The National Institutes of Health (NIH) has released funding information for 218 areas during the 2009 fiscal year. The table titled, “Estimates of Funding for Various Diseases, Conditions, and Research Areas,” shows the total funds spent in each category based on grants, contracts, and research conducted in the NIH’s own laboratories and clinics. The 218 categories included in the chart represent diseases, conditions, and research areas historically requested by and reported to Congress and the public at the end of each fiscal year. The NIH website specified that this “does not reflect the entire NIH research portfolio and budget,” that is does not “impact the way the NIH funds research or determines its research priorities”, and “does not change the way the NIH makes awards throughout the year for medical research.”

According to the chart, NIH spent $14 million on spinal muscular atrophy (SMA) research in fiscal year 2009, thanks in part to funding provided by the American Recovery & Reinvestment Act. The chart also estimates that SMA will remain at that funding level in fiscal year 2010.

Click here to view the entire “Estimates of Funding for Various Diseases, Conditions, and Research Areas” table.

From the Washington Post:

NIH authorizes use of first human embryonic stem cells under new policy

By Rob Stein
Washington Post Staff Writer
Wednesday, December 2, 2009; 1:01 PM

The Obama administration on Wednesday approved the first human embryonic stem cells for experiments by federally funded scientists under a new policy designed to dramatically expand government support for one of the most promising but also most contentious fields of biomedical research.

The National Institutes of Health authorized 11 lines of cells produced by scientists at the Children’s Hospital in Boston and two lines created by researchers at the Rockefeller University in New York. All were obtained from embryos left over by couples seeking treatment for infertility.

“This is a real change in the landscape,” NIH Director Francis Collins said. “This is the first down payment on what is going to be a much longer list . . . that will empower the scientific community to explore the potential of embryonic stem cell research.”

According to Johns Hopkins University Associate Professor of Neurology, Molecular Microbiology and Immunology, Dr. Douglas Kerr, author of multiple papers about embryonic stem cells and friend of FightSMA, “this is a significant event since it has really increased the number of embryonic stem cell lines available for researchers to study in understanding and ultimately treating human diseases.” He goes on to say, “It’s even more significant since the previously approved embryonic stem cell lines had a variety of problems including chromosomal abnormalities and culture conditions that would make them potentially unsafe in humans. These new ES lines don’t have those problems and this promises to advance research forward.”

To read the complete Washington Post article, click here.

To read the press release issued by NIH, click here.

From the National Institutes of Health press release:

NINDS Names Dr. Petra Kaufmann Director of the Office of Clinical Research

The National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health, has named Petra Kaufmann, M.D., M.Sc., as director of its Office of Clinical Research.

Dr. Kaufmann is among the foremost experts in the design and management of clinical trials for neuromuscular disorders, including spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS), and mitochondrial diseases. In her new role, Dr. Kaufmann will lead the Institute’s efforts to increase the effectiveness of clinical studies by addressing issues such as optimal trial design, ethical safe conduct of trials, and challenges in patient enrollment.

“Dr. Kaufmann has experience in all phases of clinical research, from conducting laboratory investigation and studies on disease mechanism to serving in key leadership positions on several major multicenter trials,” said Story C. Landis, Ph.D., director of NINDS. “Dr. Kaufman’s outstanding skills and expertise will allow us to make the most of the scientific opportunities ahead and to have a significant impact on clinical neuroscience.”

Dr. Kaufmann said, “I look forward to supporting excellence in clinical research at NINDS so that the advances in neuroscience can be translated into better treatments for patients.”

To read the full press release, click here.

From the National Institutes of Health:

Under the Recovery Act, the NIH has established a new program entitled Research and Research Infrastructure “Grand Opportunities” hereafter called the ”GO” grants program.This new program will support projects that address large, specific biomedical and biobehavioral research endeavors that will benefit from significant 2-year funds without the expectation of continued NIH funding beyond two years.  The research supported by the ”GO” grants program should have high short-term impact, and a high likelihood of enabling growth and investment in biomedical research and development, public health, and health care delivery.

Approximately $200 million dollars will be committed to projects resulting from this request and “only applications with budgets greater than $500,000 total costs per year for a project period of two years are expected to be considered.” This request for applications is expected to be very competitive, but it is also seen as a huge opportunity that is too important for the SMA research community to pass up.

The opening date is April 27th and the application deadline is May 27th. For more information, click here.

The National Institutes of Health (NIH) announced that Dr. Elias Zerhouni will be stepping down as NIH’s Director at the end of October. In an email to members of NIH’s extramural community, Dr. Zerhouni said, “I have had the unparalleled privilege of leading one of the great institutions in history,” and he included that he was leaving “to explore new opportunities and to devote much of my attention to writing.” Dr. Zerhouni has held the position at NIH since May of 2002.

“During his tenure, Zerhouni worked to lower barriers between disciplines of science and encourage trans-NIH collaborations,” said NIH’s press release announcing his departure. It went on to say “Zerhouni also led a major reform of the translational and clinical research system in the United States.”

To read the NIH press release and learn about Dr. Zerhouni’s accomplishments, click here.

