On October 11, 2013, the Food and Drug Administration released a pod cast detailing its investigation into the leukemia drug Iclusig ™ (ponatinib) after an increasing number of fatal blood clots reported in patients.
The drug is widely available in Europe and the United States for the treatment of several types of leukemia including chronic myeloid leukemia—chronic phase, accelerated phase or blast phase—as well as Philadelphia chromosome-positive (Ph+) acute lymphoblatic leukemia. The drug is a form of tyrosine kinase inhibitor for patients that either cannot tolerate or are resistant to other tyrosine kinase inhibitors.
According to a paper in Chemical Biology & Drug Design, the drug ponatinib works by targeting BCR-ABL, an abnormal tyrosine kinase protein that leads to the chronic production of white blood cells. Resistance to tyrosine kinase protein inhibitors is common, but research indicated ponatinib's effective use on several variations of BCR-ABL proteins, including the T315I mutant that resists most other forms of treatment.
Iclusig’s ™ manufacturer is ARIAD pharmaceuticals, headquartered in Lausanne, Switzerland and Cambridge, Massachusetts, within the great biotech belt of Greater Boston. On October 18th, the company released a press release announcing the discontinuation of its Phase 3 Trial, citing arterial thrombosis (blood clotting).
In the company’s trial, 8% of the patients developed arterial thrombosis. Other related side effects included pancreatitis (6%), hemorrhaging (5%), and congestive heart failure (4%) amongst others.
The company has been transparent about the risks involved with the medication on the drug’s website. The FDA, which had approved the drug back in July of this year, has similarly found many reports of blood and heart conditions caused by the drug.
Like any medication, talking with a doctor is the best way to make sure drug treatment options and their side affects are fully understood. Despite safety issues with the drug, the drug is still FDA-approved and still one of the best treatment options for acute and treatment-resistant forms of chronic myeloid leukemia and Ph+ lymphoblatic leukemia.