Perlstein’s real endgame

Each week, Symposium Magazine provides a blogging forum for the week’s featured article. Today, Euny Hong writes more about her piece  on Ethan Perlstein. You can also follow her on Twitter @euny.One of the most interesting things about writing this piece and watching the reactions is seeing how many readers have lashed out at Ethan Perlstein for supposedly being elitist and privileged. Just as one example, check out the comments to this post on his own web site.

In an attempt to steer the discourse away from trolls, it is important to address what Perlstein’s endgame actually is. He is a revolutionary, but he would rather be remembered for curing diseases than for annoying the academy. He is much more pragmatic and focused than his detractors give him credit for. He wants to cure some of the “7,000 rare diseases” that he cites, but it is not some vague Robin Hood aspiration. He points out that in some cases, scientific researchers already pinpointed the cause for a disease, but no one (including drug companies) picked up the baton, i.e. making an actual drug cure.

Here’s a great example he gives on his blog: “It’s been 16 years since the discovery of the ancient gene responsible for 95% of NPC [Niemann-Pick C, a neurological disease] in a 1997 Science paper by Loftus et al entitled ‘Murine Model of Niemann-Pick C Disease: Mutation in a Cholesterol Homeostasis Gene.’ But today there is still no effective treatment for NPC, despite the paper’s parting call to action.”

One of Perlstein’s goals is to make it easier to connect that final dot. He says on his blog that he is “prototyping a cheaper, modular, evolutionarily informed drug repurposing approach to rare disease research that seeks to compress the time between rare disease diagnosis and rare disease drug discovery at a fraction of the $50-$100 million that I’ve been told is the minimum cost threshold for serious drug discovery. In plain terms, I want to identify off-the-shelf candidate treatments for LSDs for a $1 million or less, and do it in less than the time it takes to review a government grant.”

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