Table 2 Going It Alone—Sometimes the Translational Scientist Is His or Her Own Best Ally

Daria Mochly-Rosen, PhD, Professor and Senior Associate Dean for Research and Director of SPARK, Stanford University’s Translational Research Program, is a protein chemist conducting translational research. Early in her career, she designed rational inhibitors that could turn off heart cell enzymes one at a time and discovered enzymes that could change the rate at which heart cells in culture beat. She thought this was an important finding that would be of interest to the heart research community, but when she presented her work at a scientific meeting, she found the audience to be disinterested in heart rate regulation. Clinicians, she had been told, already had ways of managing heart rate; they were concerned with problems such as cardiac ischemia.

When she brought her idea directly to industry, company after company turned her away. She later realized that there were good reasons for this: while her work was attractive from a basic research point of view, the barriers to executing it in patients were huge, and many steps had to be completed before reaching that goal. Mochly-Rosen reached out to her colleagues for assistance, but rather than finding support, she was discouraged from pursuing this line of inquiry and from working with industry. Translational research, she was told, is not intellectually challenging, worthwhile, or good for her career. “Career progress in academia is measured by how many papers are published and how much grant funding is received rather than, for example, attempting to produce a new drug,” she said.

Following the advice of a colleague, she invited into her laboratory a physician who wanted to learn basic research and from whom she could learn how to study more clinically relevant problems. Her work eventually led to the discovery of an inhibitor that when administered after a heart attack dramatically reduces heart damage by 70 percent and prevents subsequent heart failure, a finding that was demonstrated in mice, rats, guinea pigs, rabbits, and pigs. Patents were written and the results published. Yet no one was interested in her findings. Why would this not be useful in patients, she thought?

But she persevered, formed her own company, KAI Pharmaceuticals, and spent a year as its Chief Scientific Officer. She helped write an Investigational New Drug Application and launched a clinical trial. “Unlike my training in academia, where questions led to the research, in industry I learned to think about the final product and work backwards, to identify what research needs to support such a product,” said Mochly-Rosen. The process was humbling, but also gratifying. “There is nothing more rewarding than [treating] the first patient…or when the trial is finished, looking at the data. It’s really a true manifestation of what basic research should eventually lead to.”