Envisioning the potential of CRISPR to cure cancers, sickle cell anemia, eye diseases and possibly even Alzheimer's, members of the Senate Health, Education, Labor and Pensions (HELP) Committee searched for ways Congress could help advance the technology while ensuring ethical standards and safeguarding against unintended consequences and its use by the dark side.
They were advised to put the money where their mouth is. Developing gene-edited therapies requires long-term research that needs predictable and adequate funding, Matthew Porteus, a pediatric hematologist/oncologist and associate professor at Stanford University, told the committee Tuesday. Researchers need to know that the funding will still be there when a project is in its fifth or even 10th year.
HELP Chairman Lamar Alexander (R-Tenn.) noted that Congress has bumped NIH research funding by $2 billion each year for the past two years and the committee is pushing for a similar increase this year. He wondered aloud how much of the additional money funneled into the BRAIN Initiative is funding CRISPR projects.
Katrine Bosley, president and CEO of Editas Medicine Inc., of Cambridge, Mass., assured him that, since CRISPR is a tool being used across therapeutic spaces, some of the researchers funded through the initiative are undoubtedly using the gene-editing technology.
To provide more money for basic research involving CRISPR, Sen. Tim Kaine (D-Va.) suggested increasing the funding for universities in the higher education bill that HELP will be working on soon. Alexander reminded him that the bulk of the NIH funding already goes to university research.
It's not just research that needs funding, Bosley said, as she stressed how critical it is for the FDA to have sufficient funding. "The FDA has the appropriate authority, and they're exercising it well" in working with companies to develop gene-edited therapies, she said. However, she added, the agency must have the resources to meet the demand.
HELP members appeared enamored with CRISPR, using such words as "a miracle," "remarkable" and "truly exciting" to describe its potential. Bosley summed up the feeling in the room with her opening remarks: "There are a few times in our lives when science astonishes us, when we are suddenly able to do something that seemed like science fiction just the day before. This is one of those moments."
While others oohed and aahed over the promise of CRISPR, a few committee members tempered their excitement with concerns about the ethics and the possibility of misuse of the technology. Sen. Susan Collins (R-Maine) questioned the adequacy of current policy in overseeing CRISPR research, given the ethical issues involved and the opportunities to misuse gene-editing on the global scene. "It seems like the scientific advancement has outpaced the policy in this area," she said.
Scientific advancement generally outpaces policy in most arenas, responded Jeffrey Kahn, director of the Johns Hopkins Berman Institute of Bioethics. "In some respects, that's to be expected," he said. "We ought not to be making policy before we understand the science as it advances."
That said, Kahn agreed that while the existing U.S. oversight structures have proved robust and effective in governing gene therapies, gene editing presents new policy needs to address ethical concerns such as the potential to make human germline changes or to go beyond therapeutic uses to enhance "normal" abilities. Any new policy should involve tight controls, but it shouldn't be prohibitive, he cautioned.
"Prohibitions and bans have led not to control but rather quite the opposite," Kahn said. "So when technologies are banned in this country, scientists find places where there is either lax or no oversight. . . . A much smarter approach to policy is strict control to allow careful, responsible science to go forward . . . within our borders and not to push it out."
To illustrate his point, Kahn told the tale of two countries, Canada and the U.K. Last week, researchers called for a change to a Canadian law that criminalizes the use of gene-editing tools that could lead to heritable genetic changes in humans. The crime carries a maximum prison sentence of 10 years. As a result of the law, Canadian researchers say they are falling behind their international colleagues.
The U.K. has taken the opposite approach, Kahn said. But instead of creating a haven from oversight, it has developed strict oversight with a clear pathway for licensure. He urged U.S. lawmakers to follow the U.K. model; otherwise they risk driving science underground and ceding the country's lead in medical innovation.