Personalized medicine could soon be reality
Updated: Tue Jan. 31 2012 19:10:38
ctvwinnipeg.ca
The federal health minister says the government would like to move away from "one size fits all" medicine.
An announcement was made in Ottawa on Tuesday that more than $67 million is being invested into cutting edge research to identify markers of disease.
The aim is to get doctors to understand a person's body through their genes, family background and environment, then tailor a treatment just for them.
"With these bio markers, doctors will be able to tailor treatments based on what we know about the patient being treated," said Leona Aglukkaq, Health Minister. "this holds the potential to make many medical treatments more effective."
Some Manitobans feel it's money well spent, like Carey Tarr who has type 1 diabetes. She says she already recieves some treatment personalized for her, but says more can be done for others.
"Someone living with type one diabetes or any type diabetes may also be living with other conditions, " explained Tarr, "So there's a lot of room to look at how their body makeup and different conditions they are living with are affecting others as well."
It's believed personalized approaches will be effective in many areas including cancer.
CancerCare Manitoba says this initiative is a small step in the right direction.
"This is an initiatve that will begin to improve the specificity of our care so we aren't using a shot gun approach as we are sometimes forced to because we have no other way," said Dr. Dhali Dhaliwal, of CancerCare Manitoba.
Dr Dhaliwal says it could take at least five years before patients will see the benifits of this research.
The government says the funding is a research competition supported by three research groups, who have to match the amount of money they receive.
It's not known at this time what diseases the groups will be conducting research on.
-- with a report from CTV's Ina Sidhu
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Personalized medicine could soon be reality
Recommendation and review posted by sam
“Russ Bennett”, “Project Walk Spinal Cord Injury Recovery” – Video
25-01-2012 18:17 Spinal cord injury treatment. http://www.projectwalk.org exists to provide an improved quality of life for people with spinal cord injuries through intense exercise-based recovery programs, education, support and encouragement.
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"Russ Bennett", "Project Walk Spinal Cord Injury Recovery" - Video
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An Overview of Data Trends in Autologous Stem Cell Research and Clinical Use – James P. Watson, MD – Video
31-01-2012 13:32 James P. Watson, MD lecture sample from the 11th Clinical Applications for Age Management Medicine Conference, Fall 2011, Las Vegas, Nevada Pre-Conference Track 2: Regenerative and Cell Based Medicine This lecture focused on regenerative and cell-based medicine, Autologous Stem Cell Research. This field continues to grow in use by physicians across the world. From platelet rich plasma to culture expanded stem cells, the need for information about the applications of these therapies to treat patients has never been greater. This track will focus on the latest developments in cell-based medicine with speakers who are driving the research and using these technologies as part of their everyday practice of medicine. For more information about our upcoming conference visit our website http://www.agemed.org Or contact us at conference@agemed.org
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An Overview of Data Trends in Autologous Stem Cell Research and Clinical Use - James P. Watson, MD - Video
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Cardiovascular Drug Delivery – technologies,markets and companies
NEW YORK, Feb. 1, 2012 /PRNewswire/ -- Reportlinker.com announces that a new market research report is available in its catalogue:
Cardiovascular Drug Delivery - technologies,markets and companies
Drug delivery to the cardiovascular system is different from delivery to other systems because of the anatomy and physiology of the vascular system; it supplies blood and nutrients to all organs of the body. Drugs can be introduced into the vascular system for systemic effects or targeted to an organ via the regional blood supply. In addition to the usual formulations of drugs such as controlled release, devices are used as well. This report starts with an introduction to molecular cardiology and discusses its relationship to biotechnology and drug delivery systems.
Drug delivery to the cardiovascular system is approached at three levels: (1) routes of drug delivery; (2) formulations; and finally (3) applications to various diseases. Formulations for drug delivery to the cardiovascular system range from controlled release preparations to delivery of proteins and peptides. Cell and gene therapies, including antisense and RNA interference, are described in full chapters as they are the most innovative methods of delivery of therapeutics. Various methods of improving systemic administration of drugs for cardiovascular disorders are described including use of nanotechnology.
Cell-selective targeted drug delivery has emerged as one of the most significant areas of biomedical engineering research, to optimize the therapeutic efficacy of a drug by strictly localizing its pharmacological activity to a pathophysiologically relevant tissue system. These concepts have been applied to targeted drug delivery to the cardiovascular system. Devices for drug delivery to the cardiovascular system are also described.
Role of drug delivery in various cardiovascular disorders such as myocardial ischemia, hypertension and hypercholesterolemia is discussed. Cardioprotection is also discussed. Some of the preparations and technologies are also applicable to peripheral arterial diseases. Controlled release systems are based on chronopharmacology, which deals with the effects of circadian biological rhythms on drug actions.A full chapter is devoted to drug-eluting stents as treatment for restenosis following stenting of coronary arteries.Fifteen companies are involved in drug-eluting stents.
New cell-based therapeutic strategies are being developed in response to the shortcomings of available treatments for heart disease. Potential repair by cell grafting or mobilizing endogenous cells holds particular attraction in heart disease, where the meager capacity for cardiomyocyte proliferation likely contributes to the irreversibility of heart failure. Cell therapy approaches include attempts to reinitiate cardiomyocyte proliferation in the adult, conversion of fibroblasts to contractile myocytes, conversion of bone marrow stem cells into cardiomyocytes, and transplantation of myocytes or other cells into injured myocardium.
Advances in molecular pathophysiology of cardiovascular diseases have brought gene therapy within the realm of possibility as a novel approach to treatment of these diseases. It is hoped that gene therapy will be less expensive and affordable because the techniques involved are simpler than those involved in cardiac bypass surgery, heart transplantation and stent implantation. Gene therapy would be a more physiologic approach to deliver vasoprotective molecules to the site of vascular lesion. Gene therapy is not only a sophisticated method of drug delivery; it may at time need drug delivery devices such as catheters for transfer of genes to various parts of the cardiovascular system.
The cardiovascular drug delivery markets are estimated for the years 2011 to 2021 on the basis of epidemiology and total markets for cardiovascular therapeutics. The estimates take into consideration the anticipated advances and availability of various technologies, particularly drug delivery devices in the future. Markets for drug-eluting stents are calculated separately. Role of drug delivery in developing cardiovascular markets is defined and unmet needs in cardiovascular drug delivery technologies are identified.
Selected 80 companies that either develop technologies for drug delivery to the cardiovascular system or products using these technologies are profiled and 74 collaborations between companies are tabulated. The bibliography includes 200 selected references from recent literature on this topic. The report is supplemented with 27 tables and 7 figures
TABLE OF CONTENTS
0. Executive Summary 11
1. Cardiovascular Diseases 13
Introduction 13
History of cardiovascular drug delivery 13
Overview of cardiovascular disease 14
Coronary artery disease 14
Angina pectoris 14
Limitations of current therapies for myocardial ischemic disease 14
Cardiomyopathies 14
Cardiac arrhythmias 15
Congestive heart failure 15
Peripheral arterial disease 15
Current management 16
Atherosclerosis 16
The endothelium as a target for cardiovascular therapeutics 16
Molecular cardiology 17
Cardiogenomics 17
Cardioproteomics 17
Personalized cardiology 18
Pharmacogenomics of cardiovascular disorders 18
Modifying the genetic risk for myocardial infarction 19
Management of heart failure 19
Management of hypertension 20
Pharmacogenomics of diuretic drugs 20
Pharmacogenomics of ACE inhibitors 20
Management of hypertension by personalized approach 21
Pharmacogenetics of lipid-lowering therapies 21
Polymorphisms in genes involved in cholesterol metabolism 21
Role of eNOS gene polymorphisms 22
Important advances in cardiovascular therapeutics 22
Drug delivery, biotechnology and the cardiovascular system 23
Role of cardiovascular imaging in cardiovascular therapeutics 23
Chronopharmacotherapy of cardiovascular diseases 23
2. Methods for Drug Delivery to the Cardiovascular System 25
Introduction 25
Routes of drug delivery to the cardiovascular system 25
Local administration of drugs to the cardiovascular system 25
Intramyocardial drug delivery 25
Drug delivery via coronary venous system 26
Intrapericardial drug delivery 26
Formulations for drug delivery to the cardiovascular system 27
Sustained and controlled release 27
Programming the release at a defined time 28
Dosage formulation of calcium channel blockers 28
Sustained and controlled release verapamil 28
Methods of administration of proteins and peptides 28
Delivery of peptides by subcutaneous injection 29
Depot formulations and implants 29
Poly(ethylene glycol) technology 29
Liposomes for cardiovascular drug delivery 30
Microencapsulation for protein delivery 30
Localized delivery of biomaterials for tissue engineering 30
Oral delivery of proteins and peptides 30
DDS to improve systemic delivery of cardiovascular drugs 32
Nanotechnology-based drug delivery 32
Controlled delivery of nanoparticles to injured vasculature 33
Nanoparticles for cardiovascular imaging and targeted drug delivery 34
Nanofiber-based scaffolds with drug-release properties 34
Targeted drug delivery to the cardiovascular system 35
Immunotargeting of liposomes to activated vascular endothelial cells 35
PEGylated biodegradable particles targeted to inflamed endothelium 36
Devices for cardiovascular drug delivery 36
Local drug delivery by catheters 37
Microneedle for periarterial injection 38
Nanotechnology-based devices for the cardiovascular system 39
Liposomal nanodevices for targeted cardiovascular drug delivery 39
Nanotechnology approach to the problem of "vulnerable plaque" 40
Drug delivery in the management of cardiovascular disease 40
Drug delivery in the management of hypertension 40
Transnasal drug delivery for hypertension 41
Transdermal drug delivery for hypertension 41
Oral extended and controlled release preparations for hypertension 42
Long-acting hypertensives for 24 h blood pressure control 43
Drug delivery to control early morning blood pressure peak 43
Role of drug delivery in improving compliance with antihypertensive therapy 44
Drug delivery for congestive heart failure 44
Oral human brain-type natriuretic peptide 44
Nitric oxide-based therapies for congestive heart failure 44
Automated drug delivery system for cardiac failure 45
DDS in the management of ischemic heart disease 45
Intravenous emulsified formulations of halogenated anesthetics 46
Injectable peptide nanofibers for myocardial ischemia 46
Delivery of angiogenesis-inducing agents for myocardial ischemia 47
Drug delivery for cardioprotection 47
Drug delivery for cardiac rhythm disorders 48
Drug delivery in the treatment of angina pectoris 49
Sustained and controlled-release nitrate for angina pectoris 49
Transdermal nitrate therapy 49
Controlled release calcium blockers for angina pectoris 51
Vaccines for hypertension 51
Drug delivery in the management of pulmonary hypertension 51
Prostacyclin by inhalation 52
Endothelin receptor antagonist treatment of PAH 52
Anticoagulation in cardiovascular disease 52
Oral heparin 52
Low molecular weight heparin-loaded polymeric nanoparticles 53
Transdermal anticoagulants 53
Thrombolysis for cardiovascular disorders 53
Use of ultrasound to facilitate thrombolysis 54
Delivery of alteplase through the AngioJet rheolytic catheter 54
