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Treating Waldenstrom With Targeted Therapies

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Published on December 17, 2019

Key Takeaways

  • Two drug approvals for Waldenstrom are ibrutinib (Imbruvica) and rituximab (Rituxan)
  • In early development for Waldenstrom is a drug called venetoclax (Venclexta).
  • Genes called MYD88 and CXCR4 can make a difference in determining what drug a patient may be most responsive to.  

“For patients living with Waldenstrom, you’ve got more to talk about than ever before,” says Patient Power Co-Founder Andrew Schorr, as he discusses Waldenstrom macroglobulinemia with Dr. Frits van Rhee and Dr. Dan Vogl, two specialists who treat this rare disease. Watch this video, filmed during the 2019 American Society of Hematology (ASH) Annual Meeting & Exposition, to learn which drugs are approved for treating Waldenstrom, what other treatments are being developed, and how certain genes may allow doctors to predict which drugs will be most effective for each patient.

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Transcript | Treating Waldenstrom With Targeted Therapies

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.

Andrew Schorr:

Waldenstrom's disease, let's talk about that for a few minutes with two specialists who treat it to find out what's new, what's important for patients.  So with me is Dr. Frits van Rhee who is a specialist, also the director of the myeloma center at the University of Arkansas Medical Sciences in Little Rock.  And Dr. Dan Vogl who is at Penn Medicine, University of Pennsylvania in Philadelphia.  Thank you. 

Dr. van Rhee, things we want to take note of in Waldenstrom's, pretty rare condition, how are we doing, and what is there to talk about with patients now? 

Dr. van Rhee:

I think there is a novel drug approved for—actually two drugs approved now for Waldenstrom's.  The first one which was approved is ibrutinib, which sometimes is combined with a monoclonal antibody called rituximab (Rituxan).  And this gets away from the more old‑fashioned chemotherapy which was given, and it gives excellent results. 

Not approved and earlier in development is a drug called venetoclax (Venclexta), which also has significant activity in Waldenstrom's disease, and it may be that in future both drugs are going to be combined.  Ibrutinib (Imbruvica) although a very effective drug in 5 to 10 percent of patients causes some cardiac rhythm abnormalities called atrial fibrillation and very rarely sudden death, so it's not that suitable for patients with cardiac issues, and there is now an alternative available for those patients.  So we can get away from the old‑fashioned chemotherapy and there the opportunity to use two new drugs. 

The other thing that we understand better is who actually is going to respond to certain drugs.  There are two genes.  One is called MYD88, and other one is CXCR4, and they can each mutate it, and based on the mutational profile one can make a prediction whether the ibrutinib drug is going to be more or less effective in a specific patient. 

Andrew Schorr:

Okay.  Now, these nibs, or BTK inhibitors, some of them, so there's another one, acalabrutinib (Calquence), and I've even heard about another one, zanubrutinib (Brukinsa).  So how do you know which one when, Dr. Vogl, if they come into play? 

Dr. Vogl:

So I think the clearest answer is that ibrutinib, which is widely available and for most patients really well tolerated is probably the initial therapy of choice for patients with Waldenstrom's.  And I think it's really great to have oral medications that are generally well tolerated as the backbone of our therapy now while still having as options some of the bolder, more standard chemotherapy medications if people really need them. 

And then I think we're still learning how to incorporate the newer BTK inhibitors and medications like venetoclax which is a Bcl‑2 inhibitor into the therapy for Waldenstrom's, and we're still going to have to learn what the best medications to use are and how to switch among them or substitute over time.  Right now we don't have a lot of answers. 

Andrew Schorr:

Okay.  But for patients living with Waldenstrom's you've got more to talk about than ever before. 

Dr. Vogl:

We really do, and not just more medicines but medicines that are easier to take and more effective in the long run that what we used to have before. 

Andrew Schorr:

Okay.  Very positive story for Waldenstrom's, certainly a serious disease, and you need specialists on your team.  This is a rare condition.  Here are two of them here, Dr. Dan Vogl from Penn, thank you for being with us.  Dr. Frits van Rhee from Arkansas, thank you.  

I'm Andrew Schorr from Patient Power.  Remember, knowledge can be the best medicine of all. 

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.

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