Introduction
The goal of both a clinical trial and of a patient registry is to produce evidence for scientific advancement, regulatory approval, and/or clinical decision-making. Producing sufficient evidence will yield a best practice or a clinical guideline that establishes a standard of care.

Evidence can be produced in a variety of ways. At the top of the pyramid are clinical trials and prospective cohort studies. A clinical trial provides a test of a hypothesis to produce an efficacy statement that the intervention improves outcomes. It establishes a mechanistic efficacy associated with a clinical outcome and the intervention is safe.

In October 2018, West Virginia University neurosurgeon Ali Rezai, MD, treated the first patient in a groundbreaking US clinical trial for Alzheimer’s disease. A team of 35 medical experts at the Rockefeller Neuroscience Institute, led by Dr. Rezai, performed a phase II trial – for the first time in the United States using focused ultrasound to open the blood-brain barrier and target the hippocampus of a 61-year-old patient with early-stage Alzheimer’s disease. A second patient with Alzheimer’s underwent the procedure in January 2019 at West Virginia University.

The team used Insightec’s focused ultrasound device, Exablate Neuro.

Recently, we caught up with Dr. Rezai to talk about the first patient’s progress and his plans for future applications of focused ultrasound.

Jill O'Donnell-Tormey, PhD, is chief executive officer and director of scientific affairs at the Cancer Research Institute. In 2017, CRI and the Focused Ultrasound Foundation partnered with the goal of advancing the development of new focused ultrasound and cancer immunotherapy treatments. A major part of the partnership is a joint fund that supports research to move toward new combination therapies. That fund awarded its first research grant in June of 2018. Dr. O’Donnell-Tormey appeared at the 6th International Symposium on Focused Ultrasound in October to discuss the partnership. Below is an edited transcript of our interview with her.

Why did you partner with Focused Ultrasound Foundation?
As immunotherapy was starting to gather momentum and there were more FDA approvals of immunotherapy, the Focused Ultrasound Foundation thought there might be a place to combine immunotherapy and focused ultrasound. So that was the impetus.

Joo Ha Hwang, MD, PhD, is Professor of Medicine, Gastroenterology and Hepatology at Stanford University. He is also president of the International Society for Therapeutic Ultrasound, a partner organization of the Focused Ultrasound Foundation. Dr. Hwang became interested in focused ultrasound through his work with pancreatic cancer patients: He wanted to find a therapy that would prolong their lives or even cure them. Today he is more optimistic than ever about the possibility of using focused ultrasound to treat pancreatic cancer. Below is an edited transcript of our conversation with him.

Why are you so enthusiastic about this technology?
I've been in this field since 2000, so I've been actively doing focused ultrasound research for 18 years. Much of that research has been looking at enhancing drug delivery for pancreatic cancer using focused ultrasound. We have achieved great results, but the problem has been that there are no real effective chemotherapeutic agents. Even our best agents have very modest efficacy.

Across the medical device industry there is a very strong startup scene, and the field of focused ultrasound is no exception. According to our 2018 State of the Field Report, there are 70+ companies in the field globally; 42 of those companies are actively developing a device (or devices), with the remainder supporting other aspects of the field (OEM suppliers, distributers, etc.). The field comprises a vibrant community of innovators who are developing novel ways to solve important health problems using ultrasound technology as a therapeutic alternative to the standard of care.

Startup companies in the medical device space historically have excelled at the innovation and engineering portion of the product development process. However, they have not often been expected to go through the entire product development process. That paradigm is changing now, and again, this is true for the focused ultrasound startup community. The intricacies of getting a device through regulatory approvals and determining how to successfully commercialize it can be difficult. The challenges can be particularly difficult for novel products like focused ultrasound devices, where there is no established path for the regulatory authorities, let alone marketers.

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