NIDDK participates in novel partnership to speed validation
of disease targets
along with 10 pharmaceutical companies and several nonprofit organizations
launched an unprecedented partnership to transform the current model for
identifying and validating the most promising biological targets of disease for
new diagnostics and drug development.
Accelerating Medicines Partnership (AMP) aims to distinguish biological targets
of disease most likely to respond to new therapies and characterize biological
indicators of disease, known as biomarkers. Through the Foundation for the NIH,
AMP partners will invest more than $230 million over five years in the first
projects, which focus on Alzheimer’s disease, type 2 diabetes and the
autoimmune disorders rheumatoid arthritis and systemic lupus erythematosus
(lupus). These milestone-driven pilot projects could set the stage for
broadening AMP to other diseases and conditions.
NIDDK will have a
leadership role in advancing the type 2 diabetes project. NIDDK Director Dr. Griffin
P. Rodgers said he was immediately enthusiastic about the partnership’s
potential. “We need more targeted drug therapies to treat diabetes,” he said.
“While multiple diabetes drugs are available that help stabilize the disease,
people still progress to painful and costly complications. This novel
collaboration is an opportunity to improve the lives of people with diabetes
and those at risk.”
critical element of the partnership is the agreement that the data and analyses
generated will be made publicly available to the broad biomedical community.
we are investing a great deal of money and time in avenues with high failure
rates, while patients and their families wait. All sectors of the biomedical
enterprise agree that new approaches are sorely needed,” said NIH Director Dr. Francis
S. Collins. “Recent dramatic advances in basic research are opening new windows
of opportunity for therapeutics. … But this challenge is beyond the scope of
any one of us, and it’s time to work together in new ways to increase our
collective odds of success.”
type 2 diabetes section of the partnership will focus on two goals. The first
will encourage the building of a virtual library of DNA sequences, functional
genomic and epigenomic information, and clinical data from studies on type 2
diabetes and its heart and kidney complications. This knowledge portal will
include existing and new data from studies involving 100,000 to–150,000 people,
with the goal of helping recognize the most promising therapeutic targets for
The second type 2 diabetes-related project
will focus on DNA regions that might be critical for the development or
progression of type 2 diabetes, and will search for natural variations in
targeted populations that might predict the likelihood of success of drug
development aimed at these targets.
AMP rallies scientific key players of the innovation ecosystem in a more
unified way to address one of the key challenges to biopharma drug discovery
and development,” said Dr. Mikael Dolsten, president of Worldwide Research and
Development at Pfizer.
“We’re leveraging our expertise to quicken development of
therapies to help the 400 million or so people with diabetes worldwide and the
many more at risk for the disease,” Rodgers added. “I see this as a chance to
jumpstart diabetes drug development across the field.”
For more about
Representative visits NIH
Rep. Joe Pitts (R-PA) and staffers visited NIH on Feb. 3.
Here, NIDDK Director Dr. Griffin P. Rodgers participates in a roundtable
discussion including NIH Director Dr. Francis S. Collins and five other
institute directors or deputy directors.
Rodgers (far right) provided Pitts (fourth from left) with an update on
NIDDK-supported research toward developing artificial pancreas technologies for
people with type 1 diabetes, including how the NIDDK collaborates with the FDA.
Silver anniversary for
NIDDK’s U.S. Renal Data System
The NIDDK-supported U.S. Renal Data System (USRDS) – an
internationally emulated registry that collects, analyzes and distributes data
about chronic kidney disease (CKD) and end-stage renal disease (ESRD) –
celebrates its 25th anniversary this year. The registry receives data from the U.S.
Centers for Medicare and Medicaid Services (CMS) on all patients treated for
ESRD in the U.S., and receives CKD data from several sources. The USRDS
responds to nearly 200 data requests annually from investigators. Since its
inception, other nations have launched their own registries, yet the USRDS
remains the model and often a key starting point for kidney disease research.
Over the years, the structure of the USRDS has evolved, said
NIDDK’s Dr. Paul Eggers, co-project officer for the USRDS. “Initially one
coordinating center handled everything,” he said, but now two special study
centers act in unison with a coordinating center. “This new approach has become
the blueprint for how the USRDS functions today: a seasoned, well-oiled machine
that continues to set the pace for disease registries all over the world.”
Dr. Lawrence Agodoa is co-project officer of the USRDS and
director of NIDDK’s Office of Minority Health Research Coordination. “Before we
started the USRDS project in 1988, there were only two similar registries,” he
said. “Both relied on voluntary answers
to questionnaires they sent out, whereas the USRDS had access to 100 percent of
data” directly from CMS. Each year the USRDS team presents its newest analyses
of data, including international comparisons, at the American Society of
Nephrology’s Kidney Week Meeting. “It’s really fascinating to see,” Agodoa
said. “The USRDS is the only disease
registry to feature data compiled in one place from most of the countries that
Findings from the USRDS have improved the health of people
with kidney disease in many ways. For
example, early analysis of USRDS data showed that people with end-stage renal
disease who received catheters for dialysis had more infections and higher
death rates than those who received fistulas.
This discovery led to the requirement that dialysis centers use a
fistula when possible; the CMS Fistula First initiative helped implement the
Another finding from earlier analyses showed that African
Americans have a higher rate of ESRD than other races primarily due to
hypertension. Based on this, the NIDDK
designed the African American Study of Kidney Disease and Hypertension, which
found that African Americans who were put on calcium channel blocker
anti-hypertensive drugs progressed more quickly to end-stage renal disease than
those on ACE inhibitors and beta blockers. The results changed medical practice
for treating African Americans with hypertensive kidney disease.
