line: It’s far from clear that this technique will ever be used clinically, and the story doesn’t do enough to underscore how preliminary the findings are and how much work remains to be done.
Many drugs that may be helpful for disease cannot get into the brain because of the blood brain barrier, which normally protects the brain against toxins and infections. To have a safe method to deliver such drugs could be a significant therapeutic advance for brain tumors and other other brain pathology.
The article doesn’t mention costs at all. It’s likely a very expensive procedure since interventional radiologists are involved.
Since this is a single patient there is no quantification involved, so N/A.
This is a single patient and the story does mention that “no damage from the ultrasound was visible inside her brain.” But breaching the blood brain barrier is a big deal and likely would entail harms, especially if done repeatedly as would need to be done in many clinical treatments. There’s certainly a risk of bleeding as the news release mentions in a related Q&A. Some discussion of harms/tissue damage or lack thereof seen in animal studies would have been informative.
The story does contain information from someone who appears to be an independent voice–Dr. Gordon Li–cautioning the reader that the ability to get drugs into the brain now requires effort to determine “safe doses” of those drugs. But that issue is given just a glancing blow — not enough for a Satisfactory grade here.
This single case described at a news conference rates very low on the evidence hierarchy. And therefore the sentence: “Proving that the procedure works in people is ‘a big step forward,’” overstates what happened here. We can hardly conclude that the procedure “works” based on a single patient’s case that hasn’t been published or reviewed by other experts. And there weren’t any voices in this story who spoke up forcefully to make that point.
Several experts who, according to the story, were outside of the group that did the research were quoted. However, two of these sources are involved with the funding organization — the Focused Ultrasound Foundation. These individuals are listed as experts on the foundation website.
Although the funding source is a foundation, it receives support from numerous industry sources (as well as individuals and other sources). In addition, at least one of these experts (Ghanouni) leads other foundation-support research efforts. It’s possible that experts associated with this foundation may have an incentive to cast this foundation-supported research in a positive light. The story could have alerted readers to these links or, even better, tapped into a wider network of independent experts to provide a more critical evaluation of the research.
The article doesn’t discuss other research underway that may be trying to get drugs across the blood brain barrier. However, we think this would be beyond the scope/focus of this single case article, so we’ll rate it Not Applicable.
The article notes this is a very new, very experimental technology, and several other patients are lined up for testing. Even after that testing, further testing will be necessary to establish appropriate doses etc, so we think it’s pretty clear that this won’t be available any time soon.
The article notes this is a first-in-human case, using a creative way to use ultrasound waves. The novelty is apparent.
Although a news conference and news release were clearly what sparked the story, the author interviewed outside experts, so we can be sure there was some original reporting done.