Rare Disorders Grants

Prize4Life announced its 2-year $1 million ALS/MND Biomarker Challenge on November 6, 2006.

The Prize4Life Biomarker Prize is divided into two tracks - theoretical findings and real outcomes.

The first track was a call for scientists and researchers from all backgrounds to submit theoretical papers on how they would develop a biomarker for ALS. In May 2007, Prize4Life awarded $15,000 for each of the five best ideas submitted . This track is now closed for submissions.

The second track, currently running, involves identifying an actual ALS biomarker. Anyone can submit a solution to the second track at any time, regardless of whether they participated in the first track.

A prize of up to $1,000,000 will be awarded in track two for the first validated ALS biomarker submitted that meets the desired requirements. Those submitting a solution must provide statistically significant results as proof of the validity of the solution. This challenge will end in November 2008 if a winning solution is not received so send in your solution now!!

A biomarker is a distinctive biological or biologically derived indicator that can mark the presence and/or progression of a disease. A biomarker can take the form of a chemical marker, a mechanical device that measures relevant physiological changes, or combination of the two. Currently, there are no known biomarkers for ALS for either diagnosing or measuring the progression of the disease.

Interested scientists and researchers should REGISTER with Prize4Life.

INNOCENTIVE 4470259
ALS Biomarker
POSTED: NOV 06, 2006
DEADLINE: NOV 06, 2008
$1,000,000 USD

Cystinosis Research Foundation
Call for Funding Proposals and Postdoctoral Research Fellowships

The ultimate goal of the Cystinosis Research Foundation is a cure for this disease. Global calls for grant applications will be made two times per year, on March 15 and September 15. The deadline for the grant applications will be May 1 and November 1 respectively. Research awards and fellowship awards will be given for up to three years. Currently, the Cystinosis Research Foundation has more than $1,200,000 dollars in research and fellowship grants available. The number of awards and their value will depend on the number of outstanding proposals and the funds available at the time.

Research Proposals

The Cystinosis Research Foundation will be announcing its autumn Call for Research Proposals on September 15, 2008. The Cystinosis Research Foundation is prepared to fund proposals to improve the immediate care of children and young adults with cystinosis and to develop new understanding and treatment of cystinosis to help these children in the future.

Postdoctoral Fellowships

The Cystinosis Research Foundation has launched a postdoctoral research fellowship program to attract qualified, promising investigators to establish careers in cystinosis research. The fellow’s stipend should be the usual amount for such a trainee at that institution. With adequate justification, up to $10,000 per year can be requested to support that individual’s research. However, the total award cannot exceed $75,000 per year. Awards will generally be for a period of 2-3 years.

First priority will be given to “named” postdoctoral trainees. However, investigators who are already studying cystinosis can apply for a fellowship position with the expectation of attracting a suitable postdoctoral fellow within a year. Predoctoral students, who are already studying cystinosis, will be considered if funding is available.

REVIEW PROCESS

Proposals are evaluated by the Cystinosis Research Foundation Scientific Review Board (SRB) comprised of leading experts on cystinosis who determine the scientific validity and merit of the proposed research based on the NIH scale of standards. The SRB then advises the Cystinosis Research Foundation on the scientific merit of each proposal. The Foundation will balance the eventual funding to support clinical and bench research, and fellowships. Applicants will be notified in writing regarding the final status of their application.

PROPOSAL GUIDELINES AND FELLOWSHIP GUIDELINES

We encourage concise proposals. The research proposals and fellowship applications should describe the rationale and importance of the project and should include the specific aims and research design and methodology. Include sufficient detail in a concise manner to facilitate evaluation of the proposed work.

Research Grant Proposals: Maximum length is nine one-sided pages, single-spaced, using a standard 12-point font with 1 inch margins (2.54 cm) on all sides including figures, diagrams and drawings. Cover sheet, documentation, budget and budget justification and curriculum vitae (CV’s) are not included in the 9-page limit. Longer applications will not be evaluated.

Fellowship Grant Applications: Maximum length is six one-sided pages, single-spaced, using a standard 12-point font with 1 inch margins (2.54 cm) on all sides including figures, diagrams and drawings. Cover sheet, documentation, budget and budget justification and curriculum vitae (CV’s) are not included in the 6-page limit. Longer applications will not be evaluated.

Cover Sheet Information: Names and Affiliations of Principal and Co-Principal Investigators or and Research Fellow – It is mandatory that you complete and attach the Cystinosis Research Foundation cover sheet as part of your application. Please download and attach the applicable cover sheet – either the Research Grant Proposal Cover Sheet or Fellowship Grant Proposal Cover Sheet.

