WASHINGTON -- Members of the Senate Health Education, Labor and Pensions (HELP) Committee on Monday heard testimony from several experts in CRISPR, the gene-editing technology that has taken the genetics community by storm.
They mostly extolled the technology's promise for, yes, curing diseases that currently can only be managed as chronic conditions, often not well.
But not much was said about the ethics of altering individuals' genomes. Concerns about the technology's potential to create "designer babies," for example, were mentioned only in passing.
HELP Committee Chairman Lamar Alexander (R-Tenn.) noted in his opening remarks that CRISPR had been included on a list of "weapons of mass destruction and proliferation" by then-Director of National Security James Clapper in 2016, and that the technology poses certain "national security concerns."
However, most of the meeting was spent exploring somatic cell or non-heritable editing of the genome and its potential to "transform human health." The discussion was characterized as "a listening session."
Alexander and his colleagues credited the benefits of the 21st Century Cures Act, which includes $4.8 billion in funding for National Institutes of Health -- as well as privacy protections and data sharing provisions -- and another $2-billion bump in the NIH's budget for helping advance the science of genomics.
Katrine Bosley, CEO and president of Editas Medicine, based in Cambridge, Massachusetts, spoke of possible new treatments for diseases of the eye and liver, instead of cures -- for fear of "overpromising."
In her, Bosley described an Editas' research program that targets Leber's Congenital Amaurosis Type 10 (LCA10), a disease that causes vision loss or blindness in children. Editas is also exploring possible treatments for inherited blood disorders and therapeutics in immuno-oncology.
Editas is exclusively focused on non-heritable gene-editing -- experimentation on somatic cells, not germline cells -- she stressed, and plans to file an application with the FDA for its first candidate product for LCA10 by mid-2018, she said.
Matthew Porteus, MD, PhD, said that if his research lab, or another researcher in the scientific community, finds a cure for sickle cell disease, it might take only a few "tweaks" to then find a cure for other illnesses, such as severe combined immunodeficiency or "bubble boy disease," Porteus said.
(Porteus' lab has already found a way to correct mutations of sickle cells from patients' own blood stem cells, as he noted in )
"Because once you have cures for one disease and organ, that's the platform for hundreds of other diseases of that same organ," he said.
While there are currently no clinical trials using CRISPR-CAS9 in the U.S. or Europe, Porteus expect to see several in the next 12 to 18 months.
On the whole, questions from senators highlighted more excitement than concern about the prospects for gene editing.
Porteus, in his opening remarks opined that the current regulatory framework -- a combination of the FDA, plus the ) and institutional review boards (IRBs) -- is "completely adequate in handling the assessment of the science and ethics of doing genome editing in somatic cells."
(The RAC provides recommendations to the director of the National Institutes of Health.)
"I hope that the controversial issues that surround genome editing do not distract us from being able to stay focused and committed to developing curative therapies for devastating genetic diseases like sickle cell anemia," he said.
But Jeffrey Kahn noted a few areas where some caution was needed, in some cases even after a therapy has been approved by the FDA. For example, Kahn worried that some companies may try to expand the use of therapies beyond the approved indication -- e.g. using products for enhancement and other off-label uses.
Tools such as the FDA's Risk Evaluation and Mitigation Strategies (REMS) would become considerably more important, he noted.
"[The FDA has] the tools but they haven't ever used them in the way that they would need to [with] these kinds of technologies coming to market," Kahn told 鶹ý, after the hearing.
Sen Tim Kaine (D-Va.) also cited concerns from Kahn's own testimony regarding the lack of an international "comprehensive regulatory approach" to gene editing.
Kaine further highlighted Kahn's statement that absent a unified approach some regions will draft "lenient or nonexistent regulation" which could lead to the creation of "regulatory havens" and trigger "scientific flight" or "medical tourism," as well as a loss of competitiveness.
Kahn offered two examples of rigid versus flexible regulation. In Canada, federal law penalizes the action of using gene editing tools on cells that could lead to heritable changes with a prison sentence of up to 10 years, he noted. Britain's regulatory process, on the other hand, is "permissive with very tight controls," he said.
Too much regulation can drive the science to regulatory havens where it can't be controlled at all. Moreover, he said, "the benefits of that research disappear."
"We lose in multiple ways when we drive science underground," said Kahn. "This country has long, really forever, been the leader in science in the world and I don't think we want to cede that to anybody else."
Both Porteus and Kahn served on the Committee on Human Gene Editing for the National Academy of Sciences and the National Academy of Medicine which released a paper in February on heritable germline editing -- genetic manipulations that can be passed down to offspring.
The report outlined a very strict list of 10 criteria under which germline editing could be permitted in clinical trials in the future.
While some have criticized the criteria as overly strict, Kahn said, "If that is the case then so be it. It's a recipe for very tight control, to allow the benefits to go forward in cases where there really is no other way to achieve therapy for a particular disease."