2. Michael N. Helmus, Ph.D., Consultant
mnhelmus@msn.com
Personalized Medicine
'The molecular methods that make personalized medicine
possible include testing for variations in genes, gene
expression, proteins and metabolites, as well as new
treatments that target molecular mechanisms. Test results
are correlated with clinical factors - such as disease state,
prediction of future disease states, drug response, and
treatment prognosis - to help physicians individualize
treatment for each patient'
Personalized Medicine Coalition
www.personalizedmedicinecoalition.org/sci
encepolicy/personalmed-101_overview.php
3. Personalized Medicine to Drive New Technology
Less Invasive Therapies
Custom Implants
Biosensors
Implantable biosensors, eg CHF
Telemetered devices and implants
Molecular Diagnostics
Genomic basis of Disease
Local and Targeted Drug Delivery
Pharmacogenomics
Tissue Engineering
Cell Therapy
New Imaging, eg. Histologic Grade
OCT
Personalized Medicine:
Local and Targeted
Diagnostics and Therapeutics
to allow “individualized
treatment for each patient”
4. Michael N. Helmus, Ph.D., Consultant
mnhelmus@msn.com
Leverage Potential Disruptive Technologies
Drug Delivery
Therapeutic Polymers
Biodegradables
Tissue Engineering
Stem Cells
Smart Materials
Imaging, e.g. Molecular Imaging
Genomics
Proteomics
Glycomics
Computation
NanoStructures
MEMS
Telemetered/sensored implants
5. Michael N. Helmus, Ph.D., Consultant
mnhelmus@msn.com
Functionality-Biocompatibility
Recellularization,andneogenesisoftissue
Time
Passive Biomaterials
Bioactive, Biodegradables
& Drug Delivery
Tissue Engineering
Disruptive Biocompatibility Based Technologies
Genomics
Identifying effective Therapies
6. Michael N. Helmus, Ph.D., Consultant
mnhelmus@msn.com
Interventional Placements
of Implantable Devices and Treatments
e.g. CABG, Heart Valves, Joints
Tissue Engineered Constructs, Chordae Tendon
Repair, Biodegradable
Injectables for Heart Failure
Functionality
Time
Surgical
Interventional -
Stents
MIS
Disruptive Technology: Surgical Procedures
Niche Market: Elderly?
History of Stent Grafts
for AAA
Genomics
Identifying Effective
Therapies
7. Michael N. Helmus, Ph.D., Consultant
mnhelmus@msn.com
Nanopores for drug
delivery
Nanoenabled Diagnostics and
Therapies
Nanoparticles to cross
The blood brain barrier:
Diagnostics, drug delivery
Gold shell nanoparticles for
Tumor ablation
Nanofiber Scaffolds for
Vascular prostheses &
Tissue engineering
Nanodiagnostics for point of care
Diagnosis: infectious disease,
biomarkers
Quantum Dots for
Molecular
Imaging
Nanoporous filters: Drug delivery,
Hemodialysis, Plasmapheresis,
Oxygenation – Celgard has been
available for 30 + years
8. Michael N. Helmus, Ph.D., Consultant
mnhelmus@msn.com
Implantation of tissue
engineered construct.
Tissue Engineered Devices
Biopsy/tissue sample for autologous cell
&/or isolation of stem cells.
Culturing of cells to expand if needed.
Scaffolds: Decellularized tissue,
Polymer, Biodegradables,
Bioderived, eg collagen
Seeded scaffolds for
direct implantation or
growth of tissue in
bioreactor
Healed device
9. Michael N. Helmus, Ph.D., Consultant
mnhelmus@msn.com
Small Molecules
Proteins/Growth Factors
Gene Transfection
Remodeled Organ
In Situ Healing
Injectables to
recruit
bmc’s/tissue
stem cells to
Regenerate in
situ.
