After laying groundwork in gene therapy for a couple of decades, we are now on the cusp of reaping benefits and are actually seeing efficacy in a large number of patients.
After laying the groundwork in gene therapy for a couple of decades, we are now on the cusp of reaping benefits and are actually seeing efficacy in a large number of patients, says George Daley, MD, PhD, director of the Stem Cell Transplantation Program at Children's Hospital Boston.
At the same time, we are riding a wave of tremendous enthusiasm around stem cell biology, he said at a Food and Drug Administration (FDA) workshop in November.
But in both cases the risks within clinical studies are very real, he noted.
"One thing to note is the protection of the rights and welfare of children in clinical studies is the shared responsibility" of FDA, clinical investigators, IRBs, and parents, said Celia Whitten, MD, PhD, director of FDA's Office of Cellular, Tissue, and Gene Therapy.
"The challenges are shared, but the perspectives are different for each group," Whitten noted.
"Ensuring the adequacy of scientific review is heightened in the area of cell and gene therapy trials for a number of reasons including the complexity of these trials and often a lack of ample data on the product prior to introducing it into children," said Whitten.
Robert Nelson, MD, PhD, pediatric ethicist in the Office of Pediatric Therapeutics in the office of the FDA commissioner, told the meeting, "Over the past 15 years, we have evolved from a view that we must protect children from research to a view that we must protect children through research."
One proposal he and others in the agency have thought about, said Nelson, is a "sliding threshold."
He indicated that would point to the question of, "At what point do you have sufficient data to think you can move forward" with a trial in children. He added, "Data [whether animal or human adult] necessary to establish sufficient prospect of direct benefit to justify the risks varies with the severity of the disease and the adequacy of alternative treatments."
FDA will release a transcript of the meeting at http://www.regulations.gov/ in upcoming weeks.