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Research Grants

Clotilde Leveque;Antonio Velayes Baeza;Benedikt Bader
English

Download Research Grant Application

The Advocacy calls for research grant proposals when we are able to make seed money grants of up to $100,000 to encourage investigations into the pathophysiology and the related red blood cell abnormalities as well as the diagnostics, epidemiology and genetics of the NA syndromes. 

NA diseases are monogenic movement disorders associated with degeneration that begins in the basal ganglia. NA strikes generally

in the third or fourth decade with phenotypes resembling Huntington’s and Parkinson’s disease. The NA diseases are characterized

by acanthocytic red blood cell membranes. We hypothesize that acanthocytosis relates to a malfunction in the membrane that in neurons

may hamper normal autophagy and lead to cell death.

The rarity and wide geographical spread of the NA diseases have contributed to a number of different researchers principally in Australia, Austria, France,  Germany, Great Britain, Israel, Italy, Turkey and the United States 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. And since acanthocytosis is part of the NA diseases' 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. 

The Objective of our research is to find therapies for the NA diseases through :

  • Advancing scientific research that explores the physiology of the patients’ neurons and the effect of the gene mutation on the eventual neuronal mal-function and apoptosis, contributing to an understanding of basal ganglia degeneration that may add to progress research into Huntington’s and Parkinson’s diseases
  • Supporting clinical studies of patients who are widely and thinly dispersed around the world by sharing up-to-date patients’ case histories  in our REGISTRY that will be available through the Internet to qualified scientists
  • Encouraging younger scientists to study these diseases and bring together a wider community including the disciplines of haematology, cell biology, neuroscience and neuropsychology
  • Providing information and training in this disease group to the medical community around the world and maintain strong communication with researchers receiving grants to assure that funds are used for the intended purpose
  • Supporting families affected by these diseases with information and communication that will empower them to take initiative in finding experienced neurologists or working with neurologists who often have never diagnosed or treated any of these conditions

Grant Decision-Making Process

Application for grants is competitive. Assessment of applications is based on peer-review. External referees comments are sought on applications and are considered by the Advocacy’s Scientific Advisory Panel of qualified professionals who are not grant applicants. An interview may be a part of the assessment process. The funding committee will abide by the Code of Conduct of the Association of Medical Research Charities (AMRC) http://www.amrc.org.uk/our-work/peer-review that protects the confidentiality of the decision making process. No contact with individual committee members may be made. All requests for information must be referred to the Advocacy.

Feedback on applications may be given after decisions have been made. The Advocacy normally will provide feedback to applicants by supplying unattributed comments of the external referees and, occasionally, the views of the committee that made the funding decision. Feedback is provided solely at the Advocacy’s discretion and is intended to convey the substance of the independent scientific critique of the proposal in order to help the applicant. Although the Advocacy is normally willing to relay the view of its referees, it is not prepared to discuss the merits of the individual referees’ opinions.

Program grant applicants may have the opportunity to comment on the referees’ opinions before the meeting of the funding committee at which the applications will be considered.

Funds available 
The Advocacy expects to have total funds of $100,000 available for new research over the period 2015-16 We will seek to find joint sponsors for projects.

Time scales 
Applications for grants may be sent to the Advocacy (ginger@naadvocacy.org) throughout the year. Generally decisions are made within three months of the application.

Conditions 
Grants are made under conditions similar to those of the AMRC http://www.amrc.org.uk/publications/statement-on-the-use-of-animals-in-researchhttp://www.amrc.org.uk/publications/statement-supporting-research-universities

Reports 
Holders of Advocacy grants are required to send short interim reports at agreed intervals as well as two reports in the final months of their awards. The information provided enables the Advocacy to assess the research it has funded and is an important part of our accountability to our supporter and the public.

The two final reports required are:

  • A final report on the work undertaken, achievements and outcomes. This will include an explanation of any significant variances between sums awarded and actual expenditure under particular cost headings.
  • A financial statement of expenditure incurred against the grant.

Research Goals

The Advocacy’s medium-term goal is to identify the pathway to neurodegeneration in the NA diseases and eventually to find

therapies to delay or prevent the development of neuroacanthocytosis.

We are especially interested in:

●● The function of the protein Vps13A (chorein) that is missing in chorea-acanthocytosis (ChAc),

the most common of the NA diseases.

●● Shape, folding, and abnormal folding of Vps13A, and XK proteins

●● Genome-wide association search for other mutations in NA patients

●● Relationship of acanthocytosis to cell malfunction and death

●● Development of animal or cell models

●● Work on the established registry of neuroacanthocytosis patients

●● Study of natural history of NA diseases in comparison to Huntington’s and Parkinson’s diseases

●● Protein interactions in NA diseases

Scope of investigation

Applications from Principal Investigators not currently pursuing NA research are welcome if their specific expertise, together

with proof of project feasibility and relevance, can provide novel approaches to the identification of disease mechanisms and

therapeutic targets for NA.

Collaboration

The Advocacy’s aim and expectation is to promote close collaboration between all researchers in the field of neuroacanthocytosis,

including the investigators supported by the nine previous grants, as well as with the European Multidisciplinary Initiative on

Neuroacanthocytosis (EMINA) (www.emina2.eu). Special consideration will be given to applications that demonstrate collaboration.

If results from Advocacy funded research are not accepted for publication within 6 months after the conclusion, it is expected that

results, whether positive or negative, will be published in “PLoS Currents: Huntington Disease” (http://plosone.org).

Since 2007 we have awarded nine grants for basic research projects and one for clinical research. Progress reports of these projects

are at www.naadvocacy.org/research-grants under NANews.

Review procedure

All proposals will be assessed by our Scientific Advisory Panel that will call on scientists with expertise in the field of proposal. The

panel will consider: significance, technical merit, quality of the applicants´ previous work, relevance of goals of the research, and

complementarity with other proposals or ongoing research.

How to apply

Grant Proposals may be submitted at any time.

In the past our grants were of the order of €35,000 (US$ 38,000) per annum. The families and friends of a small community of

patients fund the research of the Advocacy

Inquiries

Please contact ginger@naadvocacy.org tel +44 20 7937 2938

ADVOCACY FOR NEUROACANTHOCYTOSIS PATIENTS

seeks proposals from academic researchers for investigations into the

etiology of basal ganglia degeneration in neuroacanthocytosis (NA).