Please format Grant and Fellowship Applications as follows:

1. Abstract/Summary of project – Explain the relevance to cystinosis. What problem does the project or fellowship address? Why is the work important to children and young adults with cystinosis? How will the project be accomplished?

2. Specific Aims – List the study or fellowship objectives and describe the specific goals of the research, including any hypotheses to be tested.

3. Preliminary Studies/Progress reports.

4. Research Design and Methods – Describe the research design and methodology that will be used to accomplish the study or fellowship aims.

Additional Information NOT part of the maximum length requirement for the Research Grant and Fellowship Grant Applications:

1. Detailed Budget – The Cystinosis Research Foundation will generally provide up to 10% of indirect costs. Requests for higher amounts must be carefully justified. Funding requests must be submitted in U.S. dollars.

2. Budget Justification – Please explain items that might be confusing or unusual to the review committee.

3. Key Personnel - Please include a current CV and indicate the percent effort that each person is expected to devote to the project.

4. Statement detailing all other available resources including other grant support available – Indicate the amount and granting organization for any other sources (current or pending) of funding for the study.

5. IRB (human subject) approval and consent forms or plan to obtain such approval, if applicable. Animal subject approval if applicable. If the proposal involves human gene therapy, NIH Recombinant DNA Advisory Committee (RAC) review or equivalent. Funds for awards will not be released until all necessary approvals for human and/or animal studies are received.

6. A statement as to why Cystinosis Research Foundation funding is essential for the execution of this project.

ADDITIONAL REQUIREMENTS FOR FELLOWSHIP APPLICANTS

In addition to the above, fellowship applications must provide a complete CV of the proposed candidate and a statement outlining his/her career goals. Please discuss why this fellowship is important to the applicant. It should be clear who the fellow’s mentor(s) will be. The mentor must provide a complete CV as well as a statement of the plan for training the fellow.

SUBMISSION INFORMATION

Applicants must submit the original of the completed application, eight printed copies of each proposal and an electronic copy (PDF format if possible) to:

Cystinosis Research Foundation
Nancy Stack, President
18802 Bardeen Avenue
Irvine, California 92612-1521
(949) 223-7610
Email: heystack4@aol.com

Sheila Essey Award - An Award for ALS Research
Presented by the AAN and the ALS Association and supported through the philanthropy of the Essey family and the ALS Association.

This award recognizes an individual who has made significant research contributions in the search for the cause, prevention of, and cure for amyotrophic lateral sclerosis (ALS). The criteria being used to determine the recipient include recent major scientific impact, long-term scientific impact, and overall impact to ALS.

Presentation
Recipient is expected to present a 20-minute lecture during an anterior horn scientific session at the 61st AAN Annual Meeting.

Recipient will receive:
Certificate of recognition and $25,000 prize to be used by recipient to continue ALS research
Complimentary registration for 61st Annual Meeting
Recognition at 2009 Awards Luncheon at 61st Annual Meeting
Eligibility
Must be actively engaged in ALS research

Application Procedure
Applicants should submit one complete set of the following materials:

Completed application form Note: Under general description: describe how scientific contributions have created a better understanding of ALS; why applicant should receive award; and why contributions represent a major advance or hold significance in ALS research in terms of identifying the cause, potential treatment, prevention, or cure.
Current curriculum vitae
Supporting information (not to exceed three pages), including:
Single page biography including previous positions and any awards or recognition received
One- to two-page listing of six to ten peer-reviewed journal references that support statements about contributions to ALS research (do not send reprints or a bibliography)

Deadline

The deadline to apply for 2009 AAN Scientific Awards is November 3, 2008. Applications will be available in August 2008.

For more information, please contact Naomi Soderbeck at nsoderbeck@aan.com or (651) 695-2814.

FUNDING FOR RESEARCH AVAILABLE

Shwachman-Diamond Syndrome (SDS) is a multi-system autosomal-recessive disorder characterized by exocrine pancreatic dysfunction, and varying degrees of marrow dysfunction with cytopenias. Many associated clinical problems have also been described, including skeletal anomalies, metaphyseal dysostosis, rib abnormalities, cleft palate, dental abnormalities and others. Shwachman-Diamond Syndrome Foundation (SDSF) is a nonprofit support organization actively helping the victims of this disease. SDSF is accepting grant requests for research relevant to this disease. Any high quality, innovative research proposal deemed relevant to SDS will be considered. The following are areas of particular interest:

Identify gene mutations of SBDS gene
Collect centralized data on bone marrow abnormalities
Evaluate predictors of bone marrow abnormalities to determine their correlation with the disease
Determine the causes of bone marrow failure and leukemia
Further delineate the pathogenesis of pancreatic disease
Determine if differences in disease complications occur dependent on persistent vs. cylic neutropenia
Investigations related to adult patient presentation, management, and care including transition from
pediatric-centered to adult care

Eligibility: Persons applying for these grants must be in a faculty position with authority to hold an independent research grant (not a training position).

