More than a decade ago, UVA Health pioneered focused ultrasound, which produced immediate, dramatic results for patients with essential tremor.
As researchers investigate more applications for the technology, it continues to gain FDA approval to treat other conditions — Parkinson's, uterine fibroids, liver tumors.
Now, numerous promising clinical trials are underway to explore it's use against a number of cancers. In this video and Q&A, Shayan Moosa, MD, shares how focused ultrasound could offer a new tool against glioblastoma and other brain cancers.
What are you working on right now?
I'm leading a clinical trial exploring the use of sonodynamic therapy to treat patients with recurrent glioblastoma. This innovative approach leverages transcranial focused ultrasound to activate an orally administered drug that selectively accumulates within tumor cells. Once triggered, this interaction induces a localized cytotoxic and immune response, subsequently enhancing cancer cell destruction.
Currently, transcranial focused ultrasound treatments require large, specialized devices in advanced medical centers, relying on MRI guidance or computerized optical tracking for precise ultrasound delivery. My lab is also pioneering new methods involving ultrasound-penetrable cranial implants that may allow these treatments to be performed more easily in the clinic or at the patient’s bedside.
What are the most intriguing potential clinical applications of your work?
Glioblastoma is one of the most aggressive and relentless brain cancers, with no known cure. Even with the most advanced treatments — surgery, radiation, and chemotherapy — patients face a devastating prognosis, with an average life expectancy of just 15 months. Unfortunately, recurrence is almost inevitable.
Sonodynamic therapy has the potential to change this reality. By seamlessly integrating with existing treatments, this innovative approach could extend the time before tumor progression and provide another noninvasive option for tackling recurrent disease.
With the ability to precisely target cancer cells while sparing healthy tissue, sonodynamic therapy represents an exciting step toward more effective and less invasive treatment strategies for glioblastoma and other aggressive cancers.
What recent discovery has impacted the way you think?
The growing body of research on immunotherapy for glioblastoma — particularly, the challenge of getting these treatments to work effectively in the brain.
Glioblastoma has long been considered 'immunologically cold,' meaning it evades immune detection, and efforts to activate the immune system have been largely unsuccessful. However, new strategies, including checkpoint inhibitors, CAR-T cell therapy, and personalized vaccines, are beginning to show promise.
What’s especially exciting is the emerging role of focused ultrasound in this space. Recent studies suggest that focused ultrasound — whether through blood-brain barrier opening, sonodynamic therapy, or direct neuromodulation — can help prime the tumor microenvironment for a more robust immune response.
For example, using focused ultrasound to open the blood-brain barrier could enhance the delivery of immunotherapeutic agents directly to the tumor, while sonodynamic therapy may induce immunogenic cell death, effectively turning the tumor into its own vaccine.
This intersection of focused ultrasound and immunotherapy is shifting how I think about glioblastoma treatment. Instead of viewing focused ultrasound as just a delivery tool, we are starting to explore its potential as an immune-modulating therapy in its own right. If we can use sound waves to make glioblastoma more responsive to immunotherapy, we may finally unlock a more effective, durable treatment for this devastating disease.
What do you wish more people knew about your area of research?
Transcranial focused ultrasound is a powerful technology with applications far beyond sonodynamic therapy. In fact, it offers multiple ways to harness the power of sound waves to interact with the brain in groundbreaking ways.
One key application is blood-brain barrier opening. Focused ultrasound can temporarily and noninvasively open this protective barrier, allowing us to deliver drugs or collect biomarkers from otherwise inaccessible regions of the brain.
We are planning a clinical trial that will use focused ultrasound to open the blood-brain barrier in a specific region of the brain in patients with Parkinson’s disease. This could pave the way for diagnosing the disease through a simple blood test — something that has never been possible before.
Another exciting application is neuromodulation, where focused ultrasound is used to alter neuronal activity without invasive surgery. We are conducting a clinical trial investigating its ability to modulate tremor circuits in the brain, potentially providing symptom relief for patients with essential tremor and Parkinson’s disease — all without creating permanent lesions.
If successful, this technique could revolutionize treatments for a wide range of conditions, including chronic pain, epilepsy, and psychiatric disorders.
With its ability to precisely target and influence the brain without incisions or implants, focused ultrasound has the potential to transform the way we diagnose and treat neurological diseases, offering safer, more effective, and less invasive solutions for patients.
What made you choose UVA Health as the place to do your research?
At UVA, students and faculty proudly refer to themselves as Hoos, and I’m what you might call a quadruple-Hoo — having completed my undergraduate degree in neuroscience, medical school, neurosurgical residency, and now serving as faculty, all at UVA.
My journey in research began during my undergraduate years when I had the incredible opportunity to join Dr. Kevin Lee’s lab, where I focused on making glioblastoma cells more sensitive to radiation therapy— work I continued into medical school.
During my neurosurgical residency, I was introduced to the transformative potential of focused ultrasound by Dr. Jeff Elias, whose pioneering work has helped revolutionize treatments for essential tremor and Parkinson’s disease. As I delved deeper, I recognized the vast potential of this technology beyond movement disorders, envisioning its application in brain tumors and other neurological diseases.
None of this would have been possible without the extraordinary mentorship I received and the unwavering support from UVA Health throughout my training. Given the profound impact this institution has had on my career, continuing my research here felt like the natural next step.