The NYU Grossman School of Medicine’s working group on Pediatric Gene Therapy and Medical Ethics (PGTME) formed in the fall of 2019 to address and propose recommendations to issues involving gene-based therapies in pediatric populations, including research activities.

“Our mission is to advance research policy, education, and medical ethics,” says Lesha D. Shah, MD, assistant professor of psychiatry, and medical director, Child, Adolescent and Family Services, at Icahn School of Medicine at Mount Sinai in New York City.

The working group’s members include physicians, medical ethics experts, patient advocates, researchers, pediatric disease authorities, and public health and ethics professors. “We’ve assembled a group of quite diverse expertise, and there is no other group like it,” Shah says. “We’re working with every stakeholder community.” (For more information, visit:

The working group received funding for three years. It will hold a conference in the fall, where the group might produce some work products, but there is no deadline for when guidance and recommendations will be published, she notes.

“We want to understand pediatric gene therapy challenges and evolving ethical challenges,” Shah says. “The plan is to have some kind of consensus guidelines in the long-term.”

The group’s five priority areas are:

• Equity in trial recruitment. The group will assess and address recruitment, enrollment, and participation in gene therapy trials, and look at gene therapies, Shah says.

• Lived experience of patients, parents, and families. “The scope could be quite broad, but we wanted a qualitative, ethnographic approach to understand their experience,” she explains.

• Informed consent. This priority addresses pediatric assent and surrogate decision-making, as well as informed consent.

• Risk and benefits. “This includes how we design clinical trials and how we educate patients and parents to make informed decisions,” Shah says. “It’s how we justify individual risk vs. societal benefit.”

• Vector immunity. Vector immunity issues are important to study design and participants’ risks. “It is not well documented in any of the literature, but it is understood that with the administration of gene therapy, you have the potential for a vector immunity-related phenomenon,” Shah says. “Gene therapy is administered by means of a viral vector. Repeat administration is precluded because of the potential immune response.”

The working group holds open-ended listening sessions with patients, parents, and advocates. The goal is to learn more about their experiences, and to bring the right people to the table before the group identifies a strategic plan, Shah says.

“Pediatric patients are special and a vulnerable population, so considerations for including them in research are important,” Shah says. “Sometimes, current guidance on pediatric enrollment does not account for nuances that pediatric gene therapy would bring to life. Also, gene therapy is a brand-new technology with a lot of implications.”