American Pediatric Society John Howland Award 1997: Acceptance

Dr. Johnston, Dr. Shapiro, Members of the American Pediatric Society and Guests: When I was called by Dick Johnson about my selection as the Howland Awardee, I couldn't help but remember the words of the late Jack Benny when he was given a special award: “I don't deserve this award but I have arthritis and I don't deserve that either.”

Thank you, Larry for your very generous introduction.

In May 1950, I was one of three of Rustin McIntosh's residents at Babies Hospital who drove nonstop from New York City to French Lick. Indiana (the home town of Larry Bird) to attend the 60th annual meeting of the American Pediatric Society. The membership consisted of 158 active and 15 emeritus members. Here gathered in one room was the heart of academic pediatrics in North America. Twenty-four papers were read all at plenary sessions, and about 30 more at the conjoint Society for Pediatric Research sessions. As they say,“We have come a long way, baby!”

We are bonded by a great tradition and stand on the shoulders of the giants in pediatrics-the pioneers in our discipline.

It has been my good fortune to have known all the Howland Awardees. Some were my teachers and mentors-Rustin McIntosh (1961), Lawson Wilkins (1963), John Caffey (1965), Richard Day (1986) or collaborators-Barton Childs (1989), others friends of many years or acquaintances. Never in my wildest dreams did I imagine that one day I would join this illustrious group. I accept this award with deep humility, gratitude, awe; and a sense of professional fulfillment. I recognize fully that I am but one of many worthy candidates. As that well known commentator of the American scene Mae West once remarked.“It is better to be looked over than overlooked.”

Rustin McIntosh at Columbia's Babies Hospital and Lawson Wilkins at Johns Hopkins' Harriet Lane Home had a profound influence on my life and my dedication to pediatrics. I am forever grateful for their strong support and encouragement in my formative years. Their contrasting personalities were like Yin and Yang.

Rusty, as he liked to be called, was reserved, kind, witty, and wise with a quiet but striking presence and enormous inner strength, who engendered incredible loyalty among his staff and generated social trust. A distinguished leader and scholar, he set high standards by example, not edict. Rusty taught me a great deal about being a pediatrician and the role of a chairman(Fig. 1).

Figure 1

Rustin McIntosh. Carpentier Professor of Pediatrics, Columbia University College of Physicians and Surgeons and Director of Pediatrics, Babies Hospital.

Lawson Wilkins, widely regarded as the father of pediatric endocrinology, had incredible vitality and zest. He was colorful, gregarious, outgoing, dramatic, possessed of a stentorian voice, burning curiosity, and an underlying devotion to his fellows and colleagues-“his boys” (and later women) as he called us. Lawson Wilkins taught us the challenges and rewards of clinical investigation and to seek out the best minds, whether in basic or clinical science, when addressing a challenging research endeavor(Fig. 2).

Figure 2

Lawson Wilkins, Professor of Pediatrics, Johns Hopkins School of Medicine, Director of Pediatric Endocrine Unit, Harriet Lane Home.

After 10 years of establishing and developing a pediatric endocrine unit at Babies Hospital as a member of the faculty at Columbia, the offer to move to the University of California. San Francisco, an unforeseen and felicitous event which I accepted with alacrity, was the greatest opportunity in my professional life. During the two decades I served as chair at UCSF-an immense privilege-I had the support and loyalty of two exceptionally wise, talented, and critical Vice Chairmen, Abe Rudolph and Moses Grossman, who must share fully in whatever success we had.

To direct and participate in an effective, competitive, and funded research program is a demanding, challenging responsibility, but a great counterpoint to the administrative activities of the chairmanship. My research activities would not have been possible without my incredible partnership for forty years with my colleague and friend Dr. Selna Kaplan. My debt to her for her unwavering support, loyalty, dedication, hard work, and as my severest critic knows no bounds.

It has been our good fortune to have been joined over the years by a host of unusually talented, imaginative, and highly motivated fellows who have gone on to distinguished careers in academic pediatrics and basic and clinical science in institutions here and abroad. They taught me much and their friendship and contributions are a continuing source of gratification.

