Credit: Jackson Smith/UVA Health System
Jeff Elias, MD
Newswise — CHARLOTTESVILLE, Va., Aug. 25, 2016 – A study published today in the prestigious New England Journal of Medicine offers the most in-depth assessment yet of the safety and effectiveness of a high-tech alternative to brain surgery to treat the uncontrollable shaking caused by the most common movement disorder. And the news is very good.
The paper outlines the results of an international clinical trial, led by Jeff Elias, MD, of the University of Virginia Health System, that evaluated the scalpel-free approach called focused ultrasound for the treatment of essential tremor (ET), a condition that afflicts an estimated 10 million Americans. Not only did the researchers determine that the procedure was safe and effective, they found that it offered a lasting benefit, reducing shaking for trial participants throughout the 12-month study period.
“This study represents a major advance for neurosurgery, treatment of brain disease and specifically the treatment of ET,” Elias said. “For the first time in a randomized controlled trial, we have shown that ultrasound can be precisely delivered through the intact human skull to treat a difficult neurological disease.”
Pioneering Tremor Trial
The multi-site clinical trial included 76 participants with moderate to severe essential tremor, a condition that often robs people of their ability to write, feed themselves and carry out their normal daily activities. The trial participants all had tried existing medications, without success. The mean age was 71, and most had suffered with their tremor for many years.
Seventy-five percent of participants received the experimental treatment using focused ultrasound guided by magnetic resonance imaging. The remaining 25 percent underwent a sham procedure, to act as the control group. (They would later be given the opportunity to undergo the real procedure.)
Participants who received the treatment showed dramatic improvement, with the beneficial effects continuing throughout the study period. The researchers employed a 32-point scale to assess tremor severity, and they found that mean tremor scores improved by 47 percent at three months and 40 percent at 12 months. Participants reported major improvements in their quality of life. People who couldn’t feed themselves soup or cereal could again do so.
Participants who received the sham procedure, on the other hand, showed no significant improvements.
“The degree of tremor control was very good overall in the study, but the most important aspects were the significant gains in disabilities and quality of life – that’s what patients really care about,” Elias said.
The most commonly reported side effects were gait disturbances and numbness in the hand or face; in most instances, these side effects were temporary but some were permanent.
FDA Approved
Based on the clinical trial led by Elias, the federal Food and Drug Administration has approved the focused ultrasound device, manufactured by InSightec Inc., for the treatment of essential tremor. The device focuses sound waves inside the brain to create heat, much like a magnifying glass focuses light. That heat can then be used to interrupt the troublesome brain connections responsible for the tremor. Elias can actually watch as patients’ tremor decreases, and the real-time imaging allows him to zone in on exactly the right spot before making any permanent changes to the brain.
The FDA approval means UVA can make the procedure available to eligible patients. UVA, however, is still working out the necessary logistics; it’s not yet clear when Elias will begin treating patients. Because the approach is so new, insurance plans will not yet cover the procedure, though that may change in the coming months. The cost at UVA has not yet been determined.
People interested in the procedure can learn more at uvahealth.com/focusedultrasound. The site includes a list of frequently asked questions and will be updated as UVA prepares to make the treatment available.
The procedure is not for everyone with essential tremor. It can’t be used in patients who cannot undergo MRI imaging, including those with implanted metallic devices such as a pacemaker. It is also not available for pregnant women, people with heart conditions or very high blood pressure, patients with kidney disease or clotting disorders, patients on blood thinners, patients with a history of strokes or brain tumors and people with substance abuse issues. There are other exclusions as well. Doctors at UVA will evaluate potential patients to determine their eligibility and then recommend the best course of treatment.
Groundbreaking Research
UVA is a world leader in focused ultrasound research. Elias and his colleagues are testing the capability of focused ultrasound to treat Parkinson’s disease, epilepsy, brain tumors and benign breast tumors.
The essential tremor research has been supported by InSightec, the Focused Ultrasound Foundation and the BIRD (US-Israel Binational Industry Research and Development) Foundation.
FOR REPORTERS: Elias will be available for interviews today, and he’ll be doing a Facebook Live broadcast at 3:15 Eastern. To arrange an interview, contact Alex Prevost at 434.906.7255 or amp3gb@virginia.edu. High-resolution images and video are available as well.
This release reports the results of a randomized, multicenter, sham-controlled clinical trial testing the safety and efficacy of a new approach to controlling essential tremors in the elderly — magnetic resonance imaging (MRI)-guided focused ultrasound — to alter the thalamus in the brain and thus control tremors. The approach would be an alternative to the current procedure — deep-brain stimulation — which is more invasive.
The study showed substantial improvement in a reduction of tremors for many patients at three-, six- and 12-months. But it also showed that about a third of the patients in the study experienced not insignificant adverse effects from the procedure. The release presents a glowing picture of the promise of this new approach while disregarding the caveats offered both in the journal paper and an accompanying editorial about the study.
