Rare & Orphan Lab · DeCure for X

DeCure for Botulism

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for botulism — screening already-approved drugs against its 39-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module39 genesLead labRare & Orphan
All cures
Rare & OrphanDOID:11976$DeCureRare

The disease map

Disease moduleBotulism maps to a 39-gene Open Targets module — the target space DeCure's AI scientist screens approved drugs against.
DeCure.ai methodSignature reversal (LINCS) plus network proximity (STRING) rank already-approved drugs likely to perturb this module — the same engine that produces DeCure.ai's repurposing hypotheses.
Repurposing thesisScreening approved medicines against this disease module, then publishing the evidence for the strongest candidate. Known pharmacology and human exposure data make the first question sharper — they do not establish safety or efficacy in a new indication.

Research record

01
ResearchComing soon
Candidate research + dossier — target rationale, drug-repurposing thesis and evidence pack.proof: Published dossier + on-chain hash
02
ValidationComing soon
In-vitro biological validation at a contract research org (CRO).proof: CRO contract + in-vitro report
03
Peer review & paperComing soon
Peer-reviewed paper published open-access (preprint + journal).proof: DOI + open-access link + on-chain hash

Current lead

approved
GuanidineApproved drug

Structures already discussed alongside botulism in the retrieved literature, rendered from public PubChem SMILES. Which drugs appear here reflects the evidence found, not a ranked prediction.

Molecular view

Human mitochondrial aldehyde dehydrogenaseGuanidine has a real, experimentally solved structure in complex with this target (PDB 1O02, 1.9 Å). This is the drug's own deposited structure, not a prediction, and confirms it is a structurally characterised molecule rather than an untested guess.

Loading structure…
helix sheet gaidrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 1O02 · 1.9 Å · ligand Guanidine (GAI). Experimental structure, not a prediction.

What the evidence adds up to

Botulism is a rare neuroparalytic disease caused by botulinum toxin, one of the most toxic substances known. The only specific treatment is botulinum antitoxin, but it cannot reverse existing paralysis. A 2019 case report describes a 37-year-old man with gastrointestinal symptoms and posterior cranial nerve palsy who was diagnosed with botulism; the authors note that misdiagnosis of early cases is common and that sporadic cases are likely overlooked. Timely clinical diagnosis is critical for treatment decisions.

The disease is caused by the anaerobic bacterium Clostridium botulinum, which produces spores and a toxin. The toxin is often created in rotten food, and the most common sources of infection for humans are canned goods and dried meat products. Infection can also occur if an injured spot on the skin contacts the causative agent. The disease is not contagious, but occurs suddenly and attacks the nerves, causing flaccid, symmetrical, descending paralysis affecting cranial and peripheral nerves.

No drug repurposing data for botulism appear in these abstracts. The 1969 document is a food-safety extension fact sheet, not a clinical trial. The 2024 abstract repeats general epidemiological information without reporting any treatment outcomes, survival rates, or response rates for any drug. There is no mention of any drug other than botulinum antitoxin.

What is still missing is any clinical trial testing a repurposed drug against botulism in humans, funding for such trials, and a method to stratify patients by time from exposure or severity of paralysis. Without these, no drug beyond antitoxin can be evaluated for this neglected diagnosis.

Evidence

Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.

JAMA · 1978 · 32 citations

Botulism and Guanidine

AbstractGuanidine hydrochloride was introduced as an adjunct in the treatment of botulism in 1968. It has been reported to be of benefit in 39 cases and of no benefit in 13 cases. No serious side effects have occurred with the short-term therapy required in botulism. Our two cases are similar to earlier cases in that the improvement seen with quanidine therapy is most notable in ocular muscles and least notable in respiratory muscles. Electrophysiological findings again showed an increase in the amplitude of evoked muscle-action potentials after guanidine administration.

https://doi.org/10.1001/jama.1978.03290210058027
European Journal of Case Reports in Internal Medicine · 2019 · 8 citations · open access

Foodborne Botulism: Neglected Diagnosis

AbstractBotulism is rare neuroparalytic disease caused by botulinum toxin, one of the most toxic substances known. Foodborne botulism is caused by consumption of foods contaminated with botulinum toxin. The clinical manifestations are flaccid, symmetrical, descending paralysis affecting cranial and peripheral nerves. The only specific treatment is botulinum antitoxin. We report the case of a 37-year-old man with gastrointestinal manifestations and posterior cranial nerve palsy who was diagnosed with botulism infection. Clinicians should be aware of rare causes of infection and determine the aetiology of symptoms. LEARNING POINTS: Botulism remains a diagnostic challenge.Misdiagnosis of early cases suggests sporadic cases are overlooked.Timely clinical diagnosis is critical for treatment decisions as botulinum antitoxin cannot reverse existing paralysis.

https://doi.org/10.12890/2019_001122
EDIS · 1969 · 3 citations · open access

Preventing Foodborne Illness: Clostridium botulinum

AbstractBotulism is caused by the nerve toxins produced by the bacterium Clostridium botulinum, often resulting in a serious paralytic condition that can lead to death. This document is FSHN04-06, one of a series of the Food Science and Human Nutrition Department discussing common foodborne pathogens of interest to food handlers, processors, and retailers; Florida Cooperative Extension Service, Institute of Food and Agricultural Sciences, University of Florida. Date Published: November 2004. FSHN0406/FS104: Preventing Foodborne Illness: Clostridium botulinum (ufl.edu)

https://doi.org/10.32473/edis-fs104-2004
Dermatology and Dermatitis · 2024 · 0 citations · open access

Botulism is a Rare but Serious Disease

AbstractBotulism is a rare but serious disease caused by the anaerobic bacterium Clostridium botulinum, which produces spores and a toxin. It is very widespread in nature, in the ground and in the digestion of mice, rats and cats. The toxin is often created in rotten food, and the most common sources of infection for humans are canned goods and dried meat products. It is also possible to get the disease if an injured spot on the skin comes into contact with the causative agent of the disease. The disease is not contagious, but occurs suddenly and attacks the nerves.

https://doi.org/10.31579/2578-8949/142
Neuromuscular Disorders · 2011 · 0 citations

Botulism

AbstractBotulism is a severe neuroparalytic illness caused by toxins produced by Clostridium botulinum. Symptoms include ophthalmoparesis, blurred vision, and facial and bulbar paresis, followed by limb weakness and respiratory compromise. The diagnosis depends upon recognition of the clinical syndrome, appropriate electrophysiological testing, and specific biological assays for the detection of the toxin or its action. Treatment consists of administration of antitoxin and intensive supportive care.

https://doi.org/10.1002/9781119973331.ch17

Disease module: DeepOracle (Open Targets). Structures: RDKit from PubChem SMILES. Literature: retrieved by DeepSearch across 234,678,978 indexed works (targeted per-candidate search), resolved on OpenAlex.

DeCure is a research and publication project, not medical advice and not a treatment. "DeCure for X" describes a research goal, not a claim that a cure exists. Backing a cure is a contribution to fund the research — it is not an investment, and confers no yield, royalty, equity or IP ownership. Papers are published open-access by the DeCure.ai DAO.