Transcript The Future of Medicine

The Future of Medicine
Danny Belkin
“Our generation may be the last to have to
accept death and taxes as inevitable."
- Commander Shaun Jones ,US Navy, DARPA
Contents
• Challenges to “traditional” big pharma, their future
• Pharmacogenomics
• Current and Future Biotechnology
• Stem Cell Therapy
• Microtechnology and nanotechnology
Challenges to Pharmaceutical Companies
• Aging and informed population
• Increased focus on preventive care / wellness
• Demand for safety and efficacy
• Serious drug side effects (Vioxx, Hormone replacement therapy)
• Prescription drugs that don’t always work
• Progress in science and technology providing other therapeutic
paradigms
• Keeping up with advances in information technology
• Lack of R&D productivity increase despite sustained investments
• Increased regulatory demands
• Unacceptable failure rates of R&D projects
…challenges which lead to changes in healthcare
• Better understanding of nutrition and its effects
• Initially specified for diseases/population groups
(problems with clinical trials for unpatentable nutrition
compounds)
• Later - per individual genetic makeup
• Pharmacogenomics and Proteomics
• Many major diseases understood at molecular level – advanced
drug design
• Smart Drugs for Cancer and Autoimmune Diseases (arthritis,
psoriasis, diabetes).
• Development of diagnostics combined with targeted therapeutics
• in silico simulations of biological systems
The “5 P’s” of Future Medicine
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Predictive Preventive Point of care Parametric -
Pharmacogenomics and Pharmacoproteomics
Acting proactively with preventive medicine
Mobile communications & ubiquitous computing
Multiple parameters, over time, referenced to patient’s
own baseline and genetic profile, compared to standard
model
• Personalized - Individual treatment for each patient
Pharmacogenomics - Personalized Medicine
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Both preventative and therapeutic
Single nucleotide polymorphisms (SNPs) - Only a fraction of these,
probably 300,000, determine everything from hair colour to disease
susceptibility and drug response.
100,000 people die each year in the US from adverse reactions to
prescription drugs, stemming from specific gene loci. Millions more
suffer painful side effects from drugs.
Knowledge of patients’ genetic profile will allow significantly
improved preventative measures as well as improved drug safety and
effectiveness (“tailor made”).
Pharmacogenomics - Personalized Medicine
Risk prediction
• Begin colonoscopy
at age 40
• Avoid high fat in
diet
Pharmacogenomics
• Drug dose of
antidepressant
determined by
drug metabolism
genetic profile
New Therapies
• Gene-based drug
therapy for
cancer
• Gene & Cell
therapy for
heart disease
Current and Future Biotechnology
• RNAi (RNA Interference) and micro RNA to disable/weaken
expression of genes
• Ageing-promoting
• Pathogens
• Cancer
• Autoimmune
• Gene Chips
• Cell Therapy
• Gene Therapy
• Combined Cell and Gene Therapy
• Human Cellular Engineering and Tissue Engineering
Stem Cells Therapy - Regenerative Medicine
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Multipotent Cells that can develop into any organ or tissue
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Embryonic stem cells – controversial and technically difficult to
manipulate
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Ideally - patient’s own stem cells, rejuvenated and then delivered
into desired tissue
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Stem cell transplants have been effective in treating cancers

Stem cell therapy now being used for degenerative diseases
Future of Stem Cell Therapy
Multipotent Blood-derived cells are available now
• Developing further depth: additional cell types
• Developing further width: treatment of additional diseases treatable by a certain cell
type
• Automation of manufacturing processes for:
• Faster, more efficient production (e.g. improved, closed-system culture systems)
• R&D – Rapid improvement of manufacturing process; manufacturing of more
effective products
• Improved transplantation techniques: microcapsules; targeted systemic infustion; slow release;
in vivo growth by mimicry of BM
• Integration of gene therapy to form a synergetic product exhibiting the benefits of
both technologies
Microtechnology and Nanotechnology
Bring the ability to detect, prevent and treat disease down
to the molecular level
First Microtechnology Pioneers
• BioMEMs – Microelectronic Mechanical Systems : microsensors,
drug delivery, disposable chips for diagnostics
• Dendrimers – synthetic polymer for disease detection and drug
delivery
• Artificial microbes - producing beneficial materials or breaking down
harmful ones
First Microtechnology Pioneers
From: Wired 14.12
The Potential of Nanotechnology
• Nanodevices can potentially:
• Be much more therapeutically effective than current drugs
• Be much more efficient than current diagnostic tests
• First generation nanoparticles are already in development:
• Magnetic particles for in vivo diagnostics are in use in the clinic
• Lipid-based nanoparticles capable of delivering drugs or genes in
advanced preclinical testing.
Nanotechnology: Information Meets the Physical World
• A technology ripening 10-20 years from now
• Massive parallel diagnostics
• Paradigm – first augmenting, then replacing
• Highly expensive, moderately efficient, then ultimately cheap and
efficient
• Upgrading the cell nucleus with a nanocomputer and nanobots
Nanotechnology: Information Meets the Physical World
• The Biological assembler
Nanotechnology: Information Meets the Physical World
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Surveillance and monitoring nanobots
Hormone delivering nanobots
Oxygen-extracting nanobots – no need for lungs
Microbivores (artificial phagocytes)
Tumor-destroying nanoparticles
Surgical Nanorobotics – vascular maintenance, gene or chromosome replacement
Metabolic nanobots – introducing nutrients directly into bloodstream; no need for
digestive system
• Augmentation of all that’s left: skeleton, skin, sensory organs, brain.
Nanobots in the bloodstream
http://www.foresight.org/Nanomedicine/Gallery/index.htm
Nanobots
Nanobots in the bloodstream
http://www.foresight.org/Nanomedicine/Gallery/index.htm
Nanobots
Clottocytes
Housekeeping Nanobots
http://www.foresight.org/Nanomedicine/Gallery/index.htm
Nanobots
Neural Augmentation/replacement
Respirocytes (mechanical RBC)
http://www.foresight.org/Nanomedicine/Gallery/index.htm
Nanobots
Surveillance and monitoring nanobots
http://www.foresight.org/Nanomedicine/Gallery/index.htm
Timeline
1990
2000
2010
Gene Therapy I
Cell Therapy I
2020
2030
Gene Therapy
II
Cell Therapy II
Combined Gene & Cell Therapy
Advanced Tissue Engineering
Advanced drug design – based on proteomics & genomics
Nanomedicine - diagnostics
Nanomedicine - interventional
Whatever happens, it will be an interesting place to be…
To Whoever is still awake…
Thank you for your attention.