On Nov. 16-17, the NHLBI held a symposium to mark the 100th anniversary of the first published scientific paper on sickle cell disease. The James B. Herrick Symposium – Sickle Cell Disease Care and Research: Past, Present and Future commemorated the history of sickle cell research, highlighted current efforts in basic and translational research in the U.S. and globally, and discussed continuing challenges and future opportunities. Over 500 registrants from around the world included people with sickle cell disease and their families, patient advocates, researchers, clinicians, and even a grandson of Dr. Herrick and a granddaughter of his intern, Dr. Irons. NIH Director Dr. Francis Collins, and NIDDK Director Dr. Griffin Rodgers were among the speakers.
"I think this symposium was extremely well designed in the sense that it was not designed just to be a purely scientific symposium nor was it just a community advocacy program," said presenter Russell Ware, a hematologist and NHLBI grantee at St. Jude Children's Research Hospital in Memphis, Tenn., in an interview. "It really brought in representatives from all aspects of the sickle cell community. I think that was intentional; I think it was wise. By having patients, families, organizations, medical professionals, basic scientists, clinicians, policymakers, government officials, it really provided the full spectrum so we could see the tour de force that really is involved in sickle cell care as we finish this century and enter the next one."
Thank you to all of the NHLBI and NIH staff who worked together with the community and speakers to plan and execute such a wonderful conference.
A complete webcast of the event can be found at:
Dr. Shurin's opening remarks from the second day of the symposium are below.
For related materials about sickle cell disease research and upcoming activities, including the NHLBI's draft sickle cell clinical guidelines, visit the NHLBI Sickle Cell Disease information center.
Opening Remarks by Dr. Susan Shurin - Wednesday, Nov. 17, 2010
Welcome back for the second day of this historic tribute to Dr. James Herrick and the progress we’ve made in sickle cell disease since Herrick’s first clinical and laboratory description of it 100 years ago.
Yesterday, we visited the history of sickle cell disease, sociocultural factors, patient experiences, and a look at the global burden of this disease and this and related hemoglobinopathies.
Today, we’re going to explore the science behind progress in sickle cell disease — advances that have made significant inroads toward understanding and battling this disease, and begin to work on where we’re going next. Forty years of biomedical research in this area has really enabled us to diagnose, prevent, and manage many of the complications of sickle cell disease — particularly those that occur in childhood.
As the first molecular disease, sickle cell disease has led the way for scientific study of genetics and molecular biology, such that it is among the best understood diseases at the cellular and genomic levels. But our most effective treatments are therapies imported from advances in treating other conditions: antibiotics, transfusion, hydroxyurea, penicillin, and bone marrow transplants.
And while these treatments build on our understanding of the clinical manifestations of the disease, they really don’t exploit our molecular understanding of it. With the foundational knowledge in hand, we can now seek more targeted and tailored therapeutic solutions. Thanks to what we’ve learned from research, we can now focus on managing morbidity, as opposed to just mortality.
My own personal and professional history spans the period of time of the most impressive advances in the science of this disorder. And I’ve had the privilege of working with some of the giants in the field who’ve been responsible for this progress — as well as with many patients.
I entered hematology two years after the Sickle Cell Act was passed in 1972. When I was in training, 13 percent of the children who had been born with sickle cell disease had died by what would have been their second birthdays. And now, with universal newborn screening, followed by education of parents and medical caregivers, the initiation of prophylactic antibiotics early on, and the progress in vaccines against pneumococcus, such deaths are really rare in the developed world.
I’ve made hundreds of trips to the hospital in the middle of the night in Cleveland, Boston, and Baltimore to see my patients, often writhing in pain, in septic shock, or fighting to breathe.
I’ll never forget sitting next to Anthony, whose wedding I had attended earlier in the day. He had been admitted with a pain crisis. Remarkably, he’d not lost his sense of humor. I remember coming in at 3:30 in the morning … he sort of squinted, looking at me and all doubled over in pain and he said, “Jeans? I’ve never seen you in jeans.”
I couldn’t imagine that this was what he would actually notice under those circumstances. And, as he and his new wife left the hospital the next week, we had a long talk about why I’d been able to shepherd him into adulthood but left him with such a burden of disease.
Anthony died of pulmonary hypertension three years after that admission. He and many hundreds of children and adults whom I’ve had the profound privilege of caring for — the privilege of saying, “I’m your doctor” — have educated me about the science, the art, and the humanity of sickle cell disease, and left me with a burning passion to make life better for them and for all those who follow them.
Today, you’ll hear the state-of-the-science in sickle cell disease research. There are some truly amazing opportunities on the horizon:
This morning, NIH Director Dr. Francis Collins will help provide context by giving us a forward-looking synthesis of what we can expect in terms of moving bench science of sickle cell disease into the clinic, and the steps NIH is taking to help us get there.
We’re still maintaining, however, a strong focus on people.
The biggest challenge for sickle cell patients is ensuring that everyone affected has the opportunity to benefit from the advances in science. We’re not quite there yet.
In the developing world, death early in life, usually from infection, from splenic sequestration, or from severe anemia, is the norm. Dr. Ohene-Frempong pointed out yesterday — and Dr. Makani confirmed — that most people born with sickle cell disease in areas of the world in which the disease is most common have actually never been diagnosed.
The demographics of the disease have changed in the developed world. The victories of antibiotic prophylaxis following newborn screening, vaccines, and patient education, have all allowed most patients to reach adulthood. But chronic organ damage, gaps in health care services, limited provider awareness, and other factors limit the extent to which all patients benefit from known therapies.
The NHLBI has been actively engaged in discussions across the department of Health and Human Services to ensure that health care delivery, payment for care, epidemiology, and drug development are coordinated and integrated with research to advance both treatment and knowledge.
Next year, the NHLBI will be issuing a set of clinical guidelines. You’ll hear a sneak preview of the first of the five chapters this morning in the next talk from Dr. Barbara Yawn and Dr. George Buchanan, who’ve been leading this process. The guidelines will be introduced with an awareness campaign, which will be done in coordination with CDC, HRSA, CMS, and professional societies including the American Society of Hematology, the American Academy of Pediatrics, and, we’re hoping, the American Academy of Family Medicine.
For the guidelines to be effective, though, we’ll need robust community participation to ensure that evidence-based treatment reaches the people who need it. Patients, families, communities, researchers, health care providers, educators, employers, and governmental and non-governmental organizations all need to be engaged in the fight against the suffering that comes from sickle cell disease.
I want to thank all of you, again, for taking the time to be here today.
Next, you’ll hear from Sonja Banks, who’s the President of the Sickle Cell Disease Association of America, one of our most important partners. She’s new to the Association, and we are thrilled to have her here today. I’d like to introduce Sonya and welcome her to the podium.