Why Penn research powers many FDA-approved treatments

Since 2017, the FDA approved more than two dozen new therapies with roots at Penn Medicine—almost half of which are first-in-class for their indications.

The news reached Abramson Cancer Center (ACC) director Robert Vonderheide at 11 a.m. on August 30, 2017. It was official: That morning, the U.S. Food and Drug Administration (FDA) approved a Penn Medicine-developed personalized cellular immune therapy.

Six hours later, Vonderheide was standing atop a coffee counter in the atrium of the Perelman Center for Advanced Medicine, addressing hundreds of jubilant faculty and staff members who had gathered for a now-iconic “flash mob” celebration of the milestone. Carl June, and a team of scientists, physicians, and other dedicated staff had together turned a dream of using patients’ own immune cells to treat their cancer into a reality.

Aerial image of Penn Medicine staff in the atrium celebrating.
On August 30, 2017, faculty and staff at the Perelman Center for Advanced Medicine gathered for a “flash mob” celebration of the the FDA approval of a Penn Medicine-developed personalized cellular immune therapy. (Image: Courtesy of Penn Medicine News)

Vonderheide called the discovery “a 20-year overnight sensation.” It would be a defining moment for Penn’s identity as a place that incubates and brings to life some of the most transformative modern medical advancements.

The FDA approval of chimeric antigen receptor (CAR) T cell therapy didn’t happen at Penn Medicine by chance. Nor did the others that followed. Since that day, other medical innovations and research that had been underway at Penn Medicine directly influenced at least two dozen more FDA approvals granted to cancer drugs and other medical technologies—giving the stamp of safety and effectiveness needed for these treatments to be widely available to patients outside of clinical trials. Eleven of the medical advancements documented to date represent the first FDA approvals in their classes: eight under cancer, two under gene therapy, and the revolutionary mRNA technology that powers COVID-19 vaccines around the world.

Penn Medicine faculty may lead this crucial work at different points across the continuum of discovery, from conducting pivotal discovery science in the lab which leads to licensing of compounds and technologies by commercial partners, to leading clinical trials of new therapies initially developed outside of Penn.

The list of Penn-linked FDA approvals is growing with each passing year, and for good reason. Science has shifted toward more discoveries based on the underlying mechanism of disease, especially in new cancer drug discovery. Through a particularly robust clinical trial and commercialization infrastructure, Penn has further worked to smooth every part of the path from idea to implementation. And to ensure that state-of-the-art treatments reach the patients who need them, Penn Medicine teams have also kept focus on equity and access during clinical trials and after therapies are approved.

Drew Weissman and Katalin Karikó in lab coats writing on a clear glass wall.
Drew Weissman and Katalin Karikó made crucial discoveries leading to the first FDA approvals for mRNA vaccines. (Image: Courtesy of Penn Medicine News)

The most ubiquitous novel advance tied to Penn Medicine happened in August 2021, when the FDA gave its first full approval to an mRNA-based COVID-19 vaccine, which uses technology discovered more than 15 years earlier by longtime research partners Drew Weissman, the Roberts Family Professor in Vaccine Research, and Katalin Karikó, an adjunct professor of neurosurgery.

Recruiting faculty who dream big is essential to drug development, but there are other reasons Penn Medicine has connections to so many cutting-edge therapies. Among the top, says Vonderheide, is a strategic emphasis on translational research and eliminating barriers between laboratories and the clinic.

“Even our building design reflects that value. If you stand in the lobby of the Perelman Center for Advanced Medicine, you're within 100 yards of the labs where discoveries are being made, the clinics where clinicians are designing clinical trials and patients are getting therapies, and the offices where executives are working out financial models,” he says. “We’re a unified, integrated system committed to making science real for patients.”

Read the full story in Penn Medicine Magazine.