June 2020

Until recently, pharmaceuticals were dominated by a single class of drugs, small molecules. Over the space of 25 years, advances in molecular biology have lead to the development of many new drug classes. Here’s my overview of pharmaceuticals, either marketed or in development, based on molecular entity, and roughly in order of the drugs physical size.

Macrophages are extraordinary cells. Not just for their phagocytic capacity, which is also shared by a few other cells, but also for their ability to dynamically adapt their function to intervene in unfolding events.

PD is a progressive degenerative neurological disease that affects about 1% of the population over age 55. Although there are many variants of the disease, a common strand that links these variants is damage to a finite group of dopaminergic (DA) neurons in the midbrain. As these neurons are lost in PD, CNS function and in particular its control over normal muscular function declines. Tremor is a well-known symptom which characterizes PD, but sleep, digestion, cognition, and mobility are also affected with increasing severity as the disease progresses. Current therapies, such as dopamine replacement therapy, only address symptoms and there is an urgent need for a disease-modifying therapy.

Cultured cells often require the addition of specific growth factors to enable proliferation, differentiation or survival. However, some recombinant proteins are expensive to use. This is either because they are difficult to make (and therefore costly) or they are simply needed in large amounts.

Everyone working in regenerative medicine today is standing in the shoulders of giants. The tools scientists have developed enable progress in the field and the creation of new therapies. Here’s my list of the regenerative medicine’s top molecular tools.