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Repurposing Research Clinical Trials
HX Refactored 2016 Boston
Dr. Bruce E. Bloom
President and Chief Science Officer
Cures Within Reach
W W W & H?
• Why I am here
• Who we are
• What types/phases of clinical trials
• How repurposing clinical trials arise
• What are our success stories
• What are clinical trial issues/challenges
• What are current/future trials changes
Cures Within Reach is a non-profit that works to catalyze repurposing
research to quickly and affordably improve patient lives. Repurposing
research seeks to create new treatments for serious unmet needs using
existing drugs, devices and nutriceuticals.
We accomplish this through collaborations that connect researchers and
funding, by facilitating crowd sourcing, building community and by
identifying alternative financing models and incentives.
Repurposing vs. de novo research
 Over 40 Academic research
institution partners
 23 projects currently in
process
 Focus on late pre-clinical or
pilot clinical proof of concept
 13 CWR repurposed
treatments in use by patients
today.
Phases of clinical trials
•Phase I-safety
•Phase II-controlled population efficacy
•Phase III-wider population efficacy
•Phase IV-post marketing surveillance
Types of Trials
Origin of Repurposing Clinical Trials
•Discovery about a disease
•Discovery about a therapy
•Clinical observation/co-morbidity
•Off-label use/patient insight
•Systematic screening
Generic
Drug
DevicesChange in
Therapy
Nutriceuticals
Successful CWR repurposing clinical trials
Generic drug saves
lives of kids with rare
auto-immune disease
6 patients clinical
trial delivers life-
saving treatment
Total $250K
3 years
Endpoints Always
Matter
Size DOESN’TAlways
Matter
Led to 5 additional
clinical trials repurposing
same drug
Change in therapy
improves success of
ASCT by 40% in rare
blood cancers
Could affect 600,000 patients
worldwide each year
Total $600K grant over
5 years
Clinical Trials
to Validate
Clinical Observations
Serendipity plus curiosity
plus persistence
Started with NHL-
Then applied to MM
and other blood cancers
$1M 7 years
6 repurposed
nutriceuticals create
100% protein levels
Supplement combo
“cures”
Familial Dysautonomia
Nature’s
“drugs”
The fastest,
cheapest way
to a new
treatment
No clinical trial
required!
Laser device repurposed for
prostate cancer treatment
eliminates side effects
$50K CWR pilot $500K NIH grant
for Phase III trial. Device moving
into SOC at major institutions
Device+PT
restores lost
function-MS
$50K CWR pilot
$4M for 510K
FDA approval
Don’t
Overlook
Devices
Faster path to
commercialization
Current CWR repurposing clinical trials
Phase III clinical
trial repurposing
BCG
Common
disease
Rare disease
repurposing
with CIHR
Cross border
co-PIs
Generics
Repurposing
England
New SIB economic
engine-pay for
success model
Open Source
Pharma
Foundation
Repurposing for
neglected disease
of poverty
Promoting
Repurposing
Collaboration
Created by
Cures Within
Reach
Launched in
June 2015
Platform built
with a grant
from the Robert
Wood Johnson
Foundation
The world’s first online,
interactive platform dedicated
to repurposing research,
testing new treatments for
unsolved diseases using
existing drugs and devices
Since
November 2015
Over
120 projects
submitted
From
52 Institutions
In
7 Countries
Oncology-45 Neuro-20
Rare diseases-23 Infectious-6
Autoimmune-6 Pediatric-5
Hematology-4 Cardiology 4
Ophthalmology-3 Pulmonary-2
GI-1 Nephrology-1
Multiple Stakeholder Participation
Future of repurposing clinical trials
•Virtual clinical research
•“Big Data” retrospective studies
•Prospective observational studies
•Clinical trial crowd research-tech driven
Join us
1. Register for CureAccelerator™
https://app.cureaccelerator.org/registration
1. Log in and start a digital discussion, post an RFP or post a
project
1. Encourage your research institution, biotech or disease
specific non-profit to become a CureAccelerator partner
To learn more
Dr. Bruce Bloom
Bruce@cureswithinreach.org
847-745-1245
www.cureswithinreach.org
www.cureaccelerator.org
Cures Within Reach is a 501(c)3 non-
profit organization, tax ID 20-3620169

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Repurposing Clinical Trials for New Treatments

  • 1. Repurposing Research Clinical Trials HX Refactored 2016 Boston Dr. Bruce E. Bloom President and Chief Science Officer Cures Within Reach
  • 2. W W W & H? • Why I am here • Who we are • What types/phases of clinical trials • How repurposing clinical trials arise • What are our success stories • What are clinical trial issues/challenges • What are current/future trials changes
  • 3. Cures Within Reach is a non-profit that works to catalyze repurposing research to quickly and affordably improve patient lives. Repurposing research seeks to create new treatments for serious unmet needs using existing drugs, devices and nutriceuticals. We accomplish this through collaborations that connect researchers and funding, by facilitating crowd sourcing, building community and by identifying alternative financing models and incentives.
