Topic
Strategy of finding new disease uses for existing approved drugs, leveraging known safety data to speed development versus novel compounds.
Researchers at Washington State University and the University of Michigan slowed endometriosis lesion spread in mice with a repackaged tapeworm drug. The treatment also reduced inflammation, and the team is working toward clinical trials for a disease with limited treatment options.
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Yale researchers report that the epilepsy drug lacosamide eased pain and slowed cartilage loss in mouse models and in human cartilage cells in the lab. The work is preclinical, and it still has to be tested in patients with osteoarthritis.
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Researchers pairing the deworming drug mebendazole with gefitinib report a median tumour doubling time of 25 days in mice, against 15.5 for gefitinib alone. The data come from one resistant EGFR-mutant model, and the tumours were not eliminated.
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Yale researchers report that lacosamide, an FDA-approved epilepsy drug, eased joint pain and reversed cartilage damage in preclinical osteoarthritis studies. No FDA-approved medicine yet does both for osteoarthritis, so the open question is whether the effect holds in people.
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University of Birmingham researchers report in Brain that blocking the P2X7 receptor halted inflammatory cytokine release in living human brain slices. That makes existing P2X7 drugs fair candidates for brain-disease trials, though the evidence so far comes from tissue kept alive outside the body.
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European researchers report that amlodipine, a blood pressure drug taken by over 70 million people in the US, eased ADHD-like traits in animal tests. Its use for the quarter of patients whom existing drugs fail still awaits human trials.
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Dana-Farber researchers safely combined paricalcitol, an approved vitamin D drug, with chemotherapy in a small trial in metastatic pancreatic cancer. Its strongest survival signal came in tumours rich in the vitamin D receptor, a possible way to choose patients that a larger trial still has to test.
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Researchers linked the tapeworm drug niclosamide to a carrier that cleared detectable endometriosis lesions in up to five of eight treated mice after one dose. It points to a non-hormonal option for a disease with no cure, though the test used eight mice per group over two weeks.
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Surrey researchers found the anti-fibrotic compound SD-208 cuts cells' vesicle release by diverting the vesicles to lysosomes. Other drugs aimed at the same target did not copy it, so the effect looks independent of SD-208's known action.
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Researchers at The Ottawa Hospital reversed autism-linked behaviours in 16p11.2 mice by restoring blood flow in brain vessels that held half the normal ATP. The drug that did it is already approved for people in Japan and South Korea, though for dry eye, not autism.
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Toronto scientists report reduced levels of a dopamine neuron marker throughout the striatum in people with long COVID, and the regional pattern tracked whether a patient's worst problem was motivation, movement or memory.
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Palomar Labs' first spinout is built on a molecule whose parent compound was tested in the 1970s and documented not to be hallucinogenic. Apathy affects about 40% of people with Parkinson's, and no drug is approved for it.
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Forty-eight healthy volunteers took a forearm injection of UV-killed E. coli, and half of them received a drug that stops the body degrading its own epoxy-oxylipins. Pain resolved faster and inflammation-linked monocytes fell.
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A Nature Communications study from Umeå University reports that stressing the lipid carrier bacteria use to build their cell wall stopped MRSA from evolving its way back to resistance. The paper's title names statins as the stressor.
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Ohio State reports that glioblastoma suppresses the enzyme PP2A using three of its own proteins, and that knocking those down in lab and animal models killed more cells and left the survivors easier to irradiate. No patients have been treated.
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Emory researchers report in Nature Microbiology that ten days of an approved blood-cancer drug, given as antiretroviral therapy began, left the intact SIV reservoir smaller nine months later. No animal was taken off ART to test rebound.
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The Hollings group puts lysyl oxidase inside the cancer cell, running its mitochondria, and reports that shutting it down leaves a backup survival pathway a second drug can close. The work is preclinical, and thinly quantified.
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The JAMA Dermatology results published August 12 push an already-approved arthritis pill toward a new indication, which turns interesting science into a prior-authorization question about who counts as a responder.
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Mount Sinai's Nature paper argues that neurons trade repair for stress management after injury. Deleting or drugging the aryl hydrocarbon receptor tipped that balance back toward rebuilding, in mice.
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Cornell researchers say single-cell sequencing separates a destructive benign canine oral tumor from its malignant lookalike, and that an ERBB4 inhibitor slowed it in live tissue.
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