In a small study, researchers modified patients’ immune cells to target their particular cancer—but it only worked for a third of volunteers.
IN A NEW step for Crispr, scientists have used the gene-editing tool to make personalized modifications to cancer patients’ immune cells to supercharge them against their tumors. In a small study published today in the journal Nature, a US team showed that the approach was feasible and safe, but was successful only in a handful of patients.
Cancer arises when cells acquire genetic mutations and divide uncontrollably. Every cancer is driven by a unique set of mutations, and each person has immune cells with receptors that can recognize these mutations and differentiate cancer cells from normal ones. But patients don’t often have enough immune cells with these receptors in order to mount an effective response against their cancer. In this Phase 1 trial, researchers identified each patient’s receptors, inserted them into immune cells lacking them, and grew more of these modified cells. Then, the bolstered immune cells were unleashed into each patient’s bloodstream to attack their tumor.
“What we’re trying to do is really harness every patient’s tumor-specific mutations,” says Stefanie Mandl, chief scientific officer at Pact Pharma and an author on the study. The company worked with experts from the University of California, Los Angeles, the California Institute of Technology, and the nonprofit Institute for Systems Biology in Seattle to design the personalized therapies.
The researchers began by separating T cells from the blood of 16 patients with solid tumors, including colon, breast, or lung cancer. (T cells are the immune system component with these receptors.) For each patient, they identified dozens of receptors capable of binding to cancer cells taken from their own tumors. The team chose up to three receptors for each patient, and using Crispr, added the genes for these receptors to the person’s T cells in the lab.
Continue reading… “This Personalized Crispr Therapy Is Designed to Attack Tumors”