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October 04, 2013

NIH and Other Public Private Partnerships to Research Treatments for Multiple Diseases

Over the past few weeks, the National Institutes of Health (NIH) has made a number of important announcements regarding collaborations with industry as well as the funding of several new research initiatives. Below is a summary of these stories.

NIH Partners With Eli Lilly and Others on Rare Diseases

FierceBiotechResearch reported that NIH selected four (4) new preclinical drug development studies to uncover new therapies for rare diseases. The projects will be funded through the Therapeutics for Rare and Neglected Diseases (TRND) program under NIH's National Center for Advancing Translational Sciences, NCATS, which we have written about previously.

"TRND is grounded in partnerships with academic, government, pharmaceutical and patient advocacy groups. Working in collaboration, scientists conduct pre-clinical development of new drugs and then advance them to first-in-human clinical trials," said NCATS Director Christopher P. Austin, M.D. "Like all NCATS programs, TRND seeks to develop new technologies and more efficient paradigms for translation, in the context of important unmet medical needs."

NIH classifies a rare disease as one that affects fewer than 200,000 Americans. There are more than 6,000 rare diseases, but effective pharmacologic treatments exist for only about 200 of these illnesses, according to NIH.

For the first time, the TRND program will collaborate with a large pharmaceutical company. NIH selected Eli Lilly as a collaborator in developing a long-acting parathyroid hormone treatment for hypoparathyroidism, a rare condition in which the body does not produce enough of a certain type of hormone.

Another project will focus on developing a potential therapeutic that targets a cardiac disorder associated with LEOPARD syndrome, an extremely rare genetic disease that affects many areas of the body. The two other projects--one involving stem cells—will investigate therapeutic approaches to treat retinitis pigmentosa, a severe form of hereditary blindness.

The TRND program does not award grant money to selected researchers. Rather, participants have access to NIH scientists and the agency's resources to help advance their projects. Because of the collaborative nature of the program, an NIH spokesman told FierceBiotech Research that the agency is not able to estimate how much the newly selected projects will cost to support.

In the last two years, TRND researchers and collaborators have moved four projects out of the lab and into human clinical trials, evaluating new therapies for sickle cell disease, chronic lymphocytic leukemia, hereditary inclusion body myopathy and Niemann-Pick Type C.

NIH Launches $45 Million Alzheimer's Research, Awards Funds to 3 Preclinical Studies

FierceBiotechResearch also reported that NIH selected 6 initial Alzheimer's projects to support, including three preclinical programs. The projects are part of the 2011 National Alzheimer's Project Act, which mandated that the Department of Health and Human Services establish a National Plan to Address Alzheimer's Disease.

The studies are among the first to be developed with direction from the 2012 NIH Alzheimer's Disease Research Summit: Path to Treatment and Prevention and reflect research goals in the National Plan to Address Alzheimer's Disease. Of the funding, $40 million is from an allocation from the Office of the NIH Director, Dr. Francis Collins, with additional funding from the National Institute on Aging (NIA), the lead Institute within NIH for Alzheimer's research.

One of those projects, headed by Dr. Philip De Jager of the Brigham and Women's Hospital, Broad Institute and Harvard University and Dr. David Bennett of Rush University Medical Center in Chicago, will to seek to find molecular networks and genes that affect a person's predisposition to cognitive decline and Alzheimer's disease. The team will receive $1.7 million this year, with the potential of $7.9 million over 5 years. Ultimately, the scientists hope to identify compounds that normalize dysfunctional activity in the brains of people with Alzheimer's and to discover drugs for novel therapeutic targets.

Prior to this announcement, the Obama Administration back in April of this year announced the public-private BRAIN Initiative (Brain Research Through Advancing Innovative Neurotechnologies). Obama proposed to allocate $100 million to find new treatments for diseases like Alzheimer's, Parkinson's and other neurological disorders. The BRAIN Initiative includes:

  • Key investments to jumpstart the effort: The National Institutes of Health, the Defense Advanced Research Projects Agency, and the National Science Foundation will support approximately $100 million in research beginning in FY 2014.
  • Strong academic leadership: The National Institutes of Health will establish a high-level working group co-chaired by Dr. Cornelia "Cori" Bargmann (The Rockefeller University) and Dr. William Newsome (Stanford University) to define detailed scientific goals for the NIH's investment, and to develop a multi-year scientific plan for achieving these goals, including timetables, milestones, and cost estimates.
  • Public-private partnerships: Federal research agencies will partner with companies, foundations, and private research institutions that are also investing in relevant neuroscience research, such as the Allen Institute, the Howard Hughes Medical Institute, the Kavli Foundation, and the Salk Institute for Biological Studies.
  • Maintaining our highest ethical standards:  Pioneering research often has the potential to raise new ethical challenges. To ensure this new effort proceeds in ways that continue to adhere to our highest standards of research protections, the President will direct his Commission for the Study of Bioethical Issues to explore the ethical, legal, and societal implications raised by this research initiative and other recent advances in neuroscience.

Recently, an NIH work group laid out initial goals of the project, which they said should focus on how complex neural circuits interact, rather than on the entire brain. An interim report from the NIH group, as reported by FierceBiotechResearch, identified 9 high-priority research areas for 2014, the first year of the project, including generating a census of brain cell types, creating structural maps of the brain, developing new neural network recording capabilities and linking neuronal activity to behavior.

