Purpose. The goal of this Funding Opportunity Announcement (FOA) is to solicit applications on lysosomal storage disorders (LSDs) focused on improving CNS treatment outcomes, enhancing the effectiveness of delivery and targeting of cells, enzymes, drugs and genes into the brain, and developing novel therapeutic modalities, such as implantable biocapsules and micro-electro-mechanical systems (MEMS)-based devices. Lysosomal storage disorders constitute a group of recessive genetic diseases resulting from cellular enzymatic deficiencies of acid hydrolases that normally catalyze the metabolism of glycoproteins, glycolipids and other macromolecules, or from defects in transporter proteins leading to pathogenic accumulation of these substances in lysosomes. Treatment modalities for LSDs are currently limited to bone marrow transplantation (BMT) and enzyme replacement therapy (ERT). These approaches while providing significant promise for treatment of the visceral manifestations of LSDs, do little to address CNS pathologies for this group of disorders. Thus this announcement specifically encourages the transition from basic studies in LSDs to translational research for improved delivery of therapeutic cells, proteins, genes, and small molecules across the blood-brain barrier.
Mechanism of Support. This FOA will utilize the Research Project Grant (R01) award mechanism, and runs in parallel with an FOA of identical scientific scope (PAS-06-202) that solicits applications under the exploratory/developmental grant award (R21) mechanism.
Funds Available and Anticipated Number of Awards. Awards issued under this FOA are contingent upon the availability of funds and the submission of a sufficient number of meritorious applications. The NINDS and ORD intend to commit a total of approximately $1,050,000 (total costs), in addition to funds available for applications sent in response to this FOA and a parallel R21 FOA (see above) that score within the NINDS payline (see NINDS Funding Strategy
http://www.ninds.nih.gov/funding/ninds_funding_strategy.htm), depending on the overall scientific merit of the applications and the availability of funds throughout the duration of this solicitation. Additional funds may also be available through the Lysosomal Storage Disease Research Consortium (
http://www.LSDresearch.org) for applications received and reviewed under this FOA. Because the nature and scope of the proposed research will vary from application to application, it is anticipated that the size and duration of each award will also vary. The total amount awarded and the number of awards will depend upon the mechanism numbers, quality, duration, and costs of the applications received. Each R01 project is limited to a duration of five years.
Eligible Institutions/Organizations. Public/State Controlled Institution of Higher Education; Private Institution of Higher Education; Nonprofit with 501(c)(3) IRS Status (Other than Institution of Higher Education); Nonprofit without 501(c)(3) IRS Status (Other than Institution of Higher Education); Small Business; For-Profit Organization (Other than Small Business); State Government; U.S. Territory or Possession; Indian/Native American Tribal Government (Federally Recognized); Indian/Native American Tribal Government (Other than Federally Recognized); Indian/Native American Tribally Designated Organization; Non-domestic (non-U.S.) Entity (Foreign Organization); Hispanic-serving Institution; Historically Black Colleges and Universities (HBCUs); Tribally Controlled Colleges and Universities (TCCUs); Alaska Native and Native Hawaiian Serving Institutions; Regional Organization; Other(s): Eligible agencies of the Federal government; Faith-based or community based organizations.