2011 Lasker~Bloomberg Public Service Award

Standard of excellence for research hospitals

The 2011 Lasker~Bloomberg Public Service Award honors an institution that has served since its inception as a model research hospital. Since 1953, the Clinical Center of the National Institutes of Health (NIH) has provided innovative therapy and high-quality patient care, treated rare and severe diseases, and produced outstanding physician-scientists whose collective work has set a standard of excellence in biomedical research. It has spearheaded major advances in a wide array of medical arenas, established an example for academic institutions across the world, and trained thousands of investigators, many of whom now lead those establishments.

On July 6, 1953, the NIH Clinical Center admitted its first patient and embarked on its mission to generate new ways to diagnose, treat, and prevent disease by connecting bedside observations with laboratory inquiries. Patterned after a similar design at the Rockefeller University Hospital (New York City), the NIH's Center scaled up the endeavor to create a larger institution with vastly more patient-recruiting power. By grappling with perplexing illnesses in a setting where laboratories and hospital beds share a building, the Center united basic-science researchers, clinicians, and patients in their shared goal of understanding and combating disease. Today, 18 of the 27 NIH Institutes and Centers, each of which focuses on a particular biomedical realm, rely on the Center. There, scientists can benefit human beings by applying knowledge they have gleaned from experiments on cells and animals. Specialists from all over the NIH join forces with each other, the highly skilled hospital staff, and the patients.

The Center has touched many conditions and countries. It tackles common plagues such as tuberculosis and rare ones such as cystinosis. Its patients suffer attacks on the entire range of the body's systems, from endocrine, neurological, blood, eye, and autoimmune disorders to adrenal problems, vitamin deficiencies, infectious diseases, and behavioral conditions such as schizophrenia and depression. The Center's success relies on patients as collaborators, and they have flocked there from 149 countries that stretch from Brazil to Bahrain to Botswana. In 2010, the Center treated 10,086 individuals and since it opened, it has ministered to almost 450,000. Its physicians are currently following 575 unique diseases. The Center provides state-of-the-art care at no cost to patients, thus facilitating their contributions to the research enterprise. Because of its geographical reach and financial resources, the Center's studies can accrue participants unusually quickly.

Medical innovations from the Center have spanned the full spectrum of human illness. For example, in the 1960s, Roscoe Brady and Elizabeth Neufeld uncovered the causes of several rare diseases in which lipids or carbohydrates, respectively, accumulate and harm tissues. The researchers pinned down specific enzyme deficiencies that underlie the maladies and laid the groundwork for diagnosis, prenatal detection, and, in some cases — such as Gaucher's disease — enzyme-replacement therapies. At the same time, scientists at the Center developed and administered combination chemotherapy for a variety of cancers, including acute lymphocytic leukemia in children. In that era, the oncology community was skeptical of this new approach, as conventional wisdom held that chemotherapy could not cure advanced cancer. Fortunately, the Center provided an environment that allowed Emil Frei and Emil J. Freirich to pursue unorthodox methods, and many of their patients achieved complete remissions. Soon afterward, they and other NIH investigators made similar advances in other cancers, including Hodgkin's disease. The Center played a central role in studying and treating AIDS as well. In the 1980s, its scientists developed AZT, the first effective drug for the disorder, and discerned key pathological features of the disease.

Activities at the Center have also enhanced the health of people who do not suffer from severe or unusual illnesses. Work there on hepatitis B and C viruses as well as HIV has radically improved the safety of blood supplies worldwide.

The Center continues to cultivate discoveries. For instance, in the last decade, Daniel Kastner has investigated a rare and devastating immunological disorder. This condition — neonatal-onset multisystem inflammatory disease — attacks the nervous system, skin, and joints. Kastner and his colleagues traced the ailment to defects in a gene that regulates inflammation and identified a compound that dramatically improves symptoms. Another recent therapeutic breakthrough involves a rare inherited disease, lipodystrophy, in which affected individuals lack fat tissue, which normally produces a hormone called leptin. The resulting leptin insufficiency leads to a severe form of type 2 diabetes, high lipid levels, and liver problems. Phillip Gorden and his colleagues reversed many of the metabolic abnormalities in these patients with leptin therapy.