Lastly, I want to thank my wife and our children Ethan, Kevin, and Anthony for their love, incredible support and understanding, and for fortifying my social conscience. My wife, Dr. Madeleine Grumbach, has a low tolerance for pretense, posturing, arrogance, and self-importance; she is possessed of indefatigable moral energy tinged with humor. Without her support and care I would not be standing before you today. Our sons' friendship and accomplishments have given me great happiness.

I now would like to turn briefly to several issues that affect the state of research in pediatric departments.

The end of a century, and even more so the impending dawn of a new millennium, is a compelling opportunity to take stock of academic pediatrics: to recognize its achievements, and the distillation of lessons and experiences we have inherited from the larger than life giants in our field; the opportunities we have missed; the lens through which academic pediatrics perceives and interprets the microcosm in which we all have a role. The era that began shortly after the end of World War II, the halcyon days of exponential growth in NIH support of research, ended in the late 1980s. We are now in a time when stable and generous funding of biomedical science is over. We are, as Harold Varmus, the Director of the National Institutes of Health, states in a “steady state,” an end to growth and an uncertain future. Aside from raising our voice in the political arena and pounding on the table, how will we adjust to the new reality after almost four decades of privilege?

I will briefly consider only one aspect that merits our most serious attention: the future of clinical investigation, scholarship, and post-residency academic training in our field and the need to reexamine and upgrade our current models. In a time when scientific and technological advances have progressed at break-neck speed, my crystal ball is cloudy. In looking to the future. I am ever mindful of the admonition of a Japanese sage.“Do not do what your ancestors did; rather seek what they sought.”

My definition of the clinical investigator is a broad one and encompasses all types of research from studies at the molecular and structural level to research on the mechanisms and treatment of diseases, to inquiry in the behavioral and social science aspects of child health, to health care delivery and health policy issues.

As for the “good old days,” when Rusty McIntosh and Lawson Wilkins made their lasting contributions to pediatrics research grants were a rarity, positions in academic pediatrics were hard to come by, and laboratory facilities were primitive. After four decades of privilege, today we face new and critical restraints on the future of academic pediatrics as we have known it. Our challenge is to address these new road blocks to creative scholarship, productivity, freedom, individuality, and the capacity to remain in the vanguard of medical progress, and to generating and translating new knowledge and new technologies into improvements in pediatric care and child health. If we do not do this, who will! The nation, at last, shows signs of beginning to focus on the health and well being of children and families. Remember it's not that research funding has been slashed-there is not much change in constant dollars but there are more talented people competing for funds.

As I look ahead to the next century, I foresee academic pediatrics as the preeminent intellectual leader in contributing to the scientific base of clinical medicine in broad and diverse ways. We have the challenge to exploit and apply the rapid advances in molecular and developmental biology and genetics. The new biology has cut a wide swath through all our subspecialty disciplines. It will provide the informed pediatric investigator the unique opportunity to apply these scientific advances to clinical pediatrics. Our insight into the multifactorial components of disease will enable us to understand more fully the origin in infancy and childhood of the afflictions of the elderly.

Pediatric investigators have been a major contributor to our knowledge and treatment of inborn errors of metabolism. Now, the dramatic advances in developmental and cellular biology are contributing significantly to our understanding of the pathogenesis of birth defects. As my colleague Charles Epstein calls it, the inborn errors of development-the new dysmorphology. These discoveries about the genesis of birth defects have advanced the field from the descriptive to the experimental stage. Although in many instances the promise of prevention and treatment are years in the future, the groundwork is being laid.

The findings in long-term epidemiologic in long-term epidemiologic studies suggest that nongenetic mechanisms affecting the growth of the fetus and newborn are an important risk factor in the development of hypertension, coronary artery disease, and diabetes in later life-an exciting new research area but not a new concept to pediatrics.

The opportunities are unlimited. Recall that the study of Burkitt's lymphoma in a child provided the first example of a human oncogene associated with cancer and that the retinoblastoma gene was the first human tumor suppressor gene isolated. Sequencing of the human genome is on track to be completed in the first decade of the new century: the genome of some bacteria, Archea, and yeast are already at hand and that of a nematode promises to be available within two years. Functional genomics is rapidly changing the landscape of biomedical research. Mutating and inserting genes in the mouse by homologous recombination by providing techniques to generate animal models of gene function have been a boon to the physiologist and clinical investigator.

