After the discovery of CRISPR in bacteria, a cut and paste DNA system used to defend against foreign genetic sequences especially those inserted by viruses, researchers transformed the discovery into a tool called CRISPR/Cas9 that can make precise cuts in the genomes of other organisms as well.
To put this tool to good use, researchers at the University of Pennsylvania have proposed a clinical trial where this gene editing technology would remove and alter human T cells that target foreign cells in cancer patients.
Since clinical trials of gene-editing therapies have already taken place and the ethical considerations have already been discussed, the researchers have cleared the first hurdle by winning unanimous approval from the Recombinant DNA Advisory Committee at the U.S. National Institutes of Health.
The proposed two-year trial will treat 18 people with myeloma, sarcoma, or melanoma who have stopped responding to existing treatments at U Penn; the University of California, San Francisco; and the University of Texas MD Anderson Cancer Center in Houston. For the next step, the researchers will need to obtain approval from the ethics review boards at their institutions and the U.S. Food and Drug Administration.
So far, the testing of the CRISPR/Cas9 system in lab animals has yielded mixed results and worked only on some cancers. However, the researchers have made improvements to the technology and will monitor immune responses and for any off-target cuts in genomes.
As Hank Greely, a Stanford law professor points out, whoever owns the therapies stand to make a lot of money. Currently there are potential financial conflicts of interest and issues with intellectual property. The Parker Institute, a foundation established by Facebook billionaire Sean Parker, is funding the trials. The trials will utilize methods based upon earlier work by Carl June of U Penn, which genetically modified cancer patients’ immune cells and would create a financial interest in the outcome.
Researchers at the University of California, Berkeley, filed a patent application in 2012. Then, researchers at the Broad Institute of MIT and Harvard filed a patent application under an expedited review program and were awarded the patent in April 2014. Until recently, the US awarded patents to those who could show that they were the first to invent, rather than the first to file. Then US switched to a first-to-file system, complicating things, and now proper licensing agreements have to be established.