The FDA Approved Two Gene Therapies: What it Means for Cancer Care

Hannah Caplan

November 29, 2017

Acute lymphoblastic leukemia (ALL) is the most common childhood cancer, accounting for about 25% of all cancer diagnoses in children under the age of 15.¹ In August, the FDA approved the first gene therapy in the United States, Kymriah, and marked the beginning a new, revolutionary approach to cancer treatment.² Kymriah is FDA-approved for pediatric and adolescent patients with ALL of B-cell origin for which initial treatment has not worked.² The success of this gene therapy, albeit in a narrow sample, suggests that it may be possible to eventually treat many forms of cancer without toxic interventions such as chemotherapy and radiation.

Kymriah is a customized gene-therapy treatment that reprograms the patient’s immune cells to identify and target specific cancer cells.² Treatment involves collecting a sample of the patient’s T-cells, which play an important role in immune response. Scientists then add a gene to the patient’s T-cells that signals the cells to recognize an ALL-specific antigen on the leukemia cell.² Once re-inserted, these genetically altered T-cells are able to identify and kill the leukemia cells. In a multi-center clinical trail of 63 pediatric and adolescent patients, the treatment achieved an 83% remission rate.² In the press announcement, FDA Commissioner Scott Gottlieb, M.D. noted that “we’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer.”²

Yet, the treatment comes at a high cost, with a cost of one-time-use currently priced at $475,000.³ Novartis justifies this price by the 83% success rate achieved among the approved population and has suggested that they will use indication-based pricing for other populations once approved.³ Perhaps most notable is how Novartis is working with the Centers for Medicare and Medicaid Services (CMS).³ If Kymriah is not successful within the first month, Novartis has said that it will not charge for the drug.³ While relapse may occur after one month, this guarantee is at least placing some of the financial risk on the drug company.

Since August, the FDA has also approved a similar gene therapy, Yescarta, for a type of lymphoma in adults who have not responded to traditional treatment.⁴ Taken together, these approvals signal that gene therapy is no longer just a promising new field with potential applications in the future. There are now proven, effective gene therapies on the US market that will reshape the way we treat disease. The demonstrated success of these two gene therapies, along with the support of the FDA, will hopefully add momentum to the human genome movement and empower researchers to continue discovering genetic linkages to diseases. As genome-modifying tools evolve they will play an increasingly powerful role in shaping the way that we approach the prevention and treatment of disease.

References

1. Childhood Acute Lymphoblastic Leukemia Treatment (PDQ®)–Patient Version. National Institutes of Health National Cancer Institute. Available at: https://www.cancer.gov/types/leukemia/patient/child-all-treatment-pdq
2. FDA approval brings first gene therapy to the United States. US Food and Drug Administration. Available at: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm574058.htm
3. Read the Fine Print on the $475,000 Price of a Cancer Breakthrough. Bloomberg BusinessWeek. Available at: https://www.bloomberg.com/news/articles/2017-08-31/novartis-kymriah-cancer-drug-475-000-but-read-the-fine-print
4. With FDA Approval for Advanced Lymphoma, Second CAR T-Cell Therapy Moves to the Clinic. US Food and Drug Administration. Available at: https://www.cancer.gov/news-events/cancer-currents-blog/2017/yescarta-fda-lymphoma

Hannah is a senior majoring in Public Health at the George Washington University Milken Institute School of Public Health.