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.

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.

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.

From the SMA Project email service (June 8, 2007):

Dear Colleague,

I am pleased to announce the following funding opportunity for researchers in the field of spinal muscular atrophy (SMA):

The SMA Project (http://www.smaproject.org) expects to release a Request for Proposals (RFP) within the next 14 days to identify and fund facilities capable of providing the necessary research and development support for an Investigational New Drug (IND) submission in a format acceptable to the U.S. Food and Drug Administration (FDA).

The intention is to award a subcontract to a facility capable of conducting safety, toxicology, pharmacokinetic/dynamic, and biodistribution studies with candidate small molecule compounds that act as therapeutic agents for SMA. These studies will be done in partial fulfillment of the requirements for submitting an IND application to the FDA. Science Applications International Corporation (SAIC), on behalf of the Government, shall supply the Offeror with a small amount of each candidate therapeutic compound.

The Offeror will be required to prepare and deliver, for each selected therapeutic compound provided by SAIC for testing, a specific Test Implementation Plan including a proposed budget for the specific testing activities. Activities within the facility shall be highly coordinated with efficacy testing activities outside the facility by SAIC. SAIC shall require from the Offeror preclinical development data that are required by the FDA for filing successful IND applications.

Offers will be due approximately 28 days from the release of the RFP. Relevant documents will be available through the Solicitations page of the SMA Project website at http://www.smaproject.org/solicitations.html.

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.

From the Department of Health & Human Services:

We are pleased to let you know that the National Institute of Neurological Disorders and Stroke (NINDS), through the NINDS Pilot Therapeutics Network (NPTUNE) program, is sponsoring two research studies to evaluate the safety and tolerability of sodium phenylbutyrate (NaPB) in subjects with Type I or Type II/III Spinal Muscular Atrophy (SMA). Both studies are Phase I dosage escalation studies that will assess safety, maximum tolerated dosage, pharmacokinetics, and bioactivity. It is anticipated that enrollment will begin soon.

Each study will be conducted in two stages. Stage 1 is the dosage escalation stage where maximum tolerated dosage will be defined. Safety, tolerability, and pharmacokinetics will be evaluated in this stage. Stage 2 will enroll different subjects who will receive NaPB at the maximum tolerated dosage identified in Stage 1. Safety, tolerability, and pharmacokinetics, as well as levels of SMN mRNA and protein, will be evaluated in Stage 2.

For additional information about these NPTUNE SMA studies, including eligibility criteria and contact information, please refer to our posting on www.ClinicalTrials.gov. The link for the Type I study is http://www.clinicaltrials.gov/ct/show/NCT00439218?order=3. The link to the Type II/III study is http://www.clinicaltrials.gov/ct/show/NCT00439569?order=4. Each participating clinical site was selected by competitive application process and will be listed on this website once ready to begin enrollment.