SHARING BIG IDEAS

MEDICINAL CHEMISTRY
A new enthusiasm has been kindled for what is, in principle, an old technique: the use of DNA tags to encode individual organic chemicals within a large “pool” of compounds. The sizes of the collections that can be made using so-called “split and mix” techniques are extraordinary and the first reports of hit compounds have appeared in the literature. There are still challenges, of course, but it seems likely to be a “hot topic” in medicinal chemistry.

 David Clark, Director of Computer-Aided Drug Design and Information Services, Early Discovery

METABOLIC DISORDERS
Two years ago, few drug companies were studying NASH (non-alcoholic steatohepatitis), a chronic liver disease associated with diabetes and obesity. Today, at least 34 companies are eyeing this growing public health problem, with the novel compound, obeticholic acid (OCA), granted FDA breakthrough status earlier this year.  NASH is a hot area right now, but a single agent likely won’t be sufficient. Disease biomarkers and/or imaging models are also clearly needed to assess NASH.

 Joe Cornicelli, Scientific Advisor, Discovery Research.

CYSTIC FIBROSIS
Organoid cultures, mini-organs able to reflect the complex cell biology of a patient’s own patient’s own tissue, are a hot topic in drug-based therapies for cystic fibrosis  (CF).  Approximately 2000 different mutations in a single gene cause CF, making it difficult to assess which targeted therapies work best in which patients. Since organoid cultures can stably recapitulate the patients’ diseased tissues in vitro they have wide application for the understanding and treatment of CF

Mary McElroy, Principal Scientist, In Vivo Discovery/Cell Therapy.