Samenvatting
Samenvatting Les 2 'Drug discovery process, therapeutic modalities'
Dit document omvat de samenvatting van Les 2 (info slides met notities) van het vak 'Preclinical drug research'.
[Meer zien]Voorbeeld 2 van de 9 pagina's
Voorbeeld 2 van de 9 pagina's
In winkelwagenVerkoper
Volgen
Voorbeeld van de inhoud
Lecture 2: 2/10Therapeu(c modali(es
Types of therapeu.cs
- Several therapeu-cal modali-es available
- Spectrum becomes broader
- Conven-onal = all types of interven-on aimed at allevia-ng and eradica-ng the effects of
disease
o Improve disease symptoms and/or prognosis
o Alleviate effects of exis-ng disease
o Directed towards disease preven-on
o Achieve permanent cure
- Non-conven-onal
o Nutraceu-cals = range of dietary prepara-ons
§ slimming diets, diets with minerals, vitamins, fiber, an-oxidants, …
§ some scien-fic ra-onale
§ not subjected to formal regulatory approval
§ Ceu-cals are oGen not regulated. Sold you as products to help to lose weight, for
example
§ In the end you can’t be sure what the effect/benefit is of nutraceu-cals
§ Also safety is not sure
o Cosmeu-cals = cosme-cs supplemented with some ac-ve (?) substances
§ reduce skin wrinkles, promote hair growth, …
§ liNle or no scien-fic ra-onale
§ free sales (but major market !!)
§ Free sale -> major market -> lot of publicity
Current therapeu.cs
Conven&onal therapeu&c drugs
- Small molecules groups
o Most of current drugs (Pharmacopoeia)
o Chemicals
o Small molecular size -> important for the phamacokine-cs, oral uptake
o Small molecular drugs/en--es
o Advantages
§ Several targets -> also a disadvantage -> less specific, side/adverse effects
§ Accepted by profession and community
§ Various routes of administra-on
§ Longterm pharmaceu-cal experience
o Disadvantages
§ It becomes more difficult to find new small molecule drugs that differen-ate
them from others
§ Broader spectrum -> lower selec-vity -> toxicity
§ Pharmacokine-c limita-ons
, • Phase I clinical trial and you need a certain plasma concentra-on -> stop
with development or change the formula-on to increase the plasma
concentra-on (and efficacy)
§ Frequent poor oral absorp-on -> improve this by adding extra substances ->
vehicles are used for this (some of this vehicles are toxic by themselves)
- Natural products or semi-synthe-cally derived)
o an-bio-cs, an-cancer, opiates, sta-ns,
Biopharmaceu&cals
- = Noval therapeu-c modali-es
- = Non small molecule drugs
- Several groups
o Pep-de/protein mediators
o Blood cloZng factors
o Enzymes
o An-bodies (an-sera, an-toxins, mAb)
o Vaccines (inac-vated, aNenuated, subunit)
o Cells/-ssues (stem cells, graG- and transplant surgery)
- Advantages
o Higher selec-vity than small molecules drugs -> open new therapeu-cal op-ons
o More straigh]orward discovery
§ You really focus on the target and design it for the target -> less toxicity
o Unexpected toxicity is less common
o Higher product quality
- Disadvantages
o Much more expensive
o Lack of oral ac-vity and short half-life
o Designed for the human pharmacological target -> no good animal model
Beyond current biopharmaceu.cals …
Gene therapy
- Correc-ng gene-c defects by altering gene-c material of cells by recombinant DNA
technology
o Introduce new genes to replace missing or dysfunc-onal ones (virus vector: non-
integrated vs. integrated DNA)
o Aim at single-gene disorders (cys-c fibrosis, haemophilia) and cancer
o Restricted to soma-c cell treatments (ban on germ-cell gene therapy experiments
- Not many gene therapy products licensed yet for clinical use
- Disappoin-ng prospects (long-term safety, gene-delivery systems, …
- Become more and more important
- OGen a rare disease -> orphan drug
- Several ways of working with this
- Broad term: for example you are going to block the transcrip-on of the gene and later the
transla-on of the mRNA to the proteins
- An-sense DNA
o oligonucleo-de sequence complementary to part of a known mRNA sequence ->
selec-ve blocking of expression
o but:
Voordelen van het kopen van samenvattingen bij Stuvia op een rij:
√ Verzekerd van kwaliteit door reviews
Stuvia-klanten hebben meer dan 700.000 samenvattingen beoordeeld. Zo weet je zeker dat je de beste documenten koopt!
Snel en makkelijk kopen
Je betaalt supersnel en eenmalig met iDeal, Bancontact of creditcard voor de samenvatting. Zonder lidmaatschap.
Focus op de essentie
Samenvattingen worden geschreven voor en door anderen. Daarom zijn de samenvattingen altijd betrouwbaar en actueel. Zo kom je snel tot de kern!
Veelgestelde vragen
Wat krijg ik als ik dit document koop?
Je krijgt een PDF, die direct beschikbaar is na je aankoop. Het gekochte document is altijd, overal en oneindig toegankelijk via je profiel.
Tevredenheidsgarantie: hoe werkt dat?
Onze tevredenheidsgarantie zorgt ervoor dat je altijd een studiedocument vindt dat goed bij je past. Je vult een formulier in en onze klantenservice regelt de rest.
Van wie koop ik deze samenvatting?
Stuvia is een marktplaats, je koop dit document dus niet van ons, maar van verkoper evagoormans. Stuvia faciliteert de betaling aan de verkoper.
Zit ik meteen vast aan een abonnement?
Nee, je koopt alleen deze samenvatting voor €7,16. Je zit daarna nergens aan vast.
Is Stuvia te vertrouwen?
4,6 sterren op Google & Trustpilot (+1000 reviews)
Afgelopen 30 dagen zijn er 84251 samenvattingen verkocht
Opgericht in 2010, al 14 jaar dé plek om samenvattingen te kopen