Gene therapy cures

Jim Human Photography LLC

From left: Gero Hütter, MD, Carl H. June, MD, Luigi Naldini, MD, PhD, Nathalie Cartier-Lecave, MD, Andrew M. Davidoff, MD, and David T. Curiel, MD, PhD

BY Cynthia Marich

Washington University School of Medicine and the Biologic Therapeutics Center (BTC) recently hosted the  William H. Danforth Scientific Symposium to focus on successful gene therapy treatment and highlight the best bench-to-bed efforts in this regard.

The symposium, titled Gene Therapy Cures, kicked off with an overview of gene therapy by David T. Curiel, MD, PhD, director of the Biologic Therapeutics Center.

“In most basic terms, gene therapy is using genes as medicine,” says Curiel, who also is a distinguished professor of radiation oncology and a professor of medicine and of obstetrics and gynecology. “From the tragedy of Jesse Gelsinger to the successful treatment of Tim Brown, this science has matured is now ready for prime time.”

One of the most emotional moments of the day came when Gero Hütter, MD, head of the Stem Cell Unit at Heidelberg University, welcomed patient Timothy Brown to the stage. Brown is known as “The Berlin Patient,” as he was treated by Hütter in his Berlin facility and is currently the first man cured of HIV. 

“In most basic terms, gene therapy is using genes as medicine.”
—David T. Curiel, MD, PhD

Brown started the Timothy Ray Brown Foundation and was recently featured in Google’s “How the World Searched 2012,” a compendium of search trends, which landed him among Austrian skydiver and world-record holder Felix Baumgartner, protests in Greece and Syria, the 2012 Summer Olympics and other important events around the globe.

“The rate of progress in science is amazing,” says Chancellor Emeritus William H. Danforth, MD, one of the many honored guests at the event.

Including Hütter, the day-long seminar featured five speakers whose research in the area of gene therapy has shown clinical efficacy in human trials. Speakers were invited from institutions worldwide and included:

• Nathalie Cartier-Lacave, MD, Associate Professor, University of Paris Descartes, Director of Research, INSERM, French National Institute of Health and Medical Research, with a research focus on Gene Therapy for Adrenoleukodystrophy;

• Andrew M. Davidoff, MD, Chair, Department of Surgery, Division Chief, General Pediatric Surgery, Director, Surgical Research, St. Jude Children’s Research Hospital, with a research focus on AAV-mediated Gene Therapy for Hemophilia B;

• Gero Hütter, MD, Head, Stem Cell Unit, Heidelberg University, with a research focus on HIV Gene Therapy;

• Carl H. June, MD, Professor of Pathology and Laboratory Medicine, University of Pennsylvania, with a research focus on engineered T Cell Therapy for Cancer and HIV; and

• Luigi Naldini, MD, PhD, Director, HSR-TIGET, San Raffaele Telethon Institute for Gene Therapy, with a research focus on Hematopoietic Stem Cell Therapy.

“Dr. Curiel is an international expert in gene therapy and therefore attracts the leading gene therapy researchers to speak at Washington University,” says Dennis E. Hallahan, MD, the Elizabeth H. and James S. McDonnell III Distinguished Professor and head of the Department of Radiation Oncology. “We look forward to continued development of gene therapy at Washington University that will lead to additional cures of human disease.”

Curiel plans to continue to showcase important breakthrough science with the Biologic Therapeutics Center's monthly Translational Research Seminar Series, now entering its third year.