We basically treat MDS patients the same today as we did 50 years ago. We can help some patients live longer, maybe by a few months or so, but we haven’t made much progress over several decades.

Kousteni: The insight into the genetic landscape that correlates with MDS development has not translated yet into an effective therapy for the disease. Patients who are treated with the available treatments eventually relapse. We do not fully understand what initiates the disease, and hence, we cannot prevent it, and we do not know, among MDS patients, who will develop AML. There is so much we still need to uncover to better understand what causes and predicts disease.

We aim to cure MDS and to cure leukemia. I am not going to settle for an improvement by two months in survival. In order to do that, the path is very clear. We investigate human samples [of MDS and AML] to find the first cell. That is our aim. That is our goal. That is what we are going to accomplish. By the time cancer declares itself it's already too late. We have to find the cancer early—the first cell—and prevent it then.

Kousteni: We’re working to understand MDS pathogenesis in multiple ways—by studying MDS cells, their precursors, and identifying the early cells that give rise to them and examining their evolution as the result of influences from all cells in their bone marrow environment. We are also studying what happens to the body as we age. Our goal is to harness knowledge from these approaches to come up with entirely new and effective therapies that are preventative or curative.

We have the ability to use the serial samples from the MDS repository to follow patients from start to finish and identify what are the biomarkers they are shedding in the blood, whether it is in the form of the first leukemia cell, the first cell, or in the form of broken pieces of protein, DNA, or RNA. What can we pick up [from these samples] before treatment and after? What markers can we pick from the serial samples of blood, serum, and saliva of these patients that will tell us the stress markers that are making some patients develop leukemia within months, and others living for 10 years without developing leukemia?

This is another strength of the center—the tissue repository, the technology we now have, the intellectual group that we have collected to ask the right questions, and then patients who we can follow in a dynamic manner.

Kousteni: I will relentlessly aim for comprehensive, novel and effective ways to prevent the disease, and when it has advanced to cure it, to manage leukemia, so people do not die from it. If we don't aim for change, for improvement, for a cure, we will never get there. I am wowed by the enthusiasm, the commitment, and the continuous presence and responsiveness of my colleagues and their trainees in the center. I believe we have the expertise, the knowledge, the tremendous will and commitment, and a game-changing collaborative spirit to achieve this as a team.