UP FRONT
The Society Pages
FDA Awards Grants to
Stimulate Development
of Orphan Drugs for Rare
Diseases
The U.S. Food and Drug Administration (FDA) has awarded 18 new
research grants totaling more than
$19 million to boost the development
of products for patients with rare
diseases through its Orphan Products
Grants Program. These grants are
intended for clinical studies evaluating the safety and effectiveness of
products that could either result in, or
substantially contribute to, the FDA
approval of products.
There are three hematology researchers among the eighteen grant
recipients:
Deepa Manwani,
MD, from Albert
Einstein College of
Medicine in New
York City, New York,
will receive $1.6 million over four years
Deepa Manwani, MD
for the “Phase 2
Study of Gamunex
(Intravenous Gammaglobulin) for the
Treatment of Sickle Cell Acute Pain”
Suzanne Lentzsch,
MD, PhD, from
Columbia University
Medical Center in New
York City, New York,
will receive $600,000
over three years for
Suzanne Lentzsch,
the “Phase 1A/B
MD, PhD
Study of 11-1F4 mAb
for the Treatment of AL Amyloidosis”
Claudia Morris, MD,
from Emory University in Atlanta,
Georgia, will receive
$1.6 million over
four years for the
“Phase 2 Study of
Claudia Morris, MD
L-Arginine Therapy
for the Treatment of
Pediatric Sickle Cell Disease Pain”
“The FDA is in a unique position to
help those who suffer from rare diseases by offering important incentives to
promote the development of products,
one of which is our grants program,”
said Gayatri R. Rao, MD, JD, director
of the FDA’s Office of Orphan Product
Development, in a press release. “The
grants awarded this year support muchneeded research in 17 different rare
diseases, many of which have little, or
no, available treatment options.”
Source: U.S. FDA press release, September 21, 2015.
MACC Fund Names Jeffrey
Medin Endowed Professor
Jeffrey A. Medin,
PhD, has been
named the MACC
Fund Professor in
the Department of
Pediatrics at the
Medical College of
Jeffrey A. Medin, PhD
Wisconsin (MCW)
in Milwaukee,
Wisconsin. Dr. Medin currently serves
as a professor in the Department of
Medical Biophysics and the Institute of
Medical Science, faculty of medicine,
at the University of Toronto in Ontario,
Canada. At MCW, Dr. Medin will serve
as vice chair of research innovation
for the Department of Pediatrics, and
research director within the Section of
Pediatric Hematology/Oncology, where
he is expected to expand the Pediatric Hematology/Oncology Transplant
Program. Dr. Medin will also serve as
director of cell processing laboratories
in the MCW Adult and Pediatric Blood
and Marrow Transplant Program, with
appointments in the MCW Cancer Center and the BloodCenter of Wisconsin’s
Blood Research Institute.
Previously, Dr. Medin was employed
with the National Institutes of Health,
where he focused on gene transcription and gene therapy, and as assistant
professor of medicine at the University
of Illinois at Chicago.
Dr. Medin will assume his full duties
on January 1, 2016.
Source: MACC Fund press release, September 24, 2015.
James Bradner Named
President of the Novartis
Institutes for BioMedical
Research
James E. Bradner, MD, of Dana-Farber
Cancer Institute and Harvard Medical
School, has been appointed president
of the Novartis Institutes for BioMedical Research (NIBR) and a member of
the Executive Committee of Novartis,
effective March 1, 2016. Dr. Bradner is
currently associate professor in the
Department of Medical Oncology at the
Dana-Farber Cancer Institute at Harvard
Medical School, where he has been on
the faculty since 2005. Dr. Bradner has
co-authored more than 130 scientific
publications and 30 United States Patent applications.
Dr. Bradner succeeds Mark C. Fishman, MD, who spent 13 years as leader
of Novartis’ drug discovery and early
clinical development efforts.
Source: Novartis press release, September 24, 2015.
ASHClinicalNews.org
CMS Awards $685 Million
to 39 Health-Care Organizations for Innovative
Clinical Practice
As part of its Transforming Clinical Practice Initiative, the Centers for Medicare
& Medicaid Services (CMS) Innovation
Center is awarding $685 million to 39
physician groups, health systems, and
other organizations for training, education, and investment in information
technology, care coordination, and quality improvement efforts. The grants are
funded under the Affordable Care Act
and are expected to provide assistance
to as many as 140,000 clinicians.
“Supporting doctors and other
health-care professionals [in changing]
the way they work is critical to improving quality and spending our health-care
dollars more wisely,” said Health and Human Services Secretary Sylvia Burwell.
“These awards will give patients more
of the information they need to make
informed decisions about their care and
give clinicians access to information and
support to improve care coordination and
quality outcomes.”
These awards will support 29 medical group practices, regional healthcare systems, and regional extension
centers in offering peer-to-peer support to primary and specialty physicians, nurse practitioners, physician
assistants, clinical pharmacists, and
their practices.
These efforts include:
• Helping providers give patients
better tools for communication
through e-mails and other information technology applications
• Providing dedicated coaches to help
practices better manage chronic
disease and offer preventive care
• Offering real-time notification
alerts for clinicians caring for highrisk patients
• Centralizing data reporting and
providing technical assistance with
quality improvement targets and
mid-course corrections
In addition, 10