Tyrosine kinase inhibitors (TKIs) are the frontline therapy for CML.
In 2002, the FDA approved imatinib mesylate (Gleevec), as a therapeutic agent for CML. Gleevec uses a technique known as molecular targeting to block the action of the protein tyrosine kinase, thought to be responsible for most cases of CML. Because it targets the specific cause of the disease, the treatment does not alter healthy tissues, and is thought to be easier on patients than other forms of treatment, such as interferon injections, chemotherapy, and bone marrow transplant. Gleevec is manufactured by Novartis Pharmaceuticals Corporation.
Other drugs that have recently been approved by the FDA are dasatinib (Sprycel) and nilotinib (Tasigna). They work in ways similar to Gleevec, by blocking tyrosine kinase.
In June of 2010, Tasigna was approved by the FDA to treat CML upon initial diagnosis. Tasigna is manufactured by Novartis Pharmaceuticals Corporation.
Sprycel was approved in October of 2010 by the FDA to treat CML when other drugs, such as Gleevec, have been ineffective. Sprycel is manufactured by Bristol Myers Squibb.
Bosulif (bosutinib) was approved by the FDA in 2012 as a treatment for patients with chronic, accelerated or blast phase Philadelphia chromosome positive CML who are resistant to or who cannot tolerate other therapies. Bosulif works by blocking the signal of the tyrosine kinase that promotes the development of abnormal and unhealthy granulocytes. Bosulif is manufactured by Pfizer.
Synribo (omacetaxine mepesuccinate) was approved by the FDA in 2012 under its accelerated approval program to treat adults with CML. This is a new treatment option for patients who are resistant to or cannot tolerate other FDA-approved drugs for chronic or accelerated phases of CML. Synribo blocks certain proteins that promote the development of cancerous cells. Synribo is manufactured by Teva Pharmaceuticals.
The following therapies have been used previously to treat CML:
The orphan drug Idarubicin HCI for injection (Idamycin) was approved by the Food and Drug Administration (FDA) in 1990 for the treatment of CML.
Interferon alfa-2a (Roferon A), administered by injection, received FDA approval for the treatment of CML in 1995.
Drugs that inhibit bone marrow activity (myelosuppressive drugs) may slow the progression of the disease. Hydroxyurea, a medication used to treat CML patients, may lower the white cell count and therefore reduce symptoms.
Radiation therapy of the spleen is another treatment option employed in only relatively few uncontrolled cases, either alone or in combination with chemotherapy, to slow the progression of the disease.
Bone marrow transplant, when performed during the early phase of the disease, can lead to remission and cure of this disease. However, this mode of treatment is not appropriate for all patients and does present some risks. The likelihood of success appears greatest among younger patients who are treated in the early stages of the disease.