View
214
Download
0
Embed Size (px)
Citation preview
““OurOur generation generation may be the may be the last to have to accept death last to have to accept death and taxes as inevitable."and taxes as inevitable."
- Commander Shaun Jones - Commander Shaun Jones ,,US NavyUS Navy,, D DARPAARPA
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 Serious drug side effects (Vioxx, Hormone
replacement therapy)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 Lack of R&D productivity increase despite sustained
investmentsinvestments
• Increased regulatory demandsIncreased regulatory demands
• Unacceptable failure rates of R&D projects 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
•Predictive - Pharmacogenomics and Pharmacoproteomics
•Preventive - Acting proactively with preventive medicine
•Point of care - Mobile communications & ubiquitous computing
•Parametric - 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
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”).
• Begin colonoscopy at age 40
• Avoid high fat in diet
•Gene-based drug therapy for cancer
•Gene & Cell therapy for heart disease
• Drug dose of antidepressant determined by drug metabolism genetic profile
Risk prediction Pharmacogenomics New Therapies
Pharmacogenomics - Personalized Medicine
• 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
Current and Future BiotechnologyCurrent and Future Biotechnology
Stem Cells Therapy - Regenerative Medicine Multipotent Cells that can develop into any organ or tissueMultipotent Cells that can develop into any organ or tissue
Embryonic stem cells – controversial and technically difficult Embryonic stem cells – controversial and technically difficult
to manipulateto manipulate
Ideally - patient’s own stem cells, rejuvenated and then Ideally - patient’s own stem cells, rejuvenated and then
delivered into desired tissuedelivered into desired tissue
Stem cell transplants have been effective in treating cancersStem cell transplants have been effective in treating cancers
Stem cell therapy now being used for degenerative diseasesStem cell therapy now being used for degenerative diseases
Future of Stem Cell TherapyFuture of Stem Cell TherapyMultipotent 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, preventBring the ability to detect, prevent and treat disease down to the and treat disease down to the molecular levelmolecular 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
•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.
The Potential of Nanotechnology
NanotechnologyNanotechnology: : Information Meets the Physical WorldInformation 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
NanotechnologyNanotechnology: : Information Meets the Physical WorldInformation Meets the Physical World
• The Biological assembler
NanotechnologyNanotechnology: : Information Meets the Physical WorldInformation Meets the Physical World
•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.
http://www.foresight.org/Nanomedicine/Gallery/index.htmhttp://www.foresight.org/Nanomedicine/Gallery/index.htm
Nanobots in the bloodstream
http://www.foresight.org/Nanomedicine/Gallery/index.htmhttp://www.foresight.org/Nanomedicine/Gallery/index.htm
Nanobots
Nanobots in the bloodstream
http://www.foresight.org/Nanomedicine/Gallery/index.htmhttp://www.foresight.org/Nanomedicine/Gallery/index.htm
Clottocytes
Housekeeping Nanobots
Nanobots
http://www.foresight.org/Nanomedicine/Gallery/index.htmhttp://www.foresight.org/Nanomedicine/Gallery/index.htm
Respirocytes (mechanical RBC)
Neural Augmentation/replacement
Nanobots
http://www.foresight.org/Nanomedicine/Gallery/index.htmhttp://www.foresight.org/Nanomedicine/Gallery/index.htm
Surveillance and monitoring nanobots
Nanobots
1990 2000 2010 2020 2030
Gene Therapy I
Cell Therapy II
Advanced Tissue Engineering
Nanomedicine - interventional
Combined Gene & Cell Therapy
Nanomedicine - diagnostics
Advanced drug design – based on proteomics & genomics
TimelineTimeline
Cell Therapy I
Gene Therapy II
Whatever happens, it will be an interesting place to be…Whatever happens, it will be an interesting place to be…