A Food and Drug Administration advisory committee will hand down a recommendation Wednesday, July 12 regarding whether a cutting edge personalized treatment will be allowed for some blood cancer patients.
The Oncologic Drugs Advisory Committee will examine the safety of CAR T-cell treatments, an immunotherapy manufactured by Novartis AG (NVS - Get Report) for treatment of children and young adult leukemia patients who have not responded to traditional treatments.
The treatments involve extracting white blood cells or T-cells which defend the immune system, from cancer patients. The cells would be frozen and sent to Novartis's facility, where they would be engineered to hunt for a protein marker called CD19 found in some blood cancers. The cells would be altered to seek out the cancer and attack it. The cells would then be refrozen and sent back to the patient. Once infused back into the patient, the cells reproduce at a fierce pace to attack the cancer.
In Novartis CTLO19 trials, 83% of the children patients were put into remission at the 90-day mark, a remarkable rate. Brad Loncar, a longtime biotech investor who runs an investment fund based on immunotherapy stocks, said, "CAR T is a revolutionary treatment that offers hope to a very advanced segment of cancer patients."
Immunotherapy has been hailed as the next great tool in fighting cancer and other life threatening diseases, but the personalized approach to medicine is not without a serious risk. By supercharging a patient's ability to battle cancer, the body is also exposed to significant side effects. In Novartis trials, 32 patients experienced cytokine release syndrome, a condition with high fever and symptoms that resemble a serious flu that can send patients into the ICU with a life threatening condition.
Another 30 patients developed neurologic symptoms that included seizure, hallucinations and delirium. In a separate study of a CAR T-cell therapy by Juno Therapeutics Inc. (JUNO) , five patients engaged in the trials died from fatal brain swelling and the study was shuddered by the FDA.
Loncar acknowledges that the push-the-envelope treatment carries risk. "While the toxicities and logistical challenges are important issues worthy of careful thought and discussion, I believe this will earn a positive panel recommendation and be approved due to the positive outcomes for patients who do respond. CAR T is a potentially curative for some patients who have an untreatable disease."
An experienced biotech investor, Loncar created the Loncar Immunotherapy Index, a collection of 30 life science companies with five large-cap and 25 growth stocks. He said the advisory committee will need to make a very human calculation. "It must be noted that the patients being treated in these studies are very sick and likely facing certain death so there is a risk/reward trade-off the FDA will have to consider."
The July 12 meeting is focused on the safety of Novartis CAR T therapy. "The overall effectiveness of this product is not the primary issue for consideration by this committee," the FDA staff preview said.
Should the committee greenlight the immunotherapy, the FDA would likely take it up for a full approval in September. But the regulator is already sounding a note of caution saying that "postmarketing considerations for long-term safety monitoring may be necessary to address the potential safety concerns."
Novartis is well aware that the regulator is taking a wary look at CAR T therapy. The pharma giant plans on a muted introduction of the drug, relying on medical centers that are familiar with the treatment from its trials as well as focused training.
The FDA has approved a type of immunotherapy before, a type of drug known as checkpoint inhibitors for use in fighting solid tumors. In all, five different checkpoint inhibitors gained approval since 2011 for use against lung and bladder cancers as well as melanoma. Opdivo by Bristol-Myers Squibb Co. (BMY - Get Report) and Keytruda from Merck & Co. (MRK - Get Report) are two high-profile examples of checkpoint inhibitors.
But the immunotherapies that have been approved by the FDA are the same for each patient, unlike the CAR T therapy being considered by the FDA committee. But both types of immunotherapies act to amplify the body's ability to fight disease.
Kite Pharma Inc. (KITE) , a Santa Monica, Calif. biotech also has a CAR T therapy before the FDA to treat lymphoma but is a couple months out.
In an investor note Loncar said, "I predict that both Novartis and Kite will earn approval in the coming months, and this will be an inflection point for cellular therapies. This unique method of treating cancer is very special and has the potential to revolutionize the treatment of many cancers in the future."
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