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Nathaniel Brooks Horwitz and Nikita Shah co-founded a company to test a new pancreatic cancer drug.
Editor’s note: In this Washington Post article, Nathaniel Brooks Horwitz wrote about his experience launching biopharmaceutical company Nivien Therapeutics in order to develop and test a new drug for pancreatic cancer patients. The drug being developed did not make it to human trials, but it’s important to reinforce that patients who participate in clinical research have better outcomes. Every treatment available today was approved through a clinical trial. The Pancreatic Cancer Action Network (PanCAN) strongly recommends clinical trials at diagnosis and during every treatment decision.
In the following interview with Horwitz, we explore what drew him to research and drug development, what he learned from his experiences with Nivien and why he feels clinical trials remain a critical step to improve outcomes for patients with pancreatic cancer and other diseases.
PanCAN: Please tell us about yourself. What drew you to biomedical research?
Horwitz: Translating biomedical research into new technologies is the best way to improve human health and happiness. From my grandfather’s work as a neurosurgeon to my mother’s treatment for advanced breast cancer when I was 8, I’ve always loved applied science. Fighting disease is personal, nonpartisan and exciting.
I began studying molecular biology at my public high school in Massachusetts. I continued this pursuit with bench research as a Harvard undergraduate in labs at Boston Children’s Hospital, the Harvard Stem Cell Institute and Harvard Medical School. Then I left Harvard during my senior year to launch Nivien Therapeutics, where we developed an experimental therapy for pancreatic cancer.
I returned to Harvard to finish my degree in molecular and cellular biology after our strategy failed in preclinical trials.
I’m an Australian-American: I grew up in Sydney, Australia and in Massachusetts and Virginia — I was born in Washington, D.C. My other passions are playing the classical harp, windsurfing and writing, especially about biotech.
PanCAN: What is the biggest lesson you learned from the Nivien experience?
Horwitz: I learned about everything from the difficulties of applied science to the challenges of managing a diverse team of chemists, biologists, statisticians and computer scientists across three continents.
The most important lesson was to engage people with the disease early in the process, while avoiding the overhyping of experimental drugs that occurs too often in public relations about biotech companies.
PanCAN: Why is it important to communicate about experimental drugs and clinical trials in a careful way, avoiding sensationalism and overpromises?
Horwitz: Nineteen in 20 experimental drugs fail between first-in-human trials and approval by the FDA [U.S. Food & Drug Administration]. However, many of the drugs that succeed improve thousands or even millions of lives.
Horwitz learned the importance of engaging people with a disease early in the process of developing a new drug.
Researchers and entrepreneurs should be clear about what the tech could do if it works, but honest about the odds.
When we were working on our experimental therapy at Nivien, we were contacted by several people with pancreatic cancer, or by their friends and family. Success in this disease is so rare that people look for anything that could help, even if the tech isn’t ready.
That means that we, as applied scientists, must be exceedingly careful to express reality without diminishing hope. As I wrote in The Washington Post, patients deserve honesty, employees deserve transparency, and investors deserve a fair bet.
Then, when the science works out, the result can be extraordinary.
PanCAN: Why are clinical trials important?
Horwitz: Clinical trials are the scientific method applied to human health. Clinical trials are our most powerful tool for discovering the truth about potential medicines.
Before clinical trials, we had no idea which drugs or devices were safe or effective. While there were fragmented attempts by doctors as early as 1820, the FDA did not require drugs to be proven safe until the Federal Food, Drug and Cosmetic Act of 1938. Proving effectiveness wasn’t required until 1962.
We still need more of the rigorous studies that generate useful answers, but we are already much better off under the current paradigm — of clinical trials for both safety and efficacy — than we were before.
Clinical trials also provide people with a disease the opportunity to test the latest applied science. New technologies often do not work any better than current treatments; they can even be harmful. However, many people benefit from early access to otherwise-unavailable treatments.
Each person, with their doctor and their family, needs to make a unique risk-benefit judgement based on their values. That is why it is so important for companies, journalists and investors to balance the promise of new tech against the possibility of failure: so that the people most affected by a disease can make the best call for themselves and their family.
It is the courage and the bravery of the people who enroll in clinical trials that drives forth medical science. Without these heroes, we would be cast back into the ignorance of early medicine.
