Groundbreaking Cancer Treatment News

In September 2017, the US Food & Drug Administration (FDA) announced approval of Novartis’s new anti-leukemia drug Kymriah. Kymriah is a patient-specific drug expected to be paramount in increasing the life expectancy of patients under 26 who have been diagnosed with a specific form of Acute Lymphoblastic Leukemia (ALL). The current prognosis for ALL indicates 10% of ALL patients will survive 5 years after diagnosis. With the use of Kymriah, 83% of patients in the trial achieved complete and prolonged remission.

What makes Kymriah unique is that it uses a patient’s own immune cells as an active ingredient in Chimeric Antigen Receptor T-cell therapy (CAR-T). The manufacturing process for Kymriah takes three to four weeks and begins by taking a sample of the patient’s blood. T-cells are separated from the blood in much the same way albumin protein is isolated. The T-cells are then sent to a manufacturing facility where a new gene is used to turn the T-cells into targeted leukemia fighting machines by using an engineered viral vector to deliver genetic information to the T-cells.

Kymriah appears to be an effective treatment for ALL, but researchers are also excited about the applications to other, more common diseases. The process used to make Kymriah essentially reprograms a patient’s immune system, so it can attack diseased cells in much the same way it would fight off an infection. Diseases that could potentially be treated in this way include other blood cancers, solid-tumor cancers, and autoimmune diseases like Type I diabetes.

The current standard of treatment for ALL is a bone marrow transplant. Kymriah is designed to be a one-time treatment. Industry analysts justify its $475,000 price tag when they compare its efficacy and advantages to those of a bone marrow transplant. Kymriah offers enhanced quality of life and longer life expectancy for patients.

Engineers will play a critical role in this level of personalized care as each individual’s cells are transformed into immunocellular drugs. This is the first gene therapy of any kind approved for use in the U.S. Moving forward, considerations will have to be made regarding the long-term safety of gene therapy as well as global regulatory issues, control, and quality assurance. The approval of Kymriah brings with it issues never before seen in the life science.

Because each drug is specific to the patient and created from their own cells, Novartis will need to figure out how to scale up production of Kymriah while maintaining quality and efficiency when no two batches of the drug will ever be the same. Besides regulatory issues, there will also be economical, logistical, and manufacturing processes to streamline as well as quality control testing to maintain throughout the process.

The FDA approval of Kymriah is amazing news for Novartis as well as the pharmaceutical industry. This opens doors for research and development of other immunocellular drugs and is a great example of the types of research and treatments Lifecycle Biotechnologies supports. To learn more about Lifecycle and how we can assist you, contact us today.