Duke researcher Louise Markert receives FDA approval for groundbreaking treatment of pediatric immune disorder

Duke alumna, scientist and pediatric immunologist Mary Louise Markert has devoted her career to developing a cure for a fatal childhood immune disease. After 30 years of research, the Food and Drug Administration approved the immunotherapy it helped develop on Oct. 8.

The breakthrough therapy is the only available treatment for pediatric patients with congenital athymia. Congenital athymia is a deadly disease in which patients are born without a functioning thymus, an essential organ where a type of immune cells develop. The immunotherapy involves culturing thymic tissue and implanting the tissue in infants with athymia.

One day in 1991, during her call, she received a call from a doctor asking if she wanted to treat a child with DiGeorge syndrome, a specific and severe type of congenital athymia.

She’s done research on mice and gene therapy before, but since that accidental phone call, she’s wondered if thymic tissue could be cultured to develop a thymus gland, and recalled previous studies of immunotherapy for pediatric patients without the thymus gland.

“After I answered the phone, there were two people I thought I should talk to: Rebecca Buckley [James Buren Sidbury distinguished professor of pediatrics], who is world renowned in treating immune-deficient children who have severe combined immune deficiency, and Barton Haynes [Frederic M. Haynes professor of medicine]who studied the thymus,” Markert recalled.

With the help of such renowned and experienced scientists in the field, she began researching cultured thymic tissue implants (CTTI).

Since 1991, Markert and colleagues have conducted many clinical studies on the effectiveness of CTTI, and the results show that the survival rate of patients enrolled in the study is significantly higher.

“Pediatricians in almost every specialty have contributed to the care of these babies with congenital athymia,” Markert said. She highlighted the team effort, including Joanne Kurtzberg, Jerome S. Harris distinguished professor of pediatrics. Markert also collaborated with researchers from the Duke Pediatrics Division of Pediatric Transplant and Cellular Therapy (PTCT) and the Divisions of Pediatric Allergy and Immunology.

“I’m so focused on this group of pediatric patients with congenital athymia, and I’ve been lucky enough to be able to do that,” Markert said.

The drug was also developed using Enzyvant Therapeutics.

Markert began her journey at Duke as a student in the MD-Ph.D. She received her doctorate in microbiology in 1981 and a medical degree in 1982. She completed fellowships in pediatrics and allergy and immunology in 1984 and 1987, respectively. Markert is currently a professor emeritus of pediatrics and professor of immunology at Duke, as well as a member of the Duke Cancer Institute .

After seeing her research come to fruition, Markert retired on October 15. Her pioneering efforts in pediatric immunology will continue to help children and families for many years to come.

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