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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 • • • • 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 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 Multipotent Cells that can develop into any organ or tissue Embryonic stem cells – controversial and technically difficult to manipulate Ideally - patient’s own stem cells, rejuvenated and then delivered into desired tissue 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 • • • • • • • 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.