Study Guide: Natural Product Drug Discovery

Single chemical entities isolated from biological sources (plants, bacteria, fungi) with relatively low molecular weight (below 2,000 g/mol).

Drugs created in labs, based on biological blueprints or completely synthesized.

The process of developing natural product drugs takes over a decade to meet FDA approval.

Natural product drugs are single agents (pure substances). Herbal remedies are mixtures of substances.

Approved drugs include small molecule drugs like natural products and synthetic drugs, along with vaccines and biologics.

Natural products are sometimes modified to improve their therapeutic effect.

Drugs that resemble the shape and structure of naturally occurring molecules, often used in drug discovery

Natural products are a significant source of drug inspiration and development, with over 50% of small molecule drugs based on them.

Natural products have evolved over millions of years, leading to structures optimized for their environment and effective interactions

Natural products tend to have more complex molecular structures than synthetic drugs, often with larger size and more chiral centers, leading to high affinity interactions with targets

Natural products occupy a larger chemical space compared to synthetic compounds due to their higher complexity and diversity

Obtaining sufficient amounts of natural products for research and development can be challenging, often needing large quantities depending on potency and production stage

A method to isolate active compounds from complex mixtures using testing during separation and purification by chromatography, targeting specific desired characteristics

The vast unexplored ocean environments hold immense microbial diversity and, therefore, untapped potential for novel drug discovery

Different organisms produce unique chemical structures. Knowing this helps predict where to look for new drugs.

An anti-cancer molecule found in a new genus of cyanobacteria (Caldora penicillata).

Extracting active compounds, determining their biological activity, identifying their targets and mechanisms of action, optimizing them, then developing the drug for clinical trials.

Issues like reliably obtaining the natural product, discovering if it works, determining if it's safe, and making enough of it (supply)

Using an organism's genetic information to find genes responsible for making valuable molecules.

Producing a molecule in a different organism (often in a lab setting) to make more of it quickly and efficiently.

Taxol is derived from the Pacific Yew tree.

Morphine is derived from the opium poppy.

Aspirin is derived from the willow tree.

Vincristine is derived from the Madagascar Periwinkle.

A class of natural products characterized by their branched 5-carbon unit structure. Often found in plants, they contribute to aromas and pigments.

Short chains of amino acids linked together by peptide bonds (amide linkages).

A diverse group of natural products formed by the condensation of acetate units. They often contain ketone and methylene groups.

Compounds containing a sugar molecule (glycone) attached to a non-sugar molecule (aglycone) through a glycosidic bond.

Naturally occurring nitrogen-containing compounds with basic properties, often found in plants. They have a lone pair of electrons on nitrogen.

Geldanamycin belongs to the polyketide class of natural products. It contains ketone and methylene groups in its structure.