Innovator Spotlight

Q: It's unusual to see a foundation created around a technology rather than a disease. What prompted you to start the foundation?
The Focused Ultrasound Foundation does seem to be a one-of-a-kind organization with a novel approach, a different kind of force in medical innovation, if you will. We don't know of any other foundation dedicated to advancing medical technology. My experience with the gamma knife was the genesis of the idea. Having been personally involved with the gamma knife's development, I witnessed firsthand that moving a new medical technology from a research prototype to a mainstream therapy is a slow, complex process that can consume decades while patients continue to suffer.

So, when I learned about focused ultrasound—often described as "medicine's best kept secret" —and its enormous potential to save lives and alleviate suffering, I thought there must be a better way. We established the Focused Ultrasound Foundation in 2006 to accelerate the process and make this revolutionary medical technology available to patients in the shortest time possible.

We began by analyzing the medical device development process from concept to worldwide standard of care, pinpointing chokepoints and identifying opportunities to accelerate progress. Then, we built an organization designed to break through the chokepoints and to catalyze cooperation among stakeholders.

The foundation concentrates on high-priority areas where we have the ability to effect change. Our initiatives include funding and coordinating research, creating effective partnerships, fostering collaboration, and building awareness. Much like an adrenaline rush, our programs provide the energy needed to speed up the global adoption of focused ultrasound.

For us, it's all about the patients. We are dedicated to exploring the potential of focused ultrasound as a mainstream therapy for cancer, brain tumors, Parkinson's disease, epilepsy, stroke, and other life-threatening conditions within years, not decades.

Q: Can you explain briefly how the technology works and what it can do?
Focused ultrasound is a noninvasive, early-stage therapeutic technology with the potential to revolutionize the treatment of many life-threatening and disabling conditions. Many believe that focused ultrasound could be a breakthrough in noninvasive surgery, an alternative or complement to radiation therapy, and a way to deliver chemotherapeutics and other drugs at higher concentrations to precise targets with less toxicity.

Much like the process of focusing sunlight through a magnifying glass, this innovative technology precisely focuses ultrasound waves to a target area within the body. Because treatment occurs only at the point where the sound waves intersect, surrounding tissue remains healthy and intact.

In addition to being noninvasive, focused ultrasound treatments do not involve general anesthesia and are typically performed on an outpatient basis in a single session. For patients, this translates into less pain and discomfort, more rapid recovery, fewer complications – like infections or blood clots – and avoidance of toxic side effects associated with drug and radiation therapies. Clinical trial data are demonstrating that focused ultrasound therapies may be safer, more effective, and less costly than comparable surgical interventions. And, very importantly, focused ultrasound produces an immediate and verifiable effect.

Researchers around the world are investigating the technology's use in treating many forms of cancer—including bone, brain, breast, liver, pancreas, prostate, and thyroid—and neurological conditions such as essential tremor, Parkinson's disease, epilepsy, obsessive compulsive disorder, and stroke. In the United States, focused ultrasound treatments for uterine fibroids have been approved by the FDA. Additional treatments for other indications are available abroad.

Q: You've chosen to launch your efforts in a handful of specific areas – can you tell us what those are and why they were chosen?

The foundation continuously monitors advances in focused ultrasound. As a trusted, independent third party that is unbiased and does not stand to achieve financial gain, the foundation has the unique ability to play the role of global connector and pinpoint areas where concerted effort can lead to advances that will resonate across the entire field.

With these ideas in mind, the foundation identified the brain as a watershed target for focused ultrasound and established our Brain Program in 2009 with the goal of driving innovation in this area. A key reason for this decision was to validate the technology by demonstrating focused ultrasound's ability to treat the brain, the most difficult organ to treat safely and effectively, and its potential to treat less sensitive tissues such as breast, liver, prostate, and bone. In addition, we focused on the brain to create awareness, as people are extremely interested in the workings of the brain and the debilitating consequences of neurological disorders.

Encouraged by the success of the Brain Program, we have also established a Liver and Pancreas Program with the goal of advancing focused ultrasound therapies for two of the most deadly, prevalent, and difficult-to-treat cancers.

However, it is important to note that the foundation also supports investigator-initiated research for a broad spectrum of conditions ranging from breast and prostate cancer to diabetes and chronic back pain though our Research Awards Program. The Research Awards Program enables us to encourage innovative, new ideas and engage with investigators at a large number of international institutions.

