Ultrasound Drug-Device Offers Diffuse Treatment for Glioblastoma Multiforme

Alpheus Medical targets brain cancer with its drug-device combination sonodynamic therapy, offering a potential new treatment for patients who have few options. The company’s device is used in combination with a cancer cell-targeting drug to induce apoptosis in solid tumors. It is headed for its first-in-human trial after the company's closing of a $16 million series A funding round. The financing was led by OrbiMed Advisors and Action Potential Venture Capital, with additional investors Medtech Convergence Fund, and investment groups of the National Brain Tumor Society, and American Cancer Society. 

The company’s initial FIH trial will involve patients with recurrent glioblastoma multiforme (rGBM), a disease which is most often fatal and has few efficacious treatments. “These patients for too long have received suboptimal treatments,” said Vijay Agarwal, MD, neurosurgeon, founder and chief executive officer of Alpheus Medical. “Over the last hundred years there have been minimal advances in the treatment of brain tumors.”

Diffuse Tumors Tough to Treat

rGBM tumors are particularly challenging as the cancer is spread throughout the tissue, and the impact to healthy brain tissue has serious consequences. “What makes surgery and radiation so dangerous is that the brain is such an intricate organ. If you damage any part of the normal brain, arguably it will cause clinical effects,” said Agarwal. Instead, a therapy that can have an effect on multiple areas and attack cancer cells precisely is needed. 

“Brain cancer is a diffuse disease, so it requires a non-focal solution,” explained Agarwal. “Surgery or targeted therapies do not treat the disease—you need to treat half the brain. Distinguishing cancer cells from normal cells and selectively targeting those cancer cells is what we are aiming to do. The goal was to develop a technology that was intelligent, [which] could pick out the brain tumor cells from the normal brain.”

Targeting Tumors

The device utilizes an FDA-approved sono-sensitizing drug used to visually distinguish tumor cells, and its noninvasive ultrasound technology to target those cells for elimination. During treatment, a patient drinks a solution with the drug that then travels to the tumor cells. Alpheus’s sonodynamic therapy device noninvasively targets the drug-laden tumor cells, causing them to release an oxygen radical which leads the cells to die.

Agarwal noted that treatments are minutes long to about an hour, have minimal impact on patients, and are repeatable as needed. “We envision this being a treatment that patients can receive over time, perhaps once a month,” said Agarwal, adding that the treatment could be a monotherapy or combined with other regimens. 

Aim to Treat the Untreatable

The device’s promise has garnered the attention of two high-profile cancer organizations—the American Cancer Society and National Brain Tumor Society—which have backed some of the funding in the company’s series A. “We’ve been fortunate to be selected by these groups to help advance sonodynamic therapy and Alpheus Medical’s treatments of brain tumors,” said Agarwal. “This really helps spread the word and helps to reinforce the message that our aim is to be the most impactful treatment in brain cancer.” The company goal is to improve survival and quality of life in cancers that have had few measurable improvements.

“We believe this could be a platform technology. Our ultimate vision is that this could be a treatment for other solid body cancers as well,” Agarwal said. “We want to take these really acute terminal diseases and have them become manageable and treatable over a long period of time. We want to be a paradigm shift in how cancer is treated.”

The funding will be used to advance the FIH trial and safety and efficacy studies as well as to examine potential in other solid body tumors.