[Note: Ben Fidler co-authored this report.] This weekend, San Diego will host the annual American Society of Hematology conference, the largest U.S. medical gathering to get the latest on blood diseases, and a venue for updates on some of the most cutting-edge biotechnologies that only a few years ago seemed like science fiction.
Using the previews of major studies that were released earlier this month, we’ll dive into five of the disease areas under scrutiny at ASH this year—leukemia/lymphoma, multiple myeloma, beta-thalassemia, hemophilia, and sickle cell disease—and give you plenty of background in case you haven’t been following each one closely.
LEUKEMIA AND LYMPHOMA
Of the 1.7 million new cancer cases and 610,000 cancer deaths in the U.S. this year, leukemia and lymphoma will account for nearly 10 percent. There are many types of each, often with different treatments and levels of success. Here’s a brief look at the major types and their expected updates at ASH.
Ninety percent of all new lymphoma cases are some form of non-Hodgkin lymphoma (NHL).
Two engineered therapies made from a patient’s own immune cells, known as CAR-T, have been approved for adults with desperate cases of NHL. But difficult logistics and high prices have kept CAR-T from making more inroads.
Novartis’s (NYSE: [[ticker:NVS]]]) tisagenlecleucel (Kymriah), one of the two approved CAR-T products, for instance, generated just $20 million in the third quarter. There’s another potential obstacle: Doctors at big cancer centers are reporting that real-world outcomes for the other approved CAR-T, axicabtagene ciloleucel (Yescarta), aren’t matching what its developer Kite Pharma, now owned by Gilead Sciences (NASDAQ: [[ticker:GILD]]), reported in clinical trials.
While meant to approximate what might await in the real world, clinical trials are always more carefully cultivated, so a falloff is not surprising, especially with CAR-T—a new kind of therapy with dangerous side effects that require special training to handle—says Lee Greenberger, the chief scientific officer of the Leukemia & Lymphoma Society in Rye Brook, NY. Training could be uneven, patients could be sicker; bottom line, says Greenberger, is that “we’re only in the first chapter. We’ve gotten [CAR-T] to work and gotten it approved. But combination approaches will be necessary. There will be patients who fail these therapies.”
The dream with CAR-T, as with most new cancer therapies, is to prove over time that it can be used ever earlier—not just for patients who have failed all other treatments. NHL will be a high hurdle, however, as an ASH paper lays out; a lot of younger people do quite well on standard chemo-based regimens.
New CAR-T flavors—CAR-T 2.0, if you will—are moving into clinical studies, such as this “armored” CAR-T at Memorial Sloan Kettering Cancer Center, hoping to address some of the first generation’s shortcomings, like severe side effects that overheat the patient’s immune system.
Beyond CAR-T, the other big immunotherapy class—antibody drugs known as checkpoint inhibitors—has been approved for some types of Hodgkin and non-Hodgkin lymphoma. Here’s an ASH update on the checkpoint drug pembrolizumab’s (Keytruda, from Merck) success in one form of NHL.
At ASH, investigators are also asking whether lymphoma patients who haven’t benefited from checkpoint treatment might go on to have success with other treatments, a clue for those thinking about combination therapies.
For chronic lymphocytic leukemia, the most prevalent type of leukemia, the big drug-related headlines are unrelated to immunotherapy. The story is ibrutinib (Imbruvica), a pill at the center of a stunning $21 billion buyout by AbbVie (NYSE: [[ticker:ABBV]]) three years ago, which has quickly become the standard of care for newly-diagnosed patients.
Whether alone or in combination, ibrutinib is