NIH Expands Nationwide Network of Vaccines


A nationwide group of institutions that conducts clinical trials of promising candidate vaccines and therapies for infectious diseases, known as the Vaccine and Treatment Evaluation Units (VTEUs), has been awarded nine contracts to strengthen and broaden the scope of its research. With these new awards, NIAID will increase the number of funded institutions from eight to nine and expand the ability of the VTEUs to conduct research in domestic and international research locations, including resource-poor settings.

Each institution has the potential to receive funding from the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, estimated to be up to $135 million annually over a seven-year period.

"The VTEUs have been an invaluable resource for testing important vaccines and treatments against deadly emerging infectious disease threats," said NIAID Director Anthony S. Fauci, M.D. "Through these new awards, we are increasing the network's capacity to study infectious diseases where they are endemic. This will allow us to learn more about the origin and evolution of emerging diseases and also improve the evaluation of diagnostics along with potential vaccines and treatments."

Established in 1962, the VTEUs have conducted hundreds of clinical trials, many of which have contributed to vaccine licensure. VTEU investigators have tested vaccines and therapeutics for diseases such as influenza, pneumonia, pertussis, Haemophilus influenzaeType B infection, cholera, anthrax, malaria and tuberculosis. Childhood vaccines and combination vaccines — the delivery of several vaccines through one inoculation — have been and remain an important part of the VTEUs' research goals.

For example, the network is evaluating the safety of and immune response generated by the pertussis vaccine Tdap in pregnant women and the effect of immunizing expectant mothers on their infants' immune responses to DTaP, the pertussis vaccine that is routinely administered to children.

In 2001, responding to biodefense concerns, the VTEUs conducted a trial that showed that stockpiled smallpox vaccine could be diluted up to five times and retain its potency, which meant that the original 15.4 million doses were actually enough to protect 77 million people from smallpox infection. More recently, when a new strain of H1N1 influenza emerged in 2009, the VTEUs initiated a series of clinical trials to assess the safety of and immune system response to various dosing regimens of candidate vaccines in healthy adults, elderly people and healthy children.

The results of these trials were made available within a few months and helped public health officials and policy makers determine the most appropriate dose of vaccine. Earlier this month, the VTEUs launched two clinical trials to evaluate an investigational vaccine against the H7N9 avian influenza virus that emerged in humans in China earlier this year.

"Launching and obtaining results from such studies quickly is possible because the VTEUs have proved that they can rapidly enroll large numbers of participants. This agility is especially important for testing vaccines designed to counteract emerging infectious diseases of public health concern," said Dr. Fauci.

Johnson & Johnson Partner to Fight Dengue Fever

J&J's Janssen recently announced that it will be partnering with the University of Leuven in Belgium and the U.K.'s Wellcome Trust in an effort to combat the world's fastest-spreading tropical disease, dengue fever, reported FierceBiotechResearch.

"The collaboration builds on an existing three-year drug discovery program at the Rega Institute and the Centre for Drug Design and Discovery (CD3) at KU Leuven, supported by a Wellcome Trust Seeding Drug Discovery Award. This effort resulted in the identification of a series of compounds that are highly potent inhibitors of dengue virus replication. Through the new partnership, researchers will use this previous work to develop candidate antiviral drugs to prevent and treat dengue infection, which mostly affects Asia but has been popping up in the Caribbean and Latin America in recent years."

There are currently no approved vaccines that can prevent the mosquito-borne infection or approved drugs that can stop the replication and spread of the virus. Available therapies only aim to alleviate symptoms, which include a severe and debilitating fever, rash, and muscle and joint pain, sometimes referred to as "breakbone fever." In some cases, infection can lead to internal hemorrhage and can be fatal.

A recent dengue fever epidemic has sparked a public health emergency in Central America, and in Florida, 8 dengue cases have been confirmed as of Aug. 30. Experts have warned that slowly rising global temperatures may be to blame, since the tropical disease favors mosquito-rich environments. The World Health Organization (WHO) estimates that there are between 50 million and 100 million infections each year.

Under the terms of the agreement, Janssen will make an upfront payment and milestone payments based on achieving development, regulatory and sales goals. KU Leuven is also eligible to receive royalty payments on sales of future products discovered or developed under the agreement. At the end of the collaboration period, Janssen will have the option to further develop the candidate drug toward regulatory approval.

The Biopharmaceutical Innovation Partnership

Monash University, located in Australia, recently announced that researchers will partner with industry on projects that will deliver advanced medicines to the world. Senator Kim Carr, Minister for Innovation, announced that the Biopharmaceutical Innovation Partnership will have its headquarters in Melbourne, and will bring together GlaxoSmithKline, AstraZeneca and CSL in partnership with Monash University to create cutting-edge science and technology. Flow-on benefits of the collaboration include employment opportunities and the generation of new scientific knowledge and drug development capabilities.

The Biopharmaceutical Innovation Partnership will have future centres in Sydney and Brisbane. Senator Carr and the Labor Government will commit up to $6 million to establish The Biopharmaceutical Innovation Partnership, to be matched with co-investment from partner organisations.

The Partnership will have access to further project funding of up to $10 million per year through the Industry Collaboration Fund for innovative projects with commercial potential.


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