For your information... sharing a press release from NORD about their new
program to develop effective treatments for rare diseases.
 
Best regards,
Gena Brumitt Gruschovnik
http://www.tomorrowthinkofme.roundtablelive.org/





Vice Treasurer, Executive Committee, DEBRA International -
www.debra-international.org
Director of Awareness & Education, DEBRA Canada - www.debracanada.org
We must be the change we wish to see in the world.  ~ Mahatma Gandhi

--- On Thu, 5/21/09, NORD <orphan@...> wrote:


From: NORD <orphan@...>
Subject: NORD President Praises New NIH Initiative
To: "otm@..." <otm@...>
Received: Thursday, May 21, 2009, 12:12 PM




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 NORD Press Release
Keeping you informed about rare diseases and orphan products
National Organization for Rare Disorders
May  2009

NORD President Praises New NIH Initiative

NORD President and CEO Peter L. Saltonstall today praised the establishment of a
new initiative at the National Institutes of Health (NIH) to encourage the
development of treatments for rare and neglected diseases. 
“There are nearly 7,000 rare diseases and only about 200 of them have an
FDA-approved therapy,” Saltonstall said.  “Every day, our staff members
assist patients and families whose lives are being impacted in very significant
ways by the fact that there is no treatment for the diseases affecting
them.” 
On Wednesday, the NIH announced the launch of a new program to address this
problem.  Called Therapeutics for Rare and Neglected Diseases (TRND), it will
create a drug development pipeline to stimulate research collaborations with
academic scientists.  The program is an attempt to bridge what is sometimes
called “the valley of death” in drug development—the phase between
promising early research and clinical trials. 
Some 80 to 90 percent of potential new drugs fail during this preclinical phase,
and the costs are high.  It takes an estimated two to four years of work and
$10 million, on average, to move a potential medicine through this phase.   
With $24 million in funding, the new NIH program has the potential to lead to
significant progress in the development of safe, effective treatments for people
with rare and neglected diseases. 
“This is a tremendously important initiative,” Saltonstall said. “Since
the enactment of the Orphan Drug Act, NORD has devoted a major share of its
advocacy efforts toward facilitating the development of safe, effective
treatments through innovative research.  Now, NORD stands ready to help in any
way we can with TRND. 
 
 
NIH’s press release about TRND: 
NIH ANNOUNCES NEW PROGRAM TO DEVELOP THERAPEUTICS FOR RARE AND NEGLECTED
DISEASES 
 
The National Institutes of Health is launching the first integrated, drug
development pipeline to produce new treatments for rare and neglected diseases.
The $24 million program jumpstarts a trans-NIH initiative called the
Therapeutics for Rare and Neglected Diseases program, or TRND. 
 
The program is unusual because TRND creates a drug development pipeline within
the NIH and is specifically intended to stimulate research collaborations with
academic scientists working on rare illnesses. The NIH Office of Rare Diseases
Research (ORDR) will oversee the program, and TRND's laboratory operations will
be administered by the National Human Genome Research Institute (NHGRI), which
also operates the NIH Chemical Genomics Center (NCGC), a principal collaborator
in TRND. Other NIH components will also participate in the initiative. 
 
A rare disease is one that affects fewer than 200,000 Americans. NIH estimates
that, in total, more than 6,800 rare diseases afflict more than 25 million
Americans. However, effective pharmacologic treatments exist for only about 200
of these illnesses. Many neglected diseases also lack treatments. Unlike rare
diseases, however, neglected diseases may be quite common in some parts of the
world, especially in developing countries where people cannot afford expensive
treatments. Private companies seldom pursue new therapies for these types of
illnesses because of high costs and failure rates and the low likelihood of
recovering investments or making a profit. 
 
"NIH is eager to begin the work to find solutions for millions of our fellow
citizens faced with rare or neglected illnesses," said NIH Acting Director
Raynard S. Kington, M.D., Ph.D. "The federal government may be the only
institution that can take the financial risks needed to jumpstart the
development of treatments for these diseases, and NIH clearly has the scientific
capability to do the work." 
 
DEVELOPING DRUGS 
 
The drug development process is complicated and expensive.  Studies suggest
that it currently takes more than a dozen years and hundreds of millions of
dollars to take a potential drug from discovery to the marketplace. And the
failure rate is high. 
 
"This initiative is really good news for patients with rare or neglected
diseases," said ORDR Director Stephen C. Groft, Pharm.D.  "While Congress has
previously taken important steps to help these patients, such as providing
incentives for drug companies under the Orphan Drug Act, this is the first time
NIH is providing support for specific, preclinical research and product
development known to be  major barriers preventing potential therapies from
entering into clinical trials for rare or neglected disorders. While we do not
underestimate the difficulty of developing treatments for people with these
illnesses, this program provides new hope to many people world-wide." 
 
Typically, drug development begins when academic researchers studying the
underlying cause of a disease discover a new molecular target or a chemical that
may have a therapeutic effect. Too often, the process gets stuck at the point of
discovery because few academic researchers can conduct all the types of studies
needed to develop a new drug. If a pharmaceutical company with the resources to
further the research does get involved, substantial preclinical work begins with
efforts to optimize the chemistry of the potential drug. This involves an
iterative series of chemical modifications and tests in progressively more
complex systems - from cell cultures to animal tests - to refine the potential
medicine for use in people. Only if these stages are successful can a potential
treatment move to clinical trials in patients. 
 
