In Which Gilead Sciences Earns Kudos — Pauline Cafferkey Is Still In Serious, But Improving Condition. . . Compassionate Early Access To GS-5734

MRK-Gil-Cafferkey-2015 About ten days ago, now, on the very same day that doctors at the Royal Free called on Gilead Sciences’ people — for access to its investigational nucleotide analogue labeled GS-5734, the folks in San Diego shipped it to London.

This is the way life saving but experimental treatments are supposed to be handled. She was, at that point apparently running out of options. . . and now she is improving. Still a very serious situation, but improving. Excellent. [More broadly, this week’s WHO Ebola situation report disclosed that more people have been lost to follow up, than are being treated as follow up cases, in Guinea — so we may yet see additional nodes there.]

Here is some of the background, but it no longer seems that hers is a case of full-on Ebola relapse — but rather a form of meningitis, of unspecified origin. Here is a bit of the story from earlier this week — (sorry I couldn’t get to it sooner) at MarketWatch:

. . . .Gilead confirmed that the company fulfilled a request for compassionate access to GS-5734, a novel nucleotide analogue in development for the potential treatment of Ebola Virus Disease (EVD). The request was received last week and drug was shipped later the same day. The compound is currently being provided to a female patient in the United Kingdom, as described earlier today by officials from the Royal Free Hospital in London.

“It is very encouraging to hear that the patient in question is doing better and is no longer in critical condition. Our thoughts and wishes for continued recovery are with her, with her family and the excellent medical team at Royal Free Hospital,” commented Norbert Bischofberger, PhD, Gilead’s Executive Vice President, Research and Development and Chief Scientific Officer. “We recognize the urgent need for treatments to address Ebola and we are working with collaborators to advance development of GS-5734 as quickly as possible.”

GS-5734 was discovered as part of Gilead’s program to screen compounds in its libraries for activity against a range of potential emerging viruses, including Ebola. In collaboration with the Centers for Disease Control and Prevention (CDC) and the United States Army Medical Research Institute of Infectious Diseases (USAMRIID), the company identified GS-5734 in vitro activity against the Ebola virus.

In animal studies conducted at USAMRIID, treatment initiated on day 3 post-infection with Ebola virus resulted in 100 percent survival of monkeys. . . .

Well. . . we are encouraged — and, mightily so. This is the opposite of the recent behavior we’ve seen from one Martin Shkreli (a Fred Hassan protégé, at one point). In any event, this is what human health science should be all about. While the Gilead pro-drug is still only being proved out in animal models, it is good to see scientists working hand in hand — and cross border regulators allowing this, in dire cases — even when the risks are high.

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