Oct. 10 (UPI) -- Researchers at Albert Einstein College of Medicine have discovered a compound that makes cancer cells self-destruct while sparing healthy cells.
The study, published Monday in Cancer Cell, showed the use of the new treatment against acute myeloid leukemia, or AML, cells.
AML accounts for nearly one-third of all new leukemia cases and kills more than 10,000 Americans each year. Patients with AML have a survival rate of 30 percent, which has remained the same for several decades.
"We're hopeful that the targeted compounds we're developing will prove more effective than current anti-cancer therapies by directly causing cancer cells to self-destruct," Dr. Evripidis Gavathiotis, an associate professor of biochemistry and medicine at the Albert Einstein College of Medicine, said in a press release. "Ideally, our compounds would be combined with other treatments to kill cancer cells faster and more efficiently -- and with fewer adverse effects, which are an all-too-common problem with standard chemotherapies."
The new compound fights cancer by triggering apoptosis, the process that rids the body of unwanted or malfunctioning cells. Some chemotherapy drugs kill cancer cells by indirectly inducing apoptosis by damaging DNA.
Apoptosis happens when BAX, the "executioner protein" in cells, is activated by pro-apoptotic proteins. BAX molecules punch lethal holes in mitochondria once activated, but cancer cells produce anti-apoptotic proteins that prevent BAX from killing them.
"Our novel compound revives suppressed BAX molecules in cancer cells by binding with high affinity to BAX's activation site," Gavathiotis said. "BAX can then swing into action, killing cancer cells while leaving healthy cells unscathed."
Researchers screened more than one million compounds looking for those with BAX-binding capabilities, evaluating the 500 most promising compounds they found.
"A compound dubbed BTSA1 [BAX Trigger Site Activator 1] proved to be the most potent BAX activator, causing rapid and extensive apoptosis when added to several different human AML cell lines," Denis Reyna, a doctoral student in Gavathiotis' lab, said.
The team generated animal models of AML by grafting human AML cells into mice. BTSA1 was given to half the AML mice while the remaining half were controls. The BTSA1 mice survived significantly longer than the control mice, 55 days to 40 days respectively, and showed no evidence of toxicity.
"This study provides proof-of-concept for direct BAX activation as a treatment strategy in AML," researchers wrote in the study.