Advancing MPN Research: How Clinical Trials Work from Patient Empowerment Network on Vimeo.

How do clinical trials advance MPN research? Dr. Angela Fleischman shares insight about the clinical trial process and the significance of clinical trials in moving MPN research forward.

Dr. Angela Fleischman is a physician scientist and assistant professor in the Department of Medicine at the University of California, Irvine. Learn more about Dr. Fleischman.

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Transcript:

Dr. Fleischman:

Well, there are multiple stages of clinical trials. One needs to have some rationale for testing a specific drug in patients. You just can’t say, I just want to take something off the shelf and see if it works for myeloproliferative neoplasms. 

There could be different ways that things sort of enter into clinical trials, either preclinical data from in vitro, meaning, in the lab, in the liquid media, with cells, that makes somebody think that it might work in humans, or that it works in a similar disease to myeloproliferative neoplasm. So, it’s a little bit of a stretch, but a very rational stretch, to then test it in a new population. 

First and foremost, safety needs to be evaluated, because as physicians, one of our primary objectives is to do no harm to patients. So, at very early stages of clinical trials, the primary objective is to see what the appropriate doses, what’s tolerated, what the side effect profile is. 

And then, moving on to efficacy. So, maybe it’s tolerated, but does it actually work at the next stage of clinical trials. Then, a much larger clinical trial would be to do a head-to-head comparison between, in most cases, standard of care versus drug X. 

And I think, for clinical trials, in particular, for myeloproliferative neoplasm, it’s very important to understand what the stated, primary end point is, in particular, for myelofibrosis patients, that myelofibrosis patients may have different problems. Some myelofibrosis patients, their primary issue may be anemia. And so, if they’re looking for a clinical trial to address their anemia, they would probably want to be looking for one whose primary end point is transfusion, freedom from transfusions, or improving the anemia, not necessarily – there was another trial that’s primarily looked at spleen reduction, but they didn’t have an enlarged spleen, that, necessarily, wouldn’t be appropriate for the patient. 

So, I think it is particularly important in myeloproliferative neoplasm to identify what the primary end point is, and whether what you’re going for is that primary end point. 

Katherine:

Mm-hmm. Any advances that are being done in MPN research require MPN patients to participate in clinical trials, right? 

Dr. Fleischman:

Of course. 

Katherine:

But what if they don’t? Why is it crucial that patients participate in trials? 

Dr. Fleischman:

Because without participation in clinical trials, we are not going to further our understanding of myeloproliferative neoplasm. Many of the drugs that we use today in myeloproliferative neoplasms, as well as other diseases, the reason why we use them today is because people 10, 20 years ago participated in the clinical trial and demonstrated a benefit of these medications. So, people don’t participate, we’re not going to have new drugs for myeloproliferative neoplasms.