Novartis Needs Special Delivery for Potent Cell Therapy

Novartis AG has a promising therapy for cancer. It’s just not sure how to get it to patients easily.

The treatment is so potent that it cleared malignant cells in about 90 percent of patients facing almost certain death from the most common form of cancer in children.

The approach involves taking T cells, part of the body’s immune system, from the blood and engineering them to identify proteins on cancer cells. When returned to the patient’s bloodstream, the revamped T cells seek and destroy cancer cells. It’s so specific that a single mistake can mean death for a patient, and so turbo-charged that Novartis plans to set up a network of hospitals versed in treating the spiking fever, chills and flu-like symptoms that may come as side effects.

“The question is really if this is the right way to go at immunotherapy,” a burgeoning field of medicine that empowers the immune system to fight diseases such as cancer, Michael Leuchten, an analyst at Barclays Plc in London, said in an interview.

Cancer cells can use proteins on their surfaces as biological cloaks of invisibility to elude detection by the immune-system cells policing the body. Immunotherapies include checkpoint agents, drugs that strip away such disguises and expose cancer cells to attack; products such as Dendreon Corp.’s Provenge, which combines a patient’s immune cells with vaccine components in an infusion; and so-called biconjugated antibodies that help immune cells anchor themselves to cancerous ones.

$35 Billion Market

The total market may amount to a $35 billion “watershed” for cancer drugs, according to Andrew Baum, a pharmaceutical analyst for Citigroup Inc. in London. Baum sees Roche Holding AG and Bristol-Myers Squibb Co., based in New York, as the field’s leaders. Roche -- like Novartis, based in Basel, Switzerland -- is developing an infused immunotherapy which blocks a protein that prevents the immune system from attacking cancer cells. Bristol-Myers sells the drug Yervoy, which helps the immune system fight melanoma.

Unlike those therapies, Novartis’s CTL019 isn’t as easy to produce and transport. For the researchers and the company, the results are worth the effort. If they find a way to deliver the treatment to the masses, CTL019, also known as CART-19, has the potential to generate $10 billion a year if approved to treat multiple forms of cancer, according to Baum.

“CART-19 gives us a huge move into immunotherapy, a first-mover advantage,” Chief Executive Officer Joe Jimenez said during a conference this year.

19 Children

Nineteen out of 22 children who had exhausted all drug treatment and bone-marrow transplant options for acute lymphoblastic leukemia went into remission after receiving the therapy, also known as CART-19, according to data presented this month at the American Society of Hematology meeting in New Orleans. Five patients later relapsed, including one whose new tumor cells produced a protein that enabled them to elude the souped-up T cells.

In chronic lymphocytic leukemia, a much larger market, the therapy provoked a response in 47 percent of patients, with half of those patients experiencing a complete remission.

“There’s never been a therapy that works after a bone-marrow transplant fails,” Carl June, one of the treatment’s developers at the University of Pennsylvania, said in a telephone interview. Novartis licensed the technology from the university.

Salvage Treatment

The therapy’s money-making potential may be hampered by the complexity of its administration, as well as the emergence of powerful new drugs for leukemia, according to Richard Parkes, a London-based analyst at Deutsche Bank AG.

“CTL019 is likely to be used as a salvage treatment and we remain skeptical on its commercial opportunity,” Parkes wrote in a Dec. 10 note to investors.

Novartis plans to first develop CTL019 in acute lymphoblastic leukemia patients, then in the chronic lymphocytic form of the disease, followed by patients with lymphoma, another type of blood cancer, according to Herve Hoppenot, who heads Novartis’s oncology unit. Hoppenot declined to comment on the treatment’s commercial prospects.

“What we are planning to do is to make it a very easy and practical thing to use,” he said in an interview. “We are certainly going to start in centers that are specialized in sophisticated treatments. They won’t find it difficult. The burden will be on us to organize and calibrate the process.”

Larger Trial

The Novartis treatment first grabbed attention last year after the doctors reported that nine of 12 patients had been pulled from death’s door.

The company bought a plant for manufacturing immunotherapies from Dendreon for $43 million last year. The Seattle-based company sold the plant because it has struggled to persuade doctors to use Provenge, which it produced there. Like CART-19, Provenge is a treatment that uses altered cells to fight cancer.

Novartis plans to conduct a larger trial next year, Jimenez said at a Morgan Stanley conference in September. If the results are positive, Novartis would then ask regulators in 2016 for permission to market the therapy.

“The effectiveness gets people really excited, including me,” Bill Chambers, interim national vice president of extramural research at the American Cancer Society, said in a telephone interview. “It seems like things are being put in place. I’m very hopeful about this.”

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