Patricia Huffman

14 posts

Patricia Huffman

Patricia Huffman

@HuffmanPat56675

Katılım Ağustos 2023
48 Takip Edilen6 Takipçiler
Patricia Huffman
Patricia Huffman@HuffmanPat56675·
@biotechbaboon I’m from the HD community and a retired RPh. Placebo effect is real and you can trust me to know. It is NOT possible in the case of AMT 130. Imagine being a patient who just had a 12 hour invasive brain procedure. You feel like you’ve been hit by MAC truck. You hope to be feeling normal at some time in the future. You don’t know the exact progression of your disease. This therapy won’t give you back what you’ve already loss. You hope that if it works you have a a few more years to enjoy life without symptoms getting more debilitating. External control may not be a gold standard but in a limited patient population it’s very good data. I know because I’m living it now. My husband’s disease progression is part of the data.
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Monkey
Monkey@biotechbaboon·
@AB1001_disciple @ThothCapital brother you are beyond retarded. et is not a valid form of control, and amt130 is clearly doing nothing. heard of placebo effect buddy? vp is right. you are a joker and should leave biotech and buy some photonic scams
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Clippy
Clippy@AB1001_disciple·
To make Clippy bleed, John Arnold sacrificed his soul to eternal hell fire.
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Patricia Huffman
Patricia Huffman@HuffmanPat56675·
@HASurfer297 your confirmation bias, the need to be right and/or defend the actions of former FDA officials keeps you from truly looking at this data objectively. Enroll HD is not “horribly flawed” because it deviates from the 10 patient control group. I would suggest it is the more accurate assessment of real patient population. Why not utilize computer resources available today instead of relying on “the way it’s always done” thinking. If the regulators thought the computer generated model was okay in 2024, why go backward in 2026. I believe it’s been established that 12 months is not enough time to evaluate the effects of gene therapy integration for AMT 130. Making a “sham surgery” control group to monitor the outcome for 3-4 years is extremely inhumane and unnecessary. I can give you a year by year breakdown down of changes and decline in my husband’s mental status but it’s not necessary because we provided that information during our Enroll HD visit. He is a real person, intelligent and whitty who is still present in a failing body. There is no “cure” for us, but he has given too much of himself over the years to have his data evaluated and deemed not valid. The clock is ticking and our community can’t wait.
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ElephantsRKewl
ElephantsRKewl@HASurfer297·
$QURE’s AMT-130 efficacy claims hinge on a historical control comparator based on “virtual patients” synthesized from the Enroll-HD database. The issue is that this is a horribly flawed comparator as it massively deviates from the placebo CTRL based on the company’s own data!!!
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Patricia Huffman
Patricia Huffman@HuffmanPat56675·
No. My hope is that it continues to maintain at a least 75% less progression. My roller coaster analogy is what I am experiencing now. Looking back to 2007, when I first noticed subtle changes until 2014, time of husband’s diagnosis, symptoms became significant. If we had maintained 2007 status, my husband could have continued to work instead of going on disability at 52 yo
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Clippy
Clippy@AB1001_disciple·
@HuffmanPat56675 So you're thinking AMT-130 goes from 75% relative slowing to 70% or?
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Clippy
Clippy@AB1001_disciple·
Forgive me as someone that's not a neurologist, but I don't believe UHDRS is sensitive enough to capture 9 vs 12 months progression over a period of 4 years.
mike@mike98572986

