On Nov. 14, the Senate health committee held a hearing on the gene editing method CRISPR to learn more about the technology from expert witnesses, which included Dr. Matthew Porteus, associate professor of Pediatrics at Stanford University; Katrine Bosley, CEO and president of Editas Medicine; and Dr. Jeffrey Kahn, director of the Johns Hopkins Berman Institute of Bioethics at Johns Hopkins School of Public Health.
In his opening statement, Chairman Lamar Alexander said, "CRISPR's use in humans is more recent, but the possibility of the diseases it could treat and the lives that could be improved is remarkable... Researchers see the possibility of treating blood diseases and sickle cell disease, improving the amount of time immune cells are active in fighting tumors, or even identifying, and then treating, a predisposition to Alzheimer's...While CRISPR has amazing potential, it is not hard to see how we can quickly get into societal and ethical issues... Part of our job on this committee is to learn about new technologies, to lead discussions with experts about the implications of these scientific advancements and to ensure that the National Institutes of Health and others have the proper authority to oversee and conduct research."
Video of Alexander's opening statement is available here.
During the hearing, Sen. Alexander asked the witnesses what he and other members of the committee could do in order to create the best environment for the development of technologies that have the potential to discover cures for diseases that currently have limited treatments or cures.
Sen. Alexander: Do any of you have a recommendation to ... this committee about what we should be doing, if anything, to create an environment in which you can succeed in an appropriate way?
Bosley: Senator, if I may comment on that, it's an excellent question I think. In many respects, the 21st Century Cures legislation, there's so many different dimensions of that legislation as well as the long history of bipartisan legislation that comes through this committee, I think implementing that robustly continues to support a fantastic environment for this technology to mature in a careful and thoughtful way.
Porteus: I would say two things. One is continued, sustained--and as a scientist of course I like to say substantial--funding to the NIH for basic science research, because as an investigator taking on real challenges, you have to know that you have the opportunity to spend five or ten or even more years on it. And if we see funding go up and down, it discourages people to taking on those long term challenges, so I think that's very important.
And then I think, and again this is consistent with [Dr.] Scott Gottlieb and Peter Marks, is having the FDA have a flexible data driven approach to the regulation of this field. It's too new to think that we know exactly which lines should be drawn in black ink and which lines should be drawn in pencil. And I think we need to have the regulators be data driven about how we adjust as we get more data from clinical trials about safety and efficacy.
Sen. Alexander: It's important for the research community to know that, with the leadership of Senator Blunt in Missouri and Senator Murray, who are the chairmen of the Appropriations subcommittee, and the support of a lot of us, we've increased funding for the National Institutes of Health by two billion dollars for two consecutive years and recommended it for a third year - plus the $4.8 billion in the 21st Century Cures Act. I mention that not to pat ourselves on the back, but I think it's important to send a signal out through the research community that we're paying attention, and we understand that it's a pretty remarkable time and we want to attract them.
Video of Alexander's questions are available here, here, and here.
Video of the full hearing is available here.