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Clinical Research Staff Shape the Success of Gene Therapy Trials

Published on May 21, 2024 in Cornerstone Blog · Last updated 1 month 2 weeks ago
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Sarah McCague

Sarah McCague, MS, is the administrative manager of CHOP’s Clinical In Vivo Gene Therapy group (CIGT), which streamlines operations for in vivo gene therapy trials — currently more than 32 — taking place at CHOP.

By Lauren Ingeno

The Clinical In Vivo Gene Therapy group (CIGT) at Children's Hospital of Philadelphia is a crucial connector for 32 active in vivo gene therapy trials taking place at the hospital across seven clinical divisions.

Behind the scenes, it is the CIGT's dedicated staff of six clinical research coordinators, two clinical research assistants, a project manager, and CIGT Administrative Manager Sarah McCague, MS, who play a critical role in the success of these trials. They require specialized knowledge about the science behind gene therapy, as well as the compliance, safety, and financial aspects of these complex studies.

"Having a core of coordinators who are specifically trained in gene therapy helps to fill in knowledge gaps and break down walls between investigators who are working in so many different clinical specialties," said McCague, who founded the CIGT alongside CHOP hematologist Lindsey George, MD, in 2021.

To centralize in vivo gene therapy research, the CIGT not only facilitates gene therapy trials, but also offers investigators a multidisciplinary clinical consult service — the so-called "Gene Team" — develops protocols for studies initiated by investigators, holds quarterly investigator meetings, and manages a student program to train the next generation of gene therapy experts.

In her role, McCague manages the CIGT's team of coordinators and leads the group's daily operations. It's a position she never could have imagined for herself a decade ago.

With no medical research experience, McCague joined CHOP as a clinical research coordinator in 2011. She was transitioning out of a career in the arts and eager to make a difference in a new sector.

"Clinical research is an entire field of STEM that you can be involved in without an MD, a PhD, or sometimes without a science background at all," McCague said.

CHOP assigned McCague to a clinical trial that was testing a new gene therapy to treat an inherited form of blindness. That experimental treatment would later become voretigene neparvovec-rzyl (Luxturna®, Spark Therapeutics) — the first gene therapy approved by the U.S. Food and Drug Administration for the treatment of a genetic disease.

The job of a CRC is especially crucial for gene therapy trials, in which patients receive a one-time, irreversible treatment. Coordinators are often responsible for educating families about the clinical trial protocol, the investigative therapy's potential side effects, and how the new approach differs from current standard treatment.

"You need to make sure families understand the risks involved, not just medically, but also emotionally," she said.

During the voretigene neparvovec-rzyl trials, patients were randomly assigned to the therapeutic group or the control group. After a year, patients who had not received the gene therapy would get the injection.

"At the beginning of the trial, we had to explain to families that they'd be going through this very intense process, and their child may not even receive the therapy for a full year," McCague said. "In that time, their vision could deteriorate even further."

As part of the trial, McCague helped to design an obstacle course that patients completed before and after receiving the therapy to test their vision. Despite the weight of responsibility that comes with managing a gene therapy clinical trial, having a front-row seat to life-changing moments makes her job worth it.

"Someone who couldn't complete the course previously, all of a sudden, they can see," McCague said. "And the parents, who I had been working with for months, are there in the room crying — it's very emotional and rewarding."

Working on the trial — as well as in the lab of Dr. George, who is developing gene therapies to treat hemophilia and other blood disorders — gave McCague the opportunity to not only manage daily trial operations, but also to help develop the framework for how to rigorously test a gene therapy, a field of investigation that was still in its infancy.

In 2019, she and Dr. George saw a need to streamline and connect the in vivo clinical trials happening at CHOP, and so, they began developing plans for the CIGT. McCague is proud of the unique role that her group plays in helping investigators navigate the complex safety, regulatory, and clinical care considerations of developing gene therapies that are changing so many patients' lives.

"I hope more people learn about clinical research as a career," McCague said. "My background as a CRC has allowed me the opportunity to help create CIGT and establish a fulfilling career in gene therapy."