CHARLOTTESVILLE, Va. — An obscure medical technique involving zapping a body part with converging beams of sound is finally getting some high-profile attention.
Focused ultrasound, as the procedure is known, is used commercially in the United States to treat just a few medical conditions, including uterine fibroids and prostate cancer. Just over two dozen hospitals and clinics across the country offer it. Hardly any insurers will pay for it.
But an irreverent, impatient, retired neurosurgeon has made it his mission to accelerate development of the treatment — and this week, he got an opportunity to do that in a big way when he was named to a panel advising Vice President Joe Biden on the national cancer moonshot initiative.
The call didn’t come out of the blue for Dr. Neal Kassell. The University of Virginia professor performed two brain surgeries on Biden to repair aneurysms in 1988. A lifelong Republican, Kassell has maintained a friendship with Biden in the years since.
They’ve talked about the therapeutic power of high-frequency sound beams, and a Biden aide even attended a recent workshop put on by Kassell’s Focused Ultrasound Foundation, according to Biden spokeswoman Meghan Dubyak.
Now, the technology — as expensive and limited as it is — “is one of the cutting-edge therapies that the VP is exploring through the moonshot,” Dubyak said.
Kassell believes focused ultrasound has the potential to “play a real role” in advancing the moonshot’s goals, such as by boosting the effects of cancer immunotherapy or delivering chemotherapy in a more targeted manner. He believes it could treat many types of cancer, as well as other diseases like Parkinson’s and perhaps even Alzheimer’s.
“The problem,” Kassell said, “is that most people have never heard of focused ultrasound. So we need to get that visible.”
He’s been working on just that.
Indefatigable at 70, Kassell — pronounced kah-SELL — talks slowly, in a deep voice that initially disguises his frequent deadpan jokes. He’s candid about the frustrations of advocacy and fundraising. He speaks with unvarnished impatience about too much red tape at organizations like the March of Dimes (“a self-perpetuating bureaucracy”) and the University of Virginia (which he jabs for “fiddling around”).
He’s such a persuasive evangelist for focused ultrasound that he inspired best-selling legal novelist John Grisham — a personal friend — to write a book championing the technology’s potential, over the concerns of his publishers. It’s been ordered or downloaded more than 250,000 times since coming out in December.
“To be involved in this is a sort of moral imperative.”
Dr. Neal Kassell
Kassell has also raised about $70 million over the past decade for his Focused Ultrasound Foundation — which is believed to be the only health charity dedicated to promoting a specific medical device. (Manufacturers of focused ultrasound machines have donated modest sums in the past, though not last year, mostly to support the foundation’s biannual research symposium.)
Kassell turns earnest when he talks about the millions of people he believes could potentially benefit from the treatment. They’re not just hypothetical patients to him: About a decade ago, his son-in-law died within months of being diagnosed with a brain tumor. Kassell now believes that focused ultrasound has potential to treat such tumors.
“To be involved in this is a sort of moral imperative,” Kassell told STAT during an interview here at the foundation’s office, a short drive from the stately University of Virginia campus.
An old technology with new promise
You might think of focused ultrasound as similar to the effect created when a magnifying glass focuses rays of sunlight to burn a hole in a leaf.
During the procedure, patients slide into an MRI machine or lie on a bed where a machine concentrates sound waves on a precisely targeted spot of tissue. It’s most often used to burn and destroy tumors or other unwanted tissue, but early studies suggest it may have promise in unleashing the immune system or activating drugs in the body. And patients don’t have to be put to sleep, go under the knife, or be exposed to harmful radiation.
First used in the clinic in the 1950s to treat pain, focused ultrasound is now predominantly used to treat prostate cancer in men and uterine fibroids in women.
The treatment is more widely accessible in Europe and Asia than in the US, but only about 25,000 patients worldwide got focused ultrasound last year.
Most patients need only a single session, but it’s costly: Treatment for prostate cancer costs about $25,000, and treatment for uterine fibroids goes for between $5,000 and $10,000. Many patients pay out of pocket, though some successfully appeal their insurers to get it covered.
The technology is also being tested in early-stage clinical trials around the world to treat other cancers, hypertension, and even neurological conditions like brain tumors, obsessive compulsive disorder, and Parkinson’s disease. Patients are getting experimental treatments at 45 sites in the US.
Independent experts and physicians say that although it’s still largely unproven, focused ultrasound has real promise and should be studied further.
“Will it replace surgery? Probably never. But the benefits of being able to treat someone without actually cutting into their body certainly appeal to a lot of people,” said Daniel Merton, who evaluates new medical technologies for the health care research organization ECRI Institute.
Despite their optimism, experts are careful to point out the technology’s limitations: It often doesn’t work on large tumors, or those that are in difficult-to-reach places. Patients risk painful burns or damage to healthy tissue. The equipment is extremely costly. And the long-term benefits for patients are as yet uncertain.
Kassell’s foundation is also careful not to oversell the technology’s potential.
In his book “The Tumor: A Non-Legal Thriller,” Grisham — who sits on the foundation’s board — imagines a future in which focused ultrasound treatment is widely available. The protagonist, Paul, is able to extend his life by more than seven years after being diagnosed with a deadly brain tumor, thanks to three rounds of focused ultrasound treatment. The projected cost: About $75,000, a quarter of the cost of the treatments Paul would get today.
But not even Grisham dares to dream that the technology can cure his hero. Paul’s brain tumor kills him eventually.
A ‘eureka’ moment
Kassell first stumbled upon therapeutic ultrasound more than a decade ago, when he was searching for a way to treat hard-to-reach brain tumors.
In the clinic, he had recently started using ultrasound to burst tiny bubbles injected into the bloodstream to measure blood flow in the brain. A eureka moment hit him in the car one day: Ultrasound might be able to treat brain tumors, too.
He realized when he got home and started scouring the scientific literature that he wasn’t the first person to have this idea. He read all the research he could find on the technology and met with a leading manufacturer of the equipment. He now thinks hundreds of thousands of people could eventually be treated annually.
“You can’t walk away from that sort of responsibility,” Kassell said. “The opportunity to be involved in a true revolution in therapy that can affect so many people — that’s a once-in-a-lifetime thing.”
His friends weren’t so sure: Some wondered why he’d shift so much of his time and energy toward an obscure and unproven medical device.
“My immediate reaction was ‘Holy cow,’” said Kassell’s longtime friend Dan Jordan, a former president of the Thomas Jefferson Foundation who now consults for nonprofits and sits on the board of Kassell’s foundation. “But I would never bet against Neal.”
“My immediate reaction was ‘Holy cow.’ But I would never bet against Neal.”
Dan Jordan, member of Focused Ultrasound Foundation board
Kassell — married with three grown daughters, three stepchildren, and six grandchildren — is smart, loyal, and charming, his friends and scientific collaborators say. It’s a personality, they say, that makes him a natural and credible salesman for a medical technology in need of an evangelist.
It certainly helps that Kassell is well-connected in the social circles of high-society Virginia — and on bigger stages, too. His foundation’s board members include a former FDA commissioner, a former CEO of Johns Hopkins Medicine, and a former CFO of Citigroup.
Last year alone, the foundation spent $3.1 million funding research, primarily targeting the technology’s potential application in the brain.
Kassell’s goal for his foundation is that it will put itself out of business, in about a decade, by advancing the field to a point where an advocacy organization is no longer needed.
“When you go to these philanthropic meetings, they talk about how to make your organization sustainable. We say, ‘Why don’t you talk about how to make it not sustainable?’” Kassell said.