Genetic treatment oversight

MARY REICHARD, HOST: Coming up next on The World and Everything in It: Who oversees new developments in the use of human DNA?

NICK EICHER, HOST: It used to be a committee of scientists on the Recombinant DNA Advisory Committee, called RAC. The committee was formed in the seventies by the National Institutes of Health. These scientists aimed to give advice on the scientific and ethical issues involved with biotech. They oversaw the gene therapy experiments that allow scientists to replace, add, or alter human DNA.

REICHARD: But last month, NIH and the Food and Drug Administration announced plans to eliminate the committee’s gene- therapy oversight role. The reason for that is the idea that once risky science is now established medicine, best regulated by the FDA.

But some bioethicists aren’t so sure it’s a good idea. WORLD Radio’s Sarah Schweinsberg brings us this report.

SARAH SCHWEINSBERG, REPORTER: Jonathan Kimmelman is a professor of biomedical ethics at McGill University. He says the Recombinant Advisory Committee has played an important role in the field of genetic experimentation during the past 45 years.

KIMMELMAN: It’s really kind of cultivated and fostered the highest possible scientific standards.

Cultivating those standards is important because genetic therapy experiments can be dangerous. In 1999, a teenage boy died after a gene therapy treatment. Since then, RAC members have tried to mitigate the risk of other gene therapy experiments going wrong.

KIMMELMAN: We’ve discovered all sorts of challenges and risks beginning with risks of the immune system reacting to the virus that’s used to deliver the genetic sequence. Genetic sequences themselves, if they insert into your DNA, can trigger the onset of cancer.

Last year, with the RAC’s oversight, the FDA approved some of the first genetic treatments for lymphoblastic leukemia, lymphoma, and vision loss.

But now the NIH and FDA say the RAC will instead take on a broader—more ambiguous—ethical advisory role. NIH Director Francis Collins said in a statement the RAC and the FDA have been reviewing the same gene therapy applications and sometimes duplicating reviews and paperwork. Collins says eliminating RAC’s oversight role will streamline gene therapy regulations. So now, the FDA will vet gene therapy experiments on its own.

David Prentice is vice president and research director for the Charlotte Lozier Institute. He says in one sense the change isn’t a big deal. It simply eliminates overlapping governmental regulations and could actually speed up the process of approving lifesaving treatments. Right now there are 700 pending gene therapy proposals. But the shift needs to be done properly.

PRENTICE: It’s also only a good thing if someone submits an application to do a gene editing trial that there isn’t just an automatic stamp. It needs a very thorough review before proceeding.

RAC members include experts in molecular genetics, molecular biology, and recombinant DNA research. The FDA doesn’t have those experts.

PRENTICE: They are going to have to increase their professional expertise in-house to give this a thorough evaluation and vetting.

The RAC was extremely open with its reviews of gene therapy experiment  applications. The FDA is different.

KIMMELMAN: We know a lot about what goes on in gene therapy because it’s been a relatively transparent area because of RAC. When FDA reviews applications, they are reviewed in strict confidence, so there’s no way for the public to get a picture about how FDA’s making decisions.

When it comes to cutting edge, possibly risky gene therapies, Kimmelman says secrecy isn’t good.

Marcy Darnovsky is the director of the Center for Genetics and Society. Her concern is different. The RAC has taken a strong stance against the genetic modification of embryos, eggs, or sperm. It remains illegal in the U-.S. to initiate a pregnancy with a genetically modified embryo. Darnovsky worries the RAC’s changing role could create an opportunity for those pushing for a change in the law.

DARNOVSKY: One of the questions that we want to ask and don’t know the answer to is whether this new move will make for weaker or stronger, um, oversight of the proposals that are coming down the line now for reproductive germline modification.

Bioethics groups and scientists will have a chance to ask their questions during an ongoing public comment period. Charlotte Lozier’s David Prentice says if the proposed change gets final approval, the FDA needs to continue the RAC’s tradition of scientific excellence and public transparency.

PRENTICE: I think it’s incumbent on them, in fact, to be completely transparent in terms of what they are doing as they move forward.

Reporting for WORLD Radio, I’m Sarah Schweinsberg.

(Photo/Associated Press, File) This Oct. 14, 2015, file photo shows the Food and Drug Administration campus in Silver Spring, Md.

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