NIH approves the first CRISPR clinical trial

The CRISPR-Cas9 genome-editing technology is inching closer towards clinical trials with the U.S. National Institutes of Health (NIH) approving a proposal to use it in cancer treatment.

On June 21, the NIH’s Recombinant DNA Advisory Committee (RAC) – a federal ethics and biosafety panel – gave its consent to the clinical trial headed by Edward Stadtmauer, a physician at the University of Pennsylvania in Philadelphia. The trial will be funded by the Parker Institute for Cancer Immunotherapy, which was founded by former Facebook president Sean Parker.    

The two-year trial will involve 18 patients with myeloma, sarcoma, or melanoma who no longer respond to the currently available treatments at the Parker Institute for Cancer Immunotherapy. The main objective of the research team will be to genetically modify the T cells i.e. the immune cells in the patient’s body to detect and attack cancer cells.

Despite the permission from the RAC for the trial to set off, Stadtmauer will need to get additional approvals – from medical centers where the trial would be conducted, the Food and Drug Administration, and the review boards at the institutions with which his team members are associated. According to Carl June, an immunologist at the University of Pennsylvania and a science adviser on the project, the trial is likely to begin by the end of the year. He also mentions that the primary challenge in using CRISPR is avoiding any unintentional mutation of genome.

Many companies are rivaling to commercialize this technology, and several institutions and research groups have expressed an interest in using it in clinical trials. While the approval of this trial has brought cheer among geneticists, it remains to be seen whether it results in better understanding about CRISPR and leads the way in the approval of more trials that use this technology.

References:

First CRISPR clinical trial gets green light from US panel

First proposed human test of CRISPR passes initial safety review