Diapositiva 1

How aremedicinesdeveloped?

What is it?

What’s inside?

What is inside a medicine?

Active ingredient

It looks for thetherapeutic target tohave an effect and curethe disease

Therapeutic target

Therapeutic target

Active ingredient

It looks for thetherapeutic target tohave an effect and curethe disease

How are medicines developed?

The search for a medicine is a long (10-25 years),complex and expensive process consisting of severalstages.

DRUG

R&D

(2-10 years)

CLINICAL DEVELOPMENT

PHASE I  PHASE II  PHASE III

Launch

of the

drug

PHASE IV

Launch

of the

drug

PHASE IV

DEVELOPMENT SEQUENCE IN THE SEARCH FOR A MEDICINE

PRECLINICAL

STUDIES

(2-6 years)

In vitro/In vivo

•A drug candidate can be discovered in differentways:

HOW IS A DRUG CANDIDATE DISCOVERED?

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A drug candidate can be discovered in different ways:

Chemical modification

Serendipitychance

Massive screening

Rational design

HOW IS A DRUG CANDIDATE DISCOVERED?

WE NOW HAVE A DRUG CANDIDATE!

•A thorough and complete preclinical study is requiredbefore we can test a drug in humans:

•These preclinical studies include:

–Stability and toxicity studies

–In-vitro tests (proteins, cells, tissues and organs)

–In-vivo tests (animals)

PRECLINICAL DEVELOPMENT

©Parc Científic Barcelona. Author: J. Planagumà

•Clinical development is the longest and most costly part ofthe search for new medicines. It consists of three phases:

PHASE I

PHASE II

PHASE III

Healthy volunteers

Safety anddose

Patients

(100-300)

(drug or placebo)

Efficacy and

side

effects

Patients

(1300-3000)

Long-term

effects

CLINICAL DEVELOPMENT

•If the drug has successfully passed the three clinicalphases, it is launched on the market, but the studycontinues (Phase IV)

PHASE II

PHASE III

PHASE IV

PHASE I

MARKET

AND FINALLY… THE DRUG IS LAUNCHED ON THE MARKET!

SEARCH for DRUGS forPARKINSON’S DISEASE

The Barcelona Science Parkresearch project

•Parkinson’s disease (PD) is a chronic and progressivemovement disorder characterised by:

–Coordination problems

–Slowed movements

–Generalised tremor

•It is the second most common neurodegenerativedisease and affects 1-2% of people over 60. There arecurrently more than 4 million people suffering from PDworldwide.

INTRODUCTION – WHAT IS PARKINSON’S DISEASE?

•Our brain is our body’s control centreand the cells responsible for it arecalled neurons. These cells self-regenerate very slowly.

•Neurotransmitters are special chemicalcompounds that enable neurones to“talk to each other” and communicate.

INTRODUCTION – PARKINSON’S DISEASE, CAUSES

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•We still do not know what causesParkinson’s disease, but we do know thatit is due to the loss or incorrectfunctioning of the neurons responsible forproducing dopamine, a neurotransmitter.

•Dopamine is responsible for transmitting the signals required tocontrol the movement of our muscles.

•A dopamine deficiency therefore leads to an imbalance in neuronaltransmission, preventing neurons from communicating properly. Thisleads to a loss of motor function.

• Dopamine  Control of movement

Low dopamine levels  Difficulties in the control

of movement

• Dopamine  Control of movement

Low dopamine levels  Difficulties in the control

of movement

INTRODUCTION – PARKINSON’S DISEASE, CAUSES

•The way to treat the disease’s progressionis by taking oral medicines.

•The most commonly used medicinetoday is levodopa, or L-dopa, whichconsists of a chemical compoundthat the brain uses to producedopamine.

INTRODUCTION – PARKINSON’S DISEASE, TREATMENT

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tyrosine

L-dopa

dopamine

vesicles with thedopamine transmitter

dopaminereceptor

receptor cell

synapse

•Other medicines, which imitate the effect ofdopamine in the brain, are also used.

–e.g. bromocriptine, lisuride, pergolide, ropinirole, etc.

receptor

imitator

Cellmembrane

dopamine

Cell response

INTRODUCTION – PARKINSON’S DISEASE, TREATMENT

•Unfortunately, these medicines have a large number ofside effects:

–Appearance of involuntary movements and tics

–Depression

–Hallucinations

•These medicines also cease to be effective with time.

•We need to produce new medicines with less side effects thatare active for longer periods.

INTRODUCTION – PARKINSON’S DISEASE, TREATMENT

•In the Barcelona Science Park, on theCombinatory Chemistry Platform, scientistsare working on the synthesis of newcompounds that can be used as therapeuticagents in the treatment ofneurodegenerative diseases such asParkinson’s or Schizophrenia that are:

–More active

–Less toxic