Steve Mathai, MD Transcript 112

Nov 17, 2017

Dr. Stephen Mathai is an Associate Professor of medicine at the Johns Hopkins University School of Medicine. His areas of clinical expertise include pulmonary disease and critical care medicine. His research interests include scleroderma-associated pulmonary hypertension, idiopathic pulmonary fibrosis and pulmonary complications of connective tissue disease. Dr. Mathai discusses how scleroderma increases your risk of the development of pulmonary hypertension. Dr. Mathai discusses clinical trials and how they're important for patients with rare diseases like scleroderma and pulmonary hypertension.

My name is Steve Mathai. I'm a pulmonologist at Johns Hopkins School of Medicine, and my clinical and research interests are in pulmonary hypertension, and specifically in scleroderma associated disease.

Today, I'd like to talk about clinical trials and how they're important for patients with rare diseases like scleroderma and pulmonary hypertension.

Clinical trials are really the backbone of advancement in the treatment of patients with any disease. In particular, in a rare disease like pulmonary arterial hypertension and a rare disease like scleroderma, I think it's particularly important to understand that advancements in our treatment armamentarium for patients really depends upon clinical trials. So, we have a pretty active clinical trial group at our center here at Hopkins, and there are many centers across the country that participate in clinical trials, either as part of pharmaceutical industry clinical trial or even clinical trials that are supported by the National Institutes of Health, the NIH, or clinical trials that are supported by local investigators. So, I think that there are many different ways in which a clinical trial can come to being.

As I mentioned, imperative that we have patients participate in these clinical trials to better understand what we observe either in the lab with animal models of pulmonary hypertension or scleroderma, or what we observe clinically in other disease states that are related potentially to scleroderma or pulmonary hypertension. If those observations about the impact of a particular medication or a particular intervention are positive, it's really important for us to understand whether that translates to what we see in humans with the disease.

So, we at our center have many patients who are involved in various forms of clinical trials. Sometimes the clinical trial does not involve an experimental drug. Sometimes it involves combinations of existing drugs. Sometimes it involves using a drug that is approved for another indication, for another disease state, and applying it in patients with either scleroderma or pulmonary hypertension, or patients who have underlying disease states that make them fall into a different category than pulmonary arterial hypertension. So, patients with interstitial lung disease, that scarring process, they can develop pulmonary hypertension, and there are ongoing studies now looking at pulmonary hypertension therapies for those patients.

So, I think clinical trials are the way that we can really understand whether something that we observe, either in the lab, in our animals that we study to understand better how pulmonary hypertension develops and progresses, or for observations that we see clinically in patients to really confirm that and understand whether those types of interventions are going to be valuable for patients with pulmonary hypertension.

I think many times patients are concerned about the time commitment that's involved and the risks that are involved. And these are valid concerns. Clinical trials aren't for everyone. And I think that you have to have an understanding that a clinical trial, depending upon the particular study that you're talking about, can last for weeks, to months, to years. And that could involve you coming back to the physician's office multiple times over that time period, and having to undergo tests or fill out forms, on a repeated basis. And that involves time and travel, and the commitment on the patient's end, which is significant. That's something that we really appreciate and value from a provider's perspective, and understand that it's a huge commitment.

I think the other thing that can happen is, is when we're talking about a new agent, a new drug, that there's some concerns that we don't know exactly what the effect is going to be. And that's really the reason to do the clinical trial. We don't know whether these medications are going to improve the outcomes for patients with this disease. The caveat to that though, is in order for these medications to reach the stage of investigation, so a clinical trial stage where we can examine these things clinically, these medications have had to go through a long and arduous testing process to assess the safety.

And I think that while we can't ever be 100% sure that what we observe in animals and in healthy volunteers who take the medication, whether the fact that there's no significant or life-threatening side effects related to the medication in those kinds of scenarios, we may not be able to extrapolate that to patients with pulmonary hypertension. I think that again is another valid concern, and really talking at length with your provider about the risks and the potential benefits of any clinical trial really would more directly address those particular concerns. But those are the two biggest things, I think the time commitment and then what's the potential impact of the medication, and what are the side effects.

One of the things that people often ask about clinical trials is, "Well how do I find out about them? And where can I go to better make myself aware or educate myself about clinical trials that are currently ongoing?" So, there is a website, www.clinicaltrials.gov. It's a search engine of all clinical trials that are currently ongoing. And there is a way to search that website for pulmonary hypertension and for scleroderma. And you can see all current clinical trials.

Not only does it describe the clinical trial and who's eligible, but it also describes and lists the centers that are involved. And I think that's a very valuable resource for patients, and periodically I myself will go and check and see what studies are ongoing or what studies have been started. Because it is really hard to keep up with all the clinical trials that are going on, even for someone who is a provider and a clinical researcher in this disease. There are things that are happening every day that change and need to be understood and need to follow.

It's important and I think a valuable resource for patients to go to the clinicaltrials.gov website to see if there are any new clinical trials or ongoing clinical trials in which you might be interested in participating in and understanding where those studies are taking place and whether or not it's reasonable for you to participate based upon your location.

My name is Steven Mathai, and I'm aware that I'm rare.

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