The National Institutes of Health “is the primary federal agency for conducting and supporting basic, clinical and translational medical research, and it investigates the causes, treatments, and cures for both common and rare diseases.” It is composed of 27 Institutes and Centers, including The National Institute of Neurological Disorders and Stroke (NINDS) which established the SMA Project, “a model translational research program to accelerate the process of developing a safe and effective treatment for spinal muscular atrophy.”

A new website has been launched to, in the words of NINDS’s Dr. John Porter, “help parents understand the how-when-where-why of clinical studies and clinical trials in their children.” While not a spinal muscular atrophy (SMA) specific site, the videos and articles address many of the concerns that parents have when considering enrolling their child in a clinical trial, including SMA trials. Topics include “Importance of Research In Kids,” “Safety and Protections,” “Questions You Should Consider Asking,” and “Effects on the Family.”

The site was developed and made possible by the support of: National Heart Lung and Blood Institute (NHLBI), NIH, National Institute of Child Health and Human Development (NICHD), Best Pharmaceuticals for Children Act (BPCA), National Marfan Foundation (NMF), National Center for Research Resources (NCRR), NIH, and Gerber Foundation.

Check out the website by visiting: www.ChildrenAndClinicalStudies.nhlbi.nih.gov.

Notice Number: NOT-FD-08-001
Release Date: September 24, 2007
Issued by: Food and Drug Administration/Office of Orphan Products Development and Food and Drug Administration

The Food and Drug Administration’s (FDA) Office of Orphan Products Development (OOPD) is pleased to announce the availability of funds for fiscal year (FY) 2009 and FY 2010 grant awards to support clinical trials on the safety and effectiveness of products for rare diseases and conditions. Contingent on availability of FY 2009 and FY 2010 funds, it is anticipated that $14.2 million will be available for new applications, competing awards, and non competing continuation awards. For FY 2009, the application receipt date is February 6, 2008, and for FY 2010, the application receipt date is February 4, 2009.

These studies are intended to provide acceptable data to the FDA that will substantially contribute to the approval of new products, or new indications for already marketed products. In the FDA OOPD grants program, products for rare diseases and conditions (orphan products) are defined as drugs, biologics, medical devices, and medical foods indicated to treat or diagnose a rare disease or condition with a prevalence of fewer than 200,000 people in the United States.

To read the complete notice, click here.

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Participating Organizations: Food and Drug Administration (FDA)

Components of Participating Organizations: Office of Orphan Products Development (OPD)

Title: Clinical Studies of Safety and Effectiveness of Orphan Products; Research Project Grant (R01)

Request for Application (RFA) Number: RFA-FD-08-001

Opening Date: January 6, 2008 (Earliest date an application may be submitted to Grants.gov)

Application Submission/Receipt Date(s): February 6, 2008; February 4, 2009

Expiration Date: February 5, 2009

For more information, click here. For additional funding opportunities from the National Institutes of Health, click here.

From the SMA Project:

Dear Colleague,

We have now posted the Supporting Documentation (including the Subcontract Agreement) for the SMA Project (http://www.smaproject.org) Request for Proposals (RFP) LD-090707. This RFP seeks to identify facilities to conduct preclinical testing of chemical compounds with the ultimate goal of submitting an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA). We have also posted an updated version of the RFP (Attachment B in the RFP has been updated).

The RFP and Supporting Documentation can be accessed through the Solicitations page of the SMA Project website at http://www.smaproject.org/solicitations.html. Proposals are due by 3 p.m. Eastern Time on October 5, 2007.

Studies to be conducted under the resulting contract will include preclinical safety, toxicology, pharmacokinetic/dynamic, and biodistribution studies with candidate small molecule compounds that act as therapeutic agents for spinal muscular atrophy (SMA). While a broad range of Good Laboratory Practice (GLP) and non-GLP studies is anticipated, capability to conduct studies according to GLP is required. Science Applications International Corporation (SAIC), on behalf of the Government, shall supply the Offeror with a small amount of each candidate therapeutic compound. For certain compounds provided to the Contractor, SAIC may request a comprehensive Product Development Plan for services to be provided by the Contractor. Facilities will be evaluated on their past experience in preclinical, IND-directed small molecule development, as well as the proposed schedule and cost of studies required.

The National Institute for Neurological Disorders and Stroke (NINDS) (http://www.ninds.nih.gov/) launched The SMA Project: A Collaborative Program to Accelerate Therapeutics Development for Spinal Muscular Atrophy in September 2003. This program funds milestone-driven research aimed at identifying and rapidly developing a treatment for SMA, a paralyzing neurodegenerative disease of childhood.

After opening with a cocktail reception the night before, the FightSMA Annual Conference hosted a full day of researcher presentations on Monday at the L’Enfant Plaza Hotel in Washington, D.C. FightSMA Science Director Dr. Christian Lorson kicked off the day at 8 o’clock in the morning with a review of the basic science of spinal muscular atrophy (SMA). This was followed by eleven scientists who represented the United States, Canada, Germany, Italy, the National Institutes of Health, university researchers, and private sector biotechnology corporations. With the contributions of the additional scientists and researchers in the audience, this diverse group openly discussed published and unpublished research, recent successes and failures, and novel tactics in the fight against SMA. The energy of the room was evident as the attendees looked towards the second day of presentations on Tuesday.