How Gene and Cell Therapy Is Developing in Dermatology

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Peter Marinkovich, MD, reflects on the significant growth in advanced therapy development, particularly in rare skin diseases.

The introduction of gene and cell therapies like beremagene geperpavec (B-VEC; VYJUVEK) and prademagene zamikeracel (pz-cel) to the treatment severe dystrophic epidermolysis bullosa (DEB) not only raises the bar for individual patient outcomes; it offers an opportunity to reengage the entire dermatology field on the potential of advanced drug classes for their difficult-to-treat patients.

In the final segment of an interview with HCPLive during the Society for Pediatric Dermatology (SPD) 2024 Pre-AAD Meeting in San Diego, CA, pz-cel investigator Peter Marinkovich, MD, director of the Stanford Bullous Disease and Psoriasis Clinics, reflected on the substantial strides made in gene and cell therapy application and research in his field.

It helps that dermatology is a “field that lends itself to research because the skin is so accessible,” Marinkovich pointed. Even so, it’s been a reward for him and his peers to see the specialty’s highly active research channels more greatly embrace and apply advanced therapies to their work.

“It used to be that we were talking about gene therapy at the dermatology research and investigational meetings, as sort of a research tool,” Marinkovich said. “But it’s so exciting to see now the integration of gene therapy into dermatology, to help your standard clinicians whose main interest is helping EB patients in their clinical care—being able to give them another tool in their armamentarium to heal wounds.”

Regarding the next breakthroughs in dermatologic gene therapy clinical research, Marinkovich said administration method is a hot topic: later-stage analyses into dosing through the eye or musical surfaces of patients with EB has been gaining steam. He also sees a greater purpose for the gene and cell therapies that have already reached these real-world patients.

“I think that EB is going to be a bit of a prototype for other indications that which these can be used,” he said. “For example, I know that ichthyosis is being treated with this same topical gene therapy approach—using genes that help to reverse the skin barrier defects these patients suffer from.”

Marinkovich also sees opportunities to streamline other modalities of care now that gene therapies are reaching the market. He advocated for free genetic testing systems for patients, as well as communication with frontline clinicians like primary care physicians regarding the clinical benefit—and very practical feasibility—of these new agents.

Expanding the involved care team would be of particular benefit for patients who had not just limited treatment options for EB, but the inconvenience of visiting specialists for their care in most cases.

“A lot of general dermatologists and pediatricians have shied away from working with EB patients because they’re so complex and there’s not a lot that they can do. Now there is something they can do,” Marinkovich said. “I think we’re going to a time now where, there’s still always going to be a need for tertiary centers in EB, but the local caregivers are going to be assuming a more important role in the management of these patients."

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