First human study of CRISPR approved by federal panel
- A federal panel at the National Institutes of Health on Tuesday approved the first in human study of the gene-editing technology known as CRISPR-cas9, reports Stat.
- Proposed by researchers at the University of Pennsylvania, the study will use CRISPR to edit genes in patient immune cells as a way to target three types of tumor cells.
- The study still needs approval from the Food and Drug Administration as well as from the medical centers where it will be carried out.
Approval from the Recombinant DNA Advisory Committee, which reviews all proposals for human trials involving recombinant DNA, could mean CRISPR is tested in humans sooner than expected.
A number of biotechs such as Editas Medicines have been developing their own CRISPR-based therapies. Editas has said it plans to test CRISPR in a human trial sometime in 2017.
The University of Pennsylvania study will be small, enrolling less than 20 patients, and will target three cancers: myeloma, melanoma, and sarcoma. Edward Stadtmauer, a professor of medicine at Penn, will lead the study and the well-known cancer scientist Carl June will be an adviser.
Internet billionaire Sean Parker is reportedly funding the research, according to the MIT Technology Review. In April, Parker donated $250 million to advance cancer immunotherapy research and announced the formation of a new institute which will collaborate with six major cancer centers in the U.S.
The study will look at using CRISPR as an autologous therapy, meaning researchers would extract T-cells from patients, genetically modify those cells to better target tumors, and then reinfuse them back into the patient.
This shares similarities to the type of cell therapy known as CAR-T, but the Penn researchers plan to use CRISPR-based editing as an additional step to improve the durability and efficacy of the treatment, reports Stat.
Other biotechs are also looking at an autologous approach to gene and cell therapy which, while highly individualized, could prove costly given the nature of the treatment.
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