Drug delivery for peripheral arterial disease 54
Delivery of thrombolytic agent to the clot through a catheter 55
Delivery of growth factors to promote angiogenesis in ischemic limbs 55
Immune modulation therapy for PAD 55
NO-based therapies for peripheral arterial disease 55
Drug delivery in the management of hypercholesterolemia 56
Controlled/sustained release formulations of statins 56
Combinations of statins with other drugs to increase efficacy 56
Controlled release fenofibrate 57
Extended release nicotinic acid 58
Intravenous infusion of lipoprotein preparations to raise HDL 59
Innovative approaches to hypercholesterolemia 59
Single dose therapy for more than one cardiovascular disorder 59
3. Cell Therapy for Cardiovascular Disorders 61
Introduction 61
Inducing the proliferation of cardiomyocytes 61
Role of stem cells in repair of the heart 61
Cell-mediated immune modulation for chronic heart disease 61
Cell therapy for atherosclerotic coronary artery disease 62
Transplantation of myoblasts for myocardial infarction 62
MyoCell™ (Bioheart) 63
Transplantation of cardiac progenitor cells for revascularization of myocardium 64
Methods of delivery of cells to the heart 64
Cellular cardiomyoplasty 64
IGF-1 delivery by nanofibers to improve cell therapy for MI 65
Intracoronary infusion of bone marrow-derived cells for AMI 65
Non-invasive delivery of cells to the heart by Morph®guide catheter 65
Transplantation of stem cells for myocardial infarction 66
Transplantation of embryonic stem cells 66
Transplantation of hematopoietic stem cells 66
Transplantation of cord blood stem cells for myocardial infarction 66
Intracoronary infusion of mobilized peripheral blood stem cells 67
Human mesenchymal stem cells for cardiac regeneration 67
Cytokine preconditioning of human fetal liver CD133+ SCs 68
Transplantation of expanded adult SCs derived from the heart 68
Transplantation of endothelial cells 68
Transplantation of genetically modified cells 69
Transplantation of cells secreting vascular endothelial growth factor 69
Transplantation of genetically modified bone marrow stem cells 69
Cell transplantation for congestive heart failure 69
Injection of adult stem cells for congestive heart failure 69
Intracoronary infusion of cardiac stem cells 70
Myoblasts for treatment of congestive heart failure 70
Role of cell therapy in cardiac arrhythmias 70
Atrioventricular conduction block 71
Ventricular tachycardia 71
ESCs for correction of congenital heart defects 72
Cardiac progenitors cells for treatment of heart disease in children 72
Stem cell therapy for peripheral arterial disease 73
Targeted delivery of endothelial progenitor cells labeled with nanoparticles 73
Clinical trials of cell therapy in cardiovascular disease 73
A critical evaluation of cell therapy for heart disease 75
Publications of clinical trials of cell therapy for CVD 76
Future directions for cell therapy of CVD 76
4. Gene Therapy for Cardiovascular Disorders 79
Introduction 79
Techniques of gene transfer to the cardiovascular system 80
Direct plasmid injection into the myocardium 80
Catheter-based systems for vector delivery 80
Ultrasound microbubbles for cardiovascular gene delivery 81
Vectors for cardiovascular gene therapy 81
Adenoviral vectors for cardiovascular diseases 81
Intravenous rAAV vectors for targeted delivery to the heart 82
Plasmid DNA-based delivery in cardiovascular disorders 82
Hypoxia-regulated gene therapy for myocardial ischemia 82
Angiogenesis and gene therapy of ischemic disorders 83
Therapeutic angiogenesis vs. vascular growth factor therapy 83
Gene painting for delivery of targeted gene therapy to the heart 84
Gene delivery to vascular endothelium 84
Targeted plasmid DNA delivery to the cardiovascular system with nanoparticles 84
Gene delivery by vascular stents 85
Gene therapy for genetic cardiovascular disorders 85
Genetic disorders predisposing to atherosclerosis 85
Familial hypercholesterolemia 86
Apolipoprotein E deficiency 87
Hypertension 87
Genetic factors for myocardial infarction 88
Acquired cardiovascular diseases 88
Coronary artery disease with angina pectoris 88
Ad5FGF-4 88
Ischemic heart disease with myocardial infarction 89
Angiogenesis for cardiovascular disease 89
Myocardial repair with IGF-1 therapy 90
miRNA gene therapy for ischemic heart disease 91
Congestive heart failure 91
Rationale of gene therapy in CHF 91
?-ARKct gene therapy 91
Intracoronary adenovirus-mediated gene therapy for CHF 92
AAV-mediated gene transfer for CHF 92
AngioCell gene therapy for CHF 93
nNOS gene transfer in CHF 93
Gene therapy for cardiac arrhythmias 93
Gene transfer for biological pacemakers 94
Management of arrhythmias due to myoblast transplantation 95
Genetically engineered cells as biological pacemakers 95
Gene therapy and heart transplantation 95
Gene therapy for peripheral arterial disease 96
Angiogenesis by gene therapy 96
HIF-1? gene therapy for peripheral arterial disease 96
HGF gene therapy for peripheral arterial disease 97
Ischemic neuropathy secondary to peripheral arterial disease 97
Maintaining vascular patency after surgery 97
Antisense therapy for cardiovascular disorders 98
Antisense therapy for hypertension 99
Antisense therapy for hypercholesterolemia 99
RNAi for cardiovascular disorders 100
RNAi for hypercholesterolemia 100
microRNA and the cardiovascular system 101
Role of miRNAs in angiogenesis 101
Role of miRNAs in cardiac hypertrophy and failure 101
Role of miRNAs in conduction and rhythm disorders of the heart 102
miRNA-based approach for reduction of hypercholesterolemia 102
miRNAs as therapeutic targets for cardiovascular diseases 103
Future prospects of miRNA in the cardiovascular therapeutics 103
Future prospects of gene therapy of cardiovascular disorders 103
Companies involved in gene therapy of cardiovascular disorders 104
5. Drug-Eluting Stents 107
Introduction 107
Percutaneous transluminal coronary angioplasty 107
Stents 107
Restenosis 107
Pathomechanism 108
Treatment 108
Nitric oxide-based therapies for restenosis 109
Carbon monoxide inhalation for preventing restenosis 109
Antisense approaches for prevention of restenosis after angioplasty 110
miRNA-based approach for restenosis following angioplasty 111
Gene therapy to prevent restenosis after angioplasty 111
Techniques of gene therapy for restenosis 112
NOS gene therapy for restenosis 113
Nonviral gene therapy to prevent intimal hyperplasia 113
HSV-1 gene therapy to prevent intimal hyperplasia 114
Drug delivery devices for restenosis 114
Local drug delivery by catheter 114
Stenosis associated with stents 115
Absorbable metal stents 115
Drug-eluting stents 115
Various types of DES 116
CYPHER® sirolimus-eluting coronary stent 116
Dexamethasone-eluting stents 116
NO-generating stents 117
Paclitaxel-eluting stents 117
Sirolimus-eluting vs paclitaxel-eluting stents 118
Novel technologies for DES 118
Absorbable DES 118
Bio-absorbable low-dose DES 119
Drug-eluting stents coated with polymer surfaces 119
Endeavour DES 119
Stents for delivery of gene therapy 120
Stem cell-based stents 121
VAN 10-4 DES 121
Nanotechnology-based stents 122
Drugs encapsulated in biodegradable nanoparticles 122
Magnetic nanoparticle-coated DES 122
Magnetic nanoparticles encapsulating paclitaxel targeted to stents 123
Nanocoated DES 123
Nanopores to enhance compatibility of DES 124
Paclitaxel-encapsulated targeted lipid-polymeric nanoparticles 124
The ideal DES 124
Companies developing drug-eluting stents 125
Clinical trials of drug-eluting stents 126
Measurements used in clinical trials of DES 126
TAXUS paclitaxel-eluting stents 126
XIENCE™ V everolimus-eluting coronary stent 127
COSTAR II clinical trial 128
Endeavor RESOLUTE zotarolimus-eluting stent system 128
CUSTOM I clinical trial 129
NOBORI CORE Trial 129
LEADERS trial 130
Comparison of DES in clinical trials 130
Comparison of DES with competing technologies 131
DES versus coronary artery bypass graft 131
DES versus bare metal stents 131
Multi-Link Vision bare metal stent vs DES 134
Guidelines for DES vs BMS 134
DES vs BMS for off-label indications 134
Role of DES in cases of bare-metal in-stent restenosis 135
DES versus balloon catheter coated with paclitaxel 135
DES versus intracoronary radiation therapy for recurrent stenosis 135
Cost-effectiveness of DES 136
Safety issues of DES 137
Adverse reactions to DES 137
Endothelial vascular dysfunction due to sirolismus 137
Risk of clotting with DES 137
Clopidogrel use and long-term outcomes of patients receiving DES 139
Prasugrel as antiplatelet agent 139
Effect of blood clot on release of drug from DES 140
Use of magnetized cell lining to prevent clotting of DES 140
Long-term safety studies of DES 140
Regulatory issues of DES 141
Future prospects for treatment of restenosis by DES 143
Future role of DES in management of cardiovascular diseases 143
Stent cost and marketing strategies 144
Improvements in stent technology 144
Bioabsorbale stent 144
DES vs drug-eluting balloons 145
6. Markets for Cardiovascular Drug Delivery 147
Introduction 147
Epidemiology of cardiovascular disease 147
Cost of care of cardiovascular disorders 148
Cardiovascular markets according to important diseases 149
Antithrombotics 149
Anticholesterol agents 149
Antihypertensive agents 150
Drugs for congestive heart failure 150
Markets for innovative technologies for cardiovascular disorders 150
Markets for cell therapy of cardiovascular disorders 150
Markets for gene therapy of cardiovascular disorders 151
Markets for drug-eluting stents 151
Major players in DES market 151
Impact of safety issues on future markets for DES 151
DES market in Asia 152
Patenting and legal issues of DES 153
The financial impact of DES on cardiovascular markets 153
Unmet needs for cardiovascular drug delivery 154
Role of DDS in developing cardiovascular markets 155
Markets for cardiovascular devices 155
Marketing of innovative cardiovascular drug delivery devices 155
Direct to consumer advertising of DES 156
Future trends in the integration of drug delivery with therapeutics 156
Future prospects of cardiovascular drug delivery 156
7. Companies involved in Cardiovascular Drug Delivery 157
Profiles of companies 157
Collaborations 243
8. References 247
List of Tables
Table 1 1: Landmarks in the historical evolution of cardiovascular drug delivery 13Table 1 2: Gene polymorphisms that alter cardiovascular response to drugs 18Table 2 1: Routes of drug delivery used for treatment of cardiovascular disorders 25Table 2 2: Formulations for drug delivery to the cardiovascular system 27Table 2 3: Improved methods of systemic drug delivery of cardiovascular drugs 32Table 2 4: Targeted delivery of therapeutic substances to the cardiovascular system 35Table 2 5: Classification of devices for drug delivery to the cardiovascular system 36Table 2 6: Various methods of delivery of therapeutic agents for hypertension 41Table 2 7: Marketed controlled/ extended release preparation for hypertension 43Table 2 8: Drug delivery in ischemic heart disease 45Table 2 9: Methods of delivery of nitrate therapy in angina pectoris 49Table 2 10: Drug delivery for peripheral arterial disorders 54Table 3 1: Clinical trials of cell therapy in cardiovascular disease 73Table 4 1: Cardiovascular disorders for which gene therapy is being considered. 79Table 4 2: Catheter-based systems for vector delivery to the cardiovascular system 80Table 4 3: Potential applications of antisense in cardiovascular disorders 99Table 4 4: Companies involved in gene therapy of cardiovascular diseases 104Table 5 1: Treatment of restenosis 108Table 5 2: Devices used for drug delivery in restenosis 114Table 5 3: Companies involved in drug-eluting stents 125Table 6 1: Prevalence of cardiovascular disorders in major markets: US 2011-2021 148Table 6 2: Prevalence of cardiovascular disorders in major markets: Europe 2011-2021 148Table 6 3: Prevalence of cardiovascular disorders in major markets: Japan 2011-2021 148Table 6 4: Values of cardiovascular DDS in major markets 2011-2021 149Table 6 5: Markets for innovative technologies for cardiovascular disorders 2011-2021 150Table 7 1: Top 5 companies in cardiovascular drug delivery 157Table 7 2: Collaborations in cardiovascular drug delivery 243
List of Figures
Figure 1 1: Drug delivery, biotechnology and cardiovascular diseases 23
Figure 2 1: MicroSyringe for periarterial injection 39
Figure 5 1: Vicious circle of vascular occlusion following angioplasty and stenting 109
Figure 5 2: Measurement of in-stent stenosis 115
Figure 5 3: Medtronic's Endeavour drug-eluting stent 120
Figure 5 4: Magnetic nanoparticle-coated stent 123
Figure 6 1: Unmet needs for cardiovascular drug delivery 154
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Cardiovascular Drug Delivery - technologies,markets and companies
Recommendation and review posted by Bethany Smith
Some nerve! Now bypass stem cells
Washington, Feb 1 (IANS) Scientists have successfully converted mouse skin cells directly into cells that become the three main parts of the nervous system, bypassing the stem cell stage, throwing up many new possibilities in the medical world.
