View
212
Download
0
Category
Tags:
Preview:
Citation preview
Stem cells and gene therapy to repair the damaged heart: will it work and is it
right?
John Martin
Professor of Cardiovascular Medicine
University College London
Small molecules
Industry funded
Heavily regulated
Mechanisms understood
Extensive toxicology performed
Intellectual property obvious
Industry total control
Gene therapy
Venture capital funded
Heavily regulated
Mechanism understood
Very extensive toxicology
Intellectual property less obvious but possible
Control shared - biotech/academia
Stem cell therapy
Difficult to fund
Less regulated
Mechanisms not understood
Little toxicology
Intellectual property difficult
Academia can control
Gene therapy trials failed
1. because of lack of understandingof basic biological problems
2. lack of safety
3. and small, uncontrolled trials
TRINAM:
EMEA orphan drug status
FDA orphan drug status and fast track status
Ark Therapeutics
October 2005Patient Safety andDose Escalation studyLow dosesuccessfully completed
Gene Therapy
Problems
1. Gene can disseminateto whole body
2. Can we get enoughwhere we want it?
Solution
1. Local gene therapy
2. Use a reservoir
Embryonic
stem cells
Bone marrow
stem cells
Resident stem cells
The whole body
Specialised tissue
(e.g. heart, blood vessels)
Repair of the organ
(e.g. in heart, repairs heart)
• Use of autologous cells in large randomised control trials in patients with:– Acute myocardial infarction– Late presentation myocardial infarction– Heart failure (both ischaemic and dilated)
• Use of autologous cells in small clinical mechanistic studies
• Studies to test use of cytokines
Stem Cell Research Plan at Barts and UCL
3 protocols approved by ethics committee
Ischaemic Heart Failure
Dilated Cardiomyopathy
Acute myocardial infarction
(Total recruitment of 700 patients)
Recommended