Letter of Intent: The potential applicant should write a one page letter of intent to the SDSF, indicating the applicant’s position and affiliation, describing their planned project, and explaining its relevance to Shwachman-Diamond Syndrome. These letters are reviewed by SDSF’s Medical and Scientific Advisory Board (MSAB) grant review process.

Purpose: This letter will be used to identify applicants who will be asked to provide a full application.

Dates: The letter is due no later than January 31st. There will be a response to all those who have written a Letter of Intent by February 28th. All Grant Applications must be submitted by April 12. The final grant decision will be announced by July 31st with a November 1 start date.

Terms of Support: Support may be provided for up to two (2) years in an amount not to exceed $30,000 per year. The second year funding will not be released until a Progress Report has been reviewed and approved by the MSAB. The date for the Progress Report is October 1. Applicants may submit only one grant at a time, and may hold only one grant at a time.

If you would like us to send you a grant application, have any questions regarding the grant process or have an interest in the disease, please contact SDSF at the address indicated on our home page, call toll free 1-877-737-4685, by fax at 1-970-255-8293 or via email at 4sskids@shwachman-diamond.org

Apply for Funding

If you are a researcher interested in applying for funding from the Jain Foundation, please read the guidelines on this page.

The explicit goal of the Jain Foundation is to expedite development of a treatment or cure for LGMD2B/Miyoshi. Your proposed project MUST have application to this disease, or it will not be considered.

We have no application deadlines and no extensive application procedures. Read the instructions below and send a detailed description of your proposed project, and any supporting work, to Dr. Plavi Mittal at plavimittal@jain-foundation.org. We generally respond to proposals within 4-6 weeks.

Instructions for submitting a proposal:

1. The project must have direct relevance to dysferlin deficiencies -- either to extend our understanding of the disease or a unique approach to a treatment for the disease.

2. The project should not have been previously performed elsewhere and sufficient due diligence should have been done to find out if the same project is currently being done in another lab. We will confirm this independently.

3. Please include a clear description of the scientific background and a demonstration of the techniques needed to make the proposed project a success. Past work in your lab and/or published papers directly related to the proposed project are helpful. Evidence of access to techniques and collaboration with others is equally important.

4. Preliminary data to assess feasibility of the proposed project are required and should be completed before the funding can be initiated.

5. Please include a detailed and aggressive timeline of experiments leading to the results of the project. Proposals without a detailed timeline will not be reviewed.

6. Please include a detailed budget (including personnel, material, and all other costs) for the proposed project. Proposals without a detailed budget will not be reviewed.

7. The average project will require a proposal of about 6-10 pages (excluding references) to cover the level of detail that we expect, though individual proposals may vary in length. The experiments should be described in enough detail for us to understand why each step in the experimental plan will require the time that is alotted for that step in the timeline.

Request For Proposals

The International Waldenstrom's Macroglobulinemia Foundation (IWMF) is a non-profit organization which offers support for basic scientific research that will further knowledge of the cause, diagnosis, treatment, and cure for the disease Waldenstrom's macroglobulinemia. Research proposals may be submitted at any time. Each proposal will receive consideration from the IWMF Research Committee, the Scientific Advisory Committee, and the IWMF Board of Trustees.

Scientific researchers interested in applying for a grant may receive an application packet by contacting:

Tom Myers
IWMF Vice President of Research
23 State Park Road
Swanton, MD 21561
Tel: 301-616-9200
Email: tom@mountaineerlog.com.

Institute for Neuroacanthocytosis Research Grants

We encourage applications for research grants for investigations into the genetics, diagnostics and epidemiology of the NA syndromes as well as the pathophysiology and the related red blood cell abnormalities.
Neuroacanthocytosis syndromes are a group of rare diseases, characterised by misshaped erythrocytes and progressive neurodegeneration, causing movement disorders and neuropsychiatric symptoms.

The rarity and wide geographical spread of the NA diseases have contributed to a number of different researchers principally in Europe and Japan pursuing specific studies of the elements of the NA phenotypes including descriptive history of the development of the disease, the abnormalities of the erthrocytes and the function of the VPS 13A protein that is absent in chorea-acanthocytosis.

Since acanthocytosis is part of the NA syndromes clinical manifestations, the study of acanthocytes may offer the possibility to progress in the disease’ knowledge, to identify new cell signalling pathways towards either membrane proteins or transport systems.

