Cancer treatment through hTERT inhibition

Title: Cancer Treatment Through hTERT Inhibition

Invention: The invention is a small molecule that reduces human telomerase reverse transcriptase (hTERT), which is highly activated in cancer cells.

Background: In 2010, approximately 73,599 U.S. patients were diagnosed with melanoma with 8,700 of those incidences resulting in death. A hallmark of cancer, and melanoma specifically, overexpression of hTERT results in cell immortalization. These types of tumors have transcription-activating mutations in the hTERT promoter region because of a pair of G-quadruplexes. The small molecule binds selectively to the G-quadruplex in the hTERT promoter mutant, reducing transcription activity. This technology has the potential to fill an unmet need in a market where only two effective drugs, with a host of side effects, are available for treating melanoma.

Applications:

• An anticancer drug for a variety of cancers with an overexpression of hTERT, especially melanoma and glioma

Advantages:

• Direct inhibition of the hTERT promoter produces apoptosis within 2-5 days versus targeting the telomeric structure
• The small molecule has greater cellular selectivity over BRACO-19, a similar agent in mechanism of action
• Poised to fill the market demand for improved survival rate and reduced drug toxicity