Translating research into better treatment: We can boost our batting average

By Naoto Ueno M.D., Ph.D.

Each year, on the basis of new basic research discoveries and clinical observations, my research group generates many excellent hypotheses that may benefit cancer patients. Unfortunately, though, our batting average in terms of translating these hypotheses into better treatment for cancer patients is low.

In my group, around 10% to 20% of our hypotheses end up being proven. Of these proven hypotheses, 30% to 40% lead to clinical trials. Of those clinical trials, 20% to 30% lead to potential adoption of new treatment strategies in the clinic. In other words, if we define a "hit" as a finding leading to new treatment for patients with cancer and an "at bat" as testing a hypothesis, our batting average is only about 0.6% to 2.4%. I suspect that our batting average is fairly typical for cancer research groups.

Of course, I wish that my own research group were more productive and that more of our hypotheses were translatable so that we could help more cancer patients.

So how can cancer research groups improve their batting average? These are my thoughts:

1. Find smarter people to replace group leaders like myself. Maybe a good idea?
2. Collaborate with other groups of smart people. Team research and team medicine are important. This means that the skill sets needed to enhance team research and team medicine must be taught to doctors and researchers.
3. Spend more time on research and less time on writing grants. This means more financial support is needed. Unfortunately, with the current fiscal crisis, we have less money for research.
4. Test good hypotheses, not just good ideas. Ideas must be translated into testable hypotheses that can be proven true or false. If you do not form a good hypothesis, you will fail not because of ideas but because of an unclear hypothesis not being proven true or false.
5. Make researchers accountable through close monitoring by grant agencies and patient advocates. Get rid of redundancy, improve efficiency, etc.
6. Allow researchers to publish negative results even if those results are not confirmed with definitive data. Proving negative results is very difficult, but publication of negative or unclear results can help cancer researchers avoid dead-end research avenues.
7. Attract or create researchers who really care about research for patients. We have people in cancer research who do research for the sake of research.

None of these things are easy to accomplish, but patient advocates and patients can promote positive changes by raising their voices to call for better-quality research with a higher batting average so that research has a more profound impact for current and future patients.

When I was diagnosed with a malignant fibrous histiocytoma sarcoma, rare for someone my age, I was frustrated about not having sufficient data about my disease and its treatment to give me 100% peace of mind about the treatment course that I ultimately chose. Don't get me wrong -- I had excellent oncologists and oncology team, and I am very happy with them. But I was unhappy that so much research remained to be done.

Naoto Ueno is a professor in MD Anderson's Department of Breast Medical Oncology and executive director of the Morgan Welch Inflammatory Breast Cancer Research Program and Clinic.

Social Media Links for Ueno

On Facebook at ntueno, NoMoreCancer and InflammatoryBreastCancer On Twitter at @teamoncology and @InflammatoryBCa