Our Objectives for Research

To pursue the fundamental physiological questions raised by NA including the:

role of the proteins affected by the mutated genes in each disease as well as their molecular and cellular function

cause and significance of the spiky red blood cell membranes that are a defining characteristic of the diseases

pathway that leads to neuronal apoptosis in each of the diseases

To promote wide collaboration in the clinical study of the diseases. Because no country has more than an estimated 100 cases of NA and most countries in the world have fewer than 10 cases, it is vital that case histories of patients from around the world be available to those studying the clinical aspects of the diseases.

To develop the tools and the skill of neurologists to diagnose and, in the future, to treat the diseases.

To collaborate effectively with research into other similar conditions especially Huntington’s disease.

To encourage new, promising projects with seed money and assist in approaching larger grantors.

Our Values

Our funding seeks to:

Promote basic, curiosity-driven, investigator–led research that relates to the phenotypes of the NA diseases.

Bring together the wide research community around the world including haematology, cell biology, neuroscience and neuropsychiatry.

Encourage the wide dissemination of and free access to the research we support. We will seek to support unrestricted access and the public benefit wherever possible by requiring electronic copies of any research papers that have been accepted for publication in a peer-reviewed journal and are supported by the Advocacy to be deposited into PubMed Central (PMC) to be made freely available as soon as possible and in any event within six months of the journal publisher’s official date of final publication.

Do every thing possible to have strong communication with researchers receiving grants to assure that funds are used for the intended purpose.

Promote good communications between those working on NA research and others concerned with other diseases of the basal ganglia.

 

Request for Research Proposals
The Barth Syndrome Foundation, Inc. (BSF) is pleased to announce the availability of funding for research on the natural history, biochemical basis, and treatment of Barth syndrome.

Background
Barth syndrome is a serious X-linked genetic condition associated with cardiomyopathy, neutropenia, skeletal muscle weakness, exercise intolerance, growth delay, and diverse biochemical abnormalities (including defects in mitochondrial metabolism and phospholipid biosynthesis). Because many clinical and biochemical abnormalities of Barth syndrome remain poorly understood, we are seeking proposals for research that may shed light on any aspect of the syndrome. We are determined to find improved treatments--and ultimately a cure--for this rare and under-diagnosed disorder.

Types of Proposals Sought
We are most interested in providing "seed money" to be used by experienced investigators for the testing of initial hypotheses and collection of preliminary data leading to successful long-term funding by the National Institutes of Health (NIH) and other major granting institutions. In addition, we are especially interested in attracting new investigators to the very interesting field of Barth syndrome research.

Funding
We anticipate awarding several one- or two-year grants of up to $40,000 each. Funds will be available as soon as the successful grant applicants have been notified.

Process
We have a simple grant process. Applications should be of 10-15 pages in length and must follow the instructions listed on the BSF website. In general terms, detailed information about the specific aims, significance, research design and methods, personnel, and budget will be required, along with evidence of application to the relevant Institutional Review Board for any work involving human subjects.

Completed proposals will be forwarded to the BSF Scientific and Medical Advisory Board (as well as outside reviewers, in certain cases) for evaluation. Based on the recommendations of the Scientific and Medical Advisory Board, the BSF Board of Directors will make the final funding decisions for the grant applications.

Deadline
The deadline for submission of completed research grant applications from interested researchers is October 31, 2008. Grants will be awarded early March 2009.

Contact Information:
Matthew J. Toth, Ph.D.
Science Director
Barth Syndrome Foundation, Inc.
mtoth[at]barthsyndrome.org

One approach the organization uses to find a cause and cure for ALS is a peer-reviewed Research Grant Program, in which the investigator selects the area and method of research. The application/review process is modeled after the grant system used by NIH. The organization solicits investigator-initiated research proposals, which are peer-reviewed by the organization's Scientific Review Committee (SRC) and competitively evaluated for their scientific merit and ALS relevance. Grants are reviewed and funded twice yearly. Criteria for funding include:

1. innovative, cutting edge research; 
2. a process which lessens or eliminates favoritism and bias; 
3. to a specific description of the intended use of the grant funds by budgets and experimental design; and 
4. accountability by way of annual progress reports.

The SRC grant process generally involves the review of 200-300 abstracts per year with 20 to 25 grants receiving funding. This NIH-style, peer-reviewed grant process provides The ALS Assocation with a credible and equitable approach for finding and supporting the best possible research into discovering the cause and cure for ALS. The ALS Assocation currently has commitments of more than $7 million to active research grants with new commitments in the range of $2-2.5 million added each fiscal year.