Limitation of animal models in drug discovery and development Candice Tahimic, Ph.D. Hence, there is...

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Limitation of animal models in drug discovery and development Candice Tahimic, Ph.D. Hence, there is a need to use human cells to test the efficacy of candidate drugs. Animal cells may not always simulate complex biochemical mechanisms of human disease Is the observed disease phenotype in the animal equivalent to that of the human counterpart?

Transcript of Limitation of animal models in drug discovery and development Candice Tahimic, Ph.D. Hence, there is...

Page 1: Limitation of animal models in drug discovery and development Candice Tahimic, Ph.D. Hence, there is a need to use human cells to test the efficacy of.

Limitation of animal models in drug discovery and development

Candice Tahimic, Ph.D.

Hence, there is a need to use human cells to test the efficacy of candidate drugs.

Animal cells may not always simulate complex biochemical mechanisms of human disease

Is the observed disease phenotype in the animal equivalent to that of the human counterpart?

Page 2: Limitation of animal models in drug discovery and development Candice Tahimic, Ph.D. Hence, there is a need to use human cells to test the efficacy of.

Midbrain dopaminergic neurons in the substantia nigra are selectively lost in Parkinson’s disease

Human ES or iPS cells can be converted into dopaminergic neurons for use as cell models of Parkinson’s disease

Certain psychiatric disorders and certain addictions (nicotine and cocaine dependence) are thought to be mediated by the dopaminergic system

Candice Tahimic, Ph.D.

Page 3: Limitation of animal models in drug discovery and development Candice Tahimic, Ph.D. Hence, there is a need to use human cells to test the efficacy of.

Human cell models for disease: the promise of hES/iPS cells

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Test compound

Rep

orte

r le

vels

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BC

DE

“On-dish” assay for efficacy

Chemical library screening

Dirrected differentiation

Candice Tahimic, Ph.D.

Human ES cell

Neurons

The iPS or hESC -derived neurons are treated with a number of candidate drugs for a particular disease. The neurons are then observed for reversal of the disease phenotype. Promising test compounds are further validated.