NIMH i PSC Assays for the Drug Pipeline - Panchision

Dr David Panchision's live presentation at the Schizophrenia Research Forum's live webinar of June 28, 2017 - http://www.schizophreniaforum.org/forums/webinar-modeling-neuropsychiatric-disorders-using-vitro-models

1. Assay Development as a Starting Point for
Biological and Therapeutics Discovery
1
Target
ID
Assay
Development
HTS Ph. I Ph. IIIHit to Lead Ph. II Ph. IV
Lead
Optimization
Disease
Target
Pre-
Clinical
Construct validity &
disease mechanism:
▼
Co-culture systems
Organoids, spheroids
Chimeras
Screening and confirmation
reporter/sensor assays:
▼
Simple monolayers
Organoids, spheroids
Other 3D systems
PD/PK, Toxicity,
Efficacy, Virtual
Trials:
▼
Tissue chips
Chimeras
•NIMH supports research to develop human cell-based assays as an alternative to animal-
based assays that have an uneven record of predictive value.
•Many 2-& 3-dimensional human cell-based assays have potential utility for different stages
in the molecular therapeutics development pipeline (examples are for illustration only).

2. NIMH Funding Initiatives: iPSCsto Assays
for Therapeutics Discovery
2
Target
ID
Assay
Development
HTS Ph. I Ph. IIIHit to Lead Ph. II Ph. IV
Lead
Optimization
Disease
Target
Pre-
Clinical
Construct validity &
disease mechanism:
▼
Co-culture systems
Organoids, spheroids
Chimeras
Screening and confirmation
reporter/sensor assays:
▼
Simple monolayers
Organoids, spheroids
Other 3D systems
PD/PK, Toxicity,
Efficacy, Virtual
Trials:
▼
Tissue chips
Chimeras
Complex Brain
Disorders
PAR-17-309 (R01), PAR-17-310 (R21)
NationalCooperative Reprogrammed
Cell Research Groups (NCRCRG)
Next-Gen
MPS
Neurobiology to Assays
PAR-15-066 (R01)
N-G
MPS
Engineering Next Generation Microphysiological
Systems PAR-16-398 (R01), PAR-16-397 (R21)

3. NIMH Repository: Resource for the Research Community
3
Biospecimen repository at
Rutgers University Cell & DNA
Repository (RUCDR) with
clinical/genetic data housed at
Washington Univ. in St. Louis
(since 1998)
https://www.nimhgenetics.org/
Stem Cell Center at RUCDR
is newest part of Repository
(since 2011)
http://nimhstemcells.org/