The 21st century has ushered in an era of incredible advancements in medicine. From the first uses of online patient portals to nanomedicine, how people are treated for their health has changed drastically. It then follows that as medicine itself develops, the regulations and protocols surrounding medicine change with it. In the United States, the Food and Drug Administration (FDA) is the gatekeeper of all new medicines. Each year, the FDA approves only dozens of novel drugs, approving fifty-five in 2023 and thirty-seven in 2022. At the end of 2023, the FDA approved the first CRISPR/Cas9-based gene therapy for treatment, implicating new questions surrounding regulations of the powerful yet controversial technology and its other applications.
CRISPR/Cas9 is the nickname for “Clustered Regularly Interspaced Short Palindromic Repeats” and “CRISPR Associated Protein 9.” It is the leading technology for editing the human genome. The applications of CRISPR are virtually limitless, from treating genetic diseases to organ transplantation to altering the genetic code of future generations. The FDA approved clinical trials of CRISPR for treating genetic diseases in 2021, effectively treating patients with sickle cell disease. In just a few short years, these treatments were greenlit for treatment in the United States. However, as CRISPR technology advances, regulations have limited its other uses. Currently, there is a ban in the United States on using federal funds for editing the genes of future generations while embryos (i.e., “germline editing”), prohibiting the FDA from approving any clinical trials for such modification and the National Institute of Health (NIH) from funding research. However, germline editing is not technically illegal in the United States—any research must use only private funding, though it is globally discouraged. The question is: are the existing regulations on germline gene editing proper?
There are clear ethical concerns about editing human embryos that make it widely acceptable amongst researchers that the brakes have been pumped on developing the technology, which isn’t quite ready for human trials. The lack of a specific ban on embryonic research has led some to push for stricter regulations to eliminate it altogether. There are concerns about genetic diversity becoming limited over time, which would not be seen within the first generation of genetically modified persons but later on. While a parent may undoubtedly want to edit a genetic disease out of their child, it becomes a public policy concern about generations down the line that may be adversely affected by dwindling genetic material. Further, unborn children cannot consent to alterations—and those alterations aren’t always accurate, and could lead to additional health issues.
Conversely, some have argued for looser regulations on germline gene editing, specifically in favor of research forthree-parent embryos. This involves using DNA from the father and the mother as well as mitochondrial DNA from a donor mother to prevent mitochondrial diseases from being passed onto the child. Properly done, it would help couples conceive while mitigating or eliminating the risk of passing on genetic diseases. This is a less controversial use of the technology already approved for clinical trials in the United Kingdom, but research has been dampened in the United States by the above regulations.
While CRISPR has recently received FDA approval for some applications, its use has been hindered in others. As the technology produces more treatments, it will gain efficiency and accuracy with increased research on the new type of medicine. With this increased accuracy, it may come time to reevaluate the current regulations regarding germline editing. Some may argue that Congress, the FDA, and the NIH have not done enough to restrict it and need to ban research on germline editing immediately. Others may argue to allow federal funds to be used for research and increase reproductive freedoms. Regardless, there is a growing conversation surrounding the use of CRISPR gene editing in medicine that has only become louder following FDA approval for one application.