One surprising outcome from evaluating our drug repurposing system is how far into the future it can generate meaningful hypotheses – years before the biomedical community recognizes them.
Here are three examples from our cystic fibrosis (CF) work.
1. Tamoxifen for CF
Predicted: 1991
First reported: 2018 (27 years later)
Our system linked Tamoxifen to CF through VIP:
Tamoxifen → VIP
• Endocrinology. 1990, 127(3):1463–1469.
The regulation of neuropeptide expression in rat anterior pituitary following chronic manipulation of estrogen status…
VIP → CF
• Am J Physiol. 1989, 257(4 Pt 1):G561–569.
Vasoactive intestinal peptide and its receptors in fetuses with cystic fibrosis.
Later literature confirmation
• Comput Struct Biotechnol J. 2018, 16:587–599.
2. Copper for CF
Predicted: 1989
First reported: 2021 (32 years later)
Our system connected Copper to CF via trypsinogen:
Copper → Trypsinogen
• J Am Coll Nutr. 1988, 7(1):27–34.
Changes in pancreatic enzyme specific activities of rats fed a high-fructose, low-copper diet.
Trypsinogen → CF
• J Pediatr. 1985, 107(3):377–381.
Age-related alterations in pancreatic lipase and cationic trypsinogen in children with cystic fibrosis.
Later literature confirmation
• Pediatr Pulmonol. 2021, 57(1):317–319.
A rare case of pancytopenia in a child with cystic fibrosis: Can copper cure it all?
3. Thalidomide for CF
Predicted: 1994
First reported: 2019 (25 years later)
The algorithm surfaced this through TNF-α:
Thalidomide → TNF-α
• Mol Cell Biochem. 1993, 129(2):195–200.
Inhibition of tumor necrosis factor-alpha by thalidomide in magnesium deficiency.
TNF-α → CF
• Pediatr Res. 1993, 34(2):162–166.
Circulating TNF-α levels and lipid abnormalities in patients with cystic fibrosis.
Later literature confirmation
• ASAIO J. 2019, 66(6):645–651.
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These examples highlight how much untapped knowledge already exists in the literature and how AI can detect meaningful signals long before they reach mainstream research.
We have many more findings like this across different diseases.
How should the scientific community incorporate early signals like these into real-world research pipelines?
I’d love to hear your thoughts.
#drugrepurposing #cysticfibrosis #drugdiscovery #AIinScience #computationalbiology