[Corrected 12/07/12, 10:18 pm. See below.] The life sciences industry has been talking up personalized medicine for more than a decade, but so far the idea that diseases will be treated with a unique drug regimen tailored to each patient has made little headway in actual clinical practice. A number of drug developers large and small may finally be making personalized medicine a reality, however, for at least one disease category—blood cancers.
Their progress will be on display at the high-profile American Society of Hematology (ASH) annual meeting in Atlanta Dec. 8-11. Data will be presented at the meeting from lab studies and clinical trials for a number of promising drugs, both newly approved and in the pipeline, for leukemia, lymphoma and myeloma.
Multiple myeloma patients already got another treatment option in July when Onyx Pharmaceuticals (NASDAQ: ONXX) of South San Francisco, CA, won FDA approval for carfilzomib (Kyprolis), a type of drug called a proteasome inhibitor that blocks cellular structures critical to cancer cell survival. Carfilzomib is meant for people who don’t respond to bortezomib (Velcade) from Millennium and lenalidomide (Revlimid) from Celgene (NASDAQ: CELG), drugs that themselves vastly improved the outlook for myeloma patients when they were introduced less than 10 years ago. But already the next generation of proteasome inhibitors, administered by pill instead of injection or IV, will be among the most closely watched drugs at ASH this year.
The rapid progress in blood cancer therapies started with Novartis’s breakthrough imatinib (Gleevec), approved in 2001 for chronic myeloid leukemia. Decades of work identifying the genes and cellular mechanisms linked to cancers that start in the blood, bone marrow, or lymphocytes led to a rapid succession of targeted therapies over the past decade, and these new drugs have led to cure rates as high as 90 percent for chronic myeloid leukemia and Hodgkin’s lymphoma, and a tripling of survival rates for myeloma, according to the Leukemia and Lymphoma Society. [An earlier version of this paragraph mistakenly said that Gleevec is a Roche drug. Xconomy regrets the error.]
“The patient population is so heterogenous” in blood cancers, Dixie Esseltine, vice president of global medical affairs for Millennium, told me. “We understand so much now at the molecular level and more so in the next ten years, that we are going to be able to have a much more refined approach to treatment, taking into account both the characteristics of their cancer and the side effects they might experience.”
Those side effects are one reason that Cambridge, MA, based-Millennium, a subsidiary of Takeda of Japan, is testing an oral proteasome inhibitor called MLN9708—often referred to in the myeloma community as “Velcade in a pill.” Velcade is delivered by injection and can cause nerve damage in some patients. Consequently, several companies, including Onyx, are developing oral proteasome inhibitors, but Millennium is furthest along, and will be presenting data from a Phase I/II study at ASH.
Shaji Kumar, lead investigator of the trial and a professor at the Mayo Clinic, told me that his goal with targeted therapies going forward is to make them both safer and more convenient for patients. Next year Millennium will test MLN9708 in Phase II and Phase III studies in combination with other types of myeloma therapies already approved.
Other drugs to watch at ASH:
—Onyx will be presenting very early clinical trial data on its own oral proteasome inhibitor, ONX 0912, as well as a number of studies evaluating the safety and efficacy of carfilzomib in various patient settings. Those results are important because the FDA, which granted carfilzomib accelerated approval, requires that Onyx submit further proof of efficacy and safety to confirm the drug’s clinical benefit.
—In July Ariad Pharmaceuticals (NASDAQ: ARIA), in Cambridge, MA, filed for FDA approval of ponatinib, the first of the next generation tyrosine kinase inhibitors meant to improve on drugs like Bristol-Meyers Squibb’s (NYSE:BMY) dasatinib (Sprycel), approved six years ago. There will be five presentations about ponatinib at ASH, including 12-month data from a key trial known as PACE testing the drug in 450 patients. The drug just got a glowing review in the Nov. 29 New England Journal of Medicine, which published a Phase I study testing the drug in patients resistant to currently available treatments. In an accompanying editorial John Goldman of Imperial College in London said ponatinib could be “the best of the bunch” of the next generation tyrosine kinase inhibitors. “Ponatinib may turn out to be another step forward in the march toward real success with molecularly targeted therapy for cancer,” he wrote.
—Celgene, in Summit, NJ, also has a drug application pending before the FDA, for pomalidomide for multiple myeloma, scheduled for review in February. Celgene’s lenalidomide is facing competition from Onyx’s carfilzomib, so the success of the next-generation pomalidomide is critical to the company. Celgene announced top-line results from a Phase III trial of pomalidomide in October but not a lot of detail; those results will be revealed at ASH.
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