Meltem Kutluer Talk 2 Therapeutic Challenges for Inherited Retinal Degenerations by Li Huang Talk 3 The TransMed consortium by Jiaming Zhou Session 2 Talk 4 How and where to test drugs by Arianna ID: 1047405
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2. Session 1Talk 1: “Taking a Closer Look at the Eye and Retinal Degenerations”, by Meltem Kutluer.Talk 2: “Therapeutic Challenges for Inherited Retinal Degenerations”, by Li Huang.Talk 3: “The TransMed consortium.”, by Jiaming Zhou.Session 2Talk 4: “How and where to test drugs”, by Arianna Tolone.Talk 5: “The journey of discovering new potential therapeutic targets in the retina.”, by Michel Rasmussen.Talk 6: “Biomarkers in ocular diseases.”, by Akanksha Roy.Talk 7: “Drug Delivery Systems – Part 1: Vehicles for the drug to reach its destination”, by Erico Himawan.Session 3Talk 8: “A Day in the Life of a Chemist: Developing a drug and its synthesis.”, by Oswaldo Perez.Talk 9: “Drug delivery systems – Part 2: How are drugs administered?”, by Dileep Urimi.Talk 10: “Last but not least: Clinical trials – Part 1.”, by Soumaya Belhadj.Talk 11: “Last but not least: Clinical trials – Part 2.”, by Laura Lorenzo.
3. Retinal degeneration： Therapies and ChallengesLi Huang
4. Retinal degenerationInherited retinal degeneration Retinitis pigmentosaMultifactorial retinal disorder Age-related macular degeneration
5. Current standing of therapies Gene therapyCell therapyDrug therapyRetinal Prostheses…….
6. Heterogeneity of genesDisease mechanismsDelivery limitationWindow of therapeutic interventionCost…… Challenges
7. Scientists Clinicians PatientsPatient organizations Funding agencies Industry…….Combined efforts
8. Thank you!
9. transMed
10. Basic researchMaking a drugGoing to clinic transMed consortium
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13. Talks resuming at 12.40!
14. How and where to test drugsArianna Tolone
15. The journey of a drug for eye disease:123
16. Photoreceptor-like cellswarm + special nutrients =cell extracted from mouse retina 1 Cell cultures:
17. dying Photoreceptor-like cellsDrug Which drug is the best? How much drug?
18. 2 Retinal explants:eyemouse witheye diseaseretina in a flower shaperetina in a dish
19. mouse with a mutationWhy retinal explants?1. Labelled dead cells2. Count and compareDRUGNO DRUGreal photoreceptors in a real tissue
20. 3 Animals4 Clinical trials
21. Thank you!any question?
22. The journey of discovering new potential therapeutic targets in the retina- Michel Rasmussen
23. Why do we need new targets?Understand the mechanism behind RDTherapeutic targets
24. cGMPThe cGMP level is increased in RP
25. Finding targets is like fishing
26. Question 1What tissue should we use?
27. Question 2What protein should we look for?
28. Question 3How do we find the proteins in the tissue?
29. TissueEntire retina
30. BaitcGMP
31. MethodAffinity Chromatography
32. MethodMass Spectrometry
33. The journey of discovering new potential therapeutic targets in the retina
34. Presented by- Akanksha Roy PhD candidatePamGene International BV, The NetherlandsBiomarkers in ocular diseases
35. What is a Biomarker? 35
36. Where are the Biomarkers? 36
37. Importance of Biomarkers 37
38. My PhD project 38Image- Arianna Tolone
39. Thank you! Any questions?39
40. Drug Delivery Systems – Part 1: Vehicles for the drug to reach its destinationERICO HIMAWANEarly Stage Researcher – Project transMed
41. Contents:WHY do we need drug delivery system? HOW they bring benefit to formulation scientist and patients?WHAT are the examples?
42. WHY do we need drug delivery system? Why we need it? The key is in the word DELIVERYRegardless of delivering a love letter, a package, a Christmas present, a person, or a drug molecule…In the process of delivery, there is always MOVEMENT involved from point A to point B
43. WHY do we need drug delivery system? Imagine you want to have Christmas holiday in Prague… Depending on where you are, you can:Drive a car?Ride a train?Fly in airplane?All these transport system can deliver you to your holiday destination! It’s the same with drug molecule… you use special “vehicles” to help it reach its destination.
