A First in the U.S.
CRISPR is currently humanity's most effective and efficient gene-editing tool, and the technology is no longer just limited to editing plants or laboratory animals. In November 2016, Chinese scientists actually tested CRISPR in a human, and now, a human CRISPR trial is about to begin in the United States.
In June 2016, an advisory board of the National Institutes of Health (NIH) gave doctors from the University of Pennsylvania Health System (Penn Medicine), led by Edward Stadtmauer, the initial approval needed to begin human CRISPR trials. According to a post in a directory of ongoing clinical tests, the team is now almost ready to begin their trial.
For this first-of-its-kind medical test in the U.S., Stadtmauer and his team plan to use CRISPR to edit human T cells, which play a central role in the immune system, to target tumors. As many as 18 patients suffering from three types of cancer — multiple myeloma, sarcoma, and melanoma — could be enrolled in the study.
For their human CRISPR trial, doctors plan to extract blood cells from the patients and then edit the cells outside the body, an approach called ex vivo gene therapy. The goal is to turn the T cells into better cancer fighters before reintroducing them into the patient's blood stream.
Using CRISPR, the doctors will delete two specific genes from the T cells. One is a so-called "checkpoint" molecule (PD-1) that cancer cells exploit to halt immune system activity. The other is a receptor that T cells use to detect dangers, such as germs or sickly tissue. They'll replace that receptor with an engineered one designed to direct T cells toward tumors.
The Parker Institute for Cancer Immunotherapy, a charity created by Napster founder Sean Parker, is helping finance the trial, which is expected to begin in January 2018 and end in January 2033, according to the clinical trial directory posting.
However, a Penn Medicine spokesperson told the MIT Technology Review that a start date hasn't been confirmed: "We are in the final steps of preparing for the trial, but cannot provide a specific projected start date.”
Still, after at least two years of planning, the U.S.'s first human CRISPR trial appears almost ready to begin, and once it does, the nation will officially join China on the very short list of places testing CRISPR on humans. Europe is expected to follow soon, though, with a human CRISPR trial using an ex vivo approach slated to begin later in 2018 courtesy of biotech firm CRISPR Therapeutics.
The results of these human clinical trials are likely to have a huge impact on the future of gene editing. Ultimately, while CRISPR does wonders in agriculture and other fields, proving its ability to cure human diseases is arguably a much larger goal, and reaching it could forever change healthcare.