(RxWiki News) A new drug under development for the treatment of the childhood leukemia T-ALL has had success in the first round of pharmaceutical testing.
An experimental new drug known as CAL-130 is a dual enzyme inhibitor, blocking two forms of an enzyme used in the out of control growth involved in leukemia.
The drug successfully stopped the enzymes phosphoinositide-3 kinase gamma and delta in testing, effectively stopping cancer growth.
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Testing of the dual inhibitor took place first in mice, and then again in samples of leukemia cells taken from patients. Data from the study showed that leukemia cell levels quickly dropped in both laboratory experiments performed on mice with leukemia, as well as samples of cancer cells taken from humans.
Blocking phosphoinositide-3 kinase gamma and delta had a clear double effect due to their close involvement in the development of the leukemia T-ALL. The leukemia cells died off rapidly, and cells that were developing abnormally into cancer were stopped from progressing further.
Results demonstrated that less than one percent of abnormal cells were alive a day later.
Trials in mice showed that treatment with CAL-130 extended lifespan for mice with leukemia from an average of 8 days to 45 days.The dual inhibitor CAL-130 was developed by Gilead Sciences.
Thomas Diacovo, MD, and his team from Columbia Medical Center initially developed the research on these enzymes when investigating the biochemistry involved in cellular inflammation several years ago. After some study, he decided to adapt his findings to developing a leukemia drug.
Out of all the forms of leukemia found in children, T-ALL is the most resistant to treatment, and relapse can occur even if treatment is successful.
"Clearly, we have a drug that is extremely effective against this type of cancer in mice,” stated Dr. Diacovo.
“If this treatment strategy can safely and selectively target the activity of these enzymes in T-ALL tumors, we might be able to reduce the need for conventional chemotherapies that more broadly affect proliferating cells, including those in healthy tissues. This would be a major advancement in helping to reduce drug toxicities in young patients.”
The study was published in the April 17 online edition of the journal Cancer Cell.
Two researchers involved in the study are employees of Gilead Sciences, which manufactures CAL-130. No other conflicts of interest were disclosed.
This research was supported by the Department of Defense, the Leukemia & Lymphoma Society Translational Research Program, and Gilead Sciences.