Medicosnext
Based on early data from the first-ever clinical trial in humans in the US, genetically editing a cancer patient’s immune cells using CRISPR/Cas9 technology and infusing them back into the patient seems to be safe and feasible. Three participants in the trial (two with multiple myeloma and one with sarcoma) have been infused so far, and it has been observed that the edited T cells expand and bind to their tumor target with no serious side effects. Researchers from the Abramson Cancer Center of the University of Pennsylvania are conducting the ongoing study in collaboration with the Parker Institute for Cancer Immunotherapy (PICI) and Tmunity Therapeutics. Early data from the trial, which aims to know whether T cells can be edited in this way, whether the resulting T cells are functional, and whether they can be safely infused into other patients, suggests that the answer may be yes in all three cases. Th e researchers say that the feasibility and safety demonstrated so far could show that the approach may be applicable across many different areas of gene therapy research. Sean Parker, the founder and chairman of PICI, said that the findings were the first step to determine if this breakthrough technology could show how cancer patients and perhaps other deadly diseases are treated in the future, adding, “CRISPR editing could be the next generation of T cell therapy.” The approach is to engineer patients’ own immune cells to fight their cancer, whereby CRISPR/ Cas9 editing is done to remove three genes from a T cell. First, two edits are done to remove a T cell’s natural receptors to ensure that the immune cells bind to the right section of the cancer cells, and then another edit is done to remove PD-1, which sometimes blocks T cells from doing their work. The CRISPR-edited T cells are not active on their own, requiring the presence of an antigen (HLA-A201). Principal investigator Edward A. Stadtmauer, MD, section chief of Hematologic Malignancies at Penn, presented the findings at the 61st American Society of Hematology Annual Meeting and Exposition in Orlando, USA in December 2019. The researchers are continuing to analyze patient samples and say they need longer clinical follow-up to study this new approach.
Ref: New Scientist, Fen 6, 2020,
