Why Is a Leading MPN Researcher Optimistic About Developing Treatments?

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Topics include: Treatment

Dr. Jason Gotlib, an MPN specialist from Stanford Cancer Institute, recently sat down with Patient Power to talk about his current research focus and new agents that he is excited about for MPNs. Dr. Gotlib elaborated on drugs in development such as new JAK inhibitors and telomerase inhibitors and shared why he’s optimistic about the future of MPN treatment and research.

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Please remember the opinions expressed on Patient Power are not necessarily the views of MD Anderson Cancer Center, its medical staff 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:

Hello and Welcome to Patient Power, I’m Andrew Schorr. As we’ve been telling you on Patient Power for quite some time, this is truly an exciting time when it comes to changing research for myeloproliferative neoplasms, or MPNs, and I have one, myelofibrosis. So we’re all glued to the latest news.  One of the leading researchers in the field is Dr. Jason Gotlib from the Stanford Cancer Institute at Stanford University near San Francisco. He joins us now to tell us about the focus of his research.

Dr. Gotlib:

So my research focus is the development of novel therapies for patients with myeloproliferative neoplasms, and that would be an umbrella for polycythemia vera, essential thrombocythemia and primary myelofibrosis.  And so I engage in looking at new treatments, both at what we call the Phase I setting where we're having patients go on these drugs for the first time, that is they're first in human trials.

Phase II trials, which are really expanding the number of patients in these trials to look at both effectiveness and seeing whether the drugs are, in fact, safe and tolerable, and this is really growing from the Phase I experience, is really meant to look at whether drugs are safe and potentially effective in humans.

And then Phase III which is really looking at large numbers of patients to see whether, in fact, effective signals and safety signals are maintained.  And these are trials that are looked at to see whether the drugs can possibly get approved by regulatory agencies such as the FDA.

Andrew Schorr

Certainly there’s a lot of research going on and a lot of new agents, as you call them, in development. What potential new medicines are you excited about?

Dr. Gotlib: 

Well, what I would say is that before even starting to talk about the new agents really the last several years I've been, of course, very excited about and have been participating in trials looking at JAK inhibitors.  There is now one commercially approved JAK inhibitor, which is called ruxolitinib, or Jakafi, and there are now several JAK inhibitors that are in clinical trials, and they go by names such as momelotinib and pacritinib, and there are others as well.

I'm excited about the fact that now there are trials looking at combination therapies where there are different drugs, both alternative mechanisms of action added to the JAK inhibitors to see if we can improve upon the response rates of those drugs. So, for example, we know that JAK inhibitors are very good at shrinking spleens. 

They're very good in mitigating symptoms of patients, but I think that we can do even better.

We want to see if we can yet further improve quality of life, have effects on the bone marrow, see whether we can transform the natural history of these diseases, that is, make people live longer and live better.

And so other drugs that we're looking at, for example, are drugs that we want to see can improve the fibrosis from the bone marrow.  These are so-called antifibrotic drugs.  There are other drugs that are from a group called the telomerase inhibitors, and there's a drug called imetelstat. 

There have been some very exciting data from the Mayo group out of Rochester showing that imetelstat can improve blood counts and not only improve blood counts but shrink spleens and also cause some morphologic remissions in the bone marrow, that is, make the bone marrow look from a myelofibrotic bone marrow to a normal bone marrow.

However, we really need to see more patients treated to further explore the safety and tolerability of the drug and to also further look at efficacy or effectiveness.  So these are new types of drugs.

We need in the next several years to see whether they're safe, tolerable and effective and also to see whether they are also in some way positive altering the natural history of these diseases.

There are other drugs, for example, histone deacetylase inhibitors, again being tried in combination with JAK inhibitors.  And we know, for example, that there are other acetylene pathways in the cells that are abnormal in patients with myeloproliferative neoplasms, particularly myelofibrosis, that are still activated that JAK inhibitors don't block.

And just to give one example, there's acetylene pathway called the PI3 kinase pathway, and just like there are inhibitors of the JAK proteins, there are inhibitors of that pathway.  And I think that drugs that can block those pathways in addition to the JAK pathway should be explored, and I think the next several years we'll look at those combinations of drugs to see if we can do a better job with patients.

Andrew Schorr

Dr. Gotlib, researchers such as yourself are our barometers for how things are going and where they’re headed and whether it’s significant for us. Doctor, are you hopeful?

Dr. Gotlib: 

I'm really excited about things.  The last seven or eight years have been really a blessing I think for patients because they've been given an opportunity to try JAK inhibitors.  We're seeing substantial improvement of quality of life.  They're getting out of bed, they're walking, they're more active, they're engaging their friends and their families.  We want to do better for them.  We want to be their partners in exciting discoveries that lay ahead with these new drugs, these possible new combinations.

And also they’re patients that are potential candidates for transplant.  Giving them these drugs, improving their quality of life, giving them a chance for transplant, these are potential avenues for both treatment investigation that are really exciting over the next several years.

Andrew Schorr

Dr. Jason Gotlib from the Stanford Cancer Institute. Thanks for all you do for patients. We wish you well with your research.

Dr. Gotlib:

You're very welcome.  It's is pleasure to be with you today. 

Andrew Schorr

I’m Andrew Schorr. Remember to be signed up for alerts on our website, so you’ll know whenever we post interviews like this one or anything new. Remember, knowledge can be the best medicine of all.

Please remember the opinions expressed on Patient Power are not necessarily the views of MD Anderson Cancer Center, its medical staff 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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Page last updated on August 20, 2015