Even though cancer rates in both men and women have been steadily declining since their peak in 1991, cancer remains the second-leading cause of death in the United States behind cardiovascular disease, according to the Centers for Disease Control and Prevention. Fortunately, the ongoing innovation boom in biotechnology has produced some game-changing new cancer treatments recently.
In 2017, for instance, the U.S. Food and Drug Administration approved dozens of new oncology drugs and line extensions for medicines already on the market. Moreover, some of these approvals were the very first of their kind for a particular therapy (CAR-Ts), or marked the first approval for a specific indication in several decades. Armed with this insight, here's a look at the three biggest breakthroughs in cancer treatment this year.
New tools in the battle against acute myeloid leukemia
Acute myeloid leukemia, or AML, isn't a single disease per se, but rather a broad collection of disorders that typically arise from the blood-forming component of bone marrow responsible for producing white blood cells. Around a quarter of all newly diagnosed adult leukemia's reportedly fall under the umbrella of AML.
On April 28, 2017, the FDA approved Novartis' (NVS -2.04%) midostaurin (brand name, Rydapt) for treating newly diagnosed FLT3-mutated AML, as well as three types of systemic mastocytosis. Rydapt is indicated for FLT3-mutated AML patients as part of a combo therapy that includes standard cytarabine and daunorubicin induction and cytarabine consolidation.
Why is Rydapt a big deal? This drug is the first medicine approved for this particular subset of AML patients in a quarter of a century. In clinical trials, Rydapt also produced a noteworthy 23% reduction in the risk of death, which is a major advancement for this particularly aggressive malignancy. On the commercial front, Wall Street analysts expect Novartis' new AML drug to rack up as much as $250 million in annual sales within a few short years.
On Aug. 1, 2017, the FDA doubled down on its fight against AML by green-lighting Celgene Corp. (CELG) and its partner Agios Pharmaceuticals' drug Idhifa, indicated for adult patients with relapsed or refractory AML with an isocitrate dehydrogenase-2 mutation as detected by an FDA-approved test. Celgene estimates that Idhifa can generate upwards of a whopping $500 million in peak sales, even as a niche AML therapy.
Taken together, Rydapt and Idhifa arguably mark a major paradigm shift in the available treatment options for AML patients. As more precise therapies advance in the clinic in the years ahead, the prognosis for this life-threatening blood and bone marrow disorder should improve markedly.
Anti-cancer gene therapies became a reality in 2017
On Aug. 30, Novartis hit another milestone in the cancer arena by gaining the first-ever regulatory approval for a chimeric antigen receptor T cell therapy (CAR-T) with tisagenlecleucel (brand name, Kymriah) for the treatment of patients up to age 25 years with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.
The fate of these promising genetically modified T cell therapies have been in question since their initial development because of their serious, and sometimes deadly, side effects. Thus, Novartis' ability to shepherd Kymriah through a highly successful clinical program, and then onto the market, is no small feat.
Not to be outdone, Gilead Sciences (GILD 0.87%), through its Kite Pharma subsidiary, quickly followed suit by gaining the second CAR-T approval this year with Yescarta (axicabtagene ciloleucel). Gilead's CAR-T therapy is indicated for adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy.
While both Novartis and Gilead's CAR-T therapies are forecast to eventually become blockbuster anti-cancer products, the more important issue is that these two landmark regulatory approvals should pave the way for a host of new cell-based therapies targeting a diverse range of hard-to treat liquid tumors.
Liver cancer's outlook is improving
On April 27, 2017, the FDA approved the first liver cancer drug in over a decade by expanding the use of Bayer AG's (BAYR.Y 2.16%) Stivarga (regorafinib) to include treatment of patients with hepatocellular carcinoma who have been previously treated with the drug sorafenib. Prior to this approval, advanced liver cancer patients that stopped responding, or never responded, to Bayer's first-line drug, sorafenib, had an exceptionally bleak prognosis, with most living less than a year.
On Sept. 22, 2017, the outlook for advanced liver cancer improved even more when the FDA granted an accelerated approval to Bristol-Myers Squibb's (BMY 0.50%) Opdivo (nivolumab) for the treatment of hepatocellular carcinoma in patients who have been previously treated with sorafenib. Bristol's Opdivo is the first and only immunotherapy approved for liver cancer.
Although Bristol's drug didn't work in the vast majority of patients in its mid-stage trial dubbed Checkmate -040, the patients that did respond showed durable responses typically lasting longer than six months, and three patients in the trial even exhibited complete responses. Moving forward, Bristol is hoping to find a way to improve Opdivo's response rate in advanced liver cancer.
While there are many therapeutic challenges remaining when it comes to the treatment of liver cancer, Bayer and Bristol's newly approved drugs are undoubtedly a step in the right direction. Several more experimental liver cancer drugs -- as well as a CAR-T therapy -- after all are already in development to hopefully bend the curve even further in 2018.