The Story of Gleevec

Recently (2001), the FDA approved of a new drug that is known as Gleevec. Gleevec is a new wonder drug that is currently being used to fight several specific types of cancer, chronic myeloid leukemia (CML) and gastrointestinal stromal tumor (GIST), a rare solid tumor. The reason for this drug's success, in part, is due to research that was conducted 40 years ago in Pennsylvannia. Scientists at the university noticed "Chromosomal abnormalities in the blood of CML patients." (FROM ARTICLE) They found that one chromosome in the blood cells of CML patients was noticeably shorter than the rest. "The stubby chromosome was nicknamed "the Philadelphia chromosome" and marked the first time that a chromosomal defect was linked to cancer." (FROM ARTICLE) For thirteen years, the ground breaking discovery had to wait for technology to catch up in order to probe the mystery further. But in 1973, the research of this chromosomal abnormality made another huge break through. At the University of Chicago, a researcher discovered the "missing" chromosomal chunk attached to the end of another chromosome. y the 1980s, scientists were able to use genetic mapping to show that the two ends of the broken chromosomes produced a cancer-causing protein, known as Bcr-Abl. In 1986 and 1987, researchers writing in Science identified the protein as a tyrosine kinase, a kind of enzyme that, among other things, helps regulate cell growth and division." (FROM ARTICLE) These scientists also determined that this protein Bcr-Abl acts by disabling the signal that tells the body to stop producing white blood cells(WBC). As a result, this protein (Bcr-Abl) causes WBC levels in the blood to be "10 to 25 times that of normal blood-around 4,000 to 10,000 per equal volumes." (FROM ARTICLE) After the identification of this protein, researchers began testing to find a molecule that would target this protein while leaving similar proteins in the cell completely alone. After two years of trials, the molecule called Gleevec was developed. The next step in the devolopment of this drug was the research required to make the molecule safe and effective for oral consumption. Once Gleevec became availabe in pill form, the drug was made available for clinical tests. The test results were spectacular, the daily pill caused a "significant reduction in the number of WBCs and a reduction or disappearance in the number of cells containing cancer-triggering chromosome." (FROM ARTICLE) Even better than the amazing improvement shown in the tests was the fact that this drug caused atmost, only minor side effects. Gleevec was approved by the FDA in 2001 as a treatment for people with the Philadelphia chromosome. Since its release on the open market, it has been hailed as "a major breakthrough by officials and scientists. Secretary of Health and Human Services Tommy Thompson called it "the wave of the future." Dr. Harmon Eyre of the American Cancer Society called it "a huge breakthrough…a great drug, a great new discovery.'" Gleevec is an amazing drug, and it caught my attention because of the perseverence and hard work that it took to bring all the information, testing, and research together into a form that is currently saving the quality, if not the lives of people who were previously suffering from CML. Another fact about this drug that is absolutely incredible is the fact that if this technique works for this particular cancer, than its potential is almost limitless as a treatment for other similar and maybe not so similar cancers. If the identity of the "bad" protein/enzyme can be determined, then a molecule similiar to Gleevec could theoretically be used as a cure for this cancer as well. Gleevec is an outstanding drug, and testament to human perseverence and diligent work.

http://www.innovation.org/index.cfm/StoriesofInnovation/InnovatorStories/The_Story_of_Gleevec