New ventures at the Center include the Undiagnosed Diseases Program, launched in 2008 to recruit individuals whose conditions have stumped the medical community. More than 4000 patient charts already have arrived at the Center. Multidisciplinary teams are puzzling out who they can most likely help and what is happening in those people's bodies. The program is beginning to make headway. For instance, researchers traced one unusual condition to alterations in a gene that normally prevents calcium from depositing in blood vessels. Now that scientists know what instigates the illness, they are developing methods to treat it.

The NIH has long attracted some of the country's best and brightest early-career physician researchers, many of whom have played pivotal roles in the Center's activities and have since carried the fruits of their training elsewhere. During the Vietnam War in particular, when virtually all male doctors were drafted, the agency drew a tremendously talented group because it allowed physicians to discharge their military service while learning how to conduct first-class research. Many of those individuals got hooked on experimental clinical science and became some of the nation's most distinguished investigators at academic and other institutions.

For almost 60 years, the Center has excelled in patient care, research, and training. Its achievements have earned high accolades; Lasker Awards have honored numerous investigators who did their clinical work there. By intermingling clinicians with scientists who untangle basic biological processes, it has sparked insights and innovations, often on sicknesses that eluded diagnosis and treatment in conventional settings. The Center has offered hope to patients and provided a template for clinical research institutions across the globe.

by Evelyn Strauss

Award presentation by Harvey Fineberg

As chair of the Lasker Foundation's Public Service Award Committee, it is my privilege to introduce the recipient of the 2011 Lasker~Bloomberg Public Service Award.

The 2011 Public Service Award goes not to an individual, but to an institution, and not for a single discovery, but for sustained and singular contributions to health. Establishment of the Clinical Center at the NIH in 1953 was a far-sighted investment by the US government: it created a model for the fully engaged, research-intensive, academic medical center, and the NIH Clinical Center went on to train some of the country's most esteemed medical scientists.

The Clinical Center of the NIH has provided decades of public service through a skillful marriage of science and clinical care. The Center and the individuals associated with it — as investigators, caregivers, and trainees — have made major progress in every area recognized by the Lasker Awards: in diagnosis, treatment, cure, and prevention of human disease.

The Clinical Center is the largest hospital in the nation devoted entirely to clinical research, and it operates on a scale that applies the benefits of research collaboration across the full spectrum of diseases. More than 400,000 patients have participated in the Center's research program over the years. All patients receive care free of charge and often participate in early trials of new drugs and other treatments.

Let me highlight a few of the contributions emanating from the work of the Clinical Center:

  • Discoveries in the 1960s by Roscoe Brady and Elizabeth Neufeld traced specific enzyme deficiencies to certain severe, inherited diseases, laying the groundwork for prenatal diagnosis and pointing the way to enzyme-replacement therapy
  • In that same decade, scientists began working on combination chemotherapy for a variety of cancers. At the time, chemotherapy was viewed mainly as a means of palliating cancer rather than as a possible cure. The Center, and the freedom it provided, enabled clinician-scientists to explore the unorthodox, even radical idea that combination chemotherapy could lead to complete remission for certain types of cancer
  • The Center's work to identify hepatitis B and C viruses as well as HIV helped to dramatically lower transmission of these viruses in the blood supply, and their scientists demonstrated the therapeutic potential of the first effective drug to combat AIDS, AZT

Today, the Clinical Center's Undiagnosed Disease Program, launched in 2008, brings in patients with medical problems that defy most experts. Since 2008, the center has reviewed more than 4,000 distinct patient records, and collaborative research on the causes of many of these unusual diseases is underway. Success in understanding the nature of a rare disease can also shed light on the etiology of common diseases, thus helping many individuals.

