Anti-Cancer Drug Discovery & Therapy (Track)


Aung Naing

Investigational Cancer Therapeutics, MD Anderson Cancer Center, Houston, TX 77030, USA


Cancer is public enemy number one for United States and worldwide. Probability of success in cancer development can be further improved.  With tremendous outpouring of resources into drug development, we have not met expectation of our patients. For example, our FDA approved drug “gemcitabine “improved survival gain only 1.5 months.

The question is “Can molecular testing make a difference in early clinical trials?”.  While it is often claimed that we need new drugs to treat cancer, a more fundamental problem may be the way we classify cancer.  We may have excellent drugs, but they work poorly because we treat patients with the wrong cancers with them.

We at early phase clinical trial group in MD Anderson believe:

(1)  Treatment is not disease-based but target-based. Diverse cancers treated with molecular profiling.
(2)  Treatment is based on early phase clinical trial.   Correlative/translational aspects are critical.
(3)  Treatment is not conventional.  Virtually all patients are on trial. 

In our department, we have 128 clinical trials and 28 % of them are first in human. We have ability to profile molecular aberrations of tumor and able to match treatment according. Here are our findings:

(1)   In patients with 1 molecular aberration, matched therapy compared with treatment without matching was associated with: Higher CR+PR rate (27% vs. 5%, p < .0001), longer TTF (median, 5.2 vs. 2.2 months, p < .0001) and longer survival (median, 13.4 vs. 9 months, p = .017)