Rare & Orphan Lab · DeCure for X

DeCure for Spastic cerebral palsy

DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for spastic cerebral palsy — screening already-approved drugs against its 1-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module1 genesLead labRare & Orphan
All cures
Rare & OrphanDOID:0050669$DeCureRare

The disease map

Disease moduleSpastic cerebral palsy maps to a 1-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 spastic 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

chromobox 8 (CBX8)CBX8 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 unxdrag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 5EQ0 · 1.18 Å · ligand UNKNOWN ATOM OR ION (UNX). Experimental structure, not a prediction.

What the evidence adds up to

In a multicentre interdisciplinary clinical trial from 1995, intrathecal baclofen infusion was investigated for relief of spasticity caused by cerebral palsy. The nursing process served as a framework for project development, protocol implementation and long-term follow-up. A 1991 letter to the editor noted that Albright et al had detailed the clinical usefulness of intrathecal baclofen as a more effective alternative to oral administration, but warned that eliminating spasticity entirely could remove a "physiological crutch" that enables some patients to walk. Excessive reduction in lower-extremity antigravity muscles may significantly diminish the ability to ambulate.

A 2019 review summarised that the common classifications of spastic cerebral palsy are the gross motor function classification system (GMFCS) and the manual ability classification system (MACS). Surgical methods are divided into soft tissue surgery, nerve surgery, and bone and joint surgery. Precise diagnosis includes qualitative, localisation, and quantitative diagnosis. Based on precise diagnosis and classification, one or more corresponding surgical methods are selected. The review concluded that manifestations are so diverse that rational surgeries must be selected based on precise diagnosis to achieve individualised treatment.

No concrete numbers for survival, response rates, or sample sizes are given in these abstracts. The 1991 letter explicitly states that eliminating functional spasticity can be a disadvantage, and no data on how many patients lose ambulation are provided. The 2019 review does not report outcomes from any specific treatment.

What is still missing: randomised controlled trial data comparing intrathecal baclofen against sham or standard care with functional outcomes measured; stratification of patients by baseline ambulatory status and GMFCS level; and funding for long-term prospective studies that track whether spasticity reduction helps or harms individual patients.

Evidence

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

Pediatric Neurosurgery · 2001 · 30 citations

Selective Posterior Rhizotomy and Intrathecal Baclofen for the Treatment of Spasticity

AbstractSpasticity occurs in children and adults due to a wide range of conditions, including cerebral palsy, head and spinal cord trauma, cerebrovascular accidents and multiple sclerosis. Multiple treatment options have been described, including medical and surgical treatments. Medical treatments include intramuscular botulinum A toxin, oral baclofen and supportive bracing. Surgical approaches include selective posterior rhizotomy, intrathecal baclofen and orthopedic procedures to address deformities. Many reports have been published on these different treatment options, but rarely has a comparison been made between them. Therefore, this review is aimed at comparing selective posterior rhizotomy and intrathecal baclofen injection for spasticity due to cerebral palsy, especially in children.

https://doi.org/10.1159/000050392
Journal of Neuroscience Nursing · 1995 · 7 citations

Intrathecal Baclofen for Spasticity of Cerebral Palsy: Project Coordination and Nursing Care

AbstractSpasticity caused by cerebral palsy is painful and disabling. Infusion of an intrathecal antispasmodic for relief is investigated in a multicenter, interdisciplinary clinical trial. Clinical nurse specialists coordinate local team endeavors. The nursing process serves as a functional framework for project development, protocol implementation and long-term patient follow-up.

https://doi.org/10.1097/01376517-199506000-00004
JAMA · 1991 · 1 citations

Intrathecal Baclofen: Not All Spasticity Is Bad

Abstract<h3>To the Editor.</h3> —Albright et al<sup>1</sup>detail the clinical usefulness of intrathecal baclofen for treating spasticity in patients with cerebral palsy as a more effective alternative to oral administration. However, one disadvantage with this approach, as with other pharmacological modes of treatment, is the potential for simultaneous elimination of "functional forms" of spasticity. Excessive spasticity reduction in the lower-extremity antigravity muscles may significantly diminish the ability of the patient with spasticity to ambulate. Aptly labeled "physiological crutch" in certain clinical instances, spasticity can enable the patient with central nervous system injury to walk and to achieve optimal functional recovery.

https://doi.org/10.1001/jama.1991.03470010070029
PubMed · 2019 · 0 citations · open access

[Precise diagnosis and treatment of spastic cerebral palsy].

AbstractOBJECTIVE: To summarize the advancement of precise diagnosis and treatment for spastic cerebral palsy in recent years. METHODS: The literature and own experiences were reviewed, and the surgical method, precise diagnosis, and personalized treatment of spastic cerebral palsy based on the classification of spastic cerebral palsy were summarized and analyzed. RESULTS: The common classification of spastic cerebral palsy are gross motor function classification system (GMFCS) and manual ability classification system (MACS). The surgical methods of spastic cerebral palsy can be divided into soft tissue surgery, nerve surgery, and bone and joint surgery. The precise diagnosis of spastic cerebral palsy includes qualitative diagnosis, localization diagnosis, and quantitative diagnosis. Based on precise diagnosis and classification, one or more corresponding surgical methods are selected for treatment. CONCLUSION: The manifestations of spastic cerebral palsy are so diverse that it is necessary to select rational surgeries based on precise diagnosis to achieve individualized treatment.

https://doi.org/10.7507/1002-1892.201903072

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.