This new study is a substantial advance over the previous paper in that it transforms the skin cells into neural precursor cells, as opposed to neurons.
While neural precursor cells can differentiate into neurons, they can also become the two other main cell types in the nervous system: astrocytes and oligodendrocytes.
The finding is an extension of a previous study by the same group from the Stanford University School of Medicine, showing that mouse and human skin cells can be turned into functional neurons or brain cells.
The multiple successes of the direct conversion method overrides the idea that pluripotency (the ability of stem cells to become nearly any cell) is necessary for a cell to transform from one type to another, the journal Proceedings of the National Academy of Sciences reports.
"We are thrilled about the prospects for potential medical use of these cells," said Marius Wernig, study co-author and assistant professor of pathology and member, Stanford's Institute for Stem Cell Biology and Regenerative Medicine, according to a Stanford statement.
Beside their greater versatility, the newly derived neural precursor cells offer another advantage over neurons because they can be cultivated in large numbers in the lab, a feature critical for their long-term usefulness in transplantation or drug screening.
"We've shown the cells can integrate into a mouse brain and produce a missing protein important for the conduction of electrical signal by the neurons," said Wernig, who co-authored the study with graduate student Ernesto Lujan.
-Indo-Asian News Service
st/pg/vm
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Some nerve! Now bypass stem cells
Recommendation and review posted by Bethany Smith
Clinical Trial for Myelofibrosis that Targets Cancer Stem Cells | CIRM Spotlight on Genomics – Video
24-01-2012 18:39 Four minute excerpt from the Spotlight on Genomics seminar presentation during the January 17th 2012 California Institute for Regnerative Medicine governing board meeting. The video features a conversation between Catriona Jamieson, director for stem cell research at UCSD Moores Cancer Center, and one of her patients who is participating in a clinical trial for the treatment of myelofibrosis, a life-threatening blood disorder.
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Clinical Trial for Myelofibrosis that Targets Cancer Stem Cells | CIRM Spotlight on Genomics - Video
Recommendation and review posted by simmons
The Use of Guided Bone Marrow Nucleated Cell Fraction Injections – Ronald W. Hanson, Jr., MD – Video
31-01-2012 13:21 Ronald W. Hanson, Jr., MD lectures at the 11th Clinical Applications for Age Management Medicine in November 2011, in Las Vegas, Nevada This focused conference track cocentrated on regenerative and cell-based medicine continue to grow in use by physicians across the world. From platelet rich plasma to culture expanded stem cells, the need for information about the applications of these therapies to treat patients has never been greater. This track will focus on the latest developments in cell-based medicine with speakers who are driving the research and using these technologies as part of their everyday practice of medicine. For more information contact conference@agemed.org Visit our website at agemed.org
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The Use of Guided Bone Marrow Nucleated Cell Fraction Injections - Ronald W. Hanson, Jr., MD - Video
Recommendation and review posted by simmons
‘Personalized medicine’ gets $67.5M research boost
The federal government is pledging up to $67.5 million for research into "personalized medicine," which tailors treatment to a patient's genetics and environment.
The funds will flow through Genome Canada, the Cancer Stem Cell Consortium and the Canadian Institutes of Health Research, the federal government's health research agency.
Federal Health Minister Leona Aglukkaq and Minister of State for Science Gary Goodyear made the announcement at the University of Ottawa's health campus Tuesday.
The field of personalized medicine is touted as having the potential to transform the way patients are treated. It looks at the genetic makeup of a person, the patient's environment and the exact course of a particular disease so that an appropriate and effective treatment can be tailored for that individual.
The idea is to move from a one-size-fits-all approach to one that is designed for a specific person and relies on the genetic signatures, or biomarkers, of both the patient and the disease.
Proponents of personalized medicine say it is likely to change the way drugs are developed, how medicines are prescribed and generally how illnesses are managed. They say it will shift the focus in health care from reaction to prevention, improve health outcomes, make drugs safer and mean fewer adverse drug reactions, and reduce costs to health-care systems.
"The potential to understand a person's genetic makeup and the specific character of their illness in order to best determine their treatment will significantly improve the quality of life for patients and their families and may show us the way to an improved health-care system and even save costs in certain circumstances," Aglukkaq said in a news release.
Research projects could last four years
The sequencing of the human genome paved the way for personalized medicine and there have been calls for more research funding so that the discoveries in laboratories can be translated further into the medical field so they will benefit patients more.
Identifying a person's genetic profile, for example, could then indicate a susceptibility to a certain disease, if the biomarkers of that disease have also been discovered. If people know they are genetically at risk of an illness they can take actions to prevent it, and their health-care providers can monitor for it.
Cancer patients could be pre-screened to determine if chemotherapy would work for them, which could not only save a lot of money on expensive treatments but also prevent pain and suffering for patients.
Genome Canada is leading the research initiative, in collaboration with Cancer Stem Cell Consortium and CIHR which on Tuesday launched its Personalized Medicine Signature Initiative. CIHR is committing up to $22.5 million to the large-scale initiative with the other two partners, but it will be providing more funding for other projects under its personalized medicine program.
The research projects are aiming to bring together biomedical, clinical, population health, health economics, ethics and policy researchers to identify areas that are best suited to personalized medicine.
Oncology, cardiovascular diseases, neurodegenerative diseases, psychiatric disorders, diabetes and obesity, arthritis, pain, and Alzheimer’s disease are all considered to be areas that hold promise for personalized medicine.
Funding will also go to projects that are aimed at developing more evidence-based and cost-effective approaches to health care.
Researchers can get up to four years of funding, but 50 per cent of their requested funding must be matched from another source, such as a provincial government or from the academic or private sectors.
Genome Canada, CIHR and the cancer consortium will invest a maximum of $5 million in each individual project.
The successful applicants for the $67.5 million worth of funding won't be announced until December.
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'Personalized medicine' gets $67.5M research boost
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World-Renowned Cell-Therapy Researcher, Doris Taylor, PhD, Joins Texas Heart Institute at St. Luke’s Episcopal Hospital
HOUSTON--(BUSINESS WIRE)-- Officials at the Texas Heart Institute (THI) at St. Luke’s Episcopal Hospital (St. Luke’s) announced today that Doris Taylor, PhD, FAHA, FACC, one of the world’s leading cell therapy and cardiac regeneration scientists, will join THI beginning March 1, 2012.
Dr. Taylor’s research includes: Cell and gene therapy for treatment of cardiovascular disease; tissue engineering of bioartificial organs and vasculature; cell-based prevention of disease; stem cells and cancer; and holistic approaches to using cell therapy for treating chronic disease.
Most recently, Dr. Taylor and her team garnered international recognition for work involving “whole organ decellularization” by showing they were able to remove existing cells from hearts of laboratory animals and even humans leaving a framework to build new organs. They repopulated the framework with other adult stem cells then provided a blood supply, and the heart regenerated with the characteristics and functions of a revitalized beating heart.
The hope is that this research is an early step toward being able to grow a fully functional human heart in the laboratory. Dr. Taylor has demonstrated that the process works for other organs as well – opening a door in the field of organ transplantation.
It is significant in that the need for transplants continues to grow, while the supply of donor organs remains critically low.
“Dr. Taylor is certainly one of the stars in the adult human stem cell field, and we feel extremely fortunate to have her join our team,” said Dr. James T. Willerson, THI’s President and Medical Director. “Her work fits very well with our mission and goals, and she certainly helps to solidify THI as a leader in cell therapy, which is one of the most promising hopes for treating cardiovascular disease.”
“The chance to work with Dr. Willerson and the THI team as colleagues is very exhilarating. From molecules, to cells, to organs and tissues, we want to create solutions for people with disease,” said Dr. Taylor. “I am confident that I am joining a regenerative medicine program that is unparalleled. And, given the breadth of innovation and science in Houston, I have every confidence that building solutions for heart diseases not only has a long history, but a bright future.”
The move to Houston will also bring her closer to her family, notes Dr. Taylor.
Dr. Taylor has been serving as director of the Center for Cardiovascular Repair and Medtronic Bakken Chair in Integrative Biology and Physiology at the University of Minnesota. Prior to that she was on the faculty as Associate Professor in Cardiology at Duke University Medical Center.
A native of Mississippi, Dr. Taylor holds a B.S. in biology from Mississippi University for Women and a Doctorate in pharmacology from the University of Texas Southwestern Medical School in Dallas.
About the Texas Heart® Institute
The Texas Heart Institute (www.texasheart.org), founded by world-renowned cardiovascular surgeon Dr. Denton A. Cooley in 1962, is a nonprofit organization dedicated to reducing the devastating toll of cardiovascular disease through innovative and progressive programs in research, education and improved patient care. Together with its clinical partner, St. Luke’s Episcopal Hospital, it has been ranked among the top 10 cardiovascular centers in the United States by U.S. News & World Report’s annual guide to “America’s Best Hospitals” for the past 21 years. The Texas Heart Institute is also affiliated with the University of Texas (UT) System, which promotes collaboration in cardiovascular research and education among UT and THI faculty at the Texas Heart Institute and other UT components.
About St. Luke’s Episcopal Health System
St. Luke’s Episcopal Health System (StLukesTexas.com) includes St. Luke’s Episcopal Hospital in the Texas Medical Center, founded in 1954 by the Episcopal Diocese of Texas; St. Luke’s The Woodlands Hospital; St. Luke’s Sugar Land Hospital; St. Luke’s Lakeside Hospital; St. Luke’s Patients Medical Center; St. Luke’s Hospital at The Vintage; and St. Luke’s Episcopal Health Charities, a charity devoted to assessing and enhancing community health, especially among the underserved. St. Luke’s Episcopal Hospital is home to the Texas Heart®Institute, which was founded in 1962 by Denton A. Cooley, MD, and is consistently ranked among the top 10 cardiology and heart surgery centers in the country by U.S. News & World Report. Affiliated with several nursing schools and three medical schools, St. Luke’s Episcopal Hospital was the first hospital in Texas named a Magnet hospital for nursing excellence, receiving the award three times.
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World-Renowned Cell-Therapy Researcher, Doris Taylor, PhD, Joins Texas Heart Institute at St. Luke’s Episcopal Hospital
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Stanford scientists bypass stem cells to create nervous system cells
Bypassing stem cells, mouse skin cells have been converted directly into cells that become the three main parts of the animal's nervous system, according to new research at the Stanford University School of Medicine.
The startling success of this method seems to refute the idea that "pluripotency" -- the ability of stem cells to become nearly any cell in the body -- is necessary for a cell to transform from one cell type to another.
It raises the possibility that embryonic stem cell research, as well as a related technique called "induced pluripotency," could be supplanted by a more direct way of generating cells for therapy or research.
"Not only do these cells appear functional in the laboratory, they also seem to be able to integrate ... in an animal model," said lead author and graduate student Ernesto Lujan.
The study was published online Jan. 30 in the Proceedings of the National Academy of Sciences.
The finding implies that it may one day be possible to generate a variety of neural-system cells for transplantation that would perfectly match a human patient.
While much research has been devoted to harnessing the potential of embryonic stem cells, taking those cells from an embryo and then implanting them in a patient could prove difficult because they would not match genetically.
The Stanford team is working to replicate the work with skin cells from adult mice and humans.
But Lujan emphasized that
much more research is needed before any human transplantation experiments could be conducted.
In the meantime, however, the ability to quickly and efficiently generate cells -- grown in mass quantities in the laboratory, and maintained over time -- will be valuable in disease and drug-targeting studies.
Contact Lisa M. Krieger at 408-920-5565.
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Stanford scientists bypass stem cells to create nervous system cells
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Skin cells turned into neural precusors, bypassing stem-cell stage
ScienceDaily (Jan. 30, 2012) — Mouse skin cells can be converted directly into cells that become the three main parts of the nervous system, according to researchers at the Stanford University School of Medicine. The finding is an extension of a previous study by the same group showing that mouse and human skin cells can be directly converted into functional neurons.
The multiple successes of the direct conversion method could refute the idea that pluripotency (a term that describes the ability of stem cells to become nearly any cell in the body) is necessary for a cell to transform from one cell type to another. Together, the results raise the possibility that embryonic stem cell research and another technique called "induced pluripotency" could be supplanted by a more direct way of generating specific types of cells for therapy or research.
This new study, published online Jan. 30 in the Proceedings of the National Academy of Sciences, is a substantial advance over the previous paper in that it transforms the skin cells into neural precursor cells, as opposed to neurons. While neural precursor cells can differentiate into neurons, they can also become the two other main cell types in the nervous system: astrocytes and oligodendrocytes. In addition to their greater versatility, the newly derived neural precursor cells offer another advantage over neurons because they can be cultivated to large numbers in the laboratory -- a feature critical for their long-term usefulness in transplantation or drug screening.
In the study, the switch from skin to neural precursor cells occurred with high efficiency over a period of about three weeks after the addition of just three transcription factors. (In the previous study, a different combination of three transcription factors was used to generate mature neurons.) The finding implies that it may one day be possible to generate a variety of neural-system cells for transplantation that would perfectly match a human patient.
"We are thrilled about the prospects for potential medical use of these cells," said Marius Wernig, MD, assistant professor of pathology and a member of Stanford's Institute for Stem Cell Biology and Regenerative Medicine. "We've shown the cells can integrate into a mouse brain and produce a missing protein important for the conduction of electrical signal by the neurons. This is important because the mouse model we used mimics that of a human genetic brain disease. However, more work needs to be done to generate similar cells from human skin cells and assess their safety and efficacy."
Wernig is the senior author of the research. Graduate student Ernesto Lujan is the first author.
While much research has been devoted to harnessing the pluripotency of embryonic stem cells, taking those cells from an embryo and then implanting them in a patient could prove difficult because they would not match genetically. An alternative technique involves a concept called induced pluripotency, first described in 2006. In this approach, transcription factors are added to specialized cells like those found in skin to first drive them back along the developmental timeline to an undifferentiated stem-cell-like state. These "iPS cells" are then grown under a variety of conditions to induce them to re-specialize into many different cell types.
Scientists had thought that it was necessary for a cell to first enter an induced pluripotent state or for researchers to start with an embryonic stem cell, which is pluripotent by nature, before it could go on to become a new cell type. However, research from Wernig's laboratory in early 2010 showed that it was possible to directly convert one "adult" cell type to another with the application of specialized transcription factors, a process known as transdifferentiation.
Wernig and his colleagues first converted skin cells from an adult mouse to functional neurons (which they termed induced neuronal, or iN, cells), and then replicated the feat with human cells. In 2011 they showed that they could also directly convert liver cells into iN cells.
"Dr. Wernig's demonstration that fibroblasts can be converted into functional nerve cells opens the door to consider new ways to regenerate damaged neurons using cells surrounding the area of injury," said pediatric cardiologist Deepak Srivastava, MD, who was not involved in these studies. "It also suggests that we may be able to transdifferentiate cells into other cell types." Srivastava is the director of cardiovascular research at the Gladstone Institutes at the University of California-San Francisco. In 2010, Srivastava transdifferentiated mouse heart fibroblasts into beating heart muscle cells.
"Direct conversion has a number of advantages," said Lujan. "It occurs with relatively high efficiency and it generates a fairly homogenous population of cells. In contrast, cells derived from iPS cells must be carefully screened to eliminate any remaining pluripotent cells or cells that can differentiate into different lineages." Pluripotent cells can cause cancers when transplanted into animals or humans.
The lab's previous success converting skin cells into neurons spurred Wernig and Lujan to see if they could also generate the more-versatile neural precursor cells, or NPCs. To do so, they infected embryonic mouse skin cells -- a commonly used laboratory cell line -- with a virus encoding 11 transcription factors known to be expressed at high levels in NPCs. A little more than three weeks later, they saw that about 10 percent of the cells had begun to look and act like NPCs.
Repeated experiments allowed them to winnow the original panel of 11 transcription factors to just three: Brn2, Sox2 and FoxG1. (In contrast, the conversion of skin cells directly to functional neurons requires the transcription factors Brn2, Ascl1 and Myt1l.) Skin cells expressing these three transcription factors became neural precursor cells that were able to differentiate into not just neurons and astrocytes, but also oligodendrocytes, which make the myelin that insulates nerve fibers and allows them to transmit signals. The scientists dubbed the newly converted population "induced neural precursor cells," or iNPCs.
In addition to confirming that the astrocytes, neurons and oligodendrocytes were expressing the appropriate genes and that they resembled their naturally derived peers in both shape and function when grown in the laboratory, the researchers wanted to know how the iNPCs would react when transplanted into an animal. They injected them into the brains of newborn laboratory mice bred to lack the ability to myelinate neurons. After 10 weeks, Lujan found that the cells had differentiated into oligodendroytes and had begun to coat the animals' neurons with myelin.
"Not only do these cells appear functional in the laboratory, they also seem to be able to integrate appropriately in an in vivo animal model," said Lujan.
The scientists are now working to replicate the work with skin cells from adult mice and humans, but Lujan emphasized that much more research is needed before any human transplantation experiments could be conducted. In the meantime, however, the ability to quickly and efficiently generate neural precursor cells that can be grown in the laboratory to mass quantities and maintained over time will be valuable in disease and drug-targeting studies.
"In addition to direct therapeutic application, these cells may be very useful to study human diseases in a laboratory dish or even following transplantation into a developing rodent brain," said Wernig.
In addition to Wernig and Lujan, other Stanford researchers involved in the study include postdoctoral scholars Soham Chanda, PhD, and Henrik Ahlenius, PhD; and professor of molecular and cellular physiology Thomas Sudhof, MD.
The research was supported by the California Institute for Regenerative Medicine, the New York Stem Cell Foundation, the Ellison Medical Foundation, the Stinehart-Reed Foundation and the National Institutes of Health.
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Journal Reference:
E. Lujan, S. Chanda, H. Ahlenius, T. C. Sudhof, M. Wernig. Direct conversion of mouse fibroblasts to self-renewing, tripotent neural precursor cells. Proceedings of the National Academy of Sciences, 2012; DOI: 10.1073/pnas.1121003109
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Skin cells turned into neural precusors, bypassing stem-cell stage
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Gene mutation is linked to accumulation of fat, other lipids in liver
Public release date: 31-Jan-2012
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Contact: Phil Sahm
phil.sahm@hsc.utah.edu
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University of Utah Health Sciences
SALT LAKE CITY -- A team of scientists from the University of Utah and the University of California at San Francisco has discovered that the mutation of a gene encoding a ketone body transporter triggers accumulation of fat and other lipids in the livers of zebrafish. This discovery, published in the Feb. 1, 2012, issue of Genes & Development, reveals that transport of ketone bodies out of the liver is a critical step in energy metabolism during fasting. It also provides a new approach for studying the development of fatty liver disease in humans.
Nonalcoholic fatty liver disease (NAFLD), or abnormally high accumulation of lipids in the liver, is the most common cause of chronic liver disease worldwide. Lipids are a broad group of molecules that include fats, triglycerides, and cholesterol. In some people, NAFLD causes no complications, but in others, excess fat in the liver can lead to inflammation or development of scar tissue, resulting in permanent liver damage or even liver failure. NAFLD may also increase the risk of heart disease in people who are overweight or obese. The increasing prevalence of NAFLD in the United States is due, in large part, to the obesity epidemic and it is estimated that more than 6 million U.S. children already have fatty liver disease.
"Currently, there are a limited number of treatment options for decreasing excess fat in the liver and there are no methods for reversing damage to liver tissue due to NAFLD," says Amnon Schlegel, M.D., Ph.D., investigator in the University of Utah Molecular Medicine program, assistant professor of internal medicine at the University of Utah School of Medicine, and senior author on the study. "By identifying and characterizing novel genes that regulate accumulation of lipids in the liver, we may be able to gain new insight into the physiological processes that lead to NAFLD."
Previous research has shown that many of the proteins known to control lipid metabolism in humans are also present in zebrafish. Schlegel and his colleagues began by identifying a zebrafish mutant known as red moon (rmn), which developed abnormal lipid accumulation in liver cells, without evidence of associated liver inflammation or liver damage, when exposed to fasting conditions. Schlegel and his colleagues then used a molecular genetic technique called positional cloning to isolate the gene disrupted by the rmn mutation. They found that the rmn mutation inactivated slc16a6a, a gene thought to encode a protein required in the transport of nutrients during fasting.
"Until now, the activity of the Slc16a6a protein has not been functionally characterized in any organism," says Schlegel, who's also an adjunct assistant professor of biochemistry at the U medical school. "Our studies indicate that Slc16a6a is a protein involved in the transport of ?-hydroxybutyrate."
?-hydroxybutyrate is a ketone body, a compound that is produced in the liver when blood glucose is low and fatty acids are broken down for energy. During periods of fasting, most body tissues can use fatty acids as an energy source, but the brain relies on ?-hydroxybutyrate and other ketone bodies for fuel. Schlegel and his colleagues discovered that, in rmn mutants deprived of nutrition, loss of Slc16a6a function disabled secretion of ketone bodies from liver cells and increased lipid accumulation in the liver. They also found that introducing the human form of the SLC16A6 protein into rmn mutant livers restored ketone body secretion.
"Our research has uncovered a previously unrecognized, but critical step, in the complicated physiology of fasting," says Schlegel. "We still don't know whether altered fasting liver metabolism influences the development of NAFLD, but knowing that Slc16a6a is required for secretion of ketone bodies from liver cells during fasting may have implications for our understanding and treatment of other medical conditions where ketone bodies play a role. These include uncontrolled type 1 diabetes, obesity, and childhood metabolic disorders caused by defects in fatty acid metabolism."
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Gene mutation is linked to accumulation of fat, other lipids in liver
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Flu Work Akin to Nuclear-Bomb Experiments, Board Says
Experts who made an unprecedented recommendation that bird-flu researchers hold back some details of their work justified the controversial decision on Tuesday, saying that the experiments were akin to the 1940s work on nuclear weapons or the first attempts at genetic engineering in the 1970s.
Members of the National Science Advisory Board for Biosecurity said that bioterrorists or rogue governments could use details of the experiments to make a global weapon of catastrophic potential.
“We found the potential risk of public harm to be of unusually high magnitude,” they wrote in a statement published jointly in the rival journals Science and Nature.
The decision, they said, is too big for the scientific community to make on its own. “Physicists faced a similar situation in the 1940s with nuclear-weapons research, and it is inevitable that other scientific disciplines will also do so.”
Since it started spreading in 2003, H5N1 bird flu has killed 344 of the 583 people it is known to have infected--a mortality rate of 59 percent. This compares to a 2.5 percent fatality rate for the 1918 flu, which killed tens of millions of people, or 30 percent for smallpox before it was eliminated in 1979. Luckily, H5N1 doesn’t infect people easily, but it spreads rapidly through flocks of chickens, infects ducks with barely a symptom, and appears to be carried by migrating wild birds. All flu viruses mutate, and most flu experts fear it is only a matter of time before H5N1 either evolves or mixes up with another flu virus to make a form that can easily infect people.
“A pandemic, or the deliberate release of a transmissible highly pathogenic influenza A/H5N1 virus, would be an unimaginable catastrophe for which the world is currently inadequately prepared,” the NSABB wrote.
Usually, when viruses acquire the ability to infect easily, they also become less lethal. So scientists are keen to find out what an H5N1 virus that could easily infect people might look like. If it transmits easily from one person to another, does it give up some of its killing power?
Two labs took a big step toward this goal last year, one in the Netherlands and one at the University of Wisconsin. They engineered forms of H5N1 that ferrets could easily pass to one another--ferrets being the closest thing in the animal world to humans when it comes to getting flu. The good news was that vaccines and drugs both worked against the new strain.
One team sent its findings to Science to be published, while the other submitted its results to Nature. The usual process would have been for the journals to ask other flu and genetics experts to critique the papers, and then they would publish them so other researchers could try to replicate the findings, adding to the world’s knowledge about H5N1, how to watch for dangerous changes, and how to make drugs and vaccines to protect people.
The flu community was atwitter about the pending news, and the potential consequences alarmed the NSABB, which was set up after the 2001 anthrax attacks and which includes heavyweight experts on bioterrorism such as Paul Keim of Northern Arizona University and Mike Osterholm of the University of Minnesota, as well as genetics experts like Claire Fraser-Liggett of the University of Maryland. They asked the two labs to hold off last year until the scientific community could agree on a way to make sure the information got into the right hands--and not into the wrong hands. The experts and the journals have agreed to wait for the time being, and the World Health Organization has set up a meeting in February in Geneva that includes experts from the two teams.
To say the decision frightened and irritated the scientific community would be an understatement. Almost everybody who is anybody in the world of viral research, bioterrorism, and scientific freedom has weighed in--most recently in eight letters to The New York Times.
Keim wrote a separate explanation for the online journal mBio, published by the American Society for Microbiology. “I carefully considered how restricting the information would compromise scientific research progress and even how it would hinder public health efforts to prevent such a horrific pandemic,” Keim wrote. “The short-term negative consequences of restricting experimental details seemed small in contrast to the large consequences of facilitating the replication of these experiments by someone with nefarious intent…. Publishing a detailed experimental protocol on how to produce a highly transmissible H5N1 virus in a highly regarded scientific journal is a very bad idea.”
Dr. Robert Webster of St. Jude Children’s Research Hospital in Memphis, Tenn., a pioneer in influenza research who doesn’t serve on the NSABB, agreed. “It has been argued that suppression of information serves no purpose, as the information will inevitably be ‘leaked.’ Although this viewpoint is likely correct, I do not believe we should publish the detailed methods of preparing transmissible H5N1,” Webster wrote in a separate commentary in mBio. But he said that the research itself must continue. “While bioterrorism is of real concern, nature has the potential to do much greater damage,” Webster pointed out.
Vincent Racaniello, a microbiologist at Columbia University College of Physicians and Surgeons, disagreed. “Bioterrorists do not want to carry out an experiment; they want to instill terror,” he wrote in mBio. “Science has always worked best when information is freely accessible. Fear has clouded the NSABB’s vision. We cannot allow fear to limit our ability to address medical problems.”
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Flu Work Akin to Nuclear-Bomb Experiments, Board Says
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Damnit I’m A Doctor Not A TSA Agent (Brainstorm Ep43) – Video
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New genetic subtype of lung cancer defined
ScienceDaily (Jan. 31, 2012) — A report from investigators at the Massachusetts General Hospital (MGH) Cancer Center has defined the role of a recently identified gene abnormality in a deadly form of lung cancer. Tumors driven by rearrangements in the ROS1 gene represent 1 to 2 percent of non-small-cell lung cancers (NSCLC), the leading cause of cancer death in the U.S. The researchers show that ROS1-driven tumors can be treated with crizotinib, which also inhibits the growth of tumors driven by an oncogene called ALK, and describe the remarkable response of one patient to crizotinib treatment.
"ROS1 encodes a protein that is important for cell growth and survival, and deregulation of ROS1 through chromosomal rearrangement drives the growth of tumors," says Alice Shaw, MD, PhD, of the MGH Cancer Center -- co-lead author of the paper which has been published online in the Journal of Clinical Oncology. "This finding is important because we have drugs that inhibit ROS1 and could lead to the sort of dramatic clinical response we describe in this paper."
The current findings add ROS1 to the list of genes known to drive NSCLC growth when altered -- a list that includes KRAS, mutations of which account for about 25 percent of cases; EGFR, accounting for 10 to 15 percent; and ALK, rearranged in about 4 percent. Altogether, known cancer-causing genetic changes have been found in a little more than half of NSCLC tumors. Originally identified in brain tumors, ROS1 rearrangement previously had been identified in one NSCLC patient and one NSCLC cell line. The current study was designed to determine the frequency of ROS1 rearrangement in NSCLC and to define the characteristics of patients with ROS1-rearranged tumors.
The investigators screened tumor samples from more than 1,000 NSCLC patients treated at the MGH, Vanderbilt University, the University of California at Irvine, and Fudan University in Shanghai, China. ROS1 rearrangement was identified in 18 tumor samples, for a prevalence of 1.7 percent; ALK rearrangements were identified in 31 samples, with no samples showing alterations in both genes. Patients with ROS1-positive tumors tended to be younger, never to have smoked and to have a type of lung cancer called adenocarcinoma -- characteristics very similar to those of ALK-positive patients.
An earlier MGH study of an experimental ALK inhibitor had found the drug suppressed the growth of a ROS1-positive cell line in addition to ALK-positive cell lines, suggesting that ROS1-positive tumors might be sensitive to the ALK-inhibitor crizotinib. This observation led corresponding author John Iafrate, MD, PhD, and his team to develop a diagnostic test that could identify ROS1-positive tumors. Around the time that test became clinically available, a lung cancer patient whose tumor had not responded to drugs targeting EGFR mutations was referred to the MGH Cancer Center for genetic testing. His tumor was negative for ALK but later proved to harbor a ROS1 rearrangement, and he was enrolled in an extension of the crizotinib clinical trial first reported in the October 28, 2010, New England Journal of Medicine.
"When he enrolled in the trial last April, this patient was extremely sick -- with significant weight loss and very low oxygen levels -- and was barely able to walk," says Shaw. "Within a few days of starting crizotinib, he felt better; and by the time we scanned his chest at seven weeks, the tumors had essentially disappeared from his lungs." Nine months after starting crizotinib therapy, this patient continues to do well. Additional ROS1-positive patients have been enrolled in this trial at MGH, at UC Irvine and at the University of Colorado.
Shaw is an assistant professor of Medicine and Iafrate is an associate professor of Pathology at Harvard Medical School. Co-lead authors are Kristin Bergethon, MGH Pathology, and Sai-Hong Ignatius Ou, MD, PhD, University of California at Irvine. The study was supported by grants from the National Institutes of Health and from Pfizer, which received FDA approval for crizotinib in August 2011.
Additional co-authors are Ryohei Katayama, Eugene Mark, Julie Batten, Eunice Kwak, Jeffrey Clark, Jeffrey Engelman, and Mari Mino Kenudson, MGH Cancer Center; Christina Siwak-Tapp, University of California at Irvine; Keith D. Wilner, Pfizer; Christine Lovly, Nerina McDonald, Pierre Massion, Adriana Gonzalez, David Carbone, and William Pao, Vanderbilt University Medical Center; Pierre Massion, Nashville Veterans Affairs Medical Center; Rong Fang and Hongbin Ji, Shanghai Institutes for Biological Sciences; and Haiquan Chen, Shanghai Medical College, Fudan University.
Massachusetts General Hospital, founded in 1811, is the original and largest teaching hospital of Harvard Medical School. The MGH conducts the largest hospital-based research program in the United States, with an annual research budget of more than $750 million and major research centers in AIDS, cardiovascular research, cancer, computational and integrative biology, cutaneous biology, human genetics, medical imaging, neurodegenerative disorders, regenerative medicine, reproductive biology, systems biology, transplantation biology and photomedicine.
