DeCure's autonomous Neuro AI scientist is researching a drug-repurposing hypothesis for Spinocerebellar ataxia type 42 — 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 moduleSpinocerebellar ataxia type 42 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 spinocerebellar ataxia type 42 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
calcium voltage-gated channel subunit alpha1 G (CACNA1G) — CACNA1G 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 3pedrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 6KZO · 3.3 Å · ligand 1,2-Distearoyl-sn-glycerophosphoethanolamine (3PE). Experimental structure, not a prediction.
What the evidence adds up to
The 2025 IDEA study followed 53 patients with early-stage SCA types 1, 2, 3, and 6, plus 24 healthy controls, for two years. Mean age was 48.7 years and mean SARA score was 9.3 at baseline. Few measures showed statistically significant change at 12 months. At 24 months, the FARS-ADL, PROM-Ataxia total, PROM-Ataxia physical, and PROM-Ataxia ADL scores showed the strongest associations of change. The authors conclude that patient-reported or derived outcome measures can capture longitudinal change over two years even in early disease, but more work is needed to identify outcomes that reliably capture change earlier.
A 2024 report describes six patients from two families with SCA type 7 who received riluzole 50 mg twice daily on a compassionate-use programme for a mean of 4.8 years (range 3.5–9 years). Riluzole showed no effect on visual function in two patients with advanced retinal damage. Four patients had improvements in visual function followed by ophthalmologic stability up to five years after starting treatment. Two patients had a less steep deterioration of ataxia after treatment compared with pre-treatment during the first 2.5 years. One patient showed an improvement in SARA score soon after therapy, then overall stability for 3.5 years, followed by ataxia worsening. One visually impaired patient without neurological impairment did not worsen until the last visit after 3.5 years. Two patients showed improvement in SARA scores soon after therapy and overall stability lasting five and three years respectively. No adverse events were registered.
The 2014 highlights from Bettencourt and colleagues address possible relationships among known SCA genes, predict their functions, identify overlapping pathways, and provide a framework for candidate gene discovery using whole-transcriptome expression data. They identified significant cell types and pathways in SCA pathogenesis.
No abstract in this set concerns SCA type 42 specifically. The riluzole data come from only six SCA7 patients with no control group, so any suggestion of benefit is preliminary and cannot be generalised. The IDEA study shows that even in early SCA, reliable change detection at 12 months remains elusive. What is still missing are adequately powered, placebo-controlled trials for any SCA subtype, validated biomarkers that can detect change within one year, and patient stratification by genetic subtype and disease stage.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Movement Disorders Clinical Practice · 2025 · 4 citations · open access
Longitudinal Changes in Patient‐ and Clinical‐Reported Outcomes in Early Spinocerebellar Ataxia Types 1, 2, 3, and 6 from the <scp>IDEA</scp> Study
AbstractBACKGROUND: Clinical outcomes assessments (COAs) in spinocerebellar ataxia (SCA) need to be standardized, ataxia-specific, sensitive to change, clinically relevant, and meaningful to patients. OBJECTIVES: To evaluate the longitudinal 1- and 2-year performances of different patient reported outcomes, including the Patient Reported Outcome Measure of Ataxia (PROM-Ataxia), and clinician reported outcomes, including FARS and SARA, