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WASHINGTON — A family in Iowa believes the Food and Drug Administration will decide whether their only surviving daughter lives or dies, and they’ve been on a monthslong crusade to break through its bureaucracy. And they’re succeeding.

Just last week, the FDA gave Jaci Hermstad, a 25-year old Iowan who is dying from a rare form of ALS, an early sign that she will receive the first dose of an experimental drug never before tested in humans. The FDA’s move, which was confirmed to STAT by Jaci’s family and doctor, is a breakthrough for the Hermstads.

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  • I understand the pain and desperation that goes with a diagnosis of ALS. The bioethicists are trying to provide a way that people can get treatment in a fair and scientific fashion, without making them vulnerable to shysters who will suck every cent out of the families, while letting their patient die. I am old enough to remember the Laetrile debacle and the cruel false hope it gave to cancer patients. There are horrible people who will tell patients that THEY have a secret supply of the drug, if their community comes up with the money
    If Jaci has a bad outcome due to toxicity, that will set the treatment by oligonucleotides of neurological degenerative diseases back a decade because no one will want to invest in the research.
    I have seen the data of this work on primates and it is very exciting. But please let the scientists nail it down before putting pressure on them to release it too soon

    • There are laws that stop
      The “terrible people”
      From telling people with als they have a secret stash of drug. If Jaci has a bad outcome, no one will react. You’re missing the point. This is giving her a chance. Without it, she’s goi g to due. STAT is biased. They have an issue with ALS.

    • Nobody who works in disease research should ever think that people ill with that disease are their family members. We would get no where. No rigorous thought; just try whatever comes to mind. If this were the case, there would be no cures.

      Jaci should be allowed to try whatever she wants, just don’t make a huge gigantic deal out of it when it doesn’t work. Because it’s not ready for even animal trials. Do not kill the chance that that it might work for others with just more work.

    • It is impossible to discuss and debate when people are only dealing with the issue emotionally.

      If everybody in the ALS community hates those working on a cure; spending all of their time working on a logical treatment; it certainly won’t bring in any researchers with new ideas…

  • Thank you for the article on ALS, I am terribly sorry for the family of the baby,
    My husband die of ALS three years ago, we did stem cells, which gave him life quality for 8 months until the illness came back again, rilutek was offered from the beginning but it didn’t do anything

  • The FDA should should let Jaci try the therapy

    Also any news on MSA advances?

  • Once again STAT is coming after the ALS community. Our loved ones are suffering and dying a horrible death. This dear family and young women have a little hope by gaining access to this potential therapy. The pharma company is supplying for free. I think this is wonderful. One has to ask why STAT is fixated with our ALS community? And not, in a good way!

  • The author seems to be interested in the ALS community. I see that he has been in some of our ALS forums trying to connect with us mainly around Right to Try. If he really understands anything about ALS or had any empathy, he would be applauding all those involved for making this a possibility for dear Jaci. Nick Florko, I canllange you to really learn about ALS and using your forum to help us. Seems to me you are preying on valunerable ALS people! Shame on you once again.

  • It seems like the “bioethicists” are creating threats to justify their own existence (and paycheck).

    • Seems like it. Holly Fernandez Lynch of UPenn: “What are we doing to patients when we advance this spirit of fighting disease at all costs?”

      Uh, saving their lives.

      These over-credentialed, under-experienced academics are incredibly good at erecting false dichotomies. It’s a good thing most of them don’t have any substantive contribution to actual policy making

  • Can you please help the ALS community as the press did in the days of HIV/AIDS? The FDA should find innovative pathways and expedite treatments for people suffering from this horrible disease.

  • How amusing, invoking God and miracles, when the answer to their prayers lies in the hands of everything they despise for most of their lives, rational scientific knowledge.

  • I really do not know where to begin. First, you and the “bioethicists’ have an issue with Right to Try. You have done all you can through the press to discourage companies and others from implementing RTT. Now, this is an example of Expanded Access which all of you have said is the pathway, but there’s an issue because the drug is in early development. Second, who do you think you are, God? Third, what is wrong with using the expanded access pathway for this 25-year-old woman suffering from ALS? I think we are making progress in ALS. Why can’t you help us change the course of this disease? I hope one of you gets ALS and then we will see ………..

    • Last sentence…..not really nice BUT we all understand…….an old expression that I don’t remember where it came from…”…don’t knock it ’til you live it”… also “…my body, my choice”…..trying to think of more….will return if I do.

    • The right to try is for potential treatments undergoing CLINICAL TRIALS. This anti-sense oligo has had almost or no testing done on it! It is years away from a clinical trial. So please, realize this first. Besides that, clinical trials should never give the participant the idea that they ARE getting a treatment.

      What is all of this anger against STAT or this author from the ALS community? Reality is what it is and the push for one person involving the senate and congress, to force the FDA’s hand can easily come back and bite the community.

      What happens if Jaci tries this random, completely untested treatment and it kills her w/in a couple of days? Rational thinking is required of everybody, excluding Jaci and her family.

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