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Conquest - Fixing Broken Genes

Making the Connection 2001-2008

Fixing Broken Genes

Date: April 20, 2010

A look at Brain Cancer and Leukemia


Brain Cancer RESEARCH
Brain cancer is among the deadliest of all cancers. The most common form of brain cancer is glioblastoma multiforme and the most lethal, with most patients surviving just 14 months from diagnosis.

TRANSLATION
Johns Hopkins Kimmel Cancer Center researchers linked the MGMT gene to glioblastoma multiforme. The investigators discovered the gene was altered by a cellular process known as hypermethylation. They discovered that the gene alteration makes
brain cancer cells more responsive to anticancer drugs known as alkylating agents. The technology is licensed by OnoMethylome Sciences.

APPLICATION

OnoMethylome Sciences gives a commercial license to LabCorp for a MGMT methylation test available to patients throughout North America.

Leukemia RESEARCH
Myelodysplastic syndrome is a preleukemic, potentially deadly disease not well understood outside of the academic research hospital setting.

TRANSLATION
Pioneering laboratory research by CRF investigators Stephen Baylin, M.D., and James Herman, M.D., revealed alterations in a cellular process known as DNA methylation. Key tumor suppressor genes were found to be turned off by too much DNA
methylation. This process has been linked to MDS and a of cancers. The work was honored in 2004 with the most outstanding research award in the National Cancer Institute's SPORE (Specialized Programs of Research Excellence) program.

APPLICATION
Clinical trials of the drug 5-aza-cytidine that blocks methylation and restores the function of tumor suppressor genes have led to complete remissions in up to half of MDS and leukemia patients treated. Results from multicenter trials led to the first FDA
approval of a demethylating agent.

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