I received this today, and thought I would put in onto the blog. More
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FOR IMMEDIATE RELEASE
First Human Dose Initiated of Promising New Friedreich's Ataxia Drug
Highlights Effectiveness of New Public-Private Partnership Model
July 15, 2008 – The Friedreich's Ataxia Research Alliance (FARA) joins
its
public and private partners in announcing that a phase I clinical trial
of a
promising new drug, designated A0001, began dosing on July 11th. This
milestone achievement illustrates the power of a new model for advancing
therapies,
especially in rare diseases.
"FARA believes in the essential nature of public-private partnerships
that
involve government agencies such as the National Institutes of Health
(NIH)
and the Food and Drug Administration (FDA), academic investigators, the
pharmaceutical industry, and multiple non-profits like FARA and the
Muscular
Dystrophy Association (MDA)," said FARA President Ron Bartek. "FARA is
grateful to
its partners that span the spectrum from discovery through clinical
development as we work together to advance promising compounds like
A0001 toward the
goal of approved treatments and cures for Friedreich's Ataxia (FA) and
other
neurodegenerative disorders."
A0001 is a small molecule that has the potential for treating defects in
the
mitochondrial respiratory chain like those believed to cause
significant
damage in FA and a number of other neurological and neuromuscular
disorders. In
April 2006, the FDA granted orphan drug designation status to A0001 for
treatment of inherited mitochondrial respiratory chain diseases.
The opening of this clinical trial demonstrates the power of the
public-private partnerships FARA has been fortunate to build and bring
to bear on the
quest for treatments and a cure for FA. FARA began its support of A0001
in 2005
with substantial research grants to and investment in Edison
Pharmaceuticals. The MDA, via Seek A Miracle/MDA, joined FARA in
co-funding the initial
research grant to Edison.
"We all know that drug development is expensive, but this is a
wonderful
example of how investments can be leveraged by multiple groups to get
more
therapies into the clinic," says Dr. Sharon Hesterlee, MDA VP for
Translational
Research.
Later in 2005, Dr. Robert Wilson of the University of Pennsylvania, FARA
and
Edison joined forces as co-applicants to the National Institutes of
Health
(NIH) program called RAID (Rapid Access to Intervention Development),
designed
to help move drugs from the discovery bench to the clinical trial
bedside.
The application was successful and contract services provided by NIH
RAID
played an important role in completing the preclinical preparations of
A0001 so
the molecule could be made ready for the clinical trial.
"This is a great example of how the various stakeholders joined forces
to
help transform a promising laboratory discovery into a therapeutic that
is now
in clinical trial," says Dr. Story Landis, NINDS Director. The NINDS
manages
the NIH RAID program and is also responsible for Friedreich's Ataxia
research
as part of its mission to reduce the burden of neurological disorders.
NINDS'
support for the A0001 pre-clinical project consisted of formulation, a
genotoxicity study, a dose escalation study, synthesis of radiolabeled
drug for
ADME and radioautography studies, dose ranging studies in two species, a
90
day GLP toxicity study, and safety pharmacology studies.
The phase I clinical trial just initiated by Penwest Pharmaceuticals
will be
conducted in healthy volunteers and is designed to evaluate the safety
and
tolerability of A0001 at various doses, as well as to collect
pharmacokinetic
data. In addition, the trial is designed to determine if there is a
maximum
tolerated dose of the drug. This data will be used in subsequent phases
of
clinical trials in patients with FA and other diseases that involve
mitochondrial dysfunction.
For futher information, contact FARA at (703) 426 1576 or
fara@cureFA.org.
About FARA
The Friedreich's Ataxia Research Alliance (FARA) is a 501(c)(3),
non-profit,
charitable organization dedicated to accelerating research leading to
treatments and a cure for Friedreich's ataxia. _http://www.CureFA.org_
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5Pjni7cG7HJjgpAdeMrfBgGceJYdw01kOAu71BttT6tU=)
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