Like any drug, side effects were known. In this case concerns especially surrounded vascular complications like blood clots, heart attacks and stroke. But the feeling was the benefits outweighed the risks. And some patients who would otherwise be very sick or face death – patients like Hans Loland of Seattle who was featured in a Patient Power video profile more than two years ago – were overjoyed. Other CML patients felt more confident that there was now a third line drug available for them should they ever need it.
A couple of weeks ago things turned “south” for ponatinib. As more data emerged it appeared the risks of severe vascular events were higher – perhaps too high to label the drug “safe.” Last week the FDA took a strong step asking Ariad to suspend marketing. Further it asked physicians to stop prescribing it and, as possible, move existing patients to other approaches. Physicians and advocacy groups quickly responded, citing the need to consider both safety and the benefits of the drug.
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Now the company is negotiating with the FDA how ponatinib may once again be available in the US for the patients who may need it to survive and where programs are set up to limit its use by others. It’s too early to tell how this will turn out. Will the FDA step back a bit from its very safety conscious and conservative approach? And what will happen in Europe where the drug is also approved? Patients and families around the world who depend upon it are surely worried. Our partner, The National CML Society, is working with the FDA and leading CML physicians to provide updates for patients which will be posted on their Facebook page as they occur and on their website.
Of course, this isn’t a story only CML patients should pay attention to. In CLL, myeloma, myeloproliferative conditions and others that are sure to be “hot topics” at ASH this year, there are many new drugs that are showing promise for having higher effectiveness with low side effects and allowing for a high quality of life. All of us need to temper our excitement about what could be great gifts from medical science. As researchers fight cancer at the genetic level we celebrate when they can “turn off” an oncogene. But we always have to wonder if there is an effect “downstream” that is too great.
I don’t mean to throw cold water on the promise of new drugs for my conditions or yours. I am truly hopeful. But cancer remains a wily foe and we have to recognize that while there is real progress all too often it comes in the form of two steps forward and one step back.