hxr 2016: to heal and to cure: digital health experiences
TRANSCRIPT
Repurposing Research Clinical Trials
HX Refactored 2016 Boston
Dr. Bruce E. BloomPresident 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
Off label Open labelRandomized
Blinded
Controlled
Blinded
Types of Trials
Controlled
Crossover
PlaceboNatural History v test
SOC v testSOC v SOC + test
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 $250K3 years
Endpoints Always Matter
Size DOESN’T Always Matter
Led to 5 additional clinical trials repurposing
same drug
Change in therapy improves success of ASCT by 40% in rare
blood cancersCould affect 600,000 patients
worldwide each year
Total $600K grant over5 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 RepurposingCollaboration
Created by Cures Within
Reach
Launched inJune 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
In7 Countries
11 RFPs
Oncology-45 Neuro-20Rare diseases-23 Infectious-6 Autoimmune-6 Pediatric-5 Hematology-4 Cardiology 4Ophthalmology-3 Pulmonary-2GI-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
2. Log in and start a digital discussion, post an RFP or post a project
3. Encourage your research institution, biotech or disease specific non-profit to become a CureAccelerator partner
To learn more
Dr. Bruce Bloom [email protected] 847-745-1245
www.cureswithinreach.org
www.cureaccelerator.org
Cures Within Reach is a 501(c)3 non-profit organization, tax ID 20-3620169