The following are three National Institutes of Health (NIH) funding opportunities that may be of interest to researchers working toward a treatment and cure for spinal muscular atrophy (SMA), a deadly crippler and the number-one inherited genetic cause of infant death.

Exceptional, Unconventional Research Enabling Knowledge Acceleration (EUREKA) (R01)
Application Due Date: October 21, 2010
Solicits proposals for exceptionally innovative research on novel hypotheses or difficult problems, solutions to which would have an extremely high impact on biomedical or biobehavioral research. Support may be requested for up to $800,000 in direct costs (excluding consortium F&A) over a four-year period, not exceeding $250,000 (direct costs, excluding consortium F&A) in any one year. (Note, that over the last three years the EUREKA mechanism has had a success rate much more in line with standard R01 applications. Each of these offers an opportunity not as easily addressed in regular NIH funding mechanisms.)

NIH Common Fund Transformative Research Projects Program (R01)
Application Due Date(s): October 27, 2010
Solicits applications proposing groundbreaking, exceptionally innovative, high risk, original and/or unconventional research with the potential to create new scientific paradigms or challenge existing ones. Budget requests should be commensurate with project needs for up to a five-year project period. Up to one third of the budget for this FOA will be reserved projects exceeding $1 million dollars in direct costs.

Scalable Assays for Unbiased Analysis of Neurobiological Function (R01)
Application Due Date: November 17, 2010
Solicits applications to develop novel, robust analytical platforms using in vitro assays to reveal changes in neuronal and/or glial function. The goal is to adapt state-of-the-art measures of basic cellular processes or molecular events that are key mediators of brain function with the intent to probe mechanisms and/or perturbations in an unbiased and efficient manner. The novel assay platforms would provide opportunities to measure neurobiological endpoints and build a pipeline to be used in the context of target identification and drug discovery.

In October, a workshop and a course will be held that may be of interest to researchers and medical professionals involved with spinal muscular atrophy (SMA).

At the beginning of October, the Center for Continuing Medical Education at Columbia University in New York will hold a course to update clinicians and caregivers about therapies in development, the new consensus on patient management, and ways to participate in upcoming clinical trials. Supported by a grant from the New York-based SMA Foundation, “Recent Advances in SMA and Other Pediatric Neuromuscular Diseases” will be held October 2nd and is “an intensive one-day course designed for medical professionals, including physicians, nurses, genetic counselors, physical, occupational, and respiratory therapists, with an interest in pediatric neuromuscular diseases.” For more information about the course, click here.

Later in the month, a three-day workshop (October 18-20) about conducting research on rare diseases and orphan products will be held in Rockville, Maryland. Sponsored by the Food and Drug Administration (FDA), agencies of National Institutes of Health (NIH), the National Organization for Rare Disorders (NORD), and Duke University Medical Center, the “Accelerating Therapies for Rare Diseases” workshop “focuses on FDAs regulatory requirements and the clinical trial issues that are especially relevant to rare disease research.” Speakers will include members of academia, government agencies, and the private sector. For more information about the workshop, click here.

On July 21, 2010, the U.S. Senate Committee on Health, Education, Labor and Pensions (HELP) heard testimony from panelists discussing Rare and Neglected Pediatric Diseases. One of these diseases was spinal muscular atrophy (SMA).

Two of the panelists were Dr. Alan Guttmacher and Mr. John F. Crowley.

Dr. Guttmacher, who is the Acting Director at the National Institute of Child Health and Human Development (NICHD), spoke on the importance of SMA and SMA research to NIH and its officials.

Mr. Crowley, CEO and Chairman of Amicus Therapeutics, spoke about patient advocacy, and the admirable work and advocacy that FightSMA has exhibited over the years.

Click here to read the transcript of Dr. Guttmacher’s testimony and here for the transcript of Mr. Crowley’s testimony. For more information about the hearing, including the testimony of other panelists and video of the hearing, click here.

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 FDA’s Office of Orphan Products Development and NIH’s Office of Rare Diseases Research:

The Office of Rare Diseases Research at the US National Institutes of Health (NIH), and the Office of Orphan Products Development at the US Food and Drug Administration (FDA), are pleased to announce the 2010 edition of their collaboratively developed course, “The Science of Small Clinical Trials.” This is a broad survey course (not a high-level statistical seminar), which is intended to heighten awareness of the methods that exist to design and analyze clinical trials using small numbers of participants. An inescapable necessity when dealing with rare diseases, the use of small trials is also rising in prominence in the context of tissue transplantation, advanced prosthetics, and individualized pharmacogenomics.

The first edition of the course, offered in 2009, was restricted to FDA and NIH staff. The 2010 edition of the course has been revised (based upon comments from 2009 participants), and is now open to ANYONE who wishes to register. The course comprises 7 2-hour lectures, presented at the Lister Hill Center Auditorium on the NIH campus in Bethesda, MD, from 16 February through 8 March, 2010; the lectures will also be available online via the Internet, live and by delayed on-demand video streaming (using freely available RealPlayer software), allowing anyone with a good Internet connection and appropriate computer to participate (questions from remote attendees will be received via a live text chat room, or via a discussion forum, on a web site dedicated to the course).

ALL participants must register. An optional self-administered open-book On- line examination will be provided at the end of the course, and individuals who pass this examination will receive a certificate from FDA’s Office of Orphan Products Development.

For more information about the course, and online registration, visit: http://small-trials.keenminds.org.

According to the course’s website, “the target audience is professionals interested in drug/device evaluation and regulatory affairs.” Currently, on-site attendance is full, so those wishing to participate must via the internet.

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.