Today at 10:40 a.m. Eastern Time, researchers from the University of Pennsylvania will ask a government panel for permission to be the first to use CRISPR, a new type of genetic engineering, to treat a disease in people.
The effort is being funded by The Parker Institute, the new philanthropy created by Facebook billionaire Sean Parker to battle cancer. The CRISPR work builds on earlier efforts by University of Pennsylvania professor Carl June.
The idea, June explained at the Forbes Philanthropy Summit earlier this month, 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. (New drugs from Merck , Bristol-Myers Squibb BMY +0.16% and Roche all target PD-1.) “This is the first example of CRISPR in a clinical trials in humans, period,” Parker said.
As radical as using CRISPR might sound, in this context it is probably not ethically different from any other gene therapy, including the existing work on T cells that has gained June renown and fascinated Parker. “From an ethics perspective, I don’t think it makes any difference,” says Hank Greely, a Stanford lawyer who follows cutting-edge biotech closely. “It’s not anything new, it’s just the normal ethics of trying to do gene therapy.”
Where he does worry, Greely says, is in the area of financial conflicts of interest. Intellectual property could be owned in a complex web between June, Penn and the Parker Institute, although Parker has said his institute will own all resulting intellectual property. What it boils down to is: Could Penn be nicer to the trial because it stands to make a lot of money.
The study, which will also be conducted at M.D. Anderson and Stanford, still has a lot of hurdles to pass. Aside from the government panel–the Recombinant DNA Advisory Committee, which is being webcast here–the study still has to get through investigational review boards at the universities where it will be conducted.
Still, June says that the Parker Institute is allowing the new therapy to move at a speed not permitted by government or industry funding. “Frankly, we’re more nimble than the big pharmas and the big biotechs.”
The new project represents a way around June’s current big pharma partner, Novartis , which licensed technology for using what are called chimeric antigen receptor T-cells, which are programmed using a construct that includes parts taken from a drug antibody. Final trials of these cells for blood cancers are in progress; they could reach the market next year.
Instead, this time the genetic manipulation is being done using a T-cell with a T-cell receptor that attacks cancer. An earlier version of this technology was licensed to Tmunity, a Penn spinout. In conversations with the Parker Institute, it was not clear who would own intellectual property around the new cells, or what that intellectual property would be.