10. Identification of Drivers for New
Technology
• Cost Containment
• New Diagnostics and Point of Care
• Infectious Disease
• Epidemic/Pandemic Surveillance
• Biomarkers for Disease
• Enablement for interventions: e.g.
vulnerable plaque
Personalized
Medicine
11. Cleveland Clinic announces its top 10 medical
innovations of 2010
1. Molecular imaging biomarker for early detection of Alzheimer's Disease
2. Targeted T-cell antibody for metastatic melanoma
3. First cancer vaccine approved by the FDA
4. Jupiter Study: Statins for healthy individuals
6. Telehealth monitoring for individuals with heart failure
7. Endoscopic weight-loss procedure
8. Exhaled nitric oxide (NO) breath analysis for diagnosing asthma
9. Oral disease modifying treatment for multiple sclerosis
10. Capsule endoscopy for diagnosis of pediatric GI disorders
http://www.cleveland.com/healthfit/index.ssf/2010/11/2010_innovations_summit_clevel.h
12. • New Therapeutics
– Cancer
– Infectious Disease
– Immune Disease
– Minimally/Less Invasive Procedures
– Implants
– Tissue Engineering
Drivers for New Technology cont.
13. Michael N. Helmus, Ph.D., Consultant
mnhelmus@msn.com
Commercializing Technology
Pulling Technology across the Valley of Death
Product Commercialization
Discovery &
Research
Development &
Engineering
Manufacturing &
Marketing
“Valley of Death”
Value
14. •The bridge to commercialization
• Proof of principle in a clinically relevant setting
• Drivers for Development
• Cost Containment, New Therapies, New
Diagnostics and Point of Care Medicine
•Intellectual Property
Commercializing
15. Michael N. Helmus, Ph.D., Consultant
mnhelmus@msn.com
Recent Examples
Personalized Medicine
* Microfluidics chip can spot rare cancer cells in blood
Mass General Hospital - microfluidics chip to detect groups of rare
tumor cells in a patient's blood sample. The technique could help
improve research into cancer metastasis and spare patients from
undergoing invasive procedures used for collecting tumor samples. MIT
Technology Review (10/5)
*J&J Invests in New Noninvasive Cancer Test
Johnson & Johnson (J&J) has announced that it is investing $30 million
in a new test that could detect—and help doctors treat—a variety of
cancers from a simple blood draw, according to reporting by Yahoo
Canada News. While experts concede that such a test is still years away,
some are predicting that it could revolutionize cancer detection and
treatment.
AdvaMed Smart Briefs
16. Michael N. Helmus, Ph.D., Consultant
mnhelmus@msn.com
Setting Expectations
How to innovate while addressing concerns.
Suggests need to establish well delineated practice
guidelines as the technology translates into the
clinic
(CNN) – Cancer breakthrough -- or nightmare?
January 11, 2011
“A simple blood test. It's able to detect minute quantities of cancer cells
that might be circulating in your bloodstream.
It's reported to be able to detect a single cell. It's intended to allow
cancer patients to start treatment much earlier.
It's supposed to save lives. It's a cancer breakthrough.
But it's not that simple. The test could just as easily start a cancer
epidemic.”
17. Personalized medicine. Personalized medicine includes the detection of disease
predisposition, screening and early disease diagnosis, prognosis assessment,
pharmacogenomic measurements of drug efficacy and risk of toxic effects, and the
monitoring of the illness until the final disease outcome is known.
JS Ross, GS Ginsburg, The Integration of Molecular Diagnostics With Therapeutics
Jeffrey S. Ross, MD, Geoffrey S. Ginsburg, MD, PhD American Journal of Clinical Pathology. 2003;119(1)
http://www.medscape.com/viewarticle/447846
19. When it becomes personal!!
Hi Fever, fainting, coughing
ER visit, immediate admission to ICU
Chest X-Ray consistent with bacterial infection
Hi dose Antibiotics
Rapid progression to BiPap and intubation and ventilator
within 4 days with continuing deterioration
20. Discussion with family
About 4 weeks prior: Exposure to Polyurethane sealant during
renovation, poor ventilation, walls exposed
2 weeks prior: reexposure to Polyurethane sealant
Immediately administered prednisone and antifungal (as precaution)
Lavage indicated no fungal or bacterial involvment
Stopped Antifungal
9 days on ventilator
Diagnosis hypersensitivity pneumonia
When it becomes personal!!
