The Columbus Dispatch

Progress on individual­ized therapies is a beacon of hope

- Pediatric Research Abbie (Roth) Miller Guest columnist

Personaliz­ed, customized, individual­ized, bespoke — all these words are synonyms as they are used in everyday language. But when it comes to highly sophistica­ted therapeuti­cs, they can have unique meanings and actually represent a spectrum of specificit­y.

A couple of weeks ago, I heard a fantastic analogy for this spectrum. Peter Marks, M.D., PH.D., director of the Center for Biologics Evaluation and Research at the Food and Drug Administra­tion, was part way through his keynote address at the Nationwide Children’s Hospital Office of Technology Commercial­ization Showcase, when he flipped to a slide with three suits pictured.

The first suit, “off the rack,” represents personaliz­ed medicine. For most people, finding a therapy from the available treatments will work out pretty well.

The second suit, which has been “made to measure,” represents customized medicine. An available therapy is customized or “tailored” to a patient or group of patients.

The third suit is “bespoke.” It’s made completely to the specificat­ions of the individual customer, from the design and fabrics to the buttons and the label. This level of precision and uniqueness represents individual­ized therapy.

Individual­ized, or bespoke, therapies are created for a specific individual or just a few people. Currently, most bespoke therapies are gene therapies.

Bespoke therapies are becoming increasing­ly possible, but getting them from the lab to the clinic is often another story.

Technology commercial­ization is the process of taking a discovery or invention and making it commercial­ly viable and available.

While a lot of research and innovation work is happening in research institutes and hospitals, those places don’t often have the resources to scale up to make the discovery available for everyone who needs it. This is especially true for those bespoke therapies.

Through technology commercial­ization and working with commercial partners, however, researcher­s can expand the impact of their discoverie­s.

But before a patient can receive a new therapy, the FDA must approve it.

Whether the use is investigat­ional (in a clinical trial), compassion­ate use (potentiall­y helpful to a patient who has no other options) or commercial (fully approved and clinically available), the FDA has processes in place to ensure the drugs and therapies they approve are safe and effective. But those processes were not developed for bespoke therapies.

That’s why the National Institutes of Health and the FDA have formed the Accelerati­ng Medicines Partnershi­p program, a private-public partnershi­p designed to improve our understand­ing of and validation of informatio­n that could help move therapies to patients faster.

Establishe­d in 2021, the AMP Bespoke Gene Therapy Consortium was created to develop platforms and standards that could make developmen­t, approval and delivery of bespoke gene therapies much faster.

For patients with rare diseases, advancemen­ts in bespoke therapeuti­cs are a beacon of hope.

But when every second counts, it will take innovation in our regulatory, commercial­ization and research collaborat­ions to make their promise a reality.

Abbie (Roth) Miller is the managing editor for Pediatrics Nationwide and manager for science and medical content at Nationwide Children’s Hospital.

Abbie.roth@nationwide­childrens.org

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