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.
For months, the family has circulated petitions, tweeted at President Trump, and called on some of Washington’s most controversial and powerful politicians — including the speaker of the House and a Republican under fire for racist comments — all to convince the FDA to waive even the most basic of studies before the drug is injected into Jaci’s already fragile immune system.
Some Right to Try and clinical trial vets who have had very encouraging results from NurOwn are in Washington this week:
I think a couple PALS will get to see Pres. Trump tomorrow (6/13) at the WH.
My prayers go out to Jaci and her family. I am 57 years old, I was just diagnosed with ALS on June 6, 2019, 10 months after I developed a slight drop foot after an episode of sciatica. The first neurologist I saw did a few test here and there and in November of 2018 he told me that he did not know what was wrong with me other that I had an abnormal EMG and come back in a year and he would repeat the EMG. The foot drop gradually got worse, so in February 2019 I was seen by another neurologist that performed another EMG and told me that I had ALS and only 9 to 12 months to live. I was crushed, he referred me to the ALS clinic at UAB in Birmingham Alabama, after countless MRI’s, LP and various blood test and the third EMG done back in April of 2019; Dr. Lee informed me of my diagnosis. I have started Riluzole, a medication that will slow the progression of the disease. I am currently waiting for my insurance to approve a IV medication, Radicava. I prayers go out and up to the heavens that there will be a cure and that more can be done to allow us the right to try.
I know what the Hermstad family is going through; I watched my sister die from ALS. Could you please confirm for me the investigational antisense oligonucleotide mentioned in your article is toferson or BIIB067 by Biogen/Ionis? Thank you
Thank you Nicholas for bringing this more to light
Art Kaplan’s comment about an larger quagmire and ethical issue is contemptuous. Is he saying that providing an investigational drug through expanded access is unethical? Isn’t that what he and his group at NYU do for a living?
Providing a drug which is toxic, even if the person is dying, is unethical. Not knowing whether a drug is toxic is unethical.
Does the author understand the Right to Try law? The title leads me to believe he does not. Right to try does not apply here because the therapy has not completed phase 1 testing and is not in active development. This is the prefect case for expanded access. Oh, it must be that he destine for RTT has to be voiced. What ever happened to unbaised journalism? Not at Stat and not with regards to RTT !
“The drug Jaci’s trying hasn’t gone through clinical trials, making it ineligible for the pathway outlined in the right-to-try law, “
I can see both sides:
On the one side, if a co-mobidity kills Jaci, the fickle, shallow investment public will dry up funds for further anti-sense oligo research.
On the other side, slow and torturous death. And a mother who has had to bury two daughters.
Is there no way the results of the treatment to be sealed?
MAY 31, 2019 AT 8:45 PM
It is utterly unsurprising that you don’t get the point. This issue is not academic for me (notwithstanding my quarter century experience in healthcare analytics).I have lost both of my daughters to two different cancers across the last 20 years. One was in a clinical trial at City of Hope that had to be terminated early. More recently, my younger daughter was found to be ineligible for a clinical trial at UCSF. The husband of one of my dear nieces is struggling with advanced ALS right now. I would love for any rational therapy that would improve his dismal chances. I find ethical dilettantes bound up in magical thinking unamusing. YOU “bugger off.”
I’m glad I got out of you at least a more thoughtful response than a juvenile “please” under every post you found unworthy.
Nothing like a bunch of dispassionate academics opining about whether folks should live or die as if we were talking about access to good schools or good vacations. McBride is especially egregious. Her rhetorical question could be applied to any dire situation, and then how ridiculous would it sound? “What does this mean for anyone else who is dying of starvation, but who maybe doesn’t have the same resources to be fed?” Not a total parallel, but enough of one to make you grateful these folks aren’t regulating access to all life-saving and sustaining substances–like, say, water or food.
“What does that mean for everyone else who is similarly in a situation that is very dire but who maybe [doesn’t] have the same resources to be able to communicate with legislators, and the media, and with the larger public through social media?” Kelly McBride Folkers, a research associate
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