$qure $clpt Matt spoke today, my connection got completely jacked up, going to work on trying to get a transcript. This is the rough notes on what my sources believed they heard this morning so take it for what you will. Could have missed some stuff or taken stuff as different context, it’s hard with no transcript or replay. Leadership mostly focused on 1 year results with pre-bla and type A meeting. Like I said, Prasad really focused on the 1 year data. The type a meeting focused on pre-specification, which he answered by saying in 2018 it would have been next to impossible to prespecify, but at 1 and 2 year data they worked with the fda before locking it in to account for that. and the other thing at the type a meeting was the 1 year data and kaputsa said any HD KOL would be able to explain that in early symptomatic patients it would be impossible to have 1 year data show anything due to the slow progression of the disease. He was asked if he expected the 4 year data to follow what they had seen, with each year getting better then previous. and he said that is what they are hoping to expect. thought big thing is he said potential for gap between treated and untreated patients to become even more meaningful The big takeaway was that Kaputsa seemed to be calling out the fda: said the MHRA understood the significant unmet medical need of HD, they had regulatory flexibility, and it was very clear to the MHRA that the 3 year evidence was worth a review. He also said there were 10s of thousands of patients in the UK/Gulf/common wealth and Latin america that would be in TAM. It sounds like they arent going to engage with the fda on an rct because they have other opportunities. It sounds like strategy was going to be put pressure on FDA to see if there really is change at leaderhsip level. This is exactly what I would do if I was Uniqure, say look, we will be releasing the 4 year data soon, happy to lock in the data beforehand , we spoke with many KOL’s, neurologists and the HD community, we do not believe it is ethically right to do a rct phase 3. We believe and many other regulators believe the data we have shown has shown an early signal that amt 130 is working, we are happy to work with the US and would love to come with a mutually beneficial agreement that makes sense for both parties. I’m very happy that it seems Uniqure might not be putting up with the phase 3 rct idea.

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Patricia Huffman
Patricia Huffman@HuffmanPat56675·
Not likely, with the group in this study. HD disease progression is a fascinating puzzle. It’s a roller coaster; everything is coasting along just fine then the bottom drops outs. This cycle repeats. It is slow at first and then things start happening much faster, the dips come more frequently. Until the end, when there isn’t much left to lose. That is why our community is so angry about the delays. We want our children and loved ones to avoid the roller coaster ride for as long as possible.
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Clippy
Clippy@AB1001_disciple·
@HuffmanPat56675 slowing maintained in the high dose should yield 3 months of progression from years 3 to 4 relative to their status at baseline. Is UHDRS sensitive enough to be that granular? I personally doubt it
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Patricia Huffman
Patricia Huffman@HuffmanPat56675·
😳tens of thousands of HD patients?! Approximately 41k symptomatic Americans have HD and 200k are at risk. Enroll HD is flawed because humans are flawed. From CHDI enroll-hd.org/for-researcher… I personally had to search for a new Enroll HD site because our previous site had a delay in recertification. Most people would not bother. I’d like to think I am thoughtful, neither naive nor cynical. I truly don’t understand someone saying AMT 130 is failed therapy when our HD scientists say it shows promise. I trust our HD scientists, because they know us personally. I don’t believe they would attach their name to something that would harm our community. I recommend reading HDBuzz for information . Written by scientists in language easy to understand en.hdbuzz.net
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ElephantsRKewl
ElephantsRKewl@HASurfer297·
@HuffmanPat56675 @Christina4HD @KitCarsonNV Has there been any external audit of Enroll HD data? Regardless you need more than 17 patients to study a drugs effect. There are literally tens of thousands of HD patients. Why didn’t the company enroll more?
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Christina D
Christina D@Christina4HD·
“We don’t approve drugs that require invasive brain surgery” — @HASurfer297 This raises an important question: who is “we” in this context?
ElephantsRKewl@HASurfer297

@Christina4HD @rachelreising96 You are correct in that it’s an experimental therapy. And it absolutely needs more investigation. We don’t approve drugs that require invasive brain surgery and show inferiority to placebo at 12 months and then claim they work based on a ghost virtual control group.