Beyond its success in clinical research, the NIH Clinical Center from the start has served an educational purpose, attracting talented, young researchers to train with eminent scientists and physicians. The Clinical Center boasts a stellar roster of alumni who, as trainees, played pivotal roles in the work of the Center and went on to become distinguished scientific leaders throughout the United States and around the world. The impact of the Center on clinical and basic science has amplified across generations and geography.

The NIH Clinical Center continues to rise to the challenge of undiagnosed disease and untested cures. In 2005, the Mark O. Hatfield Clinical Research Center opened with a flexible capacity of 240 inpatient beds and 82 day beds. Senator Mark Hatfield of Oregon was a vigorous champion for medical research throughout his congressional career. I would like to take a moment now to recognize Mr. Visko Hatfield, the late senator's son, who is with us today. We harbor the hope, that I am sure would have been dear to your late father, that the NIH Clinical Center will continue to prosper and serve as an unparalleled resource for clinical research and medical progress.

I would like to invite all those present who work at the NIH Clinical Center, those who work at any part of the NIH, and those who worked at any time as a trainee or a researcher at the Clinical Center to stand and be recognized.

Please join me now in congratulating Dr. John Gallin, director of the NIH Clinical Center, and all those responsible for the success we celebrate in today's recipient of the 2011 Lasker~Bloomberg Public Service Award, the Clinical Center of the National Institutes of Health.

The Clinical Center of the National Institutes of Health

Acceptance remarks by John Gallin on behalf of the Clinical Center of the NIH

Nature Medicine Essay

President Freire, members of the Lasker Foundation Board, Jury members, and distinguished guests,

What a wonderful gift Congress gave to the American public, and to the world, when it created the Clinical Center at NIH in 1947, a hospital literally wrapped in research laboratories to enable clinical research studies. You have heard already about the role the Clinical Center plays in serving as a model institution for training today's investigators and discovering tomorrow's cures. The Clinical Center, situated less than 10 miles from the heart of our nation's capital, is very much a living monument to our country's sustained commitment to clinical research. It is a place where the bench to bedside cycle, which translates basic science discoveries into new treatments and cures to improve public health, is a part of everyday life.

Of all the people who comprise the NIH Clinical Center community and the national clinical research enterprise, the contribution of the patients and healthy volunteers is critical. Their partnership makes clinical research possible — they enable a hopeful future of discovery in medicine. It is for this reason that I am delighted that two Clinical Center patients are with us today: Mr. Jerry Sachs, a dedicated member of the Clinical Center's Patient Advisory Group and a two-time cancer survivor, and Ms. Mandy Young, a patient of mine who was the first patient identified with a rare disease known as IRAK-4 deficiency. Jerry and Mandy represent the nearly 500,000 patients who have been seen at the Clinical Center since it first opened in 1953 and who are now studied in the magnificent Mark O. Hatfield Clinical Research Center.

Clinical research is risky business, yet human subject volunteers knowingly participate. On the one hand, clinical research promises hope and help, while, on the other, it involves risk. We need to study and understand better the sensitivities of patient volunteers because it is becoming increasingly difficult to recruit them to participate in our studies. That is why we recently developed a new survey instrument to understand their concerns. We have partnered recently with colleagues at Rockefeller University and the NIH national network of Clinical and Translational Awardees to evaluate this instrument. From the results of this new survey tool, we will learn how to adjust our interactions with our volunteers to assure we provide hope and help to each individual research subject as they enable us to do our research. We at the Clinical Center are firmly committed to assuring the vitality of the human subject volunteer population, and it is for this purpose, as well as for training young clinical investigators, that we will dedicate the generous honorarium that accompanies the Lasker Award.

I am honored and humbled to accept the 2011 Lasker~Bloomberg Public Health Service Award on behalf of the tens of thousands of public health servants and the nearly half-million patient volunteers who have partnered for nearly 60 years to make the Clinical Center such a special place.

Interview with The Clinical Center of the National Institutes of Health

Video Credit: Susan Hadary