Resume
Summary Molecular Therapy (NWI-BM078)
- Cours
- Établissement
Extensive summary of all lectures of the course molecular therapy (NWI-BM078).
[Montrer plus]
Vendeur
S'abonner
roos1397
Avis reçus
Aperçu du contenu
Molecular therapyPersonalized healthcare
Principle of personalized medicine
- The right therapy for right patient at right intensity at right time
- Molecular biomarkers as key drivers of patient selection
- = personalized healthcare, precision medicine
B-RAF and personalized medicine in melanoma
- B-RAFV600E cells always grow and become cancer cells → mutation in kinase domain that
binds ATP → mutation makes it always active
- RAF inhibitors will block pathway, block cell growth and inhibit cancers (in mice) that have B-
RAFV600E mutation
- 60% of melanoma patients have B-RAFV600E mutation
- Basis of a personalized medicine
Biomarkers
- A characteristic that is objectively measured and evaluated as an indicator of normal
biological processes, pathogenic processes, or pharmacologic responses to a therapeutic
intervention
- Or ‘whatever works in adding value’
- Molecular biomarkers provide a molecular impression of a biological system (cell, animal,
human)
- Biomarkers can be various sorts of data, or combinations thereof
Biomarker data-based translational medicine in pharma
- Exposure – Does the compound get to the site of action?
- Mechanism – Does the compound cause its intended pharmacological/functional effects?
- Efficacy – Does the compound have beneficial effects on disease or clinical pathophysiology?
- Safety – What is the therapeutic window (how safe is the drug)?
- Responders – How do sources of variability in drug response in target population affect
efficacy and safety?
Pharma strategy based on personalized medicine
- Right target
- Right tissue
- Right safety
- Right patients
- Right commercial potential
1
,Role of molecular (omics) biomarker data in personalized healthcare
The power of omics in diagnostics
- Increase diagnostic yield
- Put biomarker change in context
Personalized healthcare in rare metabolic disorders
- Technology innovation is driving impact in personalized healthcare
- Crucial to combine different molecular views to understand human health and disease (X-
omics)
- Fast translational of biomarker research to implementation needed
- On personalized diagnosis
o Metabolic screening adds strongly to genetic screening in identifying mechanisms of
disease
- On personalized medicine
o Impressive effects of uridine therapy
o Increase quality of life for patient and family
o Frequent issues regarding expensive medication versus cheap supplements
- Advantages in using holistic omics methods in clinical diagnostics
Moving to personalized health(care)
- People are more than linear pathways
- Different genetic make-up and lifestyles
- Different environments
- Different risk factors
- Different preferences
3 key aspects of personalized health(care)
- What to measure?
- How much can it change?
- What should be the follow-up for me?
3 translational innovation gaps
- Research to research
- Research to diagnostics
- Research to society
2
,Translation is key in personalized healthcare
Personalized health(care) model
Afterthought: there is no single one reflection of health
- Funhouse mirror effect
- Multiple sources of your data
o Multiple omics
o Clinical chemistry
o Electronic patient dossier
o Wearables
o Digital biomarkers
o Commercial health tests
o Social media
o Surrounding
- Each are a skewed image of you
- How to deal with all of this for your personal health(care)?
Pharmacology and drug disposition
Pharmacology
- The science that is concerned with the use, effects, and modes of action of chemicals on the
function of living system
Pharmacotherapy
- What does the patient with the drug? → pharmacokinetics
3
, - What does the drug with the patient? → pharmacodynamics
Rational vs. evidence-based pharmacotherapy
- Rational pharmacotherapy → mechanism-based pharmacotherapy
- Evidence-based pharmacotherapy → it does not matter how it works, as long as it works
Pharmacological phases in pharmacotherapy
- Exposition phase → behavior of a substance in the environment, changes in the application
form, available for uptake
- Toxicokinetic phase → absorption, distribution, metabolism (toxification, detoxification),
excretion (ADME)
- Toxicodynamic phase → interactions with receptors or other (macro) molecules at the site
of the operation
Classification of pharmacological effects
Classical drug-receptor-interactions
4
Les avantages d'acheter des résumés chez Stuvia:
Qualité garantie par les avis des clients
Les clients de Stuvia ont évalués plus de 700 000 résumés. C'est comme ça que vous savez que vous achetez les meilleurs documents.
L’achat facile et rapide
Vous pouvez payer rapidement avec iDeal, carte de crédit ou Stuvia-crédit pour les résumés. Il n'y a pas d'adhésion nécessaire.
Focus sur l’essentiel
Vos camarades écrivent eux-mêmes les notes d’étude, c’est pourquoi les documents sont toujours fiables et à jour. Cela garantit que vous arrivez rapidement au coeur du matériel.
Foire aux questions
Qu'est-ce que j'obtiens en achetant ce document ?
Vous obtenez un PDF, disponible immédiatement après votre achat. Le document acheté est accessible à tout moment, n'importe où et indéfiniment via votre profil.
Garantie de remboursement : comment ça marche ?
Notre garantie de satisfaction garantit que vous trouverez toujours un document d'étude qui vous convient. Vous remplissez un formulaire et notre équipe du service client s'occupe du reste.
Auprès de qui est-ce que j'achète ce résumé ?
Stuvia est une place de marché. Alors, vous n'achetez donc pas ce document chez nous, mais auprès du vendeur roos1397. Stuvia facilite les paiements au vendeur.
Est-ce que j'aurai un abonnement?
Non, vous n'achetez ce résumé que pour €5,99. Vous n'êtes lié à rien après votre achat.
Peut-on faire confiance à Stuvia ?
4.6 étoiles sur Google & Trustpilot (+1000 avis)
79202 résumés ont été vendus ces 30 derniers jours
Fondée en 2010, la référence pour acheter des résumés depuis déjà 14 ans