A study conducted by researchers at the Dana-Farber Cancer Institute and the Broad Institute of MIT and Harvard has shown that a variety of non-cancer drugs may also be effective as treatments for cancer. The study, which was published in the Nature Cancer journal, tested approximately 4,518 drugs against 578 cancer types and found almost 50 drugs showed some anti-cancer activity. This repurposing study is intriguing to scientists and doctors because the drugs have already been FDA-approved for safety, which means that the drugs could be accelerated into clinical trials for effectiveness against various types of cancer.
The drugs that were investigated through this study were previously used in medical areas vastly different than oncology. For example, one of the effective drugs is levonorgestrel, which is a hormonal drug used in birth control pills and emergency contraception. Also effective was disulfiram, which is used to treat alcohol dependence. The study was wide-ranging, aimed at giving researchers an understanding of which drugs could potentially be useful, or be modified to be effective, in oncology. This study is referred to as “repurposing” proven medicines from one indication to another.
The study also revealed previously unknown mechanisms for the studied drugs. For example, some of these drugs worked by activating or stabilizing existing proteins, which was effective against cancer even though most current cancer drugs work by blocking proteins. Most of the 49 drugs that did prove to have anti-cancer properties worked through a previously unidentified mechanism.
The discovery of unidentified mechanisms presents an additional opportunity for researchers. In addition to repurposing these into anti-cancer treatments, but they were also able to begin identifying other mechanisms that work against cancer that could inform new treatments for cancer. Understanding that activating or stabilizing a protein in some cases is effective against cancer opens up new avenues for brand new oncological drugs in addition to the repurposed treatments.
Overall, this repurposing study suggests an overall method to accelerate the development of new drugs to treat cancer. By finding which mechanisms work against cancer in other drugs, scientists will be able to create brand new drugs and push existing drugs into trials for effectiveness much more quickly. While this study did not produce any immediate cancer treatments, the information produced will help scientists push cancer research forward.