Researchers from the University of Florida College of Pharmacy say they have discovered a safer and more effective anticancer drug. The new treatment would target leukemia, lymphoma, and breast and lung cancers.
The drug, known currently as DT2216, affects a protein (called B-cell lymphoma-extra large), which grows malignant cells and strengthens their resistance to treatment.
There's already an inhibitor drug, but it causes a drop in blood platelets, raising the risk of bleeding. This led the U.S. Food and Drug Administration to deny approval for the earlier drug. Since then, scientists have been seeking an alternative.
According to a release on UF's website,
the new drug works better against a variety of tumor cells aided by the BCL-XL protein, and is also less toxic to blood platelets. University researchers developed the new BCL-XL-targeting anticancer drug using a technology that relies on small molecules that suppress and break down cancer-promoting proteins. They published their findings
in the journal Nature Medicine
on Dec. 2.
"These findings support the potential of DT2216 to be developed as a first-in-class BCL-XL-targeting antitumor agent," said Guangrong Zheng, Ph.D. in the release. First-in-class
refers to drugs which use a "unique mechanism of action" to work.
The researchers have so far demonstrated its effectiveness only in mathematical and mouse models, but say in those models the drug suppressed the growth of several tumors, both on its own and in combination with other drugs. The cancers affected by the drug include T-cell acute lymphoblastic leukemia and drug-resistant breast and small-cell lung cancers.
Zheng is an associate professor of medicinal chemistry in the UF College of Pharmacy, and jointly directed the research alongside Daohong Zhou, M.D., a professor of pharmacodynamics in the UF College of Pharmacy.
"We were fascinated by our findings because this was a first-of-its-kind study presenting a novel strategy to reduce the toxicity of an anti-tumor drug using PROTAC technology," said Zhou.
The study was supported by other research teams from the University of Florida, the University of Texas M.D. Anderson Cancer Center, Columbia University, Children's Hospital Los Angeles and the University of Texas at San Antonio. The team included researchers with expertise in cellular and molecular biology, drug discovery and development, medicinal chemistry, hematology and bioinformatics.
There are a few more steps and additional testing to go, before clinical trials with people can begin.
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