Can MPN Drugs Help in Myeloma?

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Andrew Schorr:

Hello and welcome from Chicago and the big American Society of Clinical Oncology, or ASCO conference. I'm Andrew Schorr.  Thousands of people here from all over the world, and among them is one of our favorite myeloma experts, Dr. James Berenson from Los Angeles.  Thanks for being with us.   

Dr. Berenson:

Thank you for having me.  

Andrew Schorr:

Okay.  So, first of all, we have a bunch of topics we want to talk about, and you can send in your questions right now.  Just put it in the comments.  But we want to talk about a number of things, and one of them is in medicine I take ruxolitinib or Jakafi for myelofibrosis, a fairly rare condition. Some people take it for a related condition, polycythemia vera.  I understand your finding may have some utility in myeloma in combination with other drugs.  

Dr. Berenson:

Very surprising that we stumbled on this, and that's why I really love being in clinic.  We happened to have a patient about four or five years ago who had polycythemia, but he came to me with myeloma.  He had been on the same drug that Andrew had been on, the ruxolitinib… 

Andrew Schorr:

Still on.   

Dr. Berenson:

…and still on, and it wasn't really working.  These people require bloodletting to keep their cancer counts in the normal range. And then he got myeloma, and I told him well, you're probably not going to need to take that roux drug anymore because your counts are going to drop, as some of you know, from the Velcade who tried it, didn't work, run the Revlimid and tried it, didn't work, and it didn't actually suppress his blood counts.  

And then we said aw, what the heck.  Let's go ahead and add back in that Jakafi, Jakafi drug, that ruxolitinib, and, oh, my God, he did great.  Blood counts normalized, and his myeloma has had a remission now for four or five years. He was a rather wealthy individual, so he said what can I do to help you?  You helped me.  I said you can give us the support, we'll go in the lab, figure it out.  We did.   

We went to the company, and then we went to FDA, and now we've completed a Phase I trial combining ruxolitinib, a drug never been effectively used in cancer of any type, Revlimid and Medrol or methylprednisolone, a steroid which we think is better tolerated than dex, and half the patients responded.  And all the responding patients were totally resistant to Revlimid, that is, they were progressing on or within eight weeks of their last dose. So this is a really big deal.  

And I think the biggest deal, and Andrew knows this, is how well these patients feel.  Now, we don't have objective measures in the trial, but I can tell you our patients, they're like, oh, I'm feeling so much better now that you added that ruxolitinib drug.  And now we're going to really objectively measure it.  

Andrew Schorr:

So, all right.  Let me see if understand.  You had a patient who was not doing well—I've been doing okay—lenalidomide (Revlimid) was no longer working for them, Velcade was no longer working for them.  You revive it, if you will, by combining it with Jakafi and now they're doing well.  

Dr. Berenson:

Yeah, even including complete remission.  We couldn't even find a trace of myeloma in patients that were doing terrible on Revlimid.  

Andrew Schorr:

Wow. 

Dr. Berenson:

And these were really heavily pretreated patients. They'd had up to 10 prior therapies, a minimum of three, average of six, all had failed not only Revlimid but almost as well, except two, and everyone had seen a proteasome inhibitor, almost everyone bortezomib (Velcade), and most had failed also carfilzomib (Kyprolis). 

Andrew Schorr:

All right.  So let's make this point.  Please validate this for me.  In oncology, that happens, where a drug gets approved for one, and they say, well, gee, whether they find it out by accident, like the polio, you know, whatever, which was discovered that way, all these things where you say, here's theoretically maybe, this from column A will work with column B, and then it does.

Dr. Berenson:

Yeah.  But the other exciting thing is this came from a single patient, went to the lab, found a lot of new mechanisms through which they drug he was on, the ruxolitinib, is working, that the company didn't have a clue was working that way, and now that's even leading to other trials.  For example, the checkpoint inhibitors, which have been tried in myeloma, the drugs like nivolumab (Opdivo), the pembrolizumab (Keytruda)s, it turns out the ruxolitinib shuts down the markers that are the targets for those drugs. We think they may be good combinations too, and we hope to study that in the clinic over the next few months.

Andrew Schorr:

Wow.

Please remember the opinions expressed on Patient Power are not necessarily the views of our sponsors, contributors, partners or Patient Power. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.