New Hope for Multiple Myeloma Treatment Why Myeloma Patients Can Expect Better Drugs

Multiple myeloma is an incurable form of cancer, but hope is on the horizon for myeloma patients. New and improved drugs are being developed, says medical oncologist Irene Ghobrial, M.D. We asked her to explain how myeloma treatment is changing... A diagnosis of multiple myeloma, an incurable cancer of plasma cells inside the bone marrow, is a dire one. But the situation is getting better, as new myeloma treatments are being developed at a rapid pace. Approximately 9,120 women and 11,400 men were diagnosed with multiple myeloma in 2011; about half will die from the disease, according to the National Cancer Institute (NCI). Typical myeloma patients once faced a life expectancy of 3-5 years, but that’s now expanded to 16-20 years, says Irene Ghobrial, M.D., a medical oncologist at the prestigious Dana-Farber Cancer Institute in Boston. That’s partly because myeloma treatment is a promising arena for new drugs, which are both extending patients’ lives and reducing side effects, she says. Myeloma patients can “usually go to work and don’t have to tell everyone, ‘Hey, I have cancer,’” she says. In this Lifescript exclusive, Ghobrial discusses the treatments available as well as new options headed for Food and Drug Administration (FDA) approval. What is multiple myeloma? It’s one type of blood cancer. There are other types, like leukemia and lymphoma. When is it diagnosed? Some myeloma patients can be diagnosed with very early stages of the disease [if they have] an incidental protein finding called monoclonal gammopathy of undetermined significance (MGUS), which is secreted by plasma cells. We don’t see the actual cells themselves, because they hide inside the bone marrow. They don’t like to circulate in the blood. If we started screening people over the age of 50 more often, we’d find 1% of the population has this protein. But the chances of those people developing actual multiple myeloma is just 1% per year. What do you do for those patients? We just watch them and see them every six months or so, because there’s active disease. That’s known as the pre-malignant stage. Although there are malignant cells, they haven’t progressed for some reason. We’re working in labs to understand what makes them stay like this, and what makes progression happen. Once the disease progresses and patients start having symptoms, we call it active multiple myeloma. That’s when it needs treatment. What are the symptoms of active multiple myeloma? They include things like anemia or lesions in the bones. [Myeloma patients] come in with bone pain, or [we measure the protein level and it’s] high enough to require myeloma treatment. Or they find an increase in an antibody called immunoglobulin, which is a marker of the disease or its progression. Or they may come in because of kidney problems, because the protein goes through the kidneys. What are other signs to watch for? Low blood counts, high calcium or bone pain. If someone has a small fall that causes a major hip fracture, we would investigate, and might find a lesion in the bone. With other people, a primary care doctor might find a high protein level in a blood sample. In general, we don’t go around and screen people for multiple myeloma. It’s a relatively uncommon disease. Who's most likely to get multiple myeloma? It’s more common in African-Americans than whites. And it’s a little more predominant in men than women, but not to a huge extent. It’s also more common over age 60. Why is it more common in African-Americans? That’s a good question. I don’t know if there are any well-defined studies saying why race is a consideration. How much less likely are women to be affected than men? It’s not a significant amount. Gender is less of a [factor]. Is there a genetic component? [Researchers are trying to find out] if there are families with multiple myeloma or if some people have a predisposition. We don’t have strong indications yet of what would predispose [someone to the disease]. Is there a cure? We don’t have a cure for it, but we’ve [extended] patients’ survival significantly. There was a time when we’d expect multiple myeloma patients to live only 3-5 years. Now people are living 16-20 years – and that’s just within the last few years [of medical advancements]. And a growing number of new drugs is being developed. What drugs were available before? [Originally] we had drugs like Memphian, a chemotherapy agent. It’s still used in high doses as chemotherapy before stem cell transplantation. When I was a fellow at Mayo Clinic, there was one new drug in development, thalidomide. It had been used when women were pregnant to control nausea or as a sleeping pill, but it was taken off the market [when it was found to cause birth defects]. But it’s also an immunomodulator and was found to cause something called anti-angiogenesis – it prevented blood vessel formation. When it was used for multiple myeloma in the 1990s, [patients] showed an improvement. It’s still used, but newer drugs from the same family have been approved, such as Lenalidomide. Then we have 2-3 related drugs that will hopefully be approved this year or next. We have 13 new drugs being tested in clinical trials, and five that have been FDA-approved for myeloma, including the drug called bortezomib (Velcade). It’s a proteasome inhibitor [It induces a process that helps cancer cells die off]. How else has multiple myeloma treatment changed? We used to use chemotherapy. Myeloma patients would lose their hair, have nausea or vomiting, and might even go into the hospital with an infection. Now almost all the drugs we use won’t make people lose their hair. Are there side effects from the new drugs? The new agents also have side effects, but don’t cause very low white blood counts. Patients can have some mild nausea or vomiting or diarrhea, but not as bad. Bortezomib and thalidomide can cause something called neuropathy – numbness and tingling in the hands and fingers. If that happens, we change the dose or frequency. But we’re developing some new drugs from the same class of agents that don’t cause neuropathy. Those new agents are used on an outpatient basis, meaning people can come in and take them as a pill for treatment. Other treatments include radiation therapy and stem cell transplants. Is radiation still used? Radiation is being used less and less, because [it can increase the risk of] fractures and other problems associated with myeloma. If a patient has spinal cord compression, meaning one of the areas of myeloma is pressing on their spine, it’s an emergency that requires radiation therapy. When are stem cell transplants considered? Stem cell transplant is one of the advances in myeloma treatment, and we still do a lot of them. We do autologous transplants, meaning we take your own cells and put them back in your body. It’s [used after] high-dose chemotherapy, which kills both bad and good cells. Now we use the newer drugs first, to get myeloma patients into a complete or very good remission, and then decide whether they need a transplant. The new drugs are so powerful that we may actually eliminate the need for stem cell transplants … but we don’t know that yet. What are the side effects of stem cell transplants? You’re wiping out the bone marrow. So blood counts become very low and you can have infections, nausea, vomiting and mouth ulcers. And a very small percentage of people can die from this. How close are we to the other drug treatments being approved by FDA? There’s another, more potent new drug related to thalidomide, called Pomalidomide, that will be approved this year or next. Then we have many new classes of agents being tested that will take time to be approved. But the studies are very promising. We’re hoping for more and more drug options to use when a patient relapses following myeloma treatment. When should myeloma patients consider clinical trials? I always encourage people to think about clinical trials because of all the new drugs coming along. And always ask your doctor what new drugs are available. How do you find out about a clinical trial? Go to clinicaltrials.gov or on the websites for the Multiple Myeloma Research Foundation and International Myeloma Foundation. How should myeloma patients prepare for a doctor’s visit? They should ask their physicians [about new drugs] and go to centers that specialize in multiple myeloma, where they have access to the clinical trials and agents. And they should read about myeloma. Is a long remission possible? Yes. But it’s not a [permanent] cure. Should patients be optimistic about all the myeloma treatment developments? It’s important to know that the prognosis and treatment options have changed dramatically in the last few years. There’s a lot of reason for hope. For more information, visit our Multiple Myeloma Health Center. How Much Do You Know About Multiple Myeloma? About 20,520 cases of multiple myeloma are diagnosed each year in the U.S., according to the American Cancer Society. Has your life been affected by a diagnosis?