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The New Jersey Life Sciences Vendors Alliance (NJLSVA) is a coalition of businesses, individuals and academia who provide goods and services to New Jersey’s life sciences companies.

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NIH Panel Approves First Test of CRISPR Editing in Humans; Study to Target Three Types of Cancer

Gene editing crisprBethesda, MD, June 22, 2016The Washington Post reports that a National Institutes of Health (NIH) advisory panel yesterday approved the first human use of the gene-editing technology CRISPR, for a study designed to target three types of cancer and funded by tech billionaire Sean Parker’s new cancer institute.

The experiment, proposed by researchers at the University of Pennsylvania, would use CRISPR-Cas9 technology to modify patients’ own T cells to make them more effective in attacking melanoma, multiple myeloma and sarcoma.

The federal Recombinant DNA Advisory Committee approved the Penn proposal unanimously, with one member abstaining.  The experiment still must be approved by the Food and Drug Administration, which regulates clinical trials.

The early stage-trial, which will involve more than a dozen patients, will be designed to test the safety of the therapy. It will be conducted at three medical institutions — Penn, MD Anderson Cancer Center in Houston and the University of California at San Francisco.

The study involves T cells, which are white blood cells essential for ridding the body of bacteria, malignancies and other invaders. Carl June and other researchers at Penn have pioneered an immune therapy, called CAR T-cell treatment, in which the cells are extracted from a patient, genetically engineered and then re-injected into the blood stream.

While the treatment shows promise, “our efforts have been hampered because the infused T cells become exhausted and die or cease to function,” the scientists said in explaining their proposal.

Under the study approved Tuesday, CRISPR would be used to remove two genes in the T cells to make the treatment last longer and reduce the chance of the cancer returning.

“Our goal is to develop a new type of immunotherapy using gene-editing technology that will enable the engineered immune cells to be more potent, survive longer and thereby kill cancer cells more effectively,” the scientists said.

For the full Washington Post story, click here.