Q: From the earliest days of the foundation you've focused on such downstream issues as FDA approval and reimbursement – why have those been important for you as a nonprofit organization?As noted earlier, our work is "all about the patients." Reimbursement and regulatory approval are chokepoints that slow down patient access to new therapies.

Here's a good example of how reimbursement can be a chokepoint. The first focused ultrasound indication to receive FDA approval was uterine fibroids. Despite having been approved in 2004, this treatment is not readily accessible to patients in the U.S. and the main reason is because few insurance companies are willing to pay for it. As a result, few physicians suggest it and few hospitals offer it. To build a case for coverage, the foundation is partnering with focused ultrasound medical device manufacturers, who view the foundation as an objective and trusted third party.

To ensure that other indications move through the regulatory process as smoothly as possible and have a high likelihood of receiving insurance reimbursement, the foundation is helping to broaden clinical study protocols. We support researchers in developing protocols that not only demonstrate safety and efficacy but also address regulatory standards and meet insurers' requirements for superior long-term benefits, quality of life, and cost-effectiveness.

We also work directly with regulatory agencies to help focused ultrasound therapy gain traction. We were delighted last year when the FDA recognized the potential of focused ultrasound to "reduce the number of invasive surgeries that patients need" and began developing test methods for focused ultrasound products and evaluating tests developed by others.

Q: What do you consider the foundation's greatest achievements to date?The foundation helped write medical history in 2011 by organizing and funding the world's first clinical trial in which focused ultrasound was used to treat essential tremor, a debilitating neurological condition. The trial produced a sea-change in the enthusiasm and interest of stakeholders in the focused ultrasound community, including physicians, scientists, manufacturers, patients, regulatory agencies, investors, payers, and the media, and represents a potential quantum leap for the field by demonstrating that focused ultrasound may offer patients a better alternative to invasive surgery or complicated drug therapies. It also laid the groundwork for new clinical trials for Parkinson's disease, brain tumors, and epilepsy, and opened the door to studying focused ultrasound therapy to treat targets more easily reached in the body, such as in the breast, prostate, or spine.

Fifteen patients were treated in this pilot study. Treatments were performed through the intact skull – no incisions were made. Patients received no anesthesia and remained wide-awake during the procedure. Before treatment, study participants could not write legibly, or drink and eat normally. Immediately following treatment, their tremors almost completely subsided. Patients and their families have called the new procedure "life-changing." As reported at the 2011 Congress of Neurological Surgeons, interim study results showed that patients had no residual disabilities in daily activities.

The trial shifted the dialogue from "if this technology can have a transformational role in therapy" to "when will this technology be widely available?" We were thrilled that the prestigious conference TEDMED included focused ultrasound on its 2011 agenda and that TIME Magazine recognized focused ultrasound as one of the "50 Best Inventions of 2011."

Q: What are your top research goals for 2012, and what will it take to reach those goals?
Our research goals for 2012 are aimed at establishing noninvasive focused ultrasound treatments for Parkinson's disease, epilepsy, as well as tumors of the breast, brain, liver, and pancreas.

Among these goals, Parkinson's research is a major priority for the foundation as it represents the next step on our R&D roadmap for movement disorders, which began with the essential tremor clinical trial. We are planning two phases of Parkinson's research. In the first, 30 patients with tremor-dominate Parkinson's disease will be treated. Once this first trial is under way, we will initiate a second clinical trial aimed at treating an additional 30 Parkinson's patients with bradykinesia/dyskinesia. The foundation needs to raise $3 million to fund these treatments. We anticipate the first patient treatments beginning in the spring.

Private support is critical to our achieving these goals. In the last five years, we have raised more than $35 million. Individual donors have contributed 70 percent of these gifts, and the balance has come from corporations and organizations. These funds have been successfully invested in high-potential research projects and programs. The foundation's challenge is to continue building momentum, and toward that end, our 2012 fundraising goal is $10 million. In addition, we are pursuing opportunities to leverage our financial investments by seeking co-funding opportunities and getting other organizations more involved in the field.

By steadfast adherence to the disciplined, entrepreneurial approach that has become the foundation's hallmark, we will ensure that 2012 is another successful year for the field of focused ultrasound and, even more importantly, for the patients who will benefit from the new treatments being developed.