FDA Recommends Approval Of Gene Therapy Which Alters Your Own Cells
Food and Drug Administration panel on Wednesday recommended approval of a first-of-its-kind cancer treatment involving gene therapy that uses patients’ revived-up immune cells to fight the disease.
If this therapy is approved, the first-ever such treatment will be the start of the new era of human medicine, in which we uses genetic engineering to boost our natural immune system and improve its ability to beat non-curable diseases.
This Gene Therapy Called as CAR-T, involves removing immune cells from a patients’ blood, reprogramming them to create an army of cells that can zero in on and destroy cancer cells and inject them back into the patient.
While this gene therapy, could be the first to reach the market, there are many more on the way. Treatments for an aggressive type of brain tumor, as well as myeloma and other varieties of leukemia, are also in the pipeline.
It’s an example of how gene therapy could revolutionize the personal medicine, in which a unique treatment for individuals is developed from his/her own cells. Which involves removal and isolation of cells from the patient and shipping them to the therapy maker, which further will process and ship back to the hospital where patient can take that therapy
Novartis, the drugmaker behind the CAR T-cell therapy, is seeking approval to use it for children and young adults whose leukemia doesn’t respond to traditional treatments — a group that numbers 600 or so patients a year in US. But the approach is also being tested for a range of diseases from non-Hodgkin lymphoma and multiple myeloma to solid tumors.
Unlike traditional gene therapy, the new treatment doesn’t replace disease-causing genes with healthy ones. Instead, it uses technology to reprogram immune cells called T cells to target and attack malignancies.
When a patient is treated with this therapy, T cells are extracted from a patient’s blood, frozen and sent to the company’s plant. There, the cells are genetically modified to attack cancer, expanded in number, refrozen and shipped back to the patient for infusion.
Once administered to the patient, the cells multiply exponentially inside the body and go hunting for the CD19 protein, which appears on a kind of white blood cell that can give rise to diseases, such as leukemia and lymphoma. The turnaround time for manufacturing the therapy, called “vein-to-vein” time, will be an estimated 22 days, Novartis officials told the committee Wednesday.
One attendee of the panel’s meeting was the first patient from the trials, Emily Whitehead, age 12. She almost died because of leukemia, which was considered fatal until she was treated at age 6. Since that time she has been living cancer free life. “We believe that when this treatment is approved it will save thousands of children’s lives around the world,” Tom Whitehead, Emily’s father, told the panel, according to The New York Times. “I hope that someday all of you on the advisory committee can tell your families for generations that you were part of the process that ended the use of toxic treatments like chemotherapy and radiation as standard treatment, and turned blood cancers into a treatable disease that even after relapse most people survive.”
Researchers at the University of Pennsylvania developed this treatment, which is now licensed to Novartis. The disease, however, is rare — affecting only about 5,000 people annually. Around 60 percent of them are young adults and children. Standard treatments can cure most children, but approximately 15 percent of patients, like Emily, do not respond to treatment or experience relapses are exceptional cases.
To ensure safety, Novartis is limiting the therapy’s availability to 30 to 35 medical centers. The company also plans to post Novartis employees at hospitals using the therapy and to follow patients for up to 15 years.
One of the big issues in CAR-T cell therapy — the cost, which analysts say could be in the hundreds of thousands of dollars as Novartis hasn’t released pricing information yet.