Companies Rush to Develop ‘Utterly Transformative’ Gene Therapies

Companies Rush to Develop ‘Utterly Transformative’ Gene Therapies
In the next year or so, he said, that approach will also be studied in both children
and adults who have acute myeloid leukemia, which he described as a “tough disease.”
Researchers at the University of Texas MD Anderson Cancer Center in Houston are trying a completely different approach to engineering cells, one
that they hope might eventually yield an “off the shelf” treatment that would not have to be tailored to each individual patient and that might be less expensive.
“This has been utterly transformative in blood cancers,” said Dr. Stephan Grupp, director of the cancer immunotherapy program at the
Children’s Hospital of Philadelphia, a professor of pediatrics at the University of Pennsylvania and a leader of major studies.
The new leukemia treatment involves removing millions of white blood cells called T cells — often referred to as the soldiers of the immune system — from the patient’s bloodstream, genetically engineering them to recognize
and kill cancer, multiplying them and then infusing them back into the patient.
There will be many such studies, Dr. Grupp predicted, but, he said, “It’s early days.”
The T cells in the Novartis products, and in the earliest ones its competitors are developing, have been engineered to seek and destroy cells
that display on their surfaces a protein called CD19 — a characteristic of many leukemias and lymphomas.
Another target is being studied, and Dr. Grupp said the next step, which he called
“superimportant,” would be to attack two cellular targets in the same patient.