Nausea and Pain
Since it has been awhile and there is some recent news, I am going to give some context. First, Sue is doing pretty well now. However, right before Passover, Sue was feeling poorly, with both nausea and tenderness on both sides. Rather than wait for the weekend with all of our wonderful family visiting, we ran over to Johns Hopkins Hospital for a quick check. Driving there and waiting for results is always anxiety provoking and worrying. But Sue’s doctor came back with an explanation for the pain and nausea and a way to deal with it.
Apparently, the wonder drug Sue has been taking, Iclusig (aka ponatinib), was the cause, drug-induced pancreatitis. Her oncologist picked up on the symptoms right away as he has occasionally seen it in other patients. So Sue was asked to step down the dosage for a little while to see if the pancreatitis went away - which it did. But still nerve-wracking.Remember, the last time Sue had to go off the drug for about a month, she had a recurrence of the brain tumor which took months of intense treatment to get rid of. We have been very wary of reducing the dosage even for a day or two despite the tough side effects. Worried,we asked if there were other patients in a similar circumstance that were on a lower effective dosage.
Context
It was about five years ago that Sue started taking Iclusig. Prior to that, there was no truly effective drug for those with ALL Ph+ (Acute Lymphoblastic Leukemia with Philadelphia chromosome present) who had CNS involvement (brain tumor). The initial wonder drugs that turned around treatment for ALL Ph+ and CML, did not seem to be effective in the central nervous system. Fortunately, there was some evidence in the animal studies that ponatinib/Iclusig did penetrate through the blood brain barrier. But nobody seems to study patients with CNS involvement due both to the infrequency and the poor prognosis. So Sue, who had tried all the rest of the drugs, got permission to try this new and not yet generally approved drug, as a study of one.
For a few years after, with no sign of a recurrence, a very, very unusual outcome, we felt that it was the drug that made the difference. But a study of one, and with many other forms of treatment also used, the effectiveness of the drug was merely speculation. Then when due to a drug interaction, the Iclusig was pulled for about a month, the tumor came roaring back. We threw everything at it we could including other cutting edge treatments. And again the tumor receded until there was no evidence of it.
Especially this time, surely this was evidence that it was Iclusig that was crucial, in that it was pulling it that coincided with the recurrence. I tried to tell folks at the LLS about it so others could learn about this potential lifesaving treatment. But, still without a study and clear evidence, this seemed to be purely speculative. There have been a few anecdotal cases written up in the journals that gave conflicting impressions of the efficacy of Iclusig/ponatinib (thanks so much for PubMed from the NIH which is itself a lifesaver: https://www.ncbi.nlm.nih.gov/pubmed/ ) And it did not seem like anyone was interested in studying this potential treatment.
Others
Although we do not know who else is being treated for this condition by name, we do know that others have been successfully treated and been disease free for quite awhile. When we asked about the lowest effective dose, to see if others were able to stay disease free at lower dosages than Sue. The answer was yes. Although there are no definitive dose efficacy numbers since so few are being treated for the same situation as Sue, just knowing that others are doing well is important information.
Apparently, there was even one person who was too sick to receive any other treatment than Iclusig/ponatinib (as it is an oral treatment and the person was not up to anything else). And just taking the drug was sufficient to get the person to remission despite a worse brain tumor than Sue. This is the closest to proof without a study that one can point to on the efficacy of the drug for CNS involvement.
Going Forward
We are grateful for the care of the doctors at Hopkins and at the effectiveness of Iclusig. Where Sue was perhaps the first patient to be successfully treated for her condition, without others being able to match the success, the treatment might not be known for others generally. And the feedback from others, may help Sue and her continuing treatment. Like all medicine, it depends on constant feedback of patients and doctors, learning, studying and relating to others the results of various treatments. We are thankful both that others seem to be given a chance due to Sue’s pioneering treatment and that her doctor is better equipped to treat Sue due to the others.
Some people view treating those who are sick as a form of charity or as a right. It seems to me that we should view treating people as the best way for a society to learn how to treat everyone else eventually. Sue and the other patients are volunteering their results, good or bad, to others so that every year more people are saved and have better quality of life. I am grateful to Sue for always volunteering for studies, always in awe of the thousands of other patients who we have seen over the last eleven years. We all have a responsibility in a society to meet the health needs of each other, as we all end up depending on each other for better health.
Current Status
Because She was so worried about even a short term lowered dosage, we went back in for a brain MRI. And, the images were the best they have been. This time both hemispheres of her brain appeared the same, with not even the scarring that had been visible last time (she got to see the scans at the appointment). Good news!