Researchers at Moffitt Cancer Center have been awarded a $22.4 million grant from the U.S. Department of War to lead groundbreaking studies and clinical trials for leptomeningeal disease, a devastating complication of breast and other cancers that spreads to the coverings of the brain and spinal cord. The competitive award represents the only grant funded among 14 national applicants and positions Moffitt as a leader in addressing this critical unmet medical need.
Grant Structure and Leadership
The four-year research initiative will be led by Peter Forsyth, M.D., chair of Moffitt's Neuro-Oncology Department. Moffitt will serve as the lead institution, receiving $18.7 million, in collaboration with Kent State University, which received $3.7 million. The funding will support two clinical trials and foundational research to better understand the disease mechanisms.
"Leptomeningeal disease is rare but extremely deadly, often giving patients only two to five months to live," Forsyth explained. "This funding allows us to launch clinical trials we believe can meaningfully change those outcomes while also building the scientific foundation to better understand why this disease develops and how to stop it."
Novel Therapeutic Approaches
The research program will test innovative treatment strategies that leverage the immune system's potential to combat cancer cells in the brain and spinal fluid. One trial will evaluate a novel dendritic cell therapy, a personalized immunotherapy that trains a patient's own immune cells to recognize and attack tumor cells in the brain and spinal fluid. A second trial will combine this approach with targeted antibody therapies and checkpoint inhibitors.
Forsyth's team has made a crucial discovery about the nature of spinal fluid in patients with leptomeningeal disease. "The exciting part is we've discovered that spinal fluid isn't just cancer cells floating in water. It's full of immune cells that are trying to fight, but they're not strong enough on their own," he explained. "Our goal is to give the immune system a helping hand, turning it from a one-armed soldier into a fully equipped defense force with memory to recognize and attack cancer if it returns."
Addressing a Critical Unmet Need
Unlike more common cancers, leptomeningeal disease has been poorly understood, with only a handful of published studies worldwide. For reasons not yet clear, breast cancer cells are particularly drawn to the brain and spinal fluid, where they can evade treatment even after tumors in the rest of the body are controlled. Forsyth and his team are among the few groups worldwide pursuing new treatments for this condition.
The research addresses a significant gap in cancer care, as current treatment options for leptomeningeal disease remain limited and largely ineffective. The disease's rarity and complexity have historically made it challenging to conduct large-scale clinical studies, making this grant particularly significant for advancing the field.
Clinical Impact and Future Outlook
The potential clinical impact of this research extends far beyond current treatment paradigms. "This grant means patients with breast cancer can live potentially years longer with leptomeningeal disease thanks to early versions of this therapy," Forsyth said. "I never want to look a patient in the eye and say we have nothing left to offer. This support brings us closer to making sure I never have to."
The grant also provides Moffitt with resources to strengthen its position as a hub for research on leptomeningeal disease, fostering collaborations that could establish the center as a national leader in this rare but devastating condition. The award underscores Moffitt's role as a leader in translational cancer research, bridging laboratory discoveries with clinical impact.