21. Disease Management
Admission
Circulating WBC Biomarkers
Circulating Antibodies
Biosensors
Radiology
Complete blood count
Complete metabolic profile
Blood gases or pulse
oximetry
Bronschoscopy, Bronchoalveolar lavage,
transbronchial biopsy
Thoracoscopic or open-lung biopsy
Radiographically guided transthoracic aspirate
Legionella, Chlamydia, Mycoplasma serology
Fungal serology
Evaluation for congestive heart failure,
pulmonary embolus, neoplasm, connective
tissue disease
Deteriorating patient without definitive
diagnosis of cause
Earlier impetus for lavage and biopsy
Earlier treatment with steroids
Eliminate diagnosis of fungal infection
Eliminate or reduce need for ventilation
More rapid recovery, mitigating DVT
Personalized Medicine Early Disease Diagnosis: Molecular Pathology
Screening
Subclinical Disease processes
Predisposition
22. Bilateral below knee DVT
Increased heparin, Vena cava filter
Indication of allergic reaction to due to skin hives/rash
Suspicion heparin allergy, change to LMW heparin
Significant bodywide rash
Warfarin therapy
Post release, discovery of hi FVIII disease
When it becomes personal!!
23. Disease Management cont.
Hospital based complications
DVT Increase heparin, Vena Cava Filter
Heparin Allergy LMW Heparin
More severe Heparin Allergy Warfarin
Personalized Medicine Mitigating Complications: Molecular Pathology
Screening Pharmacogenomics
Subclinical Disease processes
Biosensors High FVIII disease
Identification of Heparin allergy
Earlier Warfarin administration
Pharmacogenetics Titrate Warfarin dose
When can Warfarin dose be
eliminated
24. Micromechanical Sensing & Detection
Nanotechnology Approaches to Sensing and Detection
Dr. James S. Murday Dr. Richard J. Colton, Naval Research Laboratory
http://www.frtr.gov/pdf/meetings/dec04/murday_12-04.pdf
C nanotube networks: Detection via field-
induced polarization of adsorbates on
SWNT surface
BioFETs: thin for efficient sensing (~2 nm).
source drain; specific attachment of DNA or protein
Biosensors Will Drive Personalized Medicine
25. Michael N. Helmus, Ph.D., Consultant
mnhelmus@msn.com
INSTRUMENTATION/PACKAGING
• Spectrometry
• Light Scattering
• Microfluidics
• Nanosensors
• Biochips
• Thin film transistor arrays
• Scattering techniques
• Tissue culture techniques
MODELING
• Computational modeling:
- biomolecules
- crystallographic structures
- biokinetics and dosimetry
• Tissue-light interactions modeling
APPLICATIONS
• Disease Biomarkers
• DNA/Gene expression
• Chemical and Biotoxin Exposure
• Pathogen sensing
• Molecule detection
• Single molecule detection
Biosensor Development
Modified from:
http://www.ornl.gov/sci/biosensors/abstg_orgchart.pdf#search=%22Advanced%20Biomedical%20Scie
nce%20and%20Technology%20Group%22
26. Personalized Medicine to Drive New Technology
Local and Targeted
Diagnostics and Therapeutics
to allow “individualized
treatment for each patient”
Drug Delivery,
Tissue Engineering
& Cell Therapy
Biomarker &
Disease
Detection
Less Invasive
Procedures
Michael N. Helmus, Ph.D., Consultant
Medical Devices, Biomaterials
Drug Delivery, and Nanotechnology
(508) 767 0585
