For the second time in 2022, the U.S. Food and Drug Administration (FDA) green-lit a cancer cell and gene therapy. Carvykti ® (ciltacabtagene autoleucel, or cilta-cel for short) is now approved as a treatment for relapsed or refractory multiple myeloma.
The newly approved CAR T-cell therapy can be used after four unsuccessful attempts with other approved myeloma therapies. This means Carvykti is intended for myeloma cases resistant to other therapies or consistently returning after treatment ends.
This news again acknowledges the power of CAR T cells against specific types of blood cancer – in this case, multiple myeloma. Five other CAR T-cell therapies have been approved by the FDA for various blood cancers such as lymphoma, leukemia and myeloma.
There are some remaining questions that patients and providers may have: What is this new treatment, and why does the FDA believe it’s needed for patients with this cancer? On this page, Alliance for Cancer Gene Therapy has compiled a detailed analysis of what Carvykti is and how its FDA approval can help people with multiple myeloma.
What is Carvykti?
Carvykti, manufactured by partner companies Janssen (a Johnson & Johnson subsidiary) and Legend Biotech, is a type of cancer cell and gene therapy called CAR T-cell therapy. CAR stands for chimeric antigen receptor. T cells are part of the immune system and help defend the body against attack from bacteria, viruses, or cancer. However, they often need outside help to slow the growth of cancer tumors.
CAR T-cell treatment adds laboratory-created genetic code to the patient’s T cells, which are then added back into the bloodstream primed to seek out and attack the cancer. The genetic code creates a new antigen receptor, which serves as a weapon for the T cells against cancer cells.
This new protein receptor is specifically designed to target the multiple myeloma cells. Specifically, Carvykti targets the B-cell maturation antigen (BCMA), which is proven to be overexpressed on myeloma cells.
Why did the FDA approve Carvykti for multiple myeloma?
The FDA approved the therapy based on the results of the recent phase 1 and phase 2 clinical trial CARTITUDE-1. The results of the trial, presented during the 2021 American Society of Hematology, saw 74% of patients in the trial reach the two-year survival mark. Around 60% of patients did not experience any disease progression (growth) for at least two years.
There were other hopeful statistics from the multiple myeloma trial:
- 98% objective response rate (either the tumor stopped growing or shrank)
- 78% complete response rate (the tumor disappeared)
- 21.8 months median duration of response (how long the treatment was effective)
“The responses in the CARTITUDE-1 study showed durability over time, and resulted in the majority of heavily pretreated patients achieving deep responses after 18-month follow-up.” — Sundar Jagannath, MD, Director of the Center of Excellence for Multiple Myeloma
What does the FDA approval mean for multiple myeloma patients?
The FDA’s decision provides another option to patients with this disease. According to the American Society of Clinical Oncology, nearly 35,000 Americans are diagnosed with multiple myeloma each year. The five-year survival rate is 54%.
Under the current FDA approval, Carvykti is an option for people who have forms of multiple myeloma resistant to other therapies. Patients must attempt four lines of therapy from three other therapeutic classes:
- Immunotherapy agents, such as drugs that stimulate the immune system.
- Anti-CD38 monoclonal antibodies, which block the cancerous activity of the CD38 protein.
- Proteasome inhibitors, which counter the protein-recycling process needed for myeloma cells two grow.
If all three don’t provide a long-term solution, then Carvykti becomes an approved option.
Promising future ahead for CAR T-cell therapy
The FDA’s approval also shows the agency’s belief in CAR T-cell therapy as a viable cancer treatment option of the present and future. The novel approach is proven to work for blood cancers, including lymphoma and leukemia. The next frontier is solid tumor diseases, such as pancreatic cancer and glioblastoma.
CAR T-cell therapy is not the only cell and gene therapy approved for cancer. Last month, the FDA endorsed Kimmtrak ®, a T-cell receptor therapy, for a type of melanoma that forms in the eye.
Every new FDA approval gives patients access to a wider variety of treatments. These authorizations also reaffirm Alliance for Cancer Gene Therapy’s belief in cell- and gene-based therapies as the best path forward for cancer treatment. Alliance for Cancer Gene Therapy continues to champion CAR T cells and other cell and gene therapies for solid tumors by funding research projects.
With your donation today, the Alliance will be able to fund more innovative research from the brightest minds in oncology. We will use 100% of your donation toward advancing these new cancer-fighting therapies.
- Multiple Myeloma: Statistics. American Society of Clinical Oncology. Retrieved from: https://www.cancer.net/cancer-types/multiple-myeloma/statistics. Accessed: 03/01/2022.
- Survival Rates by Stage for Multiple Myeloma. American Cancer Society. Retrieved from: https://www.cancer.org/cancer/multiple-myeloma/detection-diagnosis-staging/survival-rates.html. Accessed: 03/01/2022.
- FDA Approves Cilta-cel to Treat R/R Multiple Myeloma. American Journal of Managed Care. Retrieved from: https://www.ajmc.com/view/fda-approves-cilta-cel-to-treat-r-r-multiple-myeloma. Accessed: 03/01/2022.