On Tuesday, a National Institutes of Health (NIH) panel gave the go-ahead to the first ever CRISPR clinical trial funded by internet billionaire, Sean Parker. Despite widespread speculation that the new gene editing technique could be a game-changer for fields like oncology, no other first-in-human clinical trials of the technique has been attempted.
Even with a single abstention, the NIH panel ruled unanimously in favour of allowing the first CRISPR candidate to be tested in a human clinical trial. The study will need to win additional approval from the US Food and Drug Administration (FDA), along with the medical centers where it will be conducted, before the clinical trial can begin recruiting patients.
Sean Parker – founder of Napster and the original president of Facebook – has beat numerous biotech companies to host the first CRISPR clinical trial. Parker – whose net worth is estimated at $2.4 billion – is unaffiliated with any of the pioneering companies in gene editing, including CRISPR Therapeutics, Caribou Biosciences, Edita, Intellia or Novartis.
Pending regulatory approval, the clinical trial will be conducted by a group of researchers at the University of Pennsylvania, and funded by the Parker Institute. In addition, the MD Anderson Cancer Center in Texas and the University of California, San Francisco, will also take part in the groundbreaking study.
One of the collaborators, Dr. Carl June of the University of Pennsylvania, is considered to be a major innovator in the field of T cell biology. He said that the idea behind the project is to make T cells “better than nature made them,” at the Forbes Philanthropy Summit earlier this year.
According to June, the T cells will be edited using CRISPR so that they lack a gene allowing them to respond to an immune system checkpoint protein, known as PD-1. Some cancers hijack this protein which allows them to operate outside the confines of the immune system.
Multiple drugmakers – including Merck, Bristol-Myers Squibb and Roche – have developed PD-1 drugs promising to be more effective cancer therapies. Many oncologists believe that PD-1 checkpoint inhibitors used in combination with CRISPR gene editing and CAR-T cells, represent the future world of cancer treatment.
The NIH-approved CRISPR clinical trial will use the gene editing technique to alter the T cells of approximately 15 cancer patients in an attempt to strengthen their immune systems. Cancer targets for the study include myeloma, sarcoma and melanoma.