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Patricia Huffman
Patricia Huffman@HuffmanPat56675·
@HASurfer297 please point me to the n=17 data. I am just a retired RPh who left the workforce to care for my HD spouse and don’t recall all of the data instantly. I do have some questions about accepting only data at 12 months. With your opinion based on low dose arm. What is recovery time for invasive brain surgery? Would you agree months rather than weeks? If this is true the recovery time for placebo surgery could be much less than the high dose drug injection procedure? What about inflammatory response from surgery and HD? And the biomarker Neurofilament light? We participated in Natalia Pessoa Rocha’s study of microglia in HD. So thrilled to do our part. I think the 36 month data of AMT 130 is important. We participated in many clinical trials, observational and interventional. The two interventional were both halted early. AMT 130 is different from anything we’ve had before. I have personally spoken to HD scientists about my concerns with ALZ drug debacle. I trust when they say AMT 130 shows promise.
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ElephantsRKewl
ElephantsRKewl@HASurfer297·
@rachelreising96 Garbage propaganda article. AMT-130 performed WORSE than placebo group at 12 months and AMT-130 actually accelerated Huntington's decline in the low-dose group. Approving invasive brain injections willy nilly for thousands based on unsupportive n=17 data is insane.
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Patricia Huffman retweetledi
Rod Wong, MD
Rod Wong, MD@docrodwong·
wsj has been relentless in its reporting on fda. if u haven't paid attention, this afternoon's article recaps vinay's leadership of cber especially clearly, including how it brought john arnold's (a successful energy trader) worldview into drug regulation - namely the belief that most drugs for rare disease aren't worth the expense and should be discouraged. wsj.com/opinion/marty-…
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Patricia Huffman retweetledi
CP2
CP2@LuckyPenguin10·
Dr. Sarah Tabrizi — world renowned clinician and researcher on Huntington’s disease. These comments on $QURE AMT-130 were made 6 months ago. How do you read this and call it a failed therapy? H/T @DesertDweller93
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Patricia Huffman
Patricia Huffman@HuffmanPat56675·
No trial is a failure!! All studies provide knowledge. I’m an HD caregiver and retired RPh. We have participated in many clinical trials including 2 interventional studies in almost 20 years since his first symptoms. I’ve always been the voice of reason stating safe and effective is what we want. I have had the opportunity to interact with many of our scientists and I trust them! Our community needs the chance to try . My spouse is no longer a candidate for clinical trials because of disease progression. We have however completed paperwork for a brain donation when he passes. His legacy will be his contribution throughout his HD journey.
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Razelle Kurzrock, MD
Razelle Kurzrock, MD@Dr_R_Kurzrock·
For fatal disease, Is it worse to approve agent with a possibility of it turning out to be ineffective because trial was imperfect, or is it worse to delay possibly effective agent waiting for perfect trial that requires forcing desperate pts to undergo sham brain placebo surgery
LabRadar ✡️@LabbRadar

$QURE It requires forcing desperate patients with a 100 percent fatal genetic disease to undergo invasive brain surgery solely to inject a placebo, while withholding a therapy that has demonstrated 36 months of profound disease slowing.

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Patricia Huffman
Patricia Huffman@HuffmanPat56675·
@embasic Actually, some healthcare systems have implemented a “meds to beds” program. Retail pharmacy deliveries meds to patients waiting to be discharged. This is provides continuity of care and can reduce duplicate medication therapies in the pharmacy system. Team collaboration
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Patricia Huffman
Patricia Huffman@HuffmanPat56675·
@swifferupdates @taylorswift13 Not a fan of the gift. Do they gift other wives and girlfriends with such extravagance? Taylor would probably be happy with bedazzled Chiefs swag.
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Taylor Swift Updates 💎
Taylor Swift Updates 💎@swifferupdates·
🎂| The Hunt family, co-owners of the Chiefs, gifted @taylorswift13 a $5k Judith Leiber Swarovski microphone clutch for her birthday!
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Keşfet

@biotechbaboon @AB1001_disciple @ThothCapital @HASurfer297 @Christina4HD @KitCarsonNV @rachelreising96 @DesertDweller93