What do you think are the most impressive or significant advances in MDS research and treatment over the last five years since our 25th anniversary? If you extend this back 30 years, to 1983 (when AA&MDSIF was founded), what does the longer perspective indicate?
Starting with the 30 year perspective, the two greatest advances in MDS over the past 30 years are, first, the understanding of the disease on a population level and on a biologic level. In 1983, we really weren’t sure how many people in the US and worldwide actually had myelodysplastic syndromes. Up to that point, there had only been a few hundred patients who had been reported, and it was not until database studies began and the US government in 2001 began to formally track MDS that we realized how many people actually are living with the disorder in the US. We now know that approximately 15,000 people in the US are diagnosed with MDS annually, and anywhere from 50,000 to 100,000 people in the US are living with MDS at a given time. So, especially in the past 15 years, we have gained much knowledge that we didn’t have before.
We also have realized how incredibly complex MDS really is from a biological perspective. We may divide it into four or five subtypes, or even 10 subtypes (depending on the classification system used), but MDS really represents hundred of subtypes. That has helped in our thinking about prognosis and therapies for MDS.
A second area that has advanced in the past 30 years is for developing therapies specifically for MDS. It used to be that if you were diagnosed, your only options were blood transfusions or platelet transfusions. Now, we have three FDA-approved drugs for MDS and dozens of drugs that are being testing in clinical trials. We have also moved into an era of combination therapies for MDS, and more patients each year are receiving curative therapy in the form of a bone marrow transplant for MDS. There has been a remarkable amount of progress in that time.
In the past five years, a study published in 2009 demonstrated that for the first time, an MDS drug, azacitidine, has provided a survival advantage. We have a rising number patients every year who are receiving a bone marrow transplant, and for the first time, there is recognition by Medicare of the use of bone marrow transplant in patients with MDS. There has been an explosion of information about the genetic basis for MDS. It is to the point now where we can detect genetic/molecular abnormalities in 80% of people with MDS.
At the same time, in the last five years, have there been setbacks or instances where you feel progress has not kept up with general expectations or predictions made five years ago?
Five years ago, I felt there would be another drug approved since the last one in 2006, and that has been a disappointment. Also, the flip side to our expanding knowledge of MDS is that we recognize that MDS represents a much more complex collection of diseases, that therapies therefore will never be “one size fits all,” and that truly targeted approaches will therefore only be applicable to a minority of MDS patients.
With MDS, is there a consensus on overall direction research will take or needs to take --- or is this as diverse as the areas of interest that individual researchers or research teams have?
We can agree on general themes for new investigations in MDS. We need to develop new drugs, and they should be targeted to these molecular abnormalities we are now detecting. We should be able to use these molecular abnormalities to predict which existing drugs are more or less likely to work in someone with MDS. We need to understand more about the impact of the disease on the population. Also, we need to continue to perform very complex analyses, like genomic analyses, to continue to understand the biology of the disease.
In 2009, we asked a similar panel if generalist hematologists/oncologists were adequately informed about bone marrow failure disease, and what could be done to bridge any gap between the generalists and researchers/specialists. What are your thoughts in 2013?
I think this is the same as five years ago—we must continue to emphasize to general internists or family medicine doctors that anemias or other cytopenias in older adults are not just a normal consequence of aging. These abnormalities should be explored and patients should be referred to hematologists. This may increase even further the prevalence of MDS in the US, and treatment options exist regardless of the age of a patient.
About treatment, we have no misperception about the challenges patients face traveling long distances to a specialized treatment center such as Cleveland Clinic or similar ones across the country to receive therapy. Our focus is to develop a treatment plan over one or two visits and then maximize the patient’s quality of life by having the routine or ongoing therapy be conducted closer to their home.