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U.S. FDA Approves First Chemotherapy
Overdose Reversal Agent
The U.S. Food and Drug Administration
(FDA) has approved uridine triacetate for
the emergency treatment of adults or children with cancer who receive an overdose
of the chemotherapeutic agents fluorouracil or capecitabine or who develop certain
severe or life-threating toxicities within
four days of receiving these agents. This
is the first reversal agent of its kind to be
approved by the U.S. FDA.
Uridine triacetate is administered orally and blocks cell damage and death from
chemotherapy. The manufacturers note
that uridine triacetate should be administered as soon as possible after an overdose
occurs, though it can be administered
up to four days following chemotherapy
administration.
The drug’s approval was based on
the findings of two separate trials that
included 135 adult and pediatric patients
with cancer. Patients included in the
studies had either received an overdose of
fluorouracil or capecitabine or had earlyonset, unusually severe, or life-threatening
toxicities within 96 hours of receiving the
two chemotherapy drugs. The study’s primary endpoint was survival at 30 days or,
if prior to 30 days, until the point at which
chemotherapy could be resumed.
In the study, 97 percent of patients
treated with uridine triacetate for
overdose were alive at 30 days, while 89
percent of patients treated with uridine
triacetate for toxicity were alive at 30
days. In both patient cohorts, 33 percent
of patients resumed chemotherapy in less
than 30 days.
The most common treatment-related
adverse events (AEs) included diarrhea,
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ASH Clinical News
vomiting, and nausea.
Uridine triacetate is not recommended
for treating non-emergency AEs associated with fluorouracil or capecitabine. The
safety and efficacy of uridine triacetate
initiated more than 96 hours following
chemotherapy has not yet been studied.
Source: U.S. FDA news release, December 11, 2015.
Congress Approves
Largest NIH Budget
Increase in 12 Years
With the recently approved 2016 fiscal year budget, the U.S. Congress will
increase its funding to the National
Institutes of Health (NIH) to $32.1 billion,
representing a $2 billion increase (6.6%) –
the largest increase in 12 years.
Specifically, the Consolidated Appropriations Act of 2015 includes:
• $350 million in new spending for
Alzheimer’s disease research
• $303 million increase for antimicrobial resistance research
• $200 million in funding for the
Precision Medicine Initiative
• $91 million for programs to reduce
opioid abuse
• $85 million of new funding of the
BRAIN Initiative (the project to map
the human brain)
The bill also maintains 2015 fiscal year
funding levels for numerous blood programs within the Center for Disease Control and Prevention’s Division of Blood
Disorders. The remaining funding will be
split among NIH institutes and centers,
equating to an approximately 4-percent
increase in funding overall.
The American Society of Hematology
“applauds this bipartisan action to increase vital biomedical research funding,”
according to a news release from the
Society.
“This budget signifies the importance
that Congress places on biomedical
research that saves lives, reduces human suffering, and also creates jobs,”
ASH President Charles S. Abrams, MD,
said. “In spite of this fact, overall public
health funding remains well below presequestration levels. There has never been
a more productive time in research, as our
understanding of disease on the molecular
level is translating into promising new
treatments at an unprecedented rate.”
eradicating a disease, and advance
research opportunities for rare
diseases.
3. Enhance scientific stewardship by
recruiting and retaining a biomedical research workforce.
4. Manage results by balancing outputs
with outcomes, conduct workforce
analyses, continue peer reviews,
evaluate steps to enhance reproducibility, reduce administrative burden, and track effectiveness of risk
management in decision-making.
“NIH-sponsored
research accounts for many
of the gains
that the United
States has seen
NIH Releases First
Agency-Wide Strategic in health and
Plan to Enhance Health- longevity. But
much remains
Care Research and
to be done.”
Technology
Sources: U.S. Congress, “H.R.83 – Consolidated and Further Continuing
Appropriations Act, 2015,” December 16, 2015; American Society of
Hematology press release, December 18, 2015.
The NIH has released a five-year agency-wide strategic plan for expanding
health-care research and technology, with
specific goals to further explore HIV and
influenza vaccines, cancer survival rates,
and diabetes. This is the first effort from
the agency to develop a plan that includes
all 27 institutes and centers. In addition,
the NIH included fiscal goals for 2016
through 2020.
“Strategic and technological breakthroughs that have arisen from NIHsponsored research account for many of
the gains that the United States has seen
in health and longevity,” said Francis S.
Collins, MD, PhD, director of the NIH.
“But much remains to be done.”
Four essential pillars are the guiding
principles of this initiative:
1. Advance opportunities in biomedical research in fundamental science,
treatment and cures, and health
promotion and disease prevention.
2. Foster innovation, consider burden
of disease and value of permanently
—FRANCIS S. COLLINS, MD, PhD
Highlights of the specific goals outlined by
NIH include:
• Enhance cancer survival via precision
medicine.
• Conduct clinical trials in an effort to
develop a universal influenza vaccine.
• Apply pharmacogenomics in a realworld setting for improved outcomes.
• Develop applications of certain
mobile health technologies to enhance health promotion and disease
prevention.
• Improve development of U.S. FDAapproved rare disease therapeutics.
• Conduct research on the artificial
pancreas in an effort to better manage
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February 2016