Few clinicians have been as central to the development of treatments that demonstrate the value of medicines in advancing
public health as Dr. Antonio Gotto. Currently serving as Dean of the Weill Cornell Medical College in New York, Gotto is best
known for his research to identify the central role that lipoproteins play in the development of atherosclerosis and cardiovascular
disease—the leading causes of death and disability in most countries today and thus a major driver of the healthcare cost
burden. Studies that built on his work have shown that changes in serum cholesterol levels can lower the incidence of heart
disease, coronary infarctions, and stroke, which has led in turn to the introduction of one of the most successful and cost-effective
medical interventions of the last 30 years—the statin drug.
Antonio Gotto, MD
Gotto's career is also defined by his early advocacy of translational research, which is now accepted as a given in the discovery
process but rarely figured in the clinical dialogue when he began investigating how biochemistry could be applied to understanding
the pathophysiological basis of diseases such as cancer and CVD in the 1960s. Inevitably, persistence in the face of long
lead times became a key character trait. "It required nearly two decades to structure and sequence the DNA of a key protein
in plasma lipoproteins (apolipoprotein B), work which we commenced in 1967, only concluding with the peer review publication
of our findings in 1985." According to Gotto, the policy message that should resonate from this long experience is that maintaining
a predictable climate for the conduct of research is necessary to ensure that good ideas have the solid institutional grounding
to move forward. This is a priority for Gotto as Dean at Weill Cornell, where he is spearheading a campaign to raise $1.3
billion for discovery research in eight therapeutic areas of unmet medical need.
Gotto is passionate about the contributions industry makes in driving research toward the practical endpoint of a safe and
clinically useful drug that represents an advance in treatment. "The New York Times asked me recently why I was involved in work with the drug industry. I gave them a simple answer: Because in my area, that's
where the action is." Over the years, perspectives have shifted and some initial challenges of working with companies, including
expectations that their relationship with researchers like Gotto be exclusive and proprietary, have faded. Says Gotto, "We
now have a consensus that prospects of success are greatest when we can pool resources and expertise, not just from industry
but from government and the academic centers as well. Reliance on this integrative model is bearing fruit, as seen in the
upward trend in FDA registrations so far this year."
Gotto distills from his decades in the arena the value of educating the public around basic medical interventions that work.
He prizes his long association with the late cardiovascular surgeon Michael DeBakey, with whom he established the nation's
first research center on heart disease that produced landmark work on ways that patients could improve their condition with
simple changes in behavior, such as diet and exercise. Drs. Gotto and DeBakey were among the first to introduce what we now
call a preventive and wellness agenda, as a fundamental adjunct to research. "The problem we still have today is our medical
payment system provides little incentive to advance a preventive health agenda."
Nevertheless, Gotto believes that once again good research can lead to action. "Most current R&D on lipid control is focused
on the middle-aged and older population cohort. What we need now is to see how targeting these therapies for use even earlier
in life can prevent the onset of coronary disease at a later stage. Why is it that right up to the 20th century, average levels
of LDL in the human population were much less than they are today? It may be a lifestyle issue, so if we want to get back
to these good levels we have to focus on a combination of prevention and progress in making the next generation of statins
safer and more effective for long-term use." – William Looney