Imagine developing a first-of-its-kind therapy that saved the life of one woman dying from colon cancer, only to lose the financial backing needed to bring it to market.
That’s the reality for University of Minnesota researchers who were excited last week to report early-stage results of their genetically edited immunotherapy, which halted tumor growth for a month or two in several patients and reversed cancer symptoms in one Minneapolis woman.
Intima Bioscience celebrated the results, which were featured at a national cancer research conference. But the startup company had already pivoted to other experimental therapies that could be faster, cheaper to make, and target more types of cancer.
U researchers are partnering with Intima on those cancer-fighting alternatives, but are assembling a “Minnesota homegrown” team to keep their studies of the colon cancer therapy going.
“They found it too costly and complex, but we’ve been working to make it more affordable, more rapid, more potent,” said Branden Moriarity, co-director of the U’s Center for Genome Engineering. “We are doubling down on this therapy.”
The experimental treatment has been several years in the making. U researchers used CRISPR gene-editing to deactivate a gene, called CISH, in tumor-fighting white blood cells and improve their ability to recognize and attack cancer.
“We believe that CISH is a key factor preventing T cells from recognizing and eliminating tumors,” Moriarity said.
The U-led trial was the second to receive federal approval for human testing of a CRISPR-edited cell therapy, Moriarity said. The goal was to prove that a small group of patients with severe cancers could safely tolerate the therapy, before testing its effectiveness in a larger group and then pursue approval by the U.S. Food and Drug Administration.