DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for cerebral palsy — screening already-approved drugs against its 42-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleCerebral palsy maps to a 42-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
No approved-drug candidate for cerebral palsy is corroborated in the literature DeepSearch retrieved. Some conditions are managed with non-pharmacological care — a device, surgery or physical therapy — rather than a medicine; that may be the case here, or the literature we found may simply be too sparse yet to support a drug-repurposing angle.
Molecular view
phosphomannomutase 2 (PMM2) — PMM2 is one of the genes genetically linked to this disease in Open Targets — shown as context, not as a drug target we're pursuing: no approved-drug candidate for this disease is yet corroborated in the literature we found.
Loading structure…
helix sheet g16drag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 7O58 · 1.97 Å · ligand 1,6-di-O-phosphono-alpha-D-glucopyranose (G16). Experimental structure, not a prediction.
What the evidence adds up to
A 2013 randomised controlled trial of robotic-assisted locomotor treadmill therapy (Lokomat active orthosis) in 52 children with spastic diplegic cerebral palsy found no statistically significant difference between the group that used the orthosis plus individual exercises and the control group that did only individual exercises. Improvement in mean walking speed was not significantly different between groups (p = 0.5905), and range of motion decreased slightly in both groups with no significant difference (p = 0.8676). A significant improvement in maximal range of hip flexion was seen in the study group (p = 0.0065), and a decrease in mean hip adduction correlated with increased walking speed (r = -0.53, p = 0.0011). The authors called these results preliminary and noted several limitations.
A 2017 systematic review of oral pharmacological treatments for dyskinetic cerebral palsy identified 16 eligible studies. Eight studies on trihexyphenidyl and two on levodopa showed contradictory results. Low efficacy was reported for diazepam, dantrolene sodium, perphenazine, and etybenzatropine. The review stated that tetrabenazine, gabapentin, and levetiracetam should be studied in more detail, and that the updated evidence does not support any therapeutic algorithm for dyskinetic cerebral palsy. The authors attributed the lack of evidence partly to inconsistency in patient classifications and outcome measures across studies.
A 2024 review article on early diagnosis and restorative treatment of cerebral palsy reported high efficacy for the drug Cortexin, citing its multimodal action in stimulating neuropreparation, neuroprotection, and neuroplasticity for motor, cognitive, and autonomic disorders in children with perinatal central nervous system lesions and cerebral palsy. No other abstracts in this set provide independent confirmation of that claim. A 2021 review on underlying causes noted that despite advances in genetics and molecular biology, evaluating the causes of cerebral palsy remains bleak. A 2022 book on cerebral palsy updates covered aetiology, pathophysiology, social aspects, and treatment alternatives but did not report new trial results.
What is still missing are adequately powered, randomised, double-blind, placebo-controlled trials that use consistent, disease-specific outcome measures and stratify patients by age and cerebral palsy subtype. The 2017 systematic review makes clear that even for the most studied oral drugs the evidence is contradictory or shows low efficacy. The 2013 Lokomat trial was underpowered to detect clinically meaningful gait improvements. No abstract provides data on long-term functional outcomes, quality of life, or cost-effectiveness for any intervention.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Journal of Rehabilitation Medicine · 2013 · 113 citations · open access
Functional effects of robotic-assisted locomotor treadmill thearapy in children with cerebral palsy
AbstractOBJECTIVE: The aim of this study was to assess gait in children with spastic diplegic cerebral palsy rehabilitated with the use of Lokomat active orthosis. DESIGN: A randomized controlled trial. SUBJECTS: Fifty-two children with spastic diplegic cerebral palsy. METHODS: Temporospatial parameters of gait and selected kinematic parameters were assessed. Children from the study group used active orthosis in addition to following a programme of individual exercises. Children in the control group participated only in individual exercises. RESULTS: The difference between the initial and control examinations was statistically insignificant. After the programme was finished, there was a slight improvement in walking speed in both groups. Improvement in the mean walking speed was not significantly different between the groups (p = 0.5905). Range of motion decreased slightly in both groups, and the difference between mean amounts of change was not significant (p = 0.8676). There was significant improvement in maximal range of flexion in the hip joint (p = 0.0065) in the study. It was shown that with a decrease in the mean value of adduction in hip joint, the mean walking speed increased (r = -0.53, p = 0.0011). CONCLUSION: There are several limitations to this study, therefore these results should be regarded as preliminary. Further research consistent with the above indications is needed to investigate the impact of this new treatment option in patients with cerebral palsy.
