The genetic technology revolution

One of the most exciting and promising developments in the history of medical science may bypass the U.S. if the U.S. Congress fails to act. It needs to lift the ban on federal spending on research involving human embryos.

Other governments are already responding to the progress being made in genetic technology. The U.K. has given researchers permission to use an ingenious new gene-cutting technique called Crispr-Cas9 to investigate how human embryos develop. Chinese scientists have wielded the same tool to see if it‘s possible to fix, in human embryos, the gene responsible for beta-thalassemia, a deadly blood disorder. (So far, no.)

In the U.S., such studies are allowed -- but only if they are privately funded. Not only is there the congressional ban, but the National Institutes of Health has refused to fund research that uses gene-editing technologies on human embryos.

This prohibition, unfortunate in itself, poses a greater problem: When genetic techniques advance to the point where they might be used clinically -- that is, to safely prevent disease in human embryos that progress to live births -- the Food and Drug Administration won’t be able to license them. This gives Congress even more compelling reason to change course.

The FDA has pointed out this problem: Its own expert panel has recommended that medical scientists be allowed to pursue “mitochondrial replacement therapy,” or MRT. This is an experimental genetic technique that has the potential to prevent debilitating, sometimes deadly mitochondrial diseases with “three-parent embryos” (it swaps an embryo‘s flawed mitochondrial DNA with that of a healthy donor).

For such technology to move beyond the laboratory and into actual practice, however, the FDA would have to license it, as if it were a drug or a medical device. But the agency said Congress prevents it from “using funds to review applications in which a human embryo is intentionally created or modified to include a heritable genetic modification.”

And this is precisely what MRT does: It modifies an embryo in a heritable way. By the same token, the kind of human DNA-editing that Crispr is expected to enable -- to alter genes responsible for diseases such as Tay-Sachs, Huntington’s and sickle cell anemia -- would presumably result in heritable changes. So it‘s possible to imagine a future in which there are powerful medical weapons that would be prohibited in the U.S.

It’s true that these advances raise ethical and social concerns. In the case of MRT, offspring would have genes from three people -- nuclear genes from the main parents and mitochondrial genes from a separate “mother.” And the changes this would bring about, however beneficial, would be irreversible.

The way to deal with these concerns, however, is not to ban the practice. It is to carefully regulate and monitor it, and keep all participants sufficiently informed. This is the strategy recommended by the FDA panel and being followed in the U.K., whose parliament has authorized researchers to pursue MRT.

In other words, scientists should proceed with caution -- but they should proceed. The alternative is to ignore an exhilarating opportunity to alleviate human suffering.

Bloomberg