In my research discipline of hormonal effects on growth and maturation, advances in the genetics of sex determination and differentiation, and molecular biology, neuroendocrinology, receptors, signal transduction, and hormone action are unparalleled achievements. The impact of the basic sciences on pediatrics is incredible and the promise to advance our understanding of human development unlimited. We are truly, by any yard stick, in an era of revolutionary change, a tectonic plate shift, in biomedical research. We must work to narrow the chasm between the language and methodology of the basic scientist and the clinical investigator.

The progress and opportunities in the behavioral and social sciences, the prevention of disease and the maintenance of health, issues of health policy and quality of care as related to pediatrics and especially adolescence, are striking but time does not permit their elaboration.

What of the training of our future leaders in pediatric science and clinical investigation-in disease and “dis-ease” -oriented research. How can we mentor and prepare these gifted young women and men for the increasingly competitive, dynamic world of biomedical science?

First, we must identify those candidates with integrity and creativity; with a capacity for problem solving, strong motivation and enthusiasm, intensity of commitment, ability to organize, self-discipline, and the potential for original thought (Table 1). We need excellence without elitism. Remember as well there is a life outside of medicine. Do not confuse dedication with obsession.

Table 1 Attributes sought in candidates for a career in pediatric research

We must examine our current model of training in pediatric science and asses whether it is the guiding light to our future. I firmly submit that it is not.

Our fellowship training needs to be drastically revised using new paradigms. For example, at UCSF Ph.D. students, irrespective of the basic science department that they have selected to study for their degree, enter a joint core program in molecular, cell, and developmental biology before further specialization. It provides a common language of the new biology. This program, in effect for over a decade, has been exceedingly successful. Similarly, our trainees in pediatric science need a core exposure as a group to these and other basic science disciplines before embarking on their research in a subspecialty.

In the same vein, future clinical investigators should receive training in bioethics, biostatistics, epidemiology, experimental design and hypothesis testing, informatics and computational technologies-how to store, retrieve, and manipulate data effectively, communication skills including manuscript and grant preparation, and finally, how to collaborate and develop effective collegial and cooperative relationships with basic scientists, other clinical investigators, and the biotechnology industry.

The Department has the responsibility to provide their young faculty researchers with protected time to think and develop in their chosen field. Financial support for research is essential but not sufficient. Time and scholarship lead to productivity; stimulating colleagues in the department as well as in basic sciences and other clinical departments serve as catalysts. The leadership in pediatric departments must not be averse to risk in selecting new faculty. They should be willing to take informed risks and be willing to accept the risk of failure. Try for the best! That is leadership! Moreover, we need to apply critically the principle of “selective excellence,” a ladder of values, in choosing the specific programs to invest in.

These suggestions for training afford the promise of doing much to bind together those engaged in pediatric research and to act as an intellectual and critical scientific foundation within the Department and a counterpoint to the fragmentation and isolation which subspecialization and focused research engenders.

To the young pediatricians here this morning, I have a deep and abiding faith in the next generation of academic pediatricians. You are part of a noble tradition and calling and I say to you:

• Never be too timid to question the “experts” and conventional wisdom. Remember. “the handwriting on the wall may be a forgery.”

• Consensus is often wrong-witness our quadrennial presidential selection process.

• “Never let the fear of striking out get in your way.” Joe DiMaggio only got a hit in one of three at bats and he is in the Hall of Fame.

• “Do what you can, with what you have, where you are.”

• “If you see the light at the end of the tunnel, you are going the wrong way.”

• “Whenever you fall, pick up something.”

• The first and great commandment is “Don't let them scare you.”

To close I have had a fulfilling, challenging, and happy life. My love for pediatrics and its practitioners shines brightly. The American Pediatric Society has meant a great deal to me and it is a unique privilege to have been the recipient of the John Howland Medal. I accept this Award on behalf of my colleagues, present and past, in the Department of Pediatrics at the University of California San Francisco, who have been a constant source of inspiration, collegiality, and support, who enabled me to grow and, from whom I have learned so much.

In the words of Albert Camus:

A man's work is nothing but his slow trek to rediscover, those two or three great and simple images in whose presence his heart first opened.

Thank you for this great honor.