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Journal Reference:
K. Bergethon, A. T. Shaw, S.-H. Ignatius Ou, R. Katayama, C. M. Lovly, N. T. McDonald, P. P. Massion, C. Siwak-Tapp, A. Gonzalez, R. Fang, E. J. Mark, J. M. Batten, H. Chen, K. D. Wilner, E. L. Kwak, J. W. Clark, D. P. Carbone, H. Ji, J. A. Engelman, M. Mino-Kenudson, W. Pao, A. J. Iafrate. ROS1 Rearrangements Define a Unique Molecular Class of Lung Cancers. Journal of Clinical Oncology, 2012; DOI: 10.1200/JCO.2011.35.6345
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New genetic subtype of lung cancer defined
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Myriad Genetics Reports Second Quarter Fiscal Year 2012 Results
SALT LAKE CITY, Jan. 31, 2012 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (Nasdaq:MYGN - News) today announced results for its second fiscal quarter and six months ending December 31, 2011. Revenue for the second fiscal quarter was $122.8 million, an increase of 22 percent over the $100.4 million reported in the second fiscal quarter of 2011. Earnings per diluted share were $0.33, an increase of 27 percent over the same period of the prior year.
"These results represent the second quarter in a row of 20 percent or greater year-over-year revenue growth," said Peter D. Meldrum, President and Chief Executive Officer of Myriad Genetics, Inc. "As a result, I'm pleased to announce increased guidance for fiscal 2012. We remain focused on delivering strong top-line growth and implementing our broader strategic plan to diversify revenue across multiple disease indications and geographies."
Analysis of Second Fiscal Quarter 2012
Molecular diagnostic testing revenue in the second fiscal quarter equaled $117.6 million, an increase of 17 percent compared to the prior year period. This increase was driven by strong growth across all segments and products. Oncology revenue equaled $79.8 million, an increase of 15 percent over the second quarter of 2011. Women's Health revenue totaled $37.9 million, an increase of 22 percent over the same period in the prior year. Revenue from the BRACAnalysis(R) test, which represented 82.6 percent of total revenue in the second quarter, was $101.4 million, a 14 percent increase over the same period of the prior year. Revenue from the COLARIS(R) and COLARIS AP(R) tests, which represented 8.9 percent of total revenue during the quarter, was $10.9 million, an increase of 56 percent compared to the same fiscal quarter of the prior year. Myriad's remaining molecular diagnostic tests contributed $5.3 million to second quarter revenue, an increase of 24 percent over the same period in the prior year, and accounted for 4.3 percent of total revenue. Companion diagnostic service revenue in the second fiscal quarter equaled $5.2 million and represented 4.2 percent of total company revenue. There is no prior year revenue, as the Company acquired this business in May 2011. Operating income was $45.5 million, an increase of 18 percent from the prior year period. This record level of operating income included the impact of a 68 percent increase in R&D investment to support the Company's existing molecular diagnostic tests and future product opportunities. Net income for the second fiscal quarter was $28.3 million, an increase of 17 percent over the $24.2 million reported in same period of the prior year. The Company repurchased 927,709 shares of its common stock during the quarter under its previously announced stock repurchase program. Diluted weighted average shares outstanding were 86.2 million in the second fiscal quarter as compared to 93.6 million in the same period of the prior year. The Company ended the quarter with $428.3 million in cash, cash equivalents and marketable investment securities. Days sales outstanding for Myriad's accounts receivable improved to 32 days, compared with 37 days in the same period of the prior year. Bad debt expense in the second fiscal quarter equaled 5.2 percent of revenue, compared with 4.2 percent in the same period of the prior year.
Year-to-Date Performance
Total revenue for the first half of fiscal 2012 was $233.3 million, an increase of 21 percent over $192.3 million reported for the half of fiscal 2011. Operating income for the first half of fiscal 2012 was $86.9 million, an increase of 17 percent year-over-year. Net income for the first half of fiscal 2012 equaled $53.4 million, compared to $46.7 million for the first half of the prior year, an increase of 14 percent. In the first half of fiscal 2012, diluted earnings per share increased 24 percent to $0.62 from $0.50 for the same period of the prior year.
Fiscal Year 2012 Outlook
The Company has increased its expectations for fiscal year 2012 financial performance. Total revenue is now expected to be $465 million to $475 million, an increase from the $445 million to $465 million previously announced. This level of revenue is expected to result in fully diluted earnings per share of $1.24 to $1.28, up from the original guidance of $1.20 to $1.25. Molecular diagnostic revenue is now expected to range between $440 million and $450 million and companion diagnostic service revenue continues to be expected to range between $24 million and $26 million. These projections are forward looking statements and are subject to the risks summarized in the safe harbor statement at the end of this press release. The Company will provide further detail on its business outlook during the conference call it is holding today to discuss its fiscal results for the second fiscal quarter and first half of fiscal 2012.
Conference Call and Webcast
A conference call will be held on Tuesday, January 31, 2012, at 4:30 p.m. Eastern time to discuss Myriad's second fiscal quarter and first half 2012 financial results and fiscal year 2012 outlook. The dial-in number for domestic callers is (888) 225-2734. International callers may dial (303) 223-2685. All callers will be asked to reference reservation number 21566441. An archived replay of the call will be available for seven days by dialing (800) 633-8284 and entering the reservation number above. The conference call will also be available through a live Webcast at http://www.myriad.com.
About Myriad Genetics
Myriad Genetics, Inc. (Nasdaq:MYGN - News) is a leading molecular diagnostic company dedicated to developing and marketing transformative tests to assess a person's risk of developing disease, guide treatment decisions and assess a patient's risk of disease progression and disease recurrence. Myriad's portfolio of nine molecular diagnostic tests are based on an understanding of the role genes play in human disease and were developed with a focus on improving an individual's decision making process for monitoring and treating disease. With fiscal year 2011 annual revenue of over $400 million and more than 1,000 employees, Myriad is working on strategic directives, including new product introductions, companion diagnostics, and international expansion, to take advantage of significant growth opportunities. For more information on how Myriad is making a difference, please visit the Company's website: http://www.myriad.com.
Myriad, the Myriad logo, BRACAnalysis, Colaris, Colaris AP, Melaris, TheraGuide, Prezeon, OnDose, Panexia and Prolaris are trademarks or registered trademarks of Myriad Genetics, Inc. in the United States and foreign countries. MYGN-F, MYGN-G
Safe Harbor Statement
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to the Company's focus on delivering strong top-line growth and implementing its broader strategic plan to diversify revenue across multiple disease indications and geographies; the Company's investment in R&D to support its existing molecular diagnostic tests and future product opportunities; the Company's increased guidance for fiscal year 2012 under the caption "Fiscal Year 2012 Outlook;"and the Company's strategic directives under the caption "About Myriad Genetics". These "forward-looking statements" are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by forward-looking statements. These risks and uncertainties include, but are not limited to: the risk that sales and profit margins of our existing molecular diagnostic tests and companion diagnostic services may decline or will not continue to increase at historical rates; the risk that we may be unable to expand into new markets outside of the United States; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and companion diagnostic services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and companion diagnostic services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and companion diagnostic services and any future products are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with manufacturing our products or operating our laboratory testing facilities; risks related to public concern over genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of healthcare payment systems; risks related to our ability to obtain new corporate collaborations and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we acquire; the development of competing tests and services; the risk that we or our licensors may be unable to protect the proprietary technologies underlying our tests; the risk of patent-infringement and invalidity claims or challenges of our patents; risks of new, changing and competitive technologies and regulations in the United States and internationally; and other factors discussed under the heading "Risk Factors" contained in Item 1A in our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K. All information in this press release is as of the date of the release, and Myriad undertakes no duty to update this information unless required by law.
MYRIAD GENETICS, INC. AND SUBSIDIARIES CONDENSED CONSOLIDATED INCOME STATEMENTS (Unaudited)
(in thousands, except per share amounts) Three Months Ended Six Months Ended
Dec. 31, 2011 Dec. 31, 2010 Dec. 31, 2011 Dec. 31, 2010
Molecular diagnostic testing $117,610 $100,440 $221,579 $192,298 Companion diagnostic services 5,201 -- 11,684 -- Total revenue 122,811 100,440 233,263 192,298
Costs and expenses:
Cost of molecular diagnostic testing 12,815 12,046 24,115 23,058 Cost of companion diagnostic services 3,302 -- 6,364 -- Research and development expense 10,243 6,092 18,748 11,853 Selling, general, and administrative expense 50,986 43,716 97,100 83,210 Total costs and expenses 77,346 61,854 146,327 118,121
Operating income 45,465 38,586 86,936 74,177
Other income (expense):
Interest income 1,382 548 1,856 1,269 Other (64) (80) (205) (214) Total other income 1,318 468 1,651 1,055
Income before income taxes 46,783 39,054 88,587 75,232
Income tax provision (benefit) 18,487 14,863 35,193 28,503
Net income $28,296 $24,191 $53,394 $46,729
Earnings per share:
Basic $0.33 $0.26 $0.63 $0.51 Diluted $0.33 $0.26 $0.62 $0.50
Weighted average shares outstanding
Basic 84,498 91,528 84,870 92,395 Diluted 86,231 93,647 86,602 94,178
Condensed Consolidated Balance Sheets
Dec. 31, 2011 Jun. 30, 2011
(In thousands)
Cash, cash equivalents, and marketable investment securities $428,259 $417,314
Trade receivables, net 42,988 50,272
Other receivables 1,083 575
Prepaid taxes 16,569 --
Inventory, net 10,294 8,218
Prepaid expenses 3,087 2,949
Equipment and leasehold improvements, net 24,329 23,080
Note receivable 17,667 --
Other assets 8,000 --
Intangibles, net 16,265 16,715
Goodwill 56,850 56,051
Deferred tax assets 35,867 35,653
Total assets $661,258 $610,827
Accounts payable and accrued liabilities $32,612 $33,040
Deferred revenue 2,434 1,347
Uncertain tax benefits 9,448 9,648
Stockholders' equity 616,764 566,792
Total liabilities and stockholders' equity $661,258 $610,827
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Myriad Genetics Reports Second Quarter Fiscal Year 2012 Results
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Genetics Pioneer Was UConn Professor, Mentor
Arthur Chovnick, a professor at the University of Connecticut, was a pioneer in the field of genetics whose influence was felt across the field of molecular genetics and biology.
"Arthur did something that has effectively jump-started enormous strides in the genetics of higher organisms," said Hal Krider, a former professor of genetics at UConn. "He was probably the most recognized, under-honored geneticist, but people with Nobel prizes would call and ask him for advice."
Chovnick, 84, of Chaplin, died Sept. 5.
One anecdote from his life reflected Chovnick's stature in the world of genetics. When his daughter Lisa was taking a biology course, she learned about Watson and Crick, who discovered the structure of DNA, but when the home phone rang one day and a caller identified himself as Francis Crick, Lisa hung up on him. "Quit joking," she told the Nobel Prize winner the next time he called.
Later that night, Arthur Chovnick picked up the phone himself. "Hello, Francis," he said.
"People of that stature were available to Arthur all the time," said Krider. "Everybody knew him. He was very, very well known and inordinately highly regarded."
