WASHINGTON-14, November 2017
By Julia Rapp
The cutting edge and controversial gene editing technology CRISPR-Cas9 will be part of an almost $5 billion medical Congressional investment thanks to the 21st Century Cures Act that Congress signed into law last year.
On Tuesday morning, the Committee on Health Education Labor and Pensions held a hearing about the benefits and risks of this breakthrough technology, including testimonies from its proponents who are scientists and medical professionals.
Ranking Member Sen. Patty Murray (D-WA) discussed this technology’s ability to tackle Sickle Cell disease and Huntingdon disease as well as engineer cells to fight cancer and stop infections.
“I’m proud that my home state of Washington is leading the way in advancing this technology,” Murray said, mentioning that the scientists at Seattle Children’s Ben Towne Cancer Center were using this technology through immunotherapy, a practice used to treat cancer through the immune system.
The process for gene-editing involves two methods according to Dr. Matthew Porteus who was a witness: designing a protein that will break the DNA which activates the cell to try to fix the break by gluing it back together or making a copy of an undamaged piece DNA to swap with the damaged piece of the DNA.
Gene-editing can be used to treat cancer and hereditary diseases along with modifying seeds. The process has received criticism due to its potential to create “Designer Babies” or genetically modified human embryo, and be used for biological warfare.
However, according to a survey published in the magazine Science, 59 percent of US survey respondents said that they support the use of gene editing to treat medical conditions. In comparison only 33 percent supported the use of gene editing to enhance or improve human abilities.
What makes CRISPR, which stands for Clustered Regularly Interspaced Short Palindromic Repeats special is that it can edit genetic codes more precisely.
“It is absolutely critical that in continuing to make medical advancements, our country upholds the highest standards of ethics and consumer safety and helps to ensure those standards are being followed around the globe,” said Sen. Murray.
Chairman Sen. Lamar Alexander (R-TN) acknowledged that the hearing was one of the most bi-partisan discussions he had attended, and said that both he and Murray agreed on the subject and the choice in witnesses.
“I continue to be inspired and heartened by the bi-partisan commitment to investing and supporting biomedical research,” said Sen. Murray.
Chairman Sen. Alexander introduced witness Dr. Matthew Porteus who spearheaded the Porteus Lab at Harvard University. His lab focuses specifically on genetic diseases.
“Genome-editing,” he said “…is a potentially ideal cure for these monogenic diseases.” Porteus says that it gives the ability of converting disease causing sequences in genes back to non-disease causing sequences.
Despite there being a lack of clinical trials in the U.S. or Europe that are using the CRISPR technology, the Porteus Lab team is hoping to be brought to clinic trial or being able to conduct experiments under clinical research around 2019.
Katrine Bosley, CEO and president of Editas Medince, was the second witness introduced by Sen. Elizabeth Warren (D-MA).
She argued that the CRISPR technology allows her industry to focus on treatment for a rare eye disease called LCA 10. Bosely said that she personally feels a responsibility to the patients, families and to society. “We take that responsibility very seriously,” she said.
Despite concerns and criticism over the genome-edits being inherited through offspring, Bosley says that Editas is developing medicines with non-heritable gene edits.
She acknowledged that her line of work will be subject FDA review and also public review by the NIH advisory committee RAC.
“Today’s hearing is another hallmark in this committee’s long distinguished history of overseeing biomedical research,” Bosley said.
She says that Editas is aware that CRISPR-cas9 could be a disruptive technology and believes it is the industry’s responsibility to engage with the committee and other major entities in a transparent and respectful manner.
“There are a few times in our lives when science astonishes us and when something was science fiction yesterday and now is reality-this is one of those moments,” Bosley said.
Jeffrey Kahn, director of the Berman Institute of John Hopkins Bioethics and professor in the department of health policy and management in the John Hopkins Bloomberg School of Public Health went more in depth in the containment of risks.
The risks include expanded use of therapies beyond approvals, inheritable genetic modification and challenges that genome editing poses for regulatory oversight.
“The NSA committee…noted that improvements in genome editing techniques are driving increases in the efficiency and accuracy of genome editing and also decreasing the risk of off target effects,” he said.
Kahn acknowledged that while genome-editing can be inheritable but the effects of it can be helpful.
“As a result both potential benefits and potential requirements will be multiplied under the very strict oversight if that’s ever to go forward,” he said.
Sen. Susan Collins (R-ME) was concerned over this technological method being used on an international scale.
“How do we ensure that this exciting breakthrough in gene editing is used for good by scientists in countries like China or Russia as well as in our own country?” Collins asked.
Kahn assured her that they have strict oversight for making sure that the technology is used correctly and that the National Academy of Sciences in the U.S. has a partnership with Chinese Academy of Sciences.
He said that the National Academy Consensus Committee followed an International summit in December of 2015.
Kahn also made the point that when technologies are banned in this country scientists will find places where there are no oversights to go and perform them.
“A much smarter control to policy is to allow careful, responsible, science to go forward in ways that are controlled and within our borders,” he said.
As the meeting closed, Bosley pointed out that the committee implementing the 21st Century Cure Act could help ensure a satisfactory environment for all the professional involved.
Both Porteus and Katrine Bosely have filed an application for an FDA approved treatment, the first step to be able to test on humans under FDA investigation.
Advanced Reporting Times 