It gets personal
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wrote on 7 Mar 2025, 23:15 last edited by jon-nyc 3 Aug 2025, 17:18
We are co-funding a project with the NHLBI (National Heart, Lung, and Blood Institute, one of the Institutes within NIH).
The NIH funding is a "U grant", which is a multi-center grant. NIH is funding 6 centers and we're funding 2 more, plus an overseas validation cohort (in Ireland). We're also funding some liver analyses in the original 6, as NHLBI doesn't fund liver stuff.
The project is the Alpha-1 Biomarkers Consortium. Biomarkers are just biological markers of a disease, useful ones can be predictive of prognosis or indicative of disease progression. Our main goal is to find more sensitive and less invasive clinical trial endpoints that will facilitate bringing new treatments to market.
[Quick aside on endpoints - the endpoint(s) of a trial are what you're trying to measure to prove efficacy. It might be mortality in an oncology trial - what's the median survival of the subjects in the treatment arm vs the control arm? In most disease conditions (thankfully) it's something else. Maybe lung function, liver enzymes, fibrosis scores, cholesterol levels, blood sugar levels, A1C, etc., depending on the disease. There are also 'patient reported outcomes' such as pain, shortness of breath, scores on standardized questionnaires, etc. In AATD we have shitty endpoints - spirometry for lungs and usually fibrosis/cirrhosis staging for liver. The former is very noisy and requires a large number of subjects over a long time for statistical significance (bad for a rare disease, it makes the trials unrecruitable). The latter is very invasive (liver biopsies suck) and limits trials to people with pretty advanced liver disease.]
So this project is following 270 alphas over three years (ultimately longer, but current funding is for three years). We're testing them periodically for every conceivable biomarker including some really specific stuff like obscure biological byproducts of elastin breakdown or cell death in the liver, etc. The idea is to find something sensitive so we can measure disease progression faster, and with fewer patients. We would need to show that our biomarker correlates sufficiently well with disease progression, and then convince the FDA of that.
So why am I posting this? Today the administration announced the "initial cancellation" (not sure what that means, suspension?) of all federal grants to Columbia University. $400MM in total. Part of that, we fear, is our little grant.
Columbia is just one site, we have 6 NIH funded sites. BUT, Columbia is the primary site and collects all the money and then gives it to the others. The other sites are BU, UAB, Nat'l Jewish, UNC, U of Utah. We fund Chicago and UCLA and Ireland.
This all happened hours ago so there aren't a lot of details out. I'll see our Columbia PI tomorrow at a fundraiser in Boston if she can get away. At the very least her sidekick (also MD/PhD) will be there so I can get more info.
Our foundation won't let the project die, we're two thirds through. But we may need to raise some money and fund it ourselves.
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wrote on 8 Mar 2025, 00:03 last edited by
Hopefully Trump switches from hatchet mode to surgical knife sooner rather than later. Sorry to hear. There are lots of lives being massively impacted by this in all sorts of ways for no reason other than haste and headlines.
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wrote on 8 Mar 2025, 01:55 last edited by
I read that they weren’t canceling NiH grants at Columbia. Nicht wahr?
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wrote on 8 Mar 2025, 02:32 last edited by
Not a lot of details yet but the press release came from HHS, DoE, and GSA. I think GSA is just helping craft the individual cancellation letters. I tried to reread my link but GSA site is down for ‘maintenance’.
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wrote on 8 Mar 2025, 02:32 last edited by
Having said that, there are not a lot of details released yet.
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wrote on 8 Mar 2025, 02:51 last edited by Renauda 3 Aug 2025, 02:58
Hoping your noble medical research project is saved from the pyre.
The uncertainty you are under is so wasteful and unnecessary. A dictatorship of the inept executed by mindless bean counters
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wrote on 8 Mar 2025, 03:19 last edited by
Hope things turn in the right direction for you @jon-nyc .
Killing a mosquito with a sledgehammer is not the right answer.
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wrote on 10 Mar 2025, 18:07 last edited by
Our PI has't gotten a stop work letter yet so it seems like a good sign. Some colleagues of hers have for CDC-associated work. Maybe NIH really was excluded. Though it's the 800lb gorilla of grant making institutions.
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wrote on 10 Mar 2025, 19:10 last edited by
No significant cuts on our side yet, that I’m aware of anyway.
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wrote on 15 Mar 2025, 13:21 last edited by
We lost our funding last night.
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wrote on 15 Mar 2025, 13:54 last edited by
Other possible grants?
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wrote on 15 Mar 2025, 14:23 last edited by
Try China?
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wrote on 15 Mar 2025, 15:36 last edited by
How were you funded and what percentage? Was it one grant or more?
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wrote on 15 Mar 2025, 15:52 last edited by
GoFundMe?
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wrote on 15 Mar 2025, 15:53 last edited by
Is stem cell involved?
If not, maybe the churches can step in. -
wrote on 15 Mar 2025, 16:27 last edited by jon-nyc
It was a single U Grant originally for $6MM. I'm not exactly sure how much has been billed against it, my guess is roughly half.
We're looking into the possibility that one or two other center might be able to invoice the NIH directly for their participation based on the way the grant is worded, which would help. We will also fundraise against it both from interested biotechs and patients. We will also have patients send letters, especially patients in Alabama, North Carolina, South Carolina, and Utah which all have GOP senators and affected centers.
Senator Thom Tillis may not see the logic in cutting rare disease funding at UNC because Hamilton Hall was occupied last year. Ditto Mike Lee and UU.
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wrote on 15 Mar 2025, 16:38 last edited by
@jon-nyc said in It gets personal:
We lost our funding last night.
That’s a bummer. Hopefully the work can continue.
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wrote on 15 Mar 2025, 17:06 last edited by
I’m working with both UNC and U of Utah now, plus Wake Forest.
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wrote on 15 Mar 2025, 17:31 last edited by
The work won't end, it's too important, we won't let it.
The bigger fear is what happens with research and clinical trials generally if the indirects get cut overnight. That's too much for a little foundation to backstop.