We are international

Recently in Pat Killingsworth Category

Cutting-Edge Research: How Fast Might It Reach Us Patients?

| No Comments
New Orleans, LA December 10, 2013 - Last time I wrote about exciting new research being done to help overcome multiple myeloma's ability to become drug resistant.  I shared how researchers had discovered how adding an deacetylase (HDAC) inhibitor with a proteasome inhibitor re-sensitized resistant cells, allowing proteasome inhibitors like Velcade, Kyprolis and ixazomib to work.

I went on to share how a promising new HDAC inhibitor, panobinostat, faces an uphill battle for FDA approval, because it doesn't work very well by itself.  And even though it clearly enhances the effectiveness of existing myeloma drugs, the FDA likes to see single-agent activity before approving most drugs.

The first immunotherapy drug for multiple myeloma, elotuzumab, faces a similar challenge.  It works well when combined with Revlimid, but not on its own.  That's why doctors and researchers here are so excited about daratumumab. Unlike elotuzumab, daratumumab does seem to work by itself or in combination with other myeloma drugs.

Immunotherapies are a hot topic in cancer research.  One could argue that myeloma immunotherapy research has lagged behind therapies already being used to fight breast cancer and melanoma.  But drugs like daratumumab could help change all of that.

Anti-cancer vaccines are another type of immunotherapy.  The ImMucin therapeutic vaccine for multiple myeloma shows exciting promise in early studies.  Therapies like genetically modified T cells are also making news.

When will any of this cutting-edge research reach patients?  Well daratumumab and possibly elotuzumab may only be a few years away.  Since they are a new class of drug, the FDA is more likely to fast track their approval.

Myeloma vaccines, modified T cells and other space age therapies may still be a decade away.  But maybe not!  Research in the field is advancing at break-neck speed, aided by high powered computers and willingness of researchers to share their data.  What used to take ten years now may only take five.

So stay healthy and hopeful!  Eat well, exercise and get plenty of sleep.  And a few prayers -and keeping your fingers crossed- can't hurt!

Feel good and keep smiling!  

-- Pat

New Orleans, LA December 10, 2013 - Monday is "myeloma day" here at ASH.  Non-stop oral presentations about myeloma research from 7 a.m. to 7 p.m.  My day started listening to four presentations focused on overcoming drug resistance.  

It sounds like they're making progress. But I'll be honest. Even though I read and write a lot about multiple myeloma every day, some of the research at the cellular level is way over my head.

Even so, I was able to learn that researchers are now able to overcome myeloma's resistance to proteasome inhibitors (Velcade, Kyprolis, ixazumib) by adding an HDAC inhibitor to the mix.  What the heck is an HDAC inhibitor?  That's where I start to get lost in the specifics.  But an example of a HDAC inhibitor would be panobinostat, a new experimental drug that's in Phase III trials.

Apparently, adding panobinostat to any of the proteasome inhibitors effectively re-sensitizes myeloma resistant cells. In other words, the combination of certain HDAC inhibitors helps drugs like Velcade work again.  

Great news, right?  It's this type of research that has excited myeloma experts for years.  But there's a catch.  This research is being done in mice and human cells in the lab, not in people.  

How is it working in multiple myeloma patients?  Reviews are mixed.  Like a number of other exciting new drugs, the tendency is for them to work better in the lab than in us.  No one is really sure why.  Maybe scientists haven't found just the right mix yet. 

Based on what I've read and experts I've interviewed, panobinostat therapy works.  Here come's that "but" again. There may be a problem getting the drug approved by the FDA.  Why?  Because it doesn't do much by itself.  It needs to be combined with another existing myeloma therapy.  And thus far, the FDA has only approved drugs that show significant single agent activity.  

I'll post more about some of the exciting new therapies being reviewed here at ASH next time.

Until then, feel good and keep smiling!  

-- Pat

New Orleans, LA December 9, 2013 - Attending the American Society of Hematology (ASH) annual meeting as a patient is a hopeful, yet exasperating experience.

The current meeting in New Orleans is my fifth ASH. Ironically, the first ASH I attended was here, too. I remember being amazed by the size and scope of the event; so many working so hard to help blood cancer patients live longer.  

Then as now, I found myself baffled by the dichotomy of research's progress. On one hand, you can almost feel the excitement in the air over the burgeoning development of immunotherapies and innovative, new pathways researchers can use to help destroy myeloma cells. On the other hand, doctors are still struggling with the most basic questions about when, with what and how much to administer to patients.

This would be understandable if I were referencing newly FDA-approved drugs like Kyprolis or Pomalyst. But I'm not. At the first event here at ASH, the International Myeloma Foundation's symposium, Critical Issues Need Answers: Providing Best Options for Myeloma Treatment in 2013, a panel of six world-renowned myeloma experts pleaded for more data about how to use Revlimid and Velcade.  Revlimid and Velcade? They were approved seven or eight years ago!  Did you know that Velcade is still not FDA-approved for use as maintenance therapy? Call the company and no one can tell you how often to dose patients post transplant, even though it has been used to treat patients now for over a decade.

Think of it this way. Drug company and academic researchers are frantically trying to discover the next, best thing. The result? Myeloma science is outpacing our doctors ability to apply it.  

Going back and doing clinical trials to help doctors understand the "who, when and with what" isn't sexy. It's also expensive and sometimes difficult to enroll patients in trials. Many patients are more interested in enrolling in cutting-edge trials than how much dexamethasone works best with Revlimid or Velcade. But both types of trials are important for our futures and quality of life.

I'm looking forward to sharing both the hopeful--and more challenging aspects of ASH--over the next few exciting days here in New Orleans.

Feel good and keep smiling!