The Promising New Cancer Treatment Behind Gilead's $11 Billion Deal

Gilead Sciences Inc.'s planned acquisition of Kite Pharma Inc. underscores the promise of a new type of cancer therapy that is on the brink of becoming commercially available in the U.S.

Kite's version of the treatment, which uses the body's own immune system to attack cancer, is up for Food and Drug Administration approval, while a rival treatment won a key regulatory nod in the U.S. earlier this year.

While promising, the treatments, generally called CAR-T, won't be like other drugs that win FDA approval, and then quickly wind up on pharmacy shelves and hospitals. The rollout of this new breed will be complicated by still-unresolved questions over pricing and availability.

Kite is one of the leading companies developing CAR-T, which stands for chimeric antigen receptor T-cell, therapy. Such treatments work by extracting a cancer patient's T-cells, a type of immune cell, genetically modifying them outside the body to make them more effective at hunting down and killing tumors, and then re-injecting them into the patient.

After winning unanimous approval from an advisory panel to the FDA, Switzerland's Novartis AG is expected very soon to get the first official green light to start offering the treatment.

Novartis's treatment, called CTL019, has been shown to dramatically raise the chances of survival for children and young people with leukemia and who don't respond to standard treatment, or who suffer a relapse.

So far, CAR-T therapies have been tested only in certain types of blood cancer. Kite Pharma's leading CAR-T effort is aimed at patients with aggressive non-Hodgkin lymphoma, in whom standard therapy has failed.

Kite and Novartis are both investigating several more CAR-T therapies for various forms of blood cancer. Novartis is also conducting early-stage CAR-T trials in certain types of brain and lung tumors.

Despite its limited use now, "for patients with the right disease, the response rates can be astonishing," said Stephan Grupp, who was part of the team that first developed Novartis's CTL019 at the University of Pennsylvania.

Novartis's rollout of its leukemia treatment will be closely watched for signs of how well other CAR-T treatments, including Kite's, will be received.

In the U.S., only certain specialist hospitals are qualified to carry out the Novartis procedure. Receiving CTL019 treatment is a weekslong process involving several steps. But unlike most cancer therapies, patients require just one round of treatment.

First, the patient undergoes a dialysis-like procedure to remove T-cells from their blood, which takes about a day. The T-cells are then frozen and sent by courier to a Novartis facility in New Jersey, where they are genetically modified to equip them with a molecular "warhead" designed to hunt down cancer cells in the blood. The enhanced T-cells are then multiplied in the lab until there are many millions of them. The company has said this process takes around 22 days.

The modified T-cells are sent back to the hospital, and infused into the patient. About a week before the infusion, the patient receives a round of chemotherapy to prepare them for the treatment. The chemo reduces the amount of tumor in the blood and suppresses the patient's immune system to allow the new T-cells to multiply further inside the body.

There are side effects. Most patients develop a condition called cytokine release syndrome, caused by the immune system going into overdrive. This resembles a very severe flu and usually requires hospital care to manage.

Novartis plans a gradual roll out, starting with the 13 hospitals that were involved in its clinical trials and a handful of other specialized centers. A spokeswoman said the company expects 30 to 35 sites to offer CTL019 by the end of the year, adding that the slow ramp up was in the interest of patient safety.

"Novartis has been very careful which centers they selected," said Stephen Gottschalk, who leads the bone-marrow transplant and cellular-therapy department at St. Jude Children's Research Hospital in Memphis, Tenn., which is among the centers preparing to offer CTL019. A broader rollout to nonspecialist hospitals would carry the risk of increased complications or deaths from CAR-T, he said.

Expense could also present a hurdle: A study by England's National Institute for Health and Care Excellence, an official body that analyzes the cost-effectiveness of medical treatment, said CAR-T procedures could command a price of up to GBP528,600 ($680,900) because of their potential to cure patients who are otherwise untreatable.

Novartis hasn't yet disclosed the price it will charge for CTL019. Its spokeswoman said the price would take into consideration "the value that this treatment represents for patients, society and the health-care system, both near-term and long-term." She added that Novartis was exploring payment models that would make the price dependent on patient outcomes.

The cost of manufacturing CAR-T is exponentially higher than typical cancer drugs, according to Bill Milligan, a spokesman for the International Society for Cellular Therapy, a nonprofit that supports the development of CAR-T, stem-cell therapies, and other treatments that use living cells.

Safety concerns have also cast a shadow over some development efforts. Juno Therapeutics Inc., based in Seattle, dropped its most advanced CAR-T candidate earlier this year after several patients died during a clinical trial because of swelling in the brain.

Juno plans to publish a detailed report on the deaths later this year. It continues to develop CAR-T therapies and earlier this year published the results of an early-stage trial for its now-leading product, aimed at non-Hodgkin lymphoma, in which patients experienced relatively low rates of serious side effects.

(END) Dow Jones Newswires

August 28, 2017 12:11 ET (16:11 GMT)