Two new drugs for multiple myeloma, approved in recent months, promise to extend life expectancy for patients with the blood cancer.
They follow new treatments that over the last decade have transformed the prognosis for multiple myeloma—once a short death sentence—into a manageable condition that can be survived for up to seven years or more. There is still no cure for the disease.
The new drugs were approved for patients whose blood cancer returned after developing resistance to older treatments.
Karen Countryman is one of those patients. When she was diagnosed in 2004, doctors gave her three years to live, she recalled. She underwent the standard treatment of the time, a harsh regimen of chemotherapy and a stem-cell transplant. The first of a wave of myeloma drugs had just been approved. And after her blood cancer returned in 2008, Ms. Countryman turned to one of the new therapies and then to others as her myeloma progressed.
Her myeloma remains in check, she says. "It's a miracle, and this is all due to new drugs and research," said Ms. Countryman, a 63-year-old nurse from Boise, Idaho. She is currently finishing a course of treatment with one of the newest drugs, Kyprolis.
Taken intravenously, Kyprolis was greenlighted last July. The newest myeloma treatment, a pill called Pomalyst, was approved last month.
They follow four other drugs that were cleared since 2003 to treat multiple myeloma, a cancer that starts in the soft, spongy marrow inside bones that makes blood cells. From there, myeloma spreads, damaging bones, kidneys and the immune system.
International Myeloma Foundation
After being told in 1999 that she had few treatment options for her multiple myeloma, Paula Van Riper of Branchburg, N.J., has since seen her life extended by new drugs.
What Is Multiple Myeloma
- What causes it: Multiple myeloma, a.k.a. blood cancer, occurs when plasma cells (which make antibodies that help the body fight infections) grow out of control in the bone marrow, forming tumors in bones. As these tumors grow, the ability of bone marrow to make healthy blood cells and platelets diminishes.
- How many people have it: In 2013, an estimated 22,350 cases will be diagnosed and an estimated 10,700 people will die.
- How long patients live with it: In 2001, life expectancy was 3.5 years. By 2010, it grew to 7.3 years. Today, it's believed by many researchers to be as long as 10 years, but there is no hard data yet to support that time frame.
- Cost of the newest drug regimens: Around $10,000 a month for Pomalyst; $9,950 a month for Kyprolis
Each year, nearly 22,000 people in the U.S. are diagnosed with the condition, many of whom are 65 years or older. More than 10,000 patients will die.
Yet thanks to all of the new drugs, the median survival for patients has doubled over the last decade, according to Brian Durie, co-founder and chairman of the International Myeloma Foundation.
"The focus has shifted from just keeping patients alive" to giving them a good quality of life while they manage the disease, said Dr. Durie, who is also a myeloma doctor at Cedars-Sinai Medical Center in Los Angeles. He has worked with several drug makers who sell and are researching myeloma treatments.
The wave of new drug development in myeloma dates to the late 1990s and early 2000s, when researchers were scouring old treatments for one that might have an impact on the cancer.
At that time, a myeloma prognosis could mean a few years to live. Paula Van Riper, who was diagnosed in late 1999, underwent hip replacement surgery to remove a large tumor. Afterward, she recalled being told by doctors to hold off treatment, since there weren't any good options available until the disease worsened.
"It never occurred to me that I would be around 13 years later, feeling well, going to work every day and thinking maybe I can retire," said Ms. Van Riper, 65, an assistant dean at Rutgers University in New Jersey, who is now taking Pomalyst.
A turning point in the treatment of myeloma, doctors say, was the discovery in the late 1990s that a controversial drug called thalidomide had an impact.
Thalidomide was famously pulled as a sleeping pill in 1962, after it was associated with birth defects. But it turned out to have a positive impact on myeloma, interfering with the surrounding environment in the bone marrow that the cancer cells need to flourish. Thalidomide's success drew researchers and drug makers into the hunt for even more treatments.
Velcade, approved in 2003, and Revlimid, a thalidomide successor approved in 2006, are now backbones of myeloma treatment, doctors say. The two drugs attack myeloma in different ways, and doctors tend to use them in combination and with other drugs because the combination amplifies the effect of the therapies.
Despite the new approvals, doctors like David Siegel still plan on typically starting patients with a combination of Velcade, Revlimid and a steroid called dexamethasone.
Dr. Siegel, chief of the myeloma division at John Theurer Cancer Center in Hackensack, N.J., further attacks the cancer by following up that combination with a heavy dose of a chemotherapy drug called melphalan. Dr. Siegel says he will then transplant stem cells in the bone marrow to restore the marrow killed by chemotherapy.
In most patients, these treatments will drive myeloma into hiding.
But the disease tends to return. Over the course of this cycle of relapse and remission, Dr. Siegel will draw from the full arsenal of available treatments, eventually including the two newly approved therapies.
However, for now, the U.S. Food and Drug Administration has approved Kyprolis and Pomalyst for later use by patients whose disease progressed after treatment with other therapies. Insurance companies are also steering use of the older drugs first, doctors say.
"In the end," said Dr. Siegel, "everyone feels these drugs will move closer" to early use. He has worked with several drug makers, including Celgene Corp.,CELG +2.66% the maker of Revlimid and Pomalyst; Onyx Pharmaceuticals Inc.,ONXX +2.07% which sells Kyprolis; and Takeda Pharmaceutical Co.4502.TO +0.51% Ltd.'s Millenium unit, which sells Velcade.
Treatment costs for both Kyprolis and Pomalyst run close to $10,000 a month.
A common side effect of some of the older therapies is a painful condition called peripheral neuropathy, which can cause tingling or numbness in fingers and feet. That is not an issue with Revlimid, Celgene says, and many doctors believe that won't be as much of an issue with Kyprolis and Pomalyst.
Kyprolis and Pomalyst were associated with low blood-cell counts, a common problem with most anticancer therapies, which can hurt the body's ability to fight off infections. Heart failure and shortness of breath were serious side effects seen with Kyprolis. Pomalyst shouldn't be used by pregnant women or women contemplating pregnancy because it can cause serious birth defects and life-threatening blood clots.
For all the gains in treating myeloma, the disease still lacks a cure for most patients. Doctors believe what is needed are drugs that work differently from the currently approved treatments.
"The problem in myeloma right now is many of the new drugs are in the same two classes," said Vincent Rajkumar, a myeloma specialist at the Mayo Clinic. "If you want a sea change, you want a new class," he added, because the cancer cells get smart and develop resistance to the older drugs.
Several new classes of drugs are in development. Among the furthest along in the pipeline is a monoclonal antibody called elotuzumab from Bristol-Myers SquibbCo., BMY +1.71% that several doctors singled out as promising.
The Multiple Myeloma Research Foundation, which encourages research collaborations between academia and industry and funds drug development, is also conducting a study whose results could be used to profile which patients will benefit from which treatment combinations.
(Wall Street Journal)
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