In all the talk of “disruption” and “hacking cancer,” you’d be forgiven for not taking internet billionaire Sean Parker seriously in the life sciences world. But he just got federal backing to run the world’s first-ever CRISPR/Cas9 trial, which could alter the way many diseases, especially cancer, are treated.
You’d be hard-pressed to find another first-in-human trial gain so much attention because, yes, despite all the column inches it’s generated, CRISPR has not yet entered the clinic.
But its preclinical activity has caused much hype given its gene-editing capabilities, with its core tech centering on a simple method for reengineering DNA--which may well have profound consequences in treating a number of diseases.
Late yesterday, a National Institutes of Health (NIH) panel ruled unanimously (but with one abstention) to let the first trial for a CRISPR candidate run. This is just the first step, however, as the FDA as well as the medical centers where it’ll be run will still need to clear the study before it can actually start recruiting and testing patients.
So who’s the first pioneer? CRISPR Therapeutics, Caribou Biosciences, Editas ($EDIT), or maybe Intellia ($NTLA)? Surely Novartis ($NVS) must be involved? Nope, it’s Sean Parker, founder of Napster and the first president of Facebook, who, not short of a few bucks (net worth estimated at $2.4 billion), has stolen a march on the CRISPR pioneers. (Editas was in fact down 7% at the end of play yesterday after the news, with Intellia down 4.5%.)
The trial, should it gain the regulatory nod, will be run by a group of University of Pennsylvania researchers and funded by the Parker Institute. The study would also be conducted at MD Anderson Cancer Center in Texas and the University of California, San Francisco. Penn will be its spiritual home, however, as Penn’s Dr. Carl June is one of the major innovators in T cell biology.
Speaking at the Forbes Philanthropy Summit earlier this month, June said the idea is to make T cells, a type of white blood cell, “better than nature made them.” The cells will be edited to lack several genes, including one that allows them to respond to a protein called PD-1, which acts as an off switch that some cancers hijack to evade the immune system.
PD-1 was the last next big thing in cancer, with Merck ($MRK) gaining the first approval for its PD-1 drug Keytruda in 2014, although the market leader, despite being approved second, is Bristol-Myers Squibb’s ($BMY) Opdivo. Roche ($RHHBY) also joined the party earlier this year with its PD-L1 drug Tecentriq.
Many in the oncology research field see the future as combination therapies using checkpoint inhibitors with either older tech and/or newer mechanisms of action, such as CRISPR and gene editing, as well as chimeric antigen receptor T cells--which are in the clinic and producing some stellar results in blood cancers. (In fact, Penn already has a pact with Novartis to develop CAR-T, but June says there is no relationship with the Swiss major in this trial as it will not be using CAR.)
The now-NIH-backed CRISPR study will focus on editing around 15 cancer patients' immune system T cells so that they are better at attacking cancer cells, with targets including myeloma, sarcoma, and melanoma.
Although unanimous, the NIH's Recombinant DNA Advisory Committee panelists still raised a number of concerns about the trial’s protocol and its funding. This includes questions over the financial conflicts of interest out of Penn, with a panelist asking why the study couldn't be run out of other institutions to cancel out some of those conflicts.
They also raised concerns about why cancer tests aren't included in the course of the trial--something which could exclude patients who don't have the resources to get those tests on its own. These were not deemed to be of high enough concern to stop its green-lighting of the study, however. Over to you, FDA.
Parker has been dabbling in the life sciences space over the past 12 months, in April injecting $250 million into the immuno-oncology research field in an effort to boost cancer survival rates by helping form 6 cancer centers nationwide. This builds on the $600 million Parker put up to launch the Parker Foundation last year, which has a particular focus on life sciences.
But in an unusual move, Parker’s foundation says it will control patents on research it funds and even bring treatments to market. For its CRISPR trial, however, some further clarity is needed on just who will own the IP around the new cells.
Many moneyed men have in recent years sought to get in on the lifesaving biz, including the estate of the billionaire shipping magnate Daniel Ludwig, which donated $540 million to 6 cancer centers in 2014, and Nike co-founder Phil Knight, who pledged $500 million to cancer researchers at Oregon Health & Science University in 2013.
Many in the tech space too are looking for new challenges outside of digital and into biology. Google ($GOOG) set up its own life science company Calico a few years back which, although far from transparent, has the aim of “tackling aging.”
And then there’s 23andMe, backed by Google, which is designed to help people unravel their genetics and potentially spot any warning signs for disease.
Parker, it appears, has even loftier aims of "disrupting cancer," but there are still many years and many hurdles to pass before we'll know if this trial (if the FDA passes it) is a success, with many questions over IP and the tech itself still remaining unanswered.