Looking to the future, researchers anticipate that other
findings from the USRDS, in collaboration with researchers worldwide, will help
improve clinical practices and understanding of kidney disease. One current collaboration
seeks to build or improve registries in the Caribbean and Mexico.
“When we created the USRDS, we envisioned a comprehensive
database that investigators everywhere could use to improve public health and conduct
life-changing research,” said Agodoa. “I’m proud to say that our vision has
largely been realized.”
STEP-UP lab opens in
At an official ceremony Jan. 8, the NIDDK Short-Term Research
Experience for Underrepresented Persons (STEP-UP) program opened its fifth and
final lab in the Pacific region, the Pacific molecular biology laboratory in
Palau. The goal of the STEP-UP program is to build and sustain a biomedical,
behavioral, clinical and social science research pipeline focused on NIDDK
mission areas. Pictured, local science teachers are being trained to use the
equipment. The teachers will serve as mentors, helping local STEP-UP students
gain biomedical research training without having to travel far from home.
Getting to know: Dr. Herbert Tabor
Dr. Herb Tabor, senior investigator in NIDDK’s Laboratory of
Biochemistry and Genetics, recently celebrated 70 years at NIH. In a letter,
President Barack Obama thanked Tabor for his “extraordinary commitment…to
advancing science through public service.” Tabor spoke with Krysten Carrera
about his career and accomplishments.
your first impressions of NIH when you started on September 16, 1943?
As a Public Health Service physician on transatlantic convoy duty
during World War II, I was very excited to learn that my next assignment was to
NIH – which was still just one big institute.
I was amazed by the level of teamwork among the scientists, something
that has remained constant through the years.
it like to collaborate with your wife and lab partner, the late Dr. Celia White
I was lucky to be able to work closely with Celia, who started as
an investigator at NIH in 1952, for over five decades. Together we researched
polyamines, which are short polybasic compounds present in almost all
biological materials. Our passion for science impressed upon our children: our
oldest son is a physician, and another son is a biochemist. One of our
granddaughters became a geneticist.
are you most proud of?
When I started studying polyamines, almost nothing was known of
the biosynthesis and function of the compounds spermidine and spermine. My
colleagues and I were the first to discover their biosynthetic pathway and
study the function of the genes involved, as well as to develop sensitive assay
procedures. This early research has spawned over 13,000 papers involving
spermidine or spermine from studies conducted in multiple labs. In my lab we
continue to investigate how polyamines are formed and metabolized. What we have
learned has enhanced our understanding of biological processes.
named editor-in-chief for the Journal of Biological Chemistry in 1971 and are
now editor-in-chief emeritus. How have scientific journals changed over the
The main difference is the way information is disseminated. People
now do most of their reading on the internet. The JBC has always been
forward-thinking in that regard: in 1995, we were the first biological journal
to be accessible online.
did mentors play at the beginning of your career? Do you currently serve as a
When I began at NIH, I worked with Sanford Rosenthal to
investigate saline treatments for burns, building on the work I had done
through the Public Health Service during the war. Dr. Rosenthal, a phenomenal
scientist and mentor who helped me start my career, had worked at NIH back when
it was the Hygienic Laboratory. Nowadays I am lucky to mentor superb postdocs
who are naturally inquisitive and always want to find out more – and that
curiosity is key to success in biomedical research.
IDDK director encourages
students to pursue the sciences
On February 12, NIDDK Director Dr. Griffin P. Rodgers shared his
journey through the sciences with a group of students from Buck Lodge Middle
School in Adelphi, Md. His talk and
lively quiz show highlighted how science affects the students’ daily lives and
how learning about science can improve their lives.
As part of the Nifty Fifty science speaker program sponsored by
the upcoming USA Science & Engineering Festival, Rodgers talked about how
his parents, teachers – and three of his close friends who had sickle cell
disease – inspired his passion for science and sealed his desire to become a
doctor and to focus on hematology.
Following his brief remarks, Rodgers hosted the students in an
interactive quiz show focusing on some areas of science supported by NIDDK,
including diabetes, obesity, physical activity, kidney disease and the human
microbiome. To close the event, the
students asked Rodgers questions ranging from how long it took for him to
become a doctor to if there’s a cure for diabetes.
The 3rd USA Science & Engineering Festival is the
largest science festival in the U.S. It will be held April 26-27 in Washington,
D.C. The organization sponsors contests and school programs like the Nifty
Fifty to spark the interest of youth in science, technology, engineering and
math (STEM). This was Buck Lodge Middle
School’s first time participating in the festival. More information about the 3rd
USA Science & Engineering Festival and its expo is available at: www.usasciencefestival.org.
2014 edition of NIDDK’s annual
scientific report now available
The NIDDK annual scientific report, NIDDK Recent Advances & Emerging Opportunities, is
now available. This report highlights examples of NIDDK-supported research
advances published in fiscal year 2013. It also includes stories of discovery –
which trace research progress in specific areas over an extended time frame,
personal stories of patients and scientific presentations made to the NIDDK’s
National Advisory Council during the past year. The report also contains an
overview of NIDDK’s collaborative efforts toward the development of an artificial
pancreas; features 2013 Nobel Laureate and NIDDK grantee, Dr. James Rothman;
and describes NIDDK programs such as the Drug-Induced Liver Injury Network and
the Kidney Research National Dialogue.
To read the report, go to www.niddk.nih.gov/about-niddk/strategic-plans-reports/pages/all-reports.aspx.