5 Milestones Of 2017 In The War On Cancer

It is impressive that FDA have approved 45 novel drugs in 2017 so far — more than twice as last year’s total. A dozen of those new products were in oncology, as the new year coming new choices for treating a range of cancers, including mantle cell lymphoma, Merkel cell carcinoma and follicular lymphoma.

But what’s notable about the FDA’s list of new chemical entities approved during the year is that it doesn’t include many of the other advances that were seen in the world of oncology research. That’s a separate list, which includes novel combinations of previously approved drugs, and the entry of personalized immune-cell therapies—CAR-T treatments—that have offered hope to thousands of patients who had run out of options for treating their cancers.

The bottom line is that it has been a banner year for cancer research. Here were some of the highlights:

May 23: The FDA approves the first cancer treatment that’s prescribed based on the genetic characteristics of the disease—not the tumor’s location. Merck’s immuno-oncology drug Keytruda won approval to treat patients whose tumors are defined as microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR). About 5% of colorectal tumors have one of these two characteristics, but the defects are also found in other solid tumors, such as breast cancer, gastrointestinal cancer and prostate cancer.

MSI-H and dMMR tumors are unable to properly repair the DNA inside cells. Keytruda works by blocking "checkpoints" that would normally prevent the immune system from recognizing and attacking these defective cells. Merck tested Keytruda in 149 patients with 15 different MSI-H or dMMR cancer types and charted impressive results: More than 39% of patients had at least a partial response to the drug, and 78% of those people responded well for six months or longer.

August 30: The FDA approves the first CAR-T treatment, Novartis’s Kymriah, for some young people with leukemia. When results from trials of CAR-T treatments first started emerging a few years back, it seemed too good to be true: Cure rates for previously untreatable blood cancers were 70% and higher. But the results held up, and Novartis took home the first FDA approval in this booming area of cancer research.

Kymriah is made by removing immune-boosting T cells from patients, engineering them to be able to recognize and kill their cancers and then re-infusing them. It’s a hugely complex process for a one-time treatment, and it’s priced at a whopping $475,000. But Novartis has proven to be a pioneer not just in the invention of CAR-T but also in its pricing: The company struck a deal with the Centers for Medicare and Medicaid Services (CMS) stipulating that the agency will only have to pay for the treatment when patients respond within the first month.

October 18: Kite Pharma wins approval for its CAR-T, Yescarta, just weeks after cementing its $11.9 billion purchase by Gilead. When Gilead announced its planned purchase of Kite in August—one of the biggest oncology deals of recent years—no one was surprised. Gilead had been under pressure to make a purchase that would boost its pipeline, and Novartis was on the verge of proving that personalized cell therapies can be embraced by regulators.

Yescarta was approved to treat some adult patients with large B-cell lymphoma. There have been some hiccups on the way to market, including reimbursement difficulties that have resulted in long waiting lists for the product. But analysts are optimistic about the $373,000 product, estimating it could bring in as much as $250 million next year.

December 10: Tiny Bluebird Bio stokes optimism for CAR-T in a third type of blood cancer. Shares of gene therapy startup Bluebird Bio shot up 18% to $201.80 in a day after the company announced results from a small trial of its CAR-T in multiple myeloma. The treatment, which is being co-developed by Celgene, is different from the two previously approved CAR-Ts in that it targets a protein on myeloma cells called BCMA. The 18 patients in the trial had failed multiple previous therapies, and 17 of them responded positively to Bluebird’s CAR-T. In 10 of those patients, the cancer seemed to disappear, the companies reported at the annual meeting of the American Society of Hematology (ASH).

Early successes with CAR-T have prompted researchers around the world to see if they can apply T-cell technology to multiple cancer types, including solid tumors. Novartis and Kite are among the companies that have been working on CAR-Ts for solid tumors.

December 20: The FDA approves its 12th cancer combination treatment of the year. The rise of immuno-oncology treatments brought with it a prediction that combining multiple drug modalities might make it possible to conquer previously untreatable cancers. Several companies succeeded this year in persuading the FDA that previously approved products, including immuno-oncology treatments and targeted drugs, work better in groups than do on their own. Hence 12 new cancer combinations were approved in 2017—way up from the five combos approved in 2016.

The most recent of these approvals was awarded to Roche unit Genentech, for its combination of Perjeta, a monoclonal antibody targeted at HER-2 positive breast cancer, with the similarly targeted drug Herceptin and chemotherapy, in patients who face a high risk of recurrence. Earlier in the year, the FDA granted an accelerated approval for Keytruda combined with Eli Lilly’s Alimta and carboplatin for the first-line treatment of patients with metastatic non-squamous non-small cell lung cancer.

No doubt 2018 will bring more immuno-oncology advances—and combination treatments designed to outsmart cancer from multiple sides.