The first proposed test of CRISPR gene-editing technology in human beings is being funded by Internet billionaire Sean Parker, MIT Technology Review has learned.
The novel cancer treatment, initially disclosed last week, is being reviewed Tuesday by a federal advisory panel in Washington, D.C., and could become the first clinical trial involving CRISPR, the red-hot gene modification technology.
Now we can report that Parker, a 36-year-old whose net worth is estimated at $2.4 billion, is financing the study.
Parker, best known for his role as Facebook’s first president and as co-creator of the pirate music site Napster, splashed into cancer research in April, saying he’d give $250 million in funding to six centers, including one at the University of Pennsylvania, in what he called a “Manhattan project for curing cancer with the immune system.”
Penn confirmed Parker’s charitable foundation would be funding the trial, which will use gene-editing to modify immune system T cells to attack three types of cancer: myeloma, melanoma, and sarcoma.
Parker’s backing highlights how wealthy Internet entrepreneurs think they can speed up cancer research. Parker, a one-time hacker whose resume includes a run-in with the FBI, has said he thinks of T cells as being “like little computers” that can be reprogrammed (see "10 Breakthrough Technologies 2016: Immune Engineering").
There’s no shortage of funding for immune therapy. New drugs are able to cure some cases of advanced melanoma; T cell treatments, including some pioneered at Penn, have seen dramatic success against leukemia.
But Parker’s foundation is unusual because it says it will control patents on research it funds and even bring treatments to market. Parker compared the tactic to convincing record labels to license their music to Spotify, the popular legal music-sharing service he also is an investor in.
“What if we had a system where all the [intellectual property] could be shared among the scientists?” he told Dateline NBC in a program that aired in May.
The new cancer treatment developed at Penn uses gene-editing to modify T cells so they more effectively target certain cancers other than leukemia. Penn appears to have filed a patent on the idea last October, according to European patent records.
A proposal for a small safety study will be presented Tuesday by Penn doctors Carl June, Joseph Melenhorst, and Edward Stadtmauer, according to an updated agenda of the Recombinant DNA Advisory Committee, which reviews gene therapy studies.
Up to 15 patients could receive the treatment at three sites: Penn, the University of California, San Francisco, and the MD Anderson Cancer Center. The study involves complex safety issues, such as whether the alterations achieved using gene editing will be accurate enough, and there is no guarantee it will go forward.
The treatment envisions removing a cancer patient's T cells and then re-infusing them a month later following genetic alterations to their DNA designed to cause them to zero in on, and destroy, the tumors.
“One of our key areas of focus is to develop novel approaches to modify T cells to enhance their function,” said Jenifer Haslip, a spokesperson for the billionaire’s nonprofit, called the Parker Institute for Cancer Immunotherapy, headquartered in San Francisco.
“CRISPR technology provides an opportunity to profoundly manipulate cells,” according to a statement provided by Haslip. “We’re excited to be part of the first clinical effort in the United States to combine these two powerful therapeutic approaches to treat a devastating disease like cancer."