Sukumar Nagendran, MD ’94: A Long Journey to Gene Therapy Pioneer
Robert Wood Johnson Medicine (A publication for alumni and friends of Rutgers Robert Wood Johnson Medical School)
Tirty-two years after leaving civil war–torn Sri Lanka to pick up his studies at Rutgers University, followed by Rutgers Robert Wood Johnson Medical School, Sukumar Nagendran, MD ’94, still marvels at where that journey has led him. He has worn the hats of private physician, drug developer, biotech executive, and globally recognized expert and pioneer in the burgeoning field of gene therapy. In the latter role, his multifaceted career reached new heights in May 2018 when the U.S. Food and Drug Administration approved a gene transfer treatment he helped develop, which is transforming the lives of hundreds of children stricken with spinal muscular atrophy (SMA).
Serving as his lodestar throughout this voyage was a thirst to use his acquired knowledge and training including a three-year residency in internal medicine at the Mayo Clinic School of Graduate Medical Education to touch as many lives as possible. “One of the reasons I left practice and went into the field of pharmaceuticals was the realization I wanted to have an impact on broad patient populations,” says Dr. Nagendran, who graduated first in the Class of 1994 at Robert Wood Johnson Medical School in Camden. “And the way to do that was through the pharma-biotechdiagnostics sector.”
Blessed with a disciplined mind that adapted readily to the rigorous pace and pressures of medical school, he also had the savvy to build on each opportunity his medical training and experience afforded. His third and fourth years at medical school, for example, brought him to Cooper University Hospital in Camden, where he came face-to-face with the kind of trauma and diseases often seen in developing countries. That paved the way for the Mayo Clinic, where he was drawn to treating patients with rare diseases and got the chance to interact with public health experts around the world.
That chapter folded into his first job, in Phoenix, at a high-volume private practice in diabetes and cardiovascular diseases, which set the stage eight years later for his jump to pharmaceuticals when Pfizer offered him a position as director of its U.S. diabetes and metabolism team. From that leadership post flowed other pivotal roles with Novartis, Daiichi Sankyo, Reata Pharmaceuticals, and Quest Diagnostics, where he was instrumental in building the medical affairs department of one of the world’s largest diagnostic labs.
In hindsight, those jobs might be seen as dress rehearsals for what happened in the late summer of 2015. Dr. Nagendran met in Ohio with the founders of a start-up company, AveXis, who were looking for a chief medical officer. They showed him laboratory data for a gene therapy they had developed for spinal muscular atrophy (SMA), a hereditary genetic disorder afflicting one of every 10,000 children born in the United States each year. SMA is caused by a mutation in the survival motor neuron gene 1 (SMN1), which encodes for a protein that motor neurons—the nerve cells that control muscle contraction—need to survive. Symptoms can appear within the first six months of life with SMA1, the most common and severe form of the disease. These include increasing difficulty in breathing, swallowing, speaking, and moving as the brain stops sending messages that control muscle movement. Left untreated, the muscles progressively deteriorate, leading to paralysis and death, which can occur within two years.
Dr. Nagendran remembers his reaction to the animal and early clinical data AveXis showed him: “It was incredible. I told them, ‘I’m in,’ because I think you have a cure.” So began his new role with the tiny biotech, where Dr. Nagendran was instrumental in clinical development of a one-time, intravenous therapy that delivered through a vector a fully functioning copy of the SMN1 gene to replace the mutated version. In a clinical trial, reported in the New England Journal of Medicine in November 2017, all 15 children who received a single dose of the treatment were alive and eventfree at 20 months of age, compared to a survival rate of 8 percent in a historical cohort. “This was truly a miracle for the patients and their families,” acknowledges Dr. Nagendran, who coauthored the report. He sees the novel gene therapy, marketed by Novartis under the name Zolgensma, as a cure for SMA if the disease can be detected through effective clinical screening in its presymptomatic stage—more common now that most states have newborn screening.
Following the acquisition of AveXis by Novartis in mid-2018, Dr. Nagendran left the company and stepped into an expansive new role: a much-in-demand international expert in gene transfer therapy with impressive bona fides not just in clinical development but on the business side of taking a company public and telling its remarkable story to a global audience of investors and the public. “It’s an interesting time to be using my experience in pharma and biotech to guide other companies as they look to develop therapies for difficult diseases like Duchenne muscular dystrophy and other rare disorders,” he says.
That’s not the only way Dr. Nagendran is applying his resources. He and his wife, Christine Ann, continue to give back through the Nagendran Scholarship for International Studies at Robert Wood Johnson Medical School, which allows about five students a year to experience medicine in developing countries. “I remember as a medical student in Sri Lanka, we had one needle for bone marrow aspiration in an entire 500-bed hospital,” he recounts. “The goal of our scholarship is to give medical students a better appreciation for the many resources we have here compared to other countries, and to use them to deliver the best care possible to patients.”
