Treating children with Pulmonary Hypertension – An interview with Marlene Rabinovitch, Professor at Stanford University and member of the WSPHA Scientific Board

Professor Rabinovitch, please introduce yourself.

My name is Marlene Rabinovitch and I’m a professor of pediatric cardiology at Stanford  University and I direct a program in basic research in Pulmonary Hypertension and I also direct a new program which is a combination of bioengineering and basic science to solve problems in children’s heart disease.

What, in your opinion, will be the next breakthrough for the treatment of Pulmonary Hypertension?

I think now we have tools in biology through genetics, through epigenetics, through cell biology that we didn’t have access to before and we have also very precise phenotyping of patients and I think the breakthrough in the future will come from marrying those two in understanding the specifics of a patient how their disease evolved and how we can better reverse it and treat it but I think the excitement in the new biology are the new opportunities to really discover the fundamental root cause of this abnormality.

It’s very very exciting and provocative and I think the future is very bright for our patients.

When did you become interested in Pulmonary Hypertension, and how did that affect your career?

When I began my training in pediatric cardiology it was evident that children that were not successfully repaired had major complications and in fact died after surgical correction. They had

the complication of Pulmonary Hypertension so at that time even as a trainee I recognized that it would be important to better diagnose the condition so that we could better treat it. We could put procedures in place to help these children so my first career was really establishing the guidelines for monitoring children for this complication and for treating them very aggressively in the post-operative period and for studying lung biopsy material so that we could really differentiate the children who would do well after surgery with appropriate

treatment from children who had disease that was too far advanced.

So after doing that, which was very rewarding, I decided you needed to do something also for the children who had disease that was very advanced so I began to study more about the molecular mechanisms and the pathobiology of Pulmonary Hypertension with a view that one day we could reverse even severe abnormalities that at the time precluded a successful result from a surgical treatment for children with congenital heart disease. So that’s how I began but of course, Pulmonary Hypertension doesn’t just affect children with heart disease it affects many people with different types of diseases.

So I started my own, I was doing some basic biology at Harvard but I went to Toronto to start my own laboratory to really try to understand full fundamental mechanisms and one of the mechanisms that we identified is now in a Phase One clinical trial and we hope to bring it to patients with Pulmonary Hypertension. Another treatment that was developed in our laboratory is now in a Phase Two clinical trial. So we really focus on the basic biology and what perturbs the vessel that causes this very severe fatal disease with the effort that one day we’ll be able to reverse it and to cure it.

What are the goals you wish to reach in time for the next World Symposium?

I like the idea of breaking down a rigid structure and thinking futuristically about what are the real problems that we need to address and solve in the next five years where we are going to be where does the community need answers that are new answers what are the questions that we have to begin to think about very aggressively and to really rethink what we can deliver to the community in the next five years rather than be fixated on a very rigid structure.