The Future of Medicine

1. The Future of Medicine Danny Belkin

2. “Our generationmay be the last to have to accept death and taxes as inevitable." - Commander Shaun Jones ,US Navy, DARPA

3. Contents • Challenges to “traditional” big pharma, their future • Pharmacogenomics • Current and Future Biotechnology • Stem Cell Therapy • Microtechnology and nanotechnology

4. 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

5. …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

6. 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

7. Pharmacogenomics - Personalized Medicine • Both preventative and therapeutic • Single nucleotide polymorphisms (SNPs) -Only a fractionof 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”).

8. Pharmacogenomics - Personalized Medicine Risk prediction Pharmacogenomics New Therapies • Drug dose of antidepressant determined by drug metabolism genetic profile • Begin colonoscopy at age 40 • Avoid high fat in diet • Gene-based drug therapy for cancer • Gene & Cell therapy for heart disease

9. 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

10. 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

11. 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

12. Microtechnology and Nanotechnology Bring the ability to detect, preventand treat disease down to the molecular level

13. 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

14. First Microtechnology Pioneers From: Wired 14.12

15. 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.

16. 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

17. Nanotechnology: Information Meets the Physical World • The Biological assembler

18. 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.

19. Nanobots in the bloodstream http://www.foresight.org/Nanomedicine/Gallery/index.htm

20. Nanobots http://www.foresight.org/Nanomedicine/Gallery/index.htm Nanobots in the bloodstream

21. Nanobots http://www.foresight.org/Nanomedicine/Gallery/index.htm Clottocytes Housekeeping Nanobots

22. Nanobots http://www.foresight.org/Nanomedicine/Gallery/index.htm Neural Augmentation/replacement Respirocytes (mechanical RBC)

23. Nanobots http://www.foresight.org/Nanomedicine/Gallery/index.htm Surveillance and monitoring nanobots

24. Timeline 1990 2000 2010 2020 2030 Gene Therapy I Gene Therapy II Cell Therapy I Cell Therapy II Combined Gene & Cell Therapy Advanced Tissue Engineering Advanced drug design – based on proteomics & genomics Nanomedicine - diagnostics Nanomedicine - interventional

25. Whatever happens, it will be an interesting place to be…

26. To Whoever is still awake… Thank you for your attention.