Broad Applicability of Genetic Safety Switch for Improving Cell Therapy Outcomes
Bellicum Pharmaceuticals today announced publication in The New England Journal of Medicine of clinical results using a new therapy that demonstrated rapid and complete reversal of graft-versus-host disease The paper Inducible Apoptosis as a Safety Switch for Adoptive Cell Therapy reports on the use of Bellicums CaspaCID technology to eliminate donor T cells causing GVHD a serious and sometimes fatal complication of allogeneic hematopoietic stem cell transplantation in patients with acute leukemias. The study was conducted by researchers from the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Childrens Hospital and The Methodist Hospital using the drug AP provided by Bellicum Pharmaceuticals.
CaspaCIDe technology consists of a drug-inducible suicide gene introduced into therapeutic cells that are subsequently given to patients and a drug that the physician administers if the cells cause unacceptable toxicity triggering the suicide gene and leading to their rapid self-elimination In todays reported study T cells with the genetic safety switch were infused into patients to determine if GVHD-causing T cells could be selectively eliminated, while preserving those that may support stem cell engraftment, fight infections and destroy any remaining cancer cells.
In an accompanying NEJM editorial Michel Sadelain Center for Cell Engineering Memorial Sloan-Kettering Cancer Center wrote: An effective safety switch is needed in a rising spectrum of cell therapies in order to protect against an array of toxic effects and called the clinical results with icompelling.
The study enrolled five patients between the ages of with acute leukemia some of whom had previously relapsed. All patients underwent HSCT from high GVHD risk partially HLA-mismatched, haploidentical donors In all five transplants the CaspaCIDe suicide gene was inserted into the donor T cells which were then infused into the patient Weeks later the gene-modified donor cells were detected in the peripheral blood of all five patients functioning as expected and expanding in number over time.
