Saturday, February 15, 2014
New clinical trial for Kennedy’s Disease
The National Institutes of Health (NIH) posted the following on their ‘Clinical Trials’ website.
Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy
It appears this is a two part placebo-controlled study to determine dosage and potential safety issues as well as the any change in thigh muscle volume. The trial is to begin this month.
I will post more information as it becomes available.
Tuesday, February 4, 2014
NIH, Industry and Non-Profits Join Forces to Speed Validation of Disease Targets
John Coakley, Sr., President of the
KDA, sent out the following press release from the NIH along with this
comment:
“What a great program!
We can begin to think about the
Kennedy’s Disease Association
‘partnering'
with others in the future. It's not as 'far-fetched' as you might think. Let's
all look ahead five years and open our minds to unlimited possibilities.”
For
Immediate Release: Tuesday, February 4, 2014, 8 a.m. EST
NIH,
INDUSTRY AND NON-PROFITS JOIN FORCES TO SPEED VALIDATION OF DISEASE
TARGETS
The goal is to develop new treatments
earlier, beginning with Alzheimer's, type 2
diabetes,
and autoimmune disorders.
The
National Institutes of Health, 10 biopharmaceutical companies and several non-profit organizations
today 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.
The
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 (FNIH),
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).
A
critical and groundbreaking element of the partnership is the agreement that
the data and analyses generated will be made publicly available to the
broad biomedical community. The
three- to five-year, milestone-driven pilot projects in these disease areas
could set the stage for broadening AMP to other diseases and conditions.
"Patients
and their caregivers are relying on science to find better and faster
ways to detect and treat disease and improve their quality of life,"
said NIH Director Francis S. Collins, M.D., Ph.D. "Currently, 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."
"The
good news is that recent dramatic advances in basic research are opening new
windows of opportunity for therapeutics," continued Dr. Collins. "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. We
believe this partnership is an important first step and represents the
most sweeping effort to date to tackle this vital issue."
As
a result of technological revolutions in genomics, imaging, and more, researchers
have been able to identify many changes in genes, proteins, and other
molecules that predispose to disease and influence disease progression. While
researchers have identified thousands of such biological changes that hold
promise as biomarkers and drug targets, only a small number have been pursued.
Choosing the wrong target can result in failures late in the development
process, costing time, money, and ultimately, lives. Currently, developing
a drug from early discovery through U.S.
Food and Drug Administration approval takes well over a decade and
has a failure rate of more than 95 percent. As a consequence, each success
costs more than $1 billion.
"The
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 Mikael Dolsten, M.D., Ph.D., President of Worldwide
Research and Development at Pfizer. "This type of novel collaboration
will leverage
the strengths of both industry and NIH to ensure we expedite translation of
scientific knowledge into next generation therapies to address the urgent needs
of Alzheimer's, diabetes and RA/lupus patients."
AMP
has been more than two years in the making, with intense interactions etween
scientists in the public and private sectors, progressive refinement of
the goals, strategy development support from the Boston Consulting Group,
and scientific project and partnership management by the FNIH. Through
this effort, AMP partners have developed research plans and are sharing costs,
expertise, and resources in an integrated governance structure that
enables the best informed contributions to science from all participants.
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