This story describes a recently FDA-approved device to treat essential tremor in patients who have not responded to medication. The ExAblate Neuro, made by Dallas-based InSightec, uses magnetic resonance images taken during the procedure to deliver focused ultrasound that destroys tissue in the thalamus, the part of the brain where tremors are believed to originate.
Results of a randomized clinical trial on the device’s safety and efficacy were published this month in the New England Journal of Medicine. The story and accompanying video (seen at right) vividly illustrate rapid symptom improvements, highlighting a 72-year-old woman who says the procedure changed her life by allowing her to “eat like a normal person” and easily perform basic tasks such as signing her name and folding a scarf.
This powerful patient anecdote throws the balance off: Imagine how different the story would seem if the video instead showed a patient who had improved, but also was affected by one of the serious adverse effects caused by the treatment.
Also, the story needed specific numbers on the measured benefits and harms, along with a discussion of costs and alternative treatment options.
Note: We also reviewed the news release that was issued to journalists about this study.
While not life threatening, essential tremor can be debilitating, especially affecting people in their advanced years. The ExAblate Neuro, which the FDA calls the first focused ultrasound device to treat essential tremor in patients who have not responded to medication, offers a somewhat less invasive treatment option than traditional surgical approaches. It’s certainly newsworthy. But all of the facts, including long-term efficacy data, are not in. Stories that feature glowing patient anecdotes and positive physician commentary need to be balanced with plenty of data that can help patients and their families decide whether this treatment might be worth seeking out.
The story does not say how much this new procedure costs or compare it with existing treatments for patients who don’t respond to medication. Deep brain stimulation, the current treatment standard, can run anywhere from $30,000 to $70,000, according to various online cost estimates. There also may be additional costs incurred by those patients who have adverse effects.
The story is long on description but short on numbers.
Saying the treatment “has had an immense impact on patients’ lives,” the story devotes quite a bit of print along with video footage to show rapid functional and quality-of-life improvements experienced by patients, focusing on one women who said her tremor “has almost disappeared” since she had the procedure in June. Video showing this patient before and after treatment–including her attempts to write her name, trace a spiral and pour water from one cup to another–do a great job of conveying the improvements. This is terrific news for this woman, but we’re not told how representative her experience is of overall outcomes. Is she a best-case scenario? Middle of the road?
There are also inspiring quotes from a researcher, including a description of the “remarkable moment” patients experience when they realized the tremor has subsided. “They can write their names again, they can feed themselves, they are not embarrassed about being in public. So it really is extraordinarily helpful for people,” he says.
In terms of study data, the story says researchers “found that the severity of essential tremor reduced significantly” in patients who underwent the treatment as well as reported improvements in quality of life. But there are no figures. In fact, according to the study, patients experienced a 47 percent improvement in tremor scores, dropping from 18.1 points on a 32-point scale at baseline to 9.6 points three months after the procedure. That data should have been included. Also, the story should have spelled out that 56 patients received the treatment, versus 20 who experienced a fake procedure. Also worth pointing out: We don’t know the meaning or clinical significance of a reduction of this magnitude in the total score.
The story addresses adverse effects, and that’s sufficient for a Satisfactory rating.
We do wish the story had quantified the harms that were measured in the study. The fourth paragraph says the procedure “brings lasting side effects in some patients” and later the story quotes a physician who is not involved in the research as saying, “It’s a pretty high incidence of side effects.” But nowhere do exact numbers appear.
Rather than citing data from the study, the story quotes the physician as saying that “over a third of patients actually experienced either gait disturbance or paresthesia,” or numbness. This information isn’t very useful because the story does not explain when these rates were measured. According to the study, three months after the procedure 36% of patients had gait disturbance and 38% had numbness. After 12 months, the numbers dropped, to 9 percent and 14 percent respectively.
Also worth mentioning is that one patient was reported to have permanent diminished sensation of the dominant thumb and index figure, which was categorized as a serious adverse event. That did not appear in the story.
The story clearly spells out that there isn’t data on the long-term efficacy of this procedure, and that more study is needed, so we’ll give this a barely passing Satisfactory.
Several important study limitations were not included in the story, such as the fact that the treatment did not provide significant improvement for some types of tremors, and the treatment is not appropriate for people who are unwilling or unable to undergo an MRI.
We’re also not told that the sham group was only followed for 3 months, while the experimental group was followed for a year. This is not usually recommended in clinical trials and both groups should be followed for the same length of time.
The story cites a figure from the International Essential Tremor Foundation, an advocacy group, that an estimated 10 million people in the United States suffer from the condition. However, a 2014 analysis of existing research put the number of cases at around 7 million as of 2012, or about 2.2% of the U.S. population. While this may not qualify as disease mongering, the story could have pointed out that the exact number of cases isn’t clear. Also, the story should have noted that not all people with essential tremor have symptoms severe enough to require treatment.
The story only fulfills part of our criteria. It does use one independent source, a physician who was not involved in the study. It also accurately reports the trial’s funding sources: InSightec, which developed the device; along with Focused Ultrasound Foundation, which is funded by device makers and philanthropic organizations; and the Binational Industrial Research and Development Foundation, an Israel-U.S. government partnership that receives corporate support.
However, the story falls short because it does not report the fact that nine of the study authors reported conflicts of interest, with most receiving financial compensation of some sort from device makers in the field. Lead researcher G. Rees Cosgrove, M.D. who is quoted in the story, reported receiving consulting fees from InSightec.
The story states, “Doctors usually treat essential tremor with medication, but the drugs don’t work well for all patients.” That’s true, and so we’ll give this a Satisfactory rating for at least mentioning alternatives.
But it should be noted that other procedural treatment options were not compared or even mentioned in the story and received only a brief mention in one of the videos.
The current standard for tremor patients who don’t respond to medication is deep-brain stimulation, in which a probe connected to a pacemaker is implanted in the thalamus to suppress the tremors. Gamma Knife thalamotomy, which delivers precise doses of radiation to the thalamus, is also considered to be a safe and effective therapy for patients who don’t respond to medication.
Also, some patients have mild symptoms that can be treated with relaxation techniques and the avoidance of triggers such as caffeine.
The story states that the device was approved by the FDA in July. The story could have done readers a service by giving a sense of how many medical centers around the country are geared up to perform the procedure, or will be soon.
This is indeed a new treatment option for essential tremor, though the story may leave readers with the false impression that the concept of killing off neurons in the thalamus is new. Rather, it’s the use of focused ultrasound to destroy brain tissue, rather than a probe inserted in the brain, that constitutes an advance.
The story does not appear to rely solely on the news release, which we also reviewed.