"Our results serve as another important milestone in demonstrating the potential of this treatment for patients who have no other therapeutic options," said study author Stephan A. Grupp, MD, PhD, of the Children's Hospital of Philadelphia and a professor of Pediatrics at the Perelman School of Medicine. "These data also demonstrate that these engineered 'hunter' cells greatly expand and then persist in patients, allowing for long-term disease control. We are looking forward to testing these cells in upcoming multicenter pediatric and adult trials."
During the pilot study for CLL, patients received a wide range of cell doses, but the Penn team saw no relationship between the number of cells infused and the responses or toxicities associated with the therapy. To refine the treatment approach, the Penn team launched a randomized Phase II study comparing two different doses, each of which is given as a single outpatient infusion. So far, however, the team has again seen no difference in which amount of cells is more effective or associated with greater toxicities.
Tests Pinpoint Cause, Management Technique for Side Effects
In the trials for both CLL and ALL, all responding patients experienced a cytokine release syndrome that the researchers now know marks the process of the engineered cells multiplying and attacking tumor cells in the body. During this time, patients typically experience varying degrees of flu-like symptoms, with high fevers, nausea, muscle pain, and in some cases, low blood pressure and breathing difficulties. The team has learned this reaction can be managed, if necessary, using tocilizumab, an immunosuppressant drug which tamps down
|Contact: Holly Auer|
University of Pennsylvania School of Medicine