It gets personal
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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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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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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.
