Patients with acute myeloid leukemia (AML), a type of cancer that develops in the bone marrow and affects blood cells, could soon have the option of a new and improved treatment that involves a state-of-the-art drug delivery system. The approach uses two different molecules, fused together, that provide a combining effect.
Zhen Gu, professor of bioengineering at the UCLA Samueli School of Engineering and lead author of the study, is confident his procedure will significantly augment current therapy courses. Chemotherapy, for example, is only effective in about 66 percent of cases, according to estimates from the American Cancer Society. Furthermore, many patients that exhibit signs of remission find themselves suffering from relapse — typically within two years after treatment.
The so called “cell combination drug delivery” represents a medical premiere, it’s the first time two distinct cells are joined together for therapeutic purposes. One half has blood platelets that are used to deliver immunotherapy drugs called checkpoint inhibitors, which reveal the cancerous cells to the immune system and neutralize their defenses.
“This part of the cell combination is like a delivery truck”, explained Dr. Gu. “We can package medicines or immune system boosters on the cell surface of platelets, and have them activated to unload once at the target site inside the body”.
Hematopoietic stem cells, which form the other half, have the ability to find their way into the bone marrow through specific chemical signals, delivering their “package” to places where chemotherapy does not reach.
“The hematopoietic stem cells are like a homing signal to the bone marrow. Once the stem cells guide the combo cells into the marrow, the platelets can be activated. They release immunotherapy cargoes inside the marrow to facilitate the body’s own defenses, in this case T cells, to kill leukemia cells”.
So far, the procedure has been tested only on mice, but the results were remarkable. 87.5 of subjects managed complete remission within 80 days from the initial injection. Those mice were also shown to be leukemia resistant following additional exposure to cancerous cells.
This innovative double pronged approach could represent an effective solution for leukemia as well as other diseases.