Chovnick conducted experiments on drosophila melanogaster, a relative of the humble fruit fly that, rather than being a laboratory pest, is a valuable scientific specimen used for years in genetics research. First used to study heredity, the fly is now used in the study of disease as scientists search for the genes responsible for Alzheimer's or Parkinson's or Huntington's.
Drosophila genes are nearly identical to human genes. They also reproduce very quickly, meaning mutations may be studied in weeks rather than months or years. They have only four chromosomes. Even better, no groups picket against drosophila experimentation as they do against higher-order species.
"It is easy to grow and manipulate, and they have genes like us," said Christine Rushlow, a Chovnick-trained geneticist who is a professor at New York University. "We use them as a model system to see how genes work. We share so many genes."
Chovnick, known for pioneering work in gene organization and in demonstrating the way traits cross over within a gene, could look at events that were rare and re-create them.
Chovnick, born in Brooklyn, N.Y., on Aug. 2, 1927, grew up in Queens, where he graduated from Jackson High School in 1944. He was the oldest of four children born to Fannie and Herman Chovnick, who had both emigrated from Russia. He attended Indiana University for a year before joining the U.S. Navy, where he served on a hospital ship. After he was discharged, he returned to Indiana and obtained his undergraduate degree in 1949 and his master's in 1951. He got a doctorate in genetics from Ohio State University two years later, and obtained a grant from the National Institutes of Health that continued until 1995, one of the longest continuous NIH grants.
He spent two years at the University of Connecticut doing research and teaching before going to the Cold Spring Harbor Laboratory in Long Island, first as assistant director, then as director. In 1962, he returned to UConn as a professor, where he remained until he retired in 1994. He was a fellow of the American Association for the Advancement of Science and a founding member of the Connecticut Academy of Science and Engineering.
Chovnick was revered as a mentor as well as a teacher, his colleagues said.
"He left you alone, except he would always teach or help you," Rushlow said. "He was a great analytical thinker, which he could do in his head because he was so experienced."
He helped his students design experiments that would create a certain type of drosophila — with pink eyes for example, or missing a wing — to help them create their own mutations. "You see the consequences to the fly, and what it is doing to the fly," Rushlow said.
Chovnick also did early work on cloning, providing a fly with unusual chromosomes for other scientists to study. He studied how to regulate the activity of genes.
"When things go wrong because genes are out of control, you get disease," Rowlson said. "He was at the forefront, a leader in the genetics field, and famous for the work he had done." He also understood how genes recombine and how a new DNA sequence is created with potentially new effects.
Today, as scientists intensify their search for the genetic cause of disease, Chovnick's work is significant.
"He was a seminal character in the transition from classical genetics to modern genetic cloning and gene manipulation," said Krider, his former colleague.
"He was a very careful and highly creative thinker with a keenly analytical mind," said Arthur Hilliker, a professor at York University in Toronto, who studied under and later collaborated with Chovnick.
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Genetics Pioneer Was UConn Professor, Mentor
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Silkworms may help repair damaged hearts
Although people do regularly recover from heart attacks, the heart itself never entirely "gets better." This is because cardiac muscle tissue doesn't regenerate - any that dies in the event of a heart attack will only be replaced with inactive scar tissue, and the heart's performance will be permanently compromised as a result. Scientists have responded by trying to develop heart patches made of materials that act as nanoscale scaffolds, upon which new cardiomyocytes (heart cells) can grow. Materials used for these scaffolds have included fibrin, nanofiber, gold nanowires and polymer. Now, new research is suggesting that silkworm silk may be a better choice than any of those.
For some time now, scientists from Germany's Max Planck Institute for Heart and Lung Research have been among those researching ways of growing cardiac tissue on three-dimensional scaffolds. Everything that they looked at, however, had limitations.
"Whether natural or artificial in origin, all of the tested fibers had serious disadvantages," said research group leader Felix Engel. "They were either too brittle, were attacked by the immune system or did not enable the heart muscle cells to adhere correctly to the fibers."
It turned out, however, that scientists from the Indian Institute of Technology, Kharagpur had been working on an alternative - coin-sized disks made from the cocoon of the tasar silkworm. Not only is the silk coarser than other silk fibers, making it better-suited for use as a scaffold, but its surface also contains proteins that facilitate the adhesion of cardiomyocytes. When the silk was tested at the Max Planck Institute, heart cells from rats that were seeded onto it were able to remain in communication with one another, and beat synchronously for 20 days.
Before the silk patches can ever see clinical use, however, the scientists need to figure out a safe way of procuring a sufficient amount of heart cells from the patient. Using stem cells is a possibility, although finding a way of getting those to convert into heart cells still poses a challenge.
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Silkworms may help repair damaged hearts
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Harper government invests in personalized medicine
Public release date: 31-Jan-2012
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Contact: Adele Blanchard
ablanchard@cihr.gc.ca
613-946-3308
Canadian Institutes of Health Research
This press release is available in French.
Ottawa, Ontario -- The Harper Government today announced an important investment that will help Canadians in getting more effective treatments and make the healthcare system more sustainable through personalized medicine. The announcement was made by the Honourable Leona Aglukkaq, Minister of Health, and the Honourable Gary Goodyear, Minister of State for Science and Technology.
"Our Government is committed to improving the quality of life of Canadians," Minister Aglukkaq said. "The potential to understand a person's genetic makeup and the specific character of their illness in order to best determine their treatment will significantly improve the quality of life for patients and their families and may show us the way to an improved health care system and even save costs in certain circumstances."
Personalized medicine offers the potential to transform the delivery of healthcare to patients. Healthcare will evolve from a reactive "one-size-fits-all" system towards a system of predictive, preventive, and precision care. Areas in which personalized approaches are particularly promising include oncology, cardiovascular diseases, neurodegenerative diseases, psychiatric disorders, diabetes and obesity, arthritis, pain, and Alzheimer's disease. In all of these fields, and others, a personalized molecular medicine approach is expected to lead to better health outcomes, improved treatments, and reduction in toxicity due to variable or adverse drug responses. For example, cancer patients would be screened to identify those for whom chemotherapy would be ineffective. In addition to saving on the costs of expensive drug treatments, this personalized treatment would prevent a great deal of suffering, while identifying and initiating earlier treatments that would be more effective.
"I applaud Genome Canada and the CIHR for their leadership in supporting research in personalized medicine," said Minister Goodyear. "Innovative approaches like these lead to significant health benefits, enhance our knowledge within the medical arena and can be commercialized to help so many others worldwide."
###
Genome Canada is leading the landmark research competition, with significant collaboration from the Canadian Institutes of Health Research (CIHR) and the Cancer Stem Cell Consortium (CSCC). To qualify for funding, researchers must obtain matching funding that at is least equal to that provided through the competition. Matching funding is typically derived from provincial, academic, private sector or international sources.
Fact Sheet
Further information:
Cailin Rodgers
Office of the Honourable Leona Aglukkaq
Federal Minister of Health
613-957-0200
Stephanie Thomas
Special Assistant (Communications)
Office of the Honourable Gary Goodyear
Minister of State (Science and Technology)
613-960-7728
David Coulombe
Media Relations
Canadian Institutes of Health Research
613-941-4563
Marlene Orton
Director, Media Relations
Genome Canada
613-751-4460 x119
BlackBerry: 613-295-1476
The Canadian Institutes of Health Research (CIHR) is the Government of Canada's health research investment agency. CIHR's mission is to create new scientific knowledge and to enable its translation into improved health, more effective health services and products, and a strengthened Canadian health care system. Composed of 13 Institutes, CIHR provides leadership and support to more than 14,100 health researchers and trainees across Canada. http://www.cihr-irsc.gc.ca
Genome Canada is a non-profit corporation employing an innovative business model based on funding and managing large-scale, multidisciplinary, internationally peer-reviewed genomics research projects in areas such as agriculture, forestry, fisheries, the environment and human health. For more information, visit http://www.genomecanada.ca
The Cancer Stem Cell Consortium is a not-for-profit corporation that was incorporated in 2007 to coordinate an international strategy for cancer stem cell research and related translational activities. For more information, visit http://www.cancerstemcellconsortium.ca
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Harper government invests in personalized medicine
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Bionest and PMC Address Optimization of Personalized Medicine Strategies
NEW YORK & PARIS--(BUSINESS WIRE)-- Bionest Partners, a premier strategy and management consulting firm for life science industries, and a recognized leader in personalized medicine strategy consulting, announces the publication of an article in a supplement to the Dec. 23, 2011 issue of Science. The article, co-authored by Dr. Edward Abrahams, President of the Personalized Medicine Coalition (PMC), focuses on how best to optimize decision-making for personalized medicine research and development (R&D) and commercialization strategies.
The adoption of personalized medicine strategies could bring great benefits to patients, medical service providers, payers, and the manufacturers of personalized medicine products (e.g., pharmaceutical and diagnostic companies). The article discusses the complexities and uncertainties that personalized medicine introduces for pharmaceutical and diagnostic companies, and how they can address those challenges from commercial, operational, scientific and clinical perspectives. The authors highlight the use of quantitative modeling and decision-analysis tools. In addition, they emphasize the importance of the “soft art” of communication and consensus building within the organization.
Dr. Sean X. Hu, Head of Bionest USA and Managing Director, North America, and lead author, points out that “the key to success lies in the mastery of both the science and the art of personalized medicine strategy decision-making. This article summarizes the combined approach we employ to help companies make the right decisions in a still emerging field.”
Dr. Edward Abrahams adds: “The field of personalized medicine has come a long way over the last decade, to the point that it is now embedded in nearly every major pharmaceutical and diagnostic company research or product portfolio. Nevertheless, companies are struggling with how to navigate a still uncertain regulatory and payer environment, and formulate effective market strategies. The tools and approaches we refer to in this article are an important step toward providing a ‘GPS’ for making the right strategic decisions.”
Bionest is sponsoring the upcoming PMC reception at the InterContinental San Francisco on February 22, 2012, concurrent with the Molecular Medicine TriConference. For further discussions with Drs. Hu and Abrahams on personalized medicine strategies, either at the PMC reception or at another time, please contact Dr. Rachel Laing (rlaing@bionest.com).
Bionest has become a powerhouse in personalized medicine strategy consulting, experienced in a broad spectrum of project types, from the corporate level (personalized medicine business models, commercialization capability building, R&D and commercialization business processes, and organizational structure) to development and commercialization strategies for individual drug assets and companion diagnostics.
In addition, Bionest has been driving thought leadership on personalized medicine, with many articles published or in development on the strategic, commercial and technical aspects of personalized medicine.
For more details, please visit http://www.bionest.com, (navigate to section Strategic/Strategic Practice/Personalized Medicine Strategies).
