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Developing a novel drug for neurobehavioral deficits in FASD

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R41AA029025-01
Agency Tracking Number: R41AA029025
Amount: $164,683.00
Phase: Phase I
Program: STTR
Solicitation Topic Code: 230
Solicitation Number: PA19-270
Timeline
Solicitation Year: 2019
Award Year: 2021
Award Start Date (Proposal Award Date): 2021-05-01
Award End Date (Contract End Date): 2022-04-30
Small Business Information
4621 ROSEDALE AVE
Bethesda, MD 20814-3715
United States
DUNS: 080498451
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: Yes
Principal Investigator
 MASAAKI TORII
 (202) 476-4279
 mtorii@childrensnational.org
Business Contact
 YUKA IMAMURA KAWASAWA
Phone: (203) 809-5634
Email: cogtheraso@gmail.com
Research Institution
 CHILDREN'S RESEARCH INSTITUTE
 
111 MICHIGAN AVENUE NW
WASHINGTON, DC 20010-2916
United States

 Domestic Nonprofit Research Organization
Abstract

Project Summary
Fetal Alcohol Spectrum Disorder (FASD) is a debilitating condition causing a wide range of disabilities
including behavioral and intellectual deficits, facial dysmorphology, and organ malformations. This disorder has
a profound impact on health care burden and cost; as many as 5% of children within the United States are
affected by this condition, with lifetime treatment costs reaching $2 million per individual. Despite the severity
of this disorder and its impact on public health, there currently are no therapeutic treatments available for
FASD. As such, identifying therapeutic molecules that can alleviate neurobehavioral issues associated with
FASD is critical. Our long-term objective is to identify effective treatments to ameliorate the cognitive
impairments associated with FASD. We have recently discovered that the targeting of the KCNN2 potassium
channel using a short peptide, Tamapin, improves both the gross and fine motor learning skills in our mouse
model of FASD. These findings suggest that KCNN2 blockers have the potential to reverse the
neurobehavioral problems associated with FASD and other neurodevelopmental disorders. We have already
prescreened 6 peptide candidates from the 589 designs of Tamapin variants using in silico screening, and
have custom-synthesized them. The goal of this STTR Phase I study is to select the lead drug candidate(s)
that demonstrate the best performance in safely improving behavioral deficits in the mouse model of FASD.
from the current 6 candidates. We will focus on in vitro screening of candidates through viability/cytotoxicity
assays and in vitro electrophysiology analysis (Aim 1), and defining the in vivo efficacy of the lead peptides to
improve neurobehavioral deficits in a mouse model of FASD (Aim 2). This research has immense potential in
addressing the major public health issue of neurological deficits associated with FASD through identifying
novel therapeutic peptides that may alleviate these symptoms.Project Narrative
Fetal Alcohol Spectrum Disorder (FASD) results in severe behavioral, intellectual, and developmental deficits;
however, pharmacological treatments for alleviating FASD currently do not exist. We propose to directly treat
the neurobehavioral deficits associated with FASD by developing custom peptides through computational
modeling and validation via neurophysiological and behavioral testing. This work may provide critical solutions
in addressing neurological disabilities associated with FASD.

* Information listed above is at the time of submission. *

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