To see how a news outlet covered this new research, see our review of a STAT article. Like the news release, the STAT story didn’t provide us enough quantification of the benefits or mention a potentional conflict of interest between researchers and device manufacturers. But the STAT article picked up on some things the release missed, including more discussion of the harms observed in the study.
Estimates are that essential tremors affect as many as 10 million Americans, many of whom are not helped by medications and who may not be candidates for the current surgical remedy — deep-brain stimulation. If proven safe and effective after long-term studies, this less invasive alternative would give patients and health care professionals more options for dealing with a condition that negatively affects the quality of life for many.
The release mentions cost, saying, “Because the approach is so new, insurance plans will not yet cover the procedure, though that may change in the coming months. The cost at UVA has not yet been determined.” But the procedure is still neurosurgery which is inherently expensive. Readers might benefit from knowing the cost of the current alternative surgical approach to treating this condition, deep-brain stimulation. The fact that other language in the release does come close to marketing this procedure for the medical center is also troubling.
We’ll give this release credit for including numerical data on patient improvement during the study: “they found that mean tremor scores improved by 47 percent at three months and 40 percent at 12 months. Participants reported major improvements in their quality of life. People who couldn’t feed themselves soup or cereal could again do so.” Unfortunately, it omits a host of other numerical data contained in the NEJM paper that places the patient improvement in a less-glowing perspective, issues like the reduction in improvement over time experienced by some patients and the extent of adverse effects (see below). It’s quantification data was based on relative rather than absolute improvements and it was not clear what those improvements meant functionally. The release could have described the range of the scale and given the actual tremor scores. This would have been more informative than describing a 47% reduction which could represent a very large or relatively small changedepending on what the baseline scores were.
Had the release included some of the caveats pointed to in an editorial about the same study carried in the journal, readers would have a clearer picture of just how important this new procedure was.
The release mentions harms in this statement: “The most commonly reported side effects were gait disturbances and numbness in the hand or face; in most instances, these side effects were temporary but some were permanent.” But that is by no means enough. More than a third of the patients in the study developed gait problems or numbness, or both, that in some cases persisted for 12 months. Others developed ataxia, unsteadiness and weakness that was persistent. The release seems to go overboard in downplaying these adverse effects and amplifying the positive outcomes. Releases should never cherry-pick the data from a study.
The release mentions that this clinical trial was a randomized, multi-center, sham-controlled study with a small but reasonable number of patients, given the procedure involved. It provides specifics on the patient population and the scope of their disease, as well as an explanation of how improvements were determined. The release would have been improved with a description of how improvement (or lack thereof) was measured.
The release does not appear to disease-monger.
This release identifies the funders of the study but fails to point out that the principal investigator has a financial relationship with the manufacturer of the device used in the study.
In addition, the published study notes that “Representatives of the manufacturer of the focused ultrasound system used in the study (InSightec) provided study oversight and technical support and obtained national regulatory permissions.” Allowing the device manufacturers to have “study oversight” suggests a potential for bias when most studies are obligated to keep a firewall between investigators and financial sponsors.
The release never mentions the current alternative approaches used to treat essential tremors, either the pharmacological approach with drugs such as propranolol or primidone, or the neurosurgical approach of deep-brain stimulation.
The release is clear in that the medical center is preparing to make the procedure available to qualified patients but it certainly is not available to the public at this time. It also mentions the uncertainty of insurance coverage.
The release points out that the trial is “the most in-depth assessment yet of the safety and effectiveness” of this new approach to dealing with essential tremors. And since the procedure is less invasive than the standard surgical intervention now in use, it is certainly novel enough to warrant a news release.
The release steps over the line several times in its use of hyperbole, beginning with the headline, “Scalpel-free surgery proves safe, effective for treating essential tremor,” that ignores the high proportion of adverse effects reported in the study and the limited time — 12 months — that the study covered. Saying a procedure is safe without long-term studies is an exaggeration. Further on, subheads refers to it as a “pioneering tremor trial” and “groundbreaking research.”
Systematic, Criteria-Driven
Excelente conversación entre @garyschwitzer de @HealthNewsRevu y @marynmck sobre exageración y #COVID19 en el excelente MOOC de @utknightcenter. Vale la pena verlo. https://www.youtube.com/watch?v=SkgXiPojtg4&feature=youtu.be
Words of real wisdom, as usual @garyschwitzer
And: “There were no details, charts, graphs, numbers, nothing published in a journal."
#Pharma chairman:“If you don’t put out data people will say why are you withholding the data?”
Well, yes, that is exactly what people are asking. https://twitter.com/garyschwitzer/status/1264572399523180548
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