Preventing Human Cancers

Recent literature seems to have converged on what may prove an unexpected alternative to chemotherapy – the pharmacological prevention of cancer onset.

Rapamycin is an mTORC1 inhibitor initially isolated from soils in the island, Rapa Nui. 

As chemotherapy, rapamycin did not prove overly effective. 

By contrast, rapamycin may have singular qualities as a prophylactic, counteracting cancer risk in predisposed individuals. 

Our Mission: prevent spontaneous tumorigenesis in children with Li Fraumeni syndrome (LFS).

To realize the potential of mTORC1 inhibitors in cancer prevention, we face two practical challenges:

1)

Avoiding the adverse side effects of mTORC1 inhibition

Chronic, long term, rapamycin treatment regimens may associate with adverse side effec ts, especially in pediatric patients.   

2)

The need for patient specific biomarkers of cancer risk

Clinical trials aimed at prophylactic influence may require many years of follow-up. Using dermal fibroblasts, we identify patient-specific biomarkers of drug response and cancer risk.

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