1. Building a Drug Repurposing Network (DR3N) for the CTSA Consortium:
Inspiration: Ovarian Cancer Collaboration
Drug Repurposing: What and Why
Harnessing The Power of Collaboration
DR3N: The Three R’s
Drug Rescue for stalled Pharma products
Drug Repurposing based on experimental or computational evidence
Drugs for Repositioning based on off-label indications
Clinical & Translational Science Center
CTSC

2. Inspiration: Ovarian Cancer Drug Repurposing
Concept: GTPases/Receptor Traffic Screen: Sklar Multiplex/NSAIDS Cheminformatics: Oprea Ketorolac Retrospective Trial: Cook Pain Treatment/Survival Cells/Animals: Mechanism (R-enantiomer) Prospective Trial: Muller Hits Target Outcomes: Pubs, Patents, Trials, $
Wandinger-Ness Hudson

3. Approved drugs and devices
WHY REPURPOSING?
> 6,000+
Rare diseases
> 100,000,000
people
1,000s
Approved drugs and devices 1 2 2
Success rate of 10-30% demonstrating clinical benefit for patients based on Cures Within Reach past success rate (13 successes from 50 clinical trials). Success considered when clinical results proved the repurposed therapy works and are robust enough for physicians to use to prescribe off label OR when a project moves to a phase II/III trial on the way to commercialization.
There is other support for this success rate. Repurposing generic drugs is much akin to moving a NME from phase II to phase III, since both are already proven safe for human use and have some clinical anecdotal success. Estimates of success rates for molecules moving from phase II to phase III range from 13%-32%.
See this from the Institute of Medicine:
“Drug repositioning or repurposing holds many appeals. Typically these drugs have been extensively profiled and
studied in terms of safety, efficacy and toxicity, thereby decreasing the amount of preclinical research required.
This streamlined process saves time and money, with repurposed drugs generally being approved in much
shorter timelines (3-12 years) and at about 60% of the cost of typical development. While approximately 10% of
new drug applications gain market approval, repurposed drugs approach approval rates near 30%, presenting a
significant market-driven incentive for companies that also aligns with patient desires for new therapeutics.”
Cost <$500,000 based on Cures Within Reach experience in clinical trials so far. And CWR repurposing clinical trials average is under $100,000 1= 2=

4. Network Hubs/Sites: Partners & Expertise
Leaders
Expertise
Specialization
Notable
UNM CMD
Sklar
High throughput flow cytometry; Suspension cell and molecular targets
Multiplexed and multiparameter analysis
BSL2/BSL3 access
Repurposing trials
UNM TID
Oprea
Drug discovery analytics; Target Prioritization for
Illuminating the Druggable Genome (IDG)
Databases:
DrugsDB.com
TargetCentral.ws
IDG Knowledge Management Center
KUMC/ IAMI
Weir
Pharmaceutics; GMP, formulation, drug delivery
Public-Private Partnerships /
Learning Experiences
Commercialization
UNC
Roth
Genome-wide GPCR-ome testing & de-orphanization; NIMH Psychoactive Drug Screening Program Site
Bioactives/natural products; hERG/ ion channels; efflux transporters
Drug limitations and removal for liabilities
NCATS
Simeonov
Discovery
Drug Libraries
Drug Combinations
Initiatives for drug repurposing, rare/ neglected diseases
Cures Within
Reach
Bloom
Fund raising for trials
CureAccelerator website
Economic Incentives
Overcoming Philanthropic and Commercial Barriers
Pre-competitive consortia
Impact Bonds
Sklar Oprea
Weir Roth
Simeonov Bloom

5. DR3N: A CTSA Resource to Speed and Facilitate New FIRST-IN-HUMAN STUDIES