Unfortunately, the success rate in this preclinical process is low, with 80 to
90 percent of projects failing in the preclinical phase and never making it to
clinical trials.  And the costs are high: it takes two to four years of work
and $10 million, on average, to move a potential medicine though this
preclinical process.  Drug developers colloquially call this the "Valley of
Death." 
 
TRND will work closely with disease-specific experts on selected projects,
leveraging both the in-house scientific capabilities needed to carry out much of
the preclinical development work, and contracting out other parts, as scientific
opportunities dictate. Its strategies will be similar to approaches taken by
pharmaceutical and biotechnology companies, but TRND will be working on diseases
mostly ignored by the private companies.  Importantly, TRND will also devote
some of its efforts to improving the drug development process itself, creating
new approaches to make it faster and less expensive. 
 
If a compound does survive this preclinical stage, TRND will work to find a
company willing to test the therapy in patients.  There are several stages to
the clinical trials process that can take several years before the safety and
efficacy of a new drug is determined. FDA will only approve a drug for general
use after it passes these trials. The clinical trials process is also expensive,
but the failure rate is lower at this stage. 
 
"NIH traditionally invests in basic research, which has produced important
discoveries across a wide range of illnesses," said NHGRI Acting Director Alan
E. Guttmacher, M.D. "Biotechnology and pharmaceutical companies have enormous
strength and experience in drug development, but to maximize
return-on-investment work primarily on common illnesses.  TRND will develop
promising treatments for rare diseases to the point that they are sufficiently
"de-risked" for pharmaceutical companies, disease-oriented foundations, or
others, to undertake the necessary clinical trials. NIH's goal is to get new
medications to people currently without treatment, and thus without hope." 
 
NIH already has many components of the drug development pipeline within its
research programs. TRND will begin its work in collaboration with the NIH
Chemical Genomics Center (NCGC), a center initially developed as part of the NIH
Roadmap for Medical Research. NCGC has developed a robotic, high-throughput
screening system and a library of more than 350,000 compounds that it uses to
make basic discoveries and probe cellular pathways. NCGC also has developed a
team of researchers skilled in developing assays representing disease processes
that can be tested in its screening system, and has extensive experience
building collaborative projects with investigators from across the research
community. Molecules with potential therapeutic properties that emerge from the
NCGC screening process could be fed into the TRND drug development pipeline. 
 
"With this new funding, TRND will develop teams of scientists who can do the
hard work of optimizing chemicals that we or others discover that may treat rare
diseases and turn them into actual drugs," said NCGC Director Christopher P.
Austin, M.D., who is also the Senior Advisor for Translational Research to the
NHGRI Director.  "This will still be hard work and it will take time and
produce failures. Unlike traditional drug development, however, where only
successes are published, we will publish our failures as well, so everyone in
the drug development community can learn from them. That alone could be
revolutionary." 
 
If all the preclinical hurdles can be crossed, a possible treatment must still
be tested in a series of clinical trials. TRND will seek to take advantage of
several NIH resources that can help launch human studies, including the NIH
Clinical Center, the NIH Rapid Access to Interventional Development (NIH-RAID),
and the Clinical and Translational Science Awards (CTSA) program. 
 
EXTERNAL PARTNERS 
 
Numerous obstacles impede the development of new drugs for rare and neglected
diseases. In addition to the reluctance of private companies to risk their
capital on a potentially low return, relatively few basic researchers study rare
diseases, so the underlying cause of the illness frequently remains unknown.
And, because rare diseases are rare, researchers often have difficulty
recruiting enough people with the disorder to participate in a clinical trial
once a candidate compound reaches the stage where it can be tested in people.
Moreover, for many rare diseases, the natural history of the disease is poorly
understood, so researchers lack the needed clinical measures (such as blood
pressure) that can demonstrate whether a treatment is working. 
 
To address these difficulties, TRND will seek a wide range of collaborations
with academic researchers, as well as partnerships with patient advocacy
organizations, disease-oriented foundations and others interested in treatments
for particular illnesses. TRND's leaders hope that the collaborations will help
lay the groundwork for clinical trials once that point in drug development is
reached. 
 
TRND is currently setting up an oversight process to help it decide which
projects that address thousands of rare and neglected diseases will be pursued.
 Leadership currently envisions a small number of diseases being studied each
year, with strict criteria used to determine which molecules will be studied for
which diseases. NIH expects to use existing intellectual property policies to
transfer licenses for TRND-discovered drugs to private companies or others for
development, clinical testing and marketing. 
 
Frequently asked questions about this new program are available online at: 
 
-- FAQ on the Therapeutics for Rare or Neglected Diseases (TRND) program:
<www.genome.gov/27531965> 
 
-- TRND FAQ on Neglected Diseases: <www.genome.gov/27531964> 
 
-- TRND FAQ on Rare Diseases: <www.genome.gov/27531963>

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