About Bionest Partners
Bionest Partners provides advisory and management services exclusively for the life science industries, and helps pharmaceutical, biopharmaceutical, diagnostics, biotechnology, and medical device companies and their shareholders to maximize the value of their assets and investments. The company, founded in 2003, has offices in Paris and New York. Its broad customer base includes more than 100 clients ranging from large established companies to biotech start-ups and private equity firms, such as Pfizer, Sanofi, GlaxoSmithKline, Bristol-Myers Squibb, Novartis, AstraZeneca, Johnson & Johnson, Bayer, BiogenIdec, Millennium, MedImmune, Idenix, ProStrakan, Stallergenes, Innate Pharma, Genfit, Jubilant, Sequenom, Axa Private Equity, Blackstone, Candover. The company focuses on providing actionable strategies through its expertise in management consulting, including corporate, franchise, portfolio and product commercialization, organizational and marketing strategies, due diligence, surrogate management, as well as entry into European and US markets. Bionest has a global network of consultants and associate members deployed to address specific client assignments and geographical markets in the Americas, Europe, and Asia Pacific.
About the Personalized Medicine Coalition
The Personalized Medicine Coalition (PMC), representing scientists, patients, providers and payers, promotes the understanding and adoption of personalized medicine concepts, services and products to benefit patients and the health system. For more information on the Personalized Medicine Coalition, please visit http://www.PersonalizedMedicineCoalition.org.
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Bionest and PMC Address Optimization of Personalized Medicine Strategies
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'Personalized medicine' gets $67.5M research boost
The federal government is pledging up to $67.5 million for research into "personalized medicine," which tailors treatment to a patient's genetics and environment.
The funds will flow through Genome Canada, the Cancer Stem Cell Consortium and the Canadian Institutes of Health Research, the federal government's health research agency.
Federal Health Minister Leona Aglukkaq and Minister of State for Science Gary Goodyear made the announcement at the University of Ottawa's health campus Tuesday.
The field of personalized medicine is touted as having the potential to transform the way patients are treated. It looks at the genetic makeup of a person, the patient's environment and the exact course of a particular disease so that an appropriate and effective treatment can be tailored for that individual.
The idea is to move from a one-size-fits-all approach to one that is designed for a specific person and relies on the genetic signatures, or biomarkers, of both the patient and the disease.
Proponents of personalized medicine say it is likely to change the way drugs are developed, how medicines are prescribed and generally how illnesses are managed. They say it will shift the focus in health care from reaction to prevention, improve health outcomes, make drugs safer and mean fewer adverse drug reactions, and reduce costs to health-care systems.
"The potential to understand a person's genetic makeup and the specific character of their illness in order to best determine their treatment will significantly improve the quality of life for patients and their families and may show us the way to an improved health-care system and even save costs in certain circumstances," Aglukkaq said in a news release.
Research projects could last four years
The sequencing of the human genome paved the way for personalized medicine and there have been calls for more research funding so that the discoveries in laboratories can be translated further into the medical field so they will benefit patients more.
Identifying a person's genetic profile, for example, could then indicate a susceptibility to a certain disease, if the biomarkers of that disease have also been discovered. If people know they are genetically at risk of an illness they can take actions to prevent it, and their health-care providers can monitor for it.
Cancer patients could be pre-screened to determine if chemotherapy would work for them, which could not only save a lot of money on expensive treatments but also prevent pain and suffering for patients.
Genome Canada is leading the research initiative, in collaboration with Cancer Stem Cell Consortium and CIHR which on Tuesday launched its Personalized Medicine Signature Initiative. CIHR is committing up to $22.5 million to the large-scale initiative with the other two partners, but it will be providing more funding for other projects under its personalized medicine program.
The research projects are aiming to bring together biomedical, clinical, population health, health economics, ethics and policy researchers to identify areas that are best suited to personalized medicine.
Oncology, cardiovascular diseases, neurodegenerative diseases, psychiatric disorders, diabetes and obesity, arthritis, pain, and Alzheimer’s disease are all considered to be areas that hold promise for personalized medicine.
Funding will also go to projects that are aimed at developing more evidence-based and cost-effective approaches to health care.
Researchers can get up to four years of funding, but 50 per cent of their requested funding must be matched from another source, such as a provincial government or from the academic or private sectors.
Genome Canada, CIHR and the cancer consortium will invest a maximum of $5 million in each individual project.
The successful applicants for the $67.5 million worth of funding won't be announced until December.
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'Personalized medicine' gets $67.5M research boost
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Mark Walter, MD introduces Prolotherapy and PRP: The New Field of Regenerative Medicine – Video
19-10-2011 18:19 Mark Walter, MD is an internationally recognized expert in the field of Regenerative Medicine. He is McGill trained (1980) and has specialized in Sports Medicine and healing sports-related injuries for over three decades. Dr. Walter has worked with many of the top teachers in the world in Regenerative Medicine and for five years he joined the staff of the Univ. of Wisconsin Medical School to help train residents and doctors in Central America. Dr. Walter is a member of the American Academy of Orthopedic Medicine and is founder of ProloMD, located in Sarasota, FL. He has a special interest in working with golf-related injuries and can be found on the web at http://www.PRPyourpain.com and/or ProloMD.com.
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Mark Walter, MD introduces Prolotherapy and PRP: The New Field of Regenerative Medicine - Video
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2011 Summit: Keynote Address, CIRM’s Translational Roadmap to Stem Cell Cures, Alan Trounson, PhD – Video
13-12-2011 14:45 Alan Trounson, PhD and President of the California Institute for Regenerative Medicine, offered a positive and forward looking keynote address. Trounson provided an update on CIRM's translational roadmap to regenerative medicine and outlined their accomplishments with over $300 million in funding for human embryonic stem cell research, adult stem cells, cancer stem cells, reprogrammed stem cells and endogenous stem cells for multiple treatments. He spoke about progress in these areas of research emphasizing their promise and relating how close many treatments are to becoming a reality.
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2011 Summit: Keynote Address, CIRM's Translational Roadmap to Stem Cell Cures, Alan Trounson, PhD - Video
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BioRestorative Therapies Signs License Agreement for Stem Cell Disc/Spine Procedure
JUPITER, Fla., Jan. 31, 2012 /PRNewswire/ -- BioRestorative Therapies, Inc. (OTCQB: BRTX) ("BRT") today announced that it has entered into a License Agreement with Regenerative Sciences, LLC ("RS") with respect to certain stem cell-related technology and clinical treatment procedures developed by RS. The treatment is an advanced stem cell injection procedure that may offer relief from lower back pain, buttock and leg pain, or numbness and tingling in the legs or feet as a result of bulging and herniated discs.
To date, over 40 procedures have been performed on patients. It is a minimally invasive out-patient procedure, and objective MRI data and patient outcomes for this novel injection procedure show positive results with limited patient downtime. BRT intends to utilize the existing treatment and outcome data, as well as further research, to prepare for clinical trials in the United States.
Pursuant to the agreement, BRT will obtain an exclusive license to utilize or sub-license a certain medical device for the administration of specific cells and/or cell products to the precise locations within the damaged disc and/or spine (and other parts of the body, if applicable) and an exclusive license to utilize or sublicense a certain method for culturing cells for use in repairing damaged areas. The agreement contemplates a closing of the license grant in March 2012, subject to the fulfillment of certain conditions.
Mark Weinreb, Chairman and CEO of BRT, said, "This possible alternative to back surgery represents a large market for BRT once it begins offering the procedure to patients who might be facing spinal fusions or back surgery (which often times is unsuccessful). By delivering a particular cell population using a proprietary medical device that inserts a specialized needle into the disc and injects cells for repair and re-population, BRT hopes to revolutionize how degenerative disc disease will be treated."
About BioRestorative Therapies, Inc.
BioRestorative Therapies, Inc.'s goal is to become a medical center of excellence using cell and tissue protocols, primarily involving a patient's own (autologous) adult stem cells (non-embryonic), allowing patients to undergo cellular-based treatments. In June 2011, the Company launched a technology that involves the use of a brown fat cell-based therapeutic/aesthetic program, known as the ThermoStem™ Program. The ThermoStem™ Program will focus on treatments for obesity, weight loss, diabetes, hypertension, other metabolic disorders and cardiac deficiencies and will involve the study of stem cells, several genes, proteins and/or mechanisms that are related to these diseases and disorders. As more and more cellular therapies become standard of care, the Company believes its strength will be its focus on the unity of medical and scientific explanations for clinical procedures and outcomes for future personal medical applications. The Company also plans to offer and sell facial creams and products under the Stem Pearls™ brand.
This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including those set forth in the Company's Form 10, as amended, filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.
CONTACT: Mark Weinreb, CEO, Tel: (561) 904-6070, Fax: (561) 429-5684
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BioRestorative Therapies Signs License Agreement for Stem Cell Disc/Spine Procedure
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RemedyMD Announces Investigate Cell Therapy, a Revolutionary Research Management System for Blood and Marrow …
SALT LAKE CITY, Jan. 31, 2012 /PRNewswire/ -- RemedyMD, the leading provider of Translational Research Informatics, will launch Investigate™ Cell Therapy at the American Society for Blood and Marrow Transplantation BMT Tandem Meetings Feb. 1 – Feb. 5 in San Diego, Calif. (Booth #604).
(Logo: http://photos.prnewswire.com/prnh/20120127/SF43320LOGO)
"Investigate Cell Therapy is designed to improve research efficiencies, facilitate pattern recognition and accelerate discoveries in BMT centers," said Hank Wu, Investigate product manager for RemedyMD.
Fully integrated with ComprehensiveBMT™, Investigate Cell Therapy is the world's only blood and marrow research management system capable of integrating next-generation sequencing, cross-disease registries, and long-term complications and outcomes to advance cell therapy research. Researchers benefit from:
Aggregation of disparate data types from bench to bedside data sources with an intelligent ontology that harmonizes biospecimen, registry and outcomes data 360-degree perspective of phenotypic and genotypic data for subjects, patients, diseases Configurability that keeps up with the pace of research, without waiting for software coding or database administration Multisite, real-time research collaboration without the cost and complexity of infrastructure building and maintenance A suite of reporting, analysis, and data visualization tools that enable drag-and-drop reporting by researchers, scientists, and clinicians without writing SQL
Investigate Cell Therapy leverages the same research platform implemented at the Blood Research Institute of the Blood Center of Wisconsin to integrate lab information management systems (LIMS), study design and tracking, patient registries, and data mining and visualization.
"Research is fundamentally an exercise in pattern recognition," said Gary Kennedy, Founder and CEO of RemedyMD. "Bringing the power of Investigate™ to bear on cell therapy research will provide blood and marrow researchers with the technology they need to recognize new patterns and increase the speed of discovery."
RemedyMD will demonstrate Investigate Cell Therapy at BOOTH #604 at the 2012 Tandem Meetings in San Diego, Calif. beginning Feb. 1 – Feb. 5.
This product is for research use only and is not intended for clinical or cell processing purposes.
About RemedyMD, Inc.
RemedyMD is the leading provider of registries and registry-based products for Life Science Research. RemedyMD's disease registry software is cutting-edge research management software which enables researchers, clinicians and scientists to collect, aggregate, harmonize, and analyze data from widely disparate sources and then use Mosaic™ Platform tools and applications to identify patterns that result in accelerated discoveries and better clinical data management. For more information visit: www.RemedyMD.com.
Media Contacts
Kristofer Beldin, RemedyMD, (801) 870-9407 (cell), kris.beldin@remedymd.com
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RemedyMD Announces Investigate Cell Therapy, a Revolutionary Research Management System for Blood and Marrow ...
Recommendation and review posted by Bethany Smith