Stories about: #CHBrare

Reinventing therapeutic development: Systems pharmacology

As Children’s Hospital Boston holds its 2nd Annual Rare Disease Symposium today, here’s a reminder that drug development is incredibly difficult: many initially promising drugs ultimately prove ineffective or have unacceptible toxicity—typically at a late stage of a clinical trial. But it needn’t be that way. In this 10-minute video, introducing its Initiative in Systems Pharmacology, Harvard Medical School makes the argument that focusing on a drug’s action on a single cellular target is a flawed approach — rather, we need to understand and predict its activity in all body systems.

Today’s symposium, co-sponsored by the Technology and Innovation Development Office and the Manton Center for Orphan Disease Research, will address therapeutic development and for rare, often fatal diseases, focusing on moving discoveries to clinical trial and models for partnerships between academia, industry, the FDA and NIH, foundations and patient advocacy groups. Use this link to watch the proceedings live starting at 9 a.m. You can also follow the symposium on Twitter, using the hashtag #CHBRare. Click here for the full agenda.

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