New Treatment and Drug-Delivery System Shows Promise for Leukemia

Mar. 25, 2021

A cutting-edge nanoparticle drug-delivery system can curtail acute leukemia by sending medication into the bone marrow more efficiently, a new Wilmot Cancer Institute study shows.

Young male scientist holding a microchip in a research lab
Ben Frisch, Ph.D., studies blood cancers that start in the bone marrow

So far, researchers have only evaluated this system in mice. But their data is promising and it could apply to a diverse group of leukemias and blood cancers that impact the bone marrow. Leukemia is a particularly aggressive disease because patients often relapse.

A team led by Danielle Benoit, Ph.D., and Benjamin Frisch, Ph.D., tested an approved HIV drug, delivered by a specialized nanoparticle system, and discovered that leukemia slowed considerably. In addition, the treatment partially restored the activity of the bone marrow, allowing it to once again pump out healthy blood cells instead of acting solely as a breeding ground for cancer. The study was published by the FASEB journal.

Understanding what happens in the marrow is the focus of Wilmot’s internationally known Cancer Microenvironment research program. Frisch and others investigate the interplay between cancer stem cells and other tissues in the bone marrow, to learn what suppresses the immune system and permits cancer to spread and resist treatment.

The latest study confirms the role of a key bone marrow disrupter — a protein known as CCL3. Leukemia cells secrete CCL3, which promotes inflammation. CCL3 is elevated in the majority of patients who have acute myeloid leukemia, the study said.

In the laboratory, researchers tested maraviroc, the HIV medication, which is known to interfere with the CCL3 pathway and tame inflammation. They found many potential avenues for further study, including future clinical trials in humans.

head and shoulders photo of a female scientist
Danielle Benoit, Ph.D.

Benoit invented the nanoparticle delivery system, which can bypass barriers that prevent drugs from reaching their targets. Nanoparticles are microscopic materials that act as a bridge between different structures. In this case, the system was designed to encapsulate drugs and direct them into the bone marrow.

Much of the laboratory work was conducted by first author Marian Ackun-Farmmer, Ph.D., a former doctoral student in Biomedical Engineering at the University of Rochester, who defended her thesis in October 2020 and subsequently joined the University of Maryland as a postdoctoral researcher. Co-corresponding authors are Benoit, a professor of Biomedical Engineering, and Frisch, an assistant professor of Pathology and Laboratory Medicine and Biomedical Engineering.

Funding was provided by the University of Rochester Research Award, Wilmot’s development funding program, The National Institutes of Health and National Cancer Institute, the National Science Foundation, and the UR Clinical and Translational Science Institute