Who's making this ALS drug? | SRPT Message Board Posts

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Msg  13787 of 14218  at  6/4/2019 11:13:28 AM  by


The following message was updated on 6/4/2019 11:21:46 AM.

Who's making this ALS drug?

"While a drug maker was involved in developing the drug, none of the money will go to that company, which “has nothing to do with this, at this point,” according to Shneider. Shneider declined to name the company involved, which appears to be Ionis Pharmaceuticals.

“When contacted by scientists and physicians looking for a potential treatment for Jaci, we said yes, providing some of our early research into oligonucleotides. … We also continue to advise them on antisense technology and its use in the treatment of ALS,” Frank Bennett, senior vice president of research and franchise leader for neurological programs at Ionis, told STAT in a statement."


Kind of a weird mystery. I suppose this Columbia University doctor has a reputable company manufacturing it (on Ionis's advice, I'd guess), and they are using the Ionis formulation from it sounds like years ago (maybe). Having read the article I'm not sure if toxicology was or was not done on it. Or not completed. By Ionis or anyone else? I guess, if this shot in the dark works it will be very positive for the antisense "story" ....Maybe there's a better article out there explaining more details of what's going on here ....

"While the FDA hasn’t formally accepted or denied Jaci’s expanded access petition, Columbia’s Shneider told STAT Wednesday that “there was some bending” at the FDA. He said the agency seemed willing to back off its earlier requests that the drug company conduct significant toxicology studies before Jaci’s first infusion."

WHAT drug company? How many infusions? Weekly?....

"Shneider said the money is being used to “do the very basics” needed to get Jaci treatment — things like paying a contract research organization for the remaining testing needed for the drug, as well as the costs of the infusions, hospitalizations, and Jaci’s travel to Columbia."

All I can say, is I hope that seemingly abandoned Isis drug was a good one. Weird.

She gets her first shot on June 10th supposedly.

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