FDA Landmark Decision Opens Gates for Future Gene Therapies

Allie Carter (CMC ’19)

In July 2017, the Food and Drug Administration (FDA) made the landmark decision to permit gene therapy in the United States when they approved the leukemia treatment, Kymriah. The FDA’s decision to approve Kymriah both increases treatment options for many leukemia patients, and may lead to increased options for many others for whom innovations in gene therapy will benefit. While it has been a source of controversy for over 40 years, gene therapy has the potential to exponentially revolutionize modern healthcare and medicine. Kymriah’s approval is indicative of a changing public opinion on genetic testing and modification. Further, the FDA’s approval of Kymriah is a tipping point for additional gene therapy technology and development.

Gene therapy serves as means to either replace, destroy, or introduce genes.  Described by FDA commissioner Scott Gottlieb as “a new frontier in medical innovation,” gene therapy has the potential to effectively treat diseases thought to be incurable. Because of the patient-safety concerns that accompany gene therapy, there is a streamlined application and authorization process for any company seeking FDA approval.  

Any potential gene therapy product designed for a clinical trial that aspires for subsequent commercial use must complete an Investigational New Drug (IND) application. The FDA is the only agency with the jurisdiction to authorize or reject an IND application. The FDA will authorize an IND application if the manufacturer sufficiently proves through research that provides evidence of the risks associated and accordingly, the measures enacted to protect participants. After completing an IND, a proposed gene therapy must be approved by the Institutional Review Board, which is composed of scientific and medical advisors. Thus, approval through the FDA seems to be an apolitical process.

However, gene therapy has been a source of political controversy since its creation. One of the sources of its controversy is the ethical implications of manipulating someone’s genes. Additionally, due to gene therapy’s high cost, those who oppose gene therapy view it as means to further divide society based on one’s socioeconomic standing. Even though Kymriah is not generally considered manipulating someone’s genes, it remains controversial because of the high cost associated with the treatment.

Laws and regulations concerning gene therapy have proven to fluctuate depending on the prominent political party at the time. For example, Republican President George W. Bush initiated a policy that drastically restricted National Institute of Health (NIH) funding for stem cell research and prohibited the creation of new stem cell lines. Often, conservatives opposed to genetic modification compare gene editing to the eugenics movement and believe that through gene editing, one gives up their right to autonomy. Conversely, Democratic President Barack Obama authorized an executive order to remove barriers to scientific research involving human stem cells.  President Obama’s executive order increased NIH funding for stem cell research and for the previously prohibited creation of stem cell lines. The fruits of this increased funding are evident today, and this may be a contributing factor to the FDA’s landmark approval.

Representative of the positive attitude the FDA has towards Kymriah, the director of the FDA’S Center for Biologics Evaluation and Research (CBER) Peter Marks commented that Kymriah is an extremely effective new treatment, as proven through clinical trials. For this reason, the FDA gave Kymriah Priority Review, which marks it as a Breakthrough Therapy on a fast track to approval. To approve Kymriah, the FDA evaluated the therapy employing a cross-agency method. The FDA’s Oncology Center of Excellence was responsible for observing the treatment in action, while CBER evaluated the other elements needed to be reviewed to make a holistic decision.

Currently, the FDA is reviewing a number potential gene therapies, such as voretigene neparvovec. The success of Kymriah will be a determining factor for future gene therapy implementation. Already, Kymriah is influencing the success of other gene therapies. The FDA’s approval of gene therapy is indicative of a changing public opinion of genetic testing and manipulation. As In Vitro Fertilization, stem cell therapy, and DNA genetic testing like popular 23andMe become more commonplace, they pave the way for a greater societal acceptance of gene therapy.  

Francis Crick, Nobel Prize winning biophysicist credited with the discovery of the double helix model of deoxyribonucleic acid (DNA) once said, “We used to think that our fate was in our stars.  Now we know that, in large measure, our fate is in our genes.”  If the FDA’s decision to approve Kymriah is indicative that gene therapy will soon become more commonplace, Kymriah has drastically altered the trajectory of modern science and health care. The FDA decision could very well be a tipping point for additional gene therapy technology and development.  

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