(Bloomberg) -- Novartis AG is pushing ahead with one of its most ambitious cancer therapies, a treatment that the Swiss drugmaker says has blockbuster potential as it extends that technology to a wider pool of tumor-ridden patients.
Europe’s second-biggest drugmaker is planning to test its CAR-T treatments -- which involve extracting immune cells and genetically engineering them to hunt and kill cancer cells before returning them into the patient’s body -- on lethal cancers of the brain, pancreas, colon, ovary and lung. The company has also doubled its investment in manufacturing for these treatments.
The efforts put Novartis in a race with Juno Therapeutics Inc., Kite Pharma Inc. and Bluebird Bio Inc. to master the technology, part of a class of therapies that harness the body’s defense system to attack tumors. Novartis’s initial target is an acute form of leukemia in children, and it expects to seek approval from the U.S. Food and Drug Administration early this year. Analysts estimate the therapy, CTL019, will cross $1 billion in annual sales in five years.
“This could be the most efficacious immune therapy as yet developed, and it’s just early days with this technology,” Jay Bradner, president of the Novartis Institutes for BioMedical Research, said in an interview at the company’s headquarters in Basel, Switzerland.
Novartis is trying to catch-up with rivals including Merck & Co. and Roche Holding AG in cancer immunotherapies. The company, which invested about $9 billion last year in research and development, has more than 30 cancer assets under development and is “investing massively” in immuno-oncology, according to Bruno Strigini, the oncology chief executive officer at Novartis.
Still, questions arose about Novartis’s commitment to CAR-T research after the drugmaker said in August that the work would no longer be housed in a separate division, a restructuring that included cutting 120 jobs.
The CAR-T sector itself has faced its share of skeptics: use of the treatments beyond certain blood cancers and the feasibility of large-scale manufacturing of such personalized cancer therapies are yet to be proven. And while the experimental treatments have shown dramatic results, they have also led to severe side effects, and in some cases death.
Strigini says Novartis is confident about the balance between the safety and efficacy of its therapies. The next step will be seeking approval for the use of its CAR-T therapies in patients with diffuse large B-cell lymphoma, a type of blood cancer that afflicts a larger group of people. That’s likely to happen by the end of 2017, he said.
Beyond that, Novartis, working with the University of Pennsylvania, is also exploring using its treatments on so-called solid tumors, according to Glenn Dranoff, who leads immuno-oncology efforts at the Novartis Institutes. CAR-T therapy has been shown to work against cancers in the blood but has yet to be proven against solid tumors, or cancers that occur in bones, muscle and organs.
There’s no doubt that treating solid tumors with CAR-T cells will be more challenging, partly because T cells have to make a bigger effort to get into the tumor from the blood, according to Marcela Maus, director of cellular immunotherapy at Massachusetts General Hospital’s cancer center. And there may be better approaches, she said.
“T-cell therapies are a new platform — the trick now will be extending it to more cancers and having a better understanding of how and which patients can benefit the most,” Maus said.
Competitors are also seeing signs of success. Kite in December said it’s seeking FDA approval for its CAR-T therapy in a type of lymphoma. The same month, Juno said most patients with a form of blood cancer responded to its experimental treatment with manageable side effects, which may help invigorate the company after a different trial was halted due to patient deaths.
Bradner, who joined Novartis a year ago from Harvard Medical School and the Dana-Farber Cancer Institute, said the company doesn’t expect any regulatory surprises for its therapy based on discussions so far. More than 80 percent of patients went into remission in a mid-stage study published in December evaluating its CAR-T therapy in children and young adults with acute lymphoblastic leukemia, a fast-growing disease.
Some patients are likely to be free of the disease for five years or more following treatment with CAR-T, Bradner predicted.
“It’s tantalizingly close to ‘cure’ though it’s hard to know with certainty when that happens or how frequently patients will have a long interval where they can feel and be cancer free,” Maus said.
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