  • 4. Repurposing vs. de novo research
  • 5.  Over 40 Academic research institution partners  23 projects currently in process  Focus on late pre-clinical or pilot clinical proof of concept  13 CWR repurposed treatments in use by patients today.
  • 6. Phases of clinical trials •Phase I-safety •Phase II-controlled population efficacy •Phase III-wider population efficacy •Phase IV-post marketing surveillance
  • 8.
  • 9. Origin of Repurposing Clinical Trials •Discovery about a disease •Discovery about a therapy •Clinical observation/co-morbidity •Off-label use/patient insight •Systematic screening
  • 11. Generic drug saves lives of kids with rare auto-immune disease 6 patients clinical trial delivers life- saving treatment Total $250K 3 years Endpoints Always Matter Size DOESN’TAlways Matter Led to 5 additional clinical trials repurposing same drug
  • 12. Change in therapy improves success of ASCT by 40% in rare blood cancers Could affect 600,000 patients worldwide each year Total $600K grant over 5 years Clinical Trials to Validate Clinical Observations Serendipity plus curiosity plus persistence Started with NHL- Then applied to MM and other blood cancers
  • 13. $1M 7 years 6 repurposed nutriceuticals create 100% protein levels Supplement combo “cures” Familial Dysautonomia Nature’s “drugs” The fastest, cheapest way to a new treatment No clinical trial required!
  • 14. Laser device repurposed for prostate cancer treatment eliminates side effects $50K CWR pilot $500K NIH grant for Phase III trial. Device moving into SOC at major institutions Device+PT restores lost function-MS $50K CWR pilot $4M for 510K FDA approval Don’t Overlook Devices Faster path to commercialization
  • 15. Current CWR repurposing clinical trials Phase III clinical trial repurposing BCG Common disease Rare disease repurposing with CIHR Cross border co-PIs Generics Repurposing England New SIB economic engine-pay for success model Open Source Pharma Foundation Repurposing for neglected disease of poverty
  • 17. Created by Cures Within Reach Launched in June 2015 Platform built with a grant from the Robert Wood Johnson Foundation The world’s first online, interactive platform dedicated to repurposing research, testing new treatments for unsolved diseases using existing drugs and devices
  • 18. Since November 2015 Over 120 projects submitted From 52 Institutions In 7 Countries Oncology-45 Neuro-20 Rare diseases-23 Infectious-6 Autoimmune-6 Pediatric-5 Hematology-4 Cardiology 4 Ophthalmology-3 Pulmonary-2 GI-1 Nephrology-1
  • 20.
  • 21. Future of repurposing clinical trials •Virtual clinical research •“Big Data” retrospective studies •Prospective observational studies •Clinical trial crowd research-tech driven
  • 22. Join us 1. Register for CureAccelerator™ https://app.cureaccelerator.org/registration 1. Log in and start a digital discussion, post an RFP or post a project 1. Encourage your research institution, biotech or disease specific non-profit to become a CureAccelerator partner
  • 23. To learn more Dr. Bruce Bloom Bruce@cureswithinreach.org 847-745-1245 www.cureswithinreach.org www.cureaccelerator.org Cures Within Reach is a 501(c)3 non- profit organization, tax ID 20-3620169