The Challenge of Translation: Reflections of a Cancer Patient-Scientist

The diversity of molecular phenotypes present in cancers provides significant scientific challenges for developing therapies in a cost effective and timely manner. In addition to repurposing across cancer types, repurposing from non-cancer indications, such as metabolic diseases, has the potential to improve patient outcomes, but comes with its own challenges.

My work with the investigational drug dichloroacetate (DCA) as a cancer therapy has highlighted how the path beyond the lab to clinical outcomes is slow, hard and complex. The mechanism of DCA is via metabolic changes, inhibiting pyruvate dehydrogenase kinases, thereby activating pyruvate dehydrogenase promoting oxidative phosphorylation over anerobic glycolysis that cancers in general prefer. Through this mechanism, DCA has shown promise in a broad range of diseases in decades of animal model and patient studies, but is yet to have FDA approval for any indication. One barrier is the lack of traditional intellectual property around this molecule and consequently lack of finances for the “standard” approach for drug development, but there are many other more subtle barriers. An alternative route for development of medicines is needed.

My experience as a breast cancer patient in 2022 has reinforced this perspective and led me to ask: Why is common breast cancer treated primarily with drugs first approved around 50 years ago? Why does it take so long for scientific innovation to reach the patient?

Here I offer some reflections on the limitations and simplicity of our scientific model systems and organisational mindsets, and the complexity of moving our science beyond the lab to have real impact.

By combining the perspectives of a scientist, patient and entrepreneur to study the repurposing ecosystem with examples such as DCA I hope to find some small ways to improve the translational process.