Developmental Medicine & Child Neurology · 2017 · 32 citations · open access
Efficacy of oral pharmacological treatments in dyskinetic cerebral palsy: a systematic review
AbstractAIM: To evaluate the actual evidence of efficacy of oral pharmacological treatments in the management of dyskinetic cerebral palsy (CP). METHOD: A systematic review was performed according to the American Academy for Cerebral Palsy and Developmental Medicine (AACPDM) and Preferred Reporting Items for Systematic Reviews and Meta-Analyses methodology. Articles were searched for in PubMed/MEDLINE, Scopus, Web of Science, Cochrane Library, Database of Reviews of Effectiveness, OTSeeker, Physiotherapy Evidence Database, REHABDATA, and ClinicalTrials.gov. RESULTS: Sixteen articles met the eligibility criteria. Eight studies on trihexyphenidyl and two on levodopa showed contradictory results. Low efficacy was reported for diazepam, dantrolene sodium, perphenazine, and etybenzatropine. Tetrabenazine, gabapentin and levetiracetam should be studied in more detail. The updated available evidence does not support any therapeutic algorithm for the management of dyskinetic CP. INTERPRETATION: This lack of evidence is partially owing to the inconsistency of classifications of patients and of outcome measures used in the reviewed studies. Further randomized, double-blind, placebo-controlled pharmacological trials, optimized for different age groups, based on valid, reliable, and disease-specific rating scales are strongly needed. Outcome measures should be selected within the framework of the International Classification of Functioning, Disability and Health. WHAT THIS PAPER ADDS: Evidence to prove (or disprove) the efficacy of oral drugs in dyskinetic cerebral palsy is low. The most investigated drugs, trihexyphenidyl and levodopa, show contradictory results. Tetrabenazine, levetiracetam, and gabapentin efficacy should be studied in more detail. Lack of evidence is partially due to the inconsistency of classifications and outcome measures used. Outcome measures should be selected within the framework of the International Classification of Functioning, Disability and Health in next clinical trials.
S S Korsakov Journal of Neurology and Psychiatry · 2024 · 6 citations
Early differential diagnosis and restorative treatment of cerebral palsy
AbstractThe article is devoted to an urgent problem of modern neurology - early diagnosis and complex restorative treatment of cerebral palsy (cerebral palsy). Etiological factors and pathogenetic aspects of the formation of various forms of cerebral palsy are considered in detail, as well as modern possibilities of differential diagnosis in children of the first years of life of cerebral palsy and a wide range of pathological conditions (somatic, endocrine, hereditary-conditioned, including hereditary-metabolic and neuromuscular diseases). The leading directions of complex rehabilitation of cerebral palsy are widely presented, taking into account modern standards and clinical recommendations. The high efficacy of the drug Cortexin has been shown, due to its positive multimodal action (stimulation of the processes of neuropreparation, neuroprotection, neuroplasticity) in the treatment of motor, cognitive and autonomic disorders in children with perinatal lesions of the central nervous system and cerebral palsy.
Folia Neuropathologica · 2021 · 4 citations · open access
Underlying causes of cerebral palsy: public health perspectives
AbstractCerebral palsy (CP) is a neurological pathology that is characterized by a combination of signs and symptoms that occur in neurodegenerative or metabolic disorder during the first few years of life. It is a complex pathology orchestrated by a plethora of different causes. The current diagnostic regimen for CP involves brain magnetic resonance imaging (MRI), and antenatal and perinatal insult. Despite advances in the field of genetics and molecular biology, the evaluating the underlying causes of this severe pathology are still bleak. In this review we have attempted to provide a landscape of the underlying mechanisms of cerebral palsy. We have partitioned this review broadly into genetic and proteomic-based studies, which have enriched our understanding about the pathogenesis of CP.
IntechOpen eBooks · 2022 · 0 citations · open access
Cerebral Palsy - Updates
AbstractCerebral palsy is a debilitating disease that affects the everyday life of patients and their caregivers. Understanding its pathophysiology, preventing avoidable factors, and effectively treating the disease with the most appropriate approach is paramount in the management of patients with cerebral palsy. This book presents up-to-date information about the etiology, pathophysiology, social aspects, and optimum care and treatment alternatives of this chronic condition.
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.
