What is screening?
Screening means sorting amongst known natural molecules or synthetic substances using tests to identify those which are most likely to have the desired activity.
These tests can be performed on human cell cultures in vitro or animal models such as C. elegans, a nematode worm which shares more than half its genome with humans.
Today, most screening platforms perform high-throughput robotic screening. This consists in passing as many molecules as possible through a biological test as quickly as possible. Today, this high-throughput corresponds to one molecule per second. These molecules are set out on ready-to-use plates with a "96 well" format (8 rows x 12 columns).
A screening platform for neuromuscular diseases
AFM-Téléthon supports a large pharmacological screening platform for neuromuscular diseases (CNRS, Lyon and Inserm, Paris). In 2007, 20 molecules were identified which are of potential interest for Duchenne muscular dystrophy and are currently being validated in mice.
Other platforms have been set up with AFM-Téléthon’s support, such as I-Stem, with a "new generation" molecular screening robot. This robot, which is unique in France, can perform 10,000 to 40,000 tests per screening campaign! An initial test of therapeutic screening, performed in 2009, tested 1,500 molecules and small interfering RNAs (siRNA) for use in myotonic dystrophy (Steinert's disease).
Pleotherapy and network pharmacology
AFM-Téléthon is supporting the French biotech company Pharnext (with a repayable advance) to develop its "Pléothérapie™" platform. This platform is based on network pharmacology. It is based on the principle that a biological function depends on intricate molecular networks and an anomaly in this function is more likely to be treated with a combination of molecules than with a single drug. It therefore intends to examine the molecule network that is disturbed in a given disease, then screen the molecules which could act on this network. This strategy is being evaluated using animal models for Charcot-Marie-Tooth disease.