When High Hopes for New Cancer Drugs Are In Limbo

November 6, 2013
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Iclusig (Ponatinib)There was tremendous excitement last year at the American Society of Hematology (ASH) meeting about promising new leukemia drugs.

Iclusig (Ponatinib)There was tremendous excitement last year at the American Society of Hematology (ASH) meeting about promising new leukemia drugs. One drug for chronic myelogenous leukemia (CML), ponatinib (brand name Iclusig) made big news as leading experts shared later stage study data showing great effectiveness for patients with the life-threatening T315i mutation and for those who did not respond to already approved drugs for CML. The buzz was that ponatinib might not only help the few patients with T315i and who were almost out of options, but also a bigger group of patients earlier in their CML journey. Within weeks the FDA crowned the excitement with approval for marketing of the drug under the brand name of Iclusig and gave a huge boost the to the biotech company that developed it, Ariad Pharmaceuticals.

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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cancer patientsThis is another example of how excitement about promising new drugs – even ones that are approved – need to be measured. Certainly, ponatinib is not the first drug to be limited in this way. As more patients take a medicine after it is approved, more than in the earlier studies, a clearer picture of effectiveness – and also risks – can emerge. Also, since physicians may prescribe an approved drug “off label” to a wider group of patients who were in the original studies, it is possible some patients may take on unnecessary risk.

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.

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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.