[Disclaimer: I am an employee of Celgene. The views reported here are my own.]
On a recent family vacation to Cumberland Island, a 9,800-acre barrier island off the coast of Georgia, I was mesmerized by the dense forest of live oak trees covered with Spanish moss. Upon first glance, the branches of these magnificent trees extend chaotically in all directions, and it is difficult to discern where the trees begin and end. But upon closer inspection, the root structure can be identified, moss disentangled, and the overall complexity unraveled.
These craggy oak trees serve as metaphor for our complex human biological ecosystem: a dense forest of molecules with gnarled branches of pathways meandering in all directions, without an obvious root structure of human disease. Extending the metaphor further, the oak trees make the point that I see as one of the most difficult aspect of drug discovery and development: understanding root cause of disease, and matching therapeutic modality and biological mechanism to prevent or cure devastating illness.
In this blog, I highlight two recent publications that underscore the importance of matching modality and mechanism. The first article, published in the New England Journal of Medicine, reported clinical data on 22 patients with beta-thalassemia treated with ex vivo gene therapy (here).…
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Last week Alnylam reported positive news on Phase 3 outcomes for their RNA interference (RNAi) therapy to treat patients with a rare genetic cause of amyloidosis with polyneuropathy (see here). I tweeted the following:
The 20-year journey from scientific discovery to positive Phase 3 clinical trial data got me thinking about other novel therapeutic modalities. Was twenty years a long time or typical for an innovative therapeutic modality? Where are other promising modalities on their journey to regulatory approval? Is the biopharmaceutical industry on the cusp of a series of innovative modalities that could change the therapeutic landscape for patients? How will these new modalities improve our ability to test therapeutic hypotheses?
[Disclaimer: I am an employee of Celgene. The views expressed here are my own.]
To explore these questions, I decided to review different novel therapeutic modalities, which I am defining as those other than small molecules, protein therapeutics (e.g., insulin) and traditional vaccines. This decision was practical, as the amount of literature in these modalities is expansive.
For each new modality, I asked whether a drug has been approved by either a European or US regulatory agency (EMA and FDA, respectively). If a drug has been approved, I reviewed the time from seminal scientific discovery (which sometimes is clear, sometimes is not) to the approved therapy.…
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