44. HOW drug delivery system bring benefit to formulation scientist and patients?Benefit? Simple... Imagine again that you are going to Prague for Christmas holiday, but this time you are walking! No car, no train, no airplane!What do you think will happen? Unless you are really close to the border of Prague – you may not reach itNow, using drug without proper delivery system is the same with walking instead of riding a vehicle to reach a destination..It’s possible, but may cost you too much time, energy and still have high chance of failure.
45. HOW drug delivery system bring benefit to formulation scientist and patients?With a proper delivery system, formulation scientist can help drug to reach the target faster and in more efficient way.This may leads to higher chance for treatment to be successful, and in many cases, help reduce the cost of treatment due to increase in efficiency!
46. WHAT are examples for “vehicles” for drug?Just like there are many types vehicles to help you travel to Prague (e.g. car, train, airplane) - there are many types of vehicles that can help drug reach its target…Some examples for these vehicles includes but not limited to:Liposomes, solid lipid nanoparticles, and cyclodextrin nanoparticles.One type maybe better than the other depends on the circumstances! There is no such thing as perfect delivery system
47. THANK YOU!
48. Talks resuming at 13.30!
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50. A Day in the Life of a (Process) Chemist
51. Looking backDrugIRDsThe retinaChallengesTargeting disease with drugsTesting drugsHelping drugs reach target
52. What IS a Drug?Definition:Causes biological effect = The DriverTreats, cures, prevents, diagnoses.Really:A molecule with a unique structure.
53. Our drugStructure:Biological Effect: Prolongs the life of photoreceptor cells
54. Process Development:Best possible recipe for drug.A Process should be: Scalable
55. Process Development:Best possible recipe for drug.A Process should be: Safe
56. Cooking pasta:Good process?
57. Thank you!
58. Drug delivery systems – Part 2: How are drugs administeredDileep UrimiPhD studentRISE Research Institutes of Sweden
59. Routes of drug administrationSimply, it is the way drugs are introduced into the bodyExamplesTablets, capsules, syrups – Oral routeInjections – Injection/Parenteral routeOintments/creams – transdermal route
60. Each route has its own purpose Selection mainly depends on Body siteType and severity of diseasePatient comfort Type of drug and drug formulationWhy different routes? How to select best route?
61. Drug delivery to the eyeRoutine injection to armEye dropsInjections into the eyeComfort may be compromised by requirement in case of retinal drug delivery
62. Thank You
63. Last but not least: Clinical Trials - Part 1Soumaya Belhadj
64. “Clinical trials are a type of research that studies new tests and treatments and evaluates their effects on human health outcomes”
65. James Lind (1747): 1st Systematic Clinical TrialCider Elixir of vitriol 25 drops Vinegar 6 spoonfulsSeawater½ a pint 2 oranges 1 lemonSpicy pasteBarley waterRecovered!!Improvement!!
66. Methodology John Haygarth: Importance of a control group for identification of the placebo effectRonald A. Fisher: Importance of randomization, replication, blocking 1946-1947: First randomized trial: Streptomycin for pulmonary tuberculosis
67. Ethical and regulatory framework Hippocratic Oath: prime duty of a physician: avoid harming the patientNuremberg Code (1947): Informed Consent1862: Food and Drug Administration1995: European Medicines Agency
68. Pre-clinical PhaseAnimal studiesSubmitted for approval if the drug is to be further tested in human subjects Drug’s safety in doses equivalent to approximated human exposuresPharmacodynamics and pharmacokinetics
69. Last but not least: Clinical Trials - Part 2
70. Clinical PhasesGlobal Medical Institute
71. Clinical Phase I - SafetyFirst evaluation of safety in humansWhat is the safe dose of the drug?Are there any side effects?Weeks or months
72. Clinical Phase II - EffectivenessLarger group of participants The drug is safe Further monitoring of side effectsSeveral years
73. Clinical Phase IIIDifferent population and dosesCombination with other drugsCompared with common treatmentsPositive results for FDA approval
74. Clinical Phase IVDrug has been approvedIdentify less common adverse reactionsEvaluate cost-effectiveness4% of drugs are withdrawn
75. Participation in clinical trialsAccess to new treatmentsRegular medical attentionContribution to scientific and medical progressPotential access to low cost or free treatmentsBenefits
76. Participation in clinical trialsRandom assignation of treatmentNon-effectives of the treatmentNew or more severe side effectsFrequent doctor visits and monitoringRisks