How a New Pill Can ‘Melt Away’ Your Leukemia Cells

Breakthrough pill boosts survival in CLL patients

mystery pill in pill pack

A pill that “melts away” cancer cells may replace chemotherapy often used to treat relapsed chronic lymphocytic leukemia (CLL).

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The U.S. Food and Drug Administration (FDA) recently approved the drug, idelalisib (Zydelig™), to treat CLL. 

In a recent study of the drug, researchers found that it was effective in treating patients with CLL. The study used this pill along with another drug to treat the disease.

Researchers saw improved survival rates with less severe side effects in patients who used the pill. One researcher described how it worked, saying patients’ cancer “quickly melted away.” 

Idelalisib is the second new pill the FDA has approved to treat CLL in the last 12 months. The agency approved the first drug, ibrutinib (Imbruvica®), in November 2013. 

Who will benefit? 

The FDA approved idelalisib and ibrutinib for use in patients who have already had chemotherapy and have relapsed or who have refractory CLL, which means it has been less responsive to treatment. Both drugs provide targeted therapy, as opposed to traditional therapy. This means the pills kill cancer cells without killing healthy cells. 

Neither drug has yet been approved as an initial treatment for CLL.

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“There are some clinical trials going on right now to compare ibrutinib head-to-head with traditional chemotherapy treatments, but we don’t know yet how effective this will be,” says oncologist Brian Hill, MD, PhD. “It may be a few years down the road before we do know. As of now, chemotherapy is still the standard of care for frontline treatment.” 

A major breakthrough 

Dr. Hill says FDA approval of these two medications is a significant advance in cancer treatment. 

“Ibrutinib has already been shown to improve overall survival rates in patients who had relapsed,” he says. 

Researchers in the idelalisib study expect the new drug to change the lives of many patients. The long-term toxic effects of chemotherapy lead to bone marrow failure, infections and a risk of death. Researchers say the next step is moving these therapies up front in the treatment regimen and providing it to all patients.

Dr. Hill says physicians are also using the recently approved pills to treat certain types of lymphomas. However, he warns that the pills are not effective for treating acute leukemias, such as acute lymphocytic leukemia or acute myeloid leukemia. 

“The way these drugs work is to interfere with the signals that are telling the cancerous B cells to grow,” he says. “Those cells are not present in acute leukemias.” 

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Possible side effects 

You may have side effects when you take any medication. But Dr. Hill says idelalisib and ibrutinib have fewer side effects than drugs usually used to treat CLL.

If you do have side effects from idelalisib, they’ll most likely be things like diarrhea, fever, fatigue, nausea, cough, pneumonia, abdominal pain, chills or rash. Side effects from ibrutinib can include diarrhea, nausea or vomiting, fatigue, bleeding, bruising, skin rash, shortness of breath, bone or muscle pain, or swelling in the hands, legs or feet. 

However, for either of these drugs, you won’t lose your hair or develop severe nausea and vomiting, anemia or bone marrow failure, as you might with chemotherapy drugs.

“Both drugs are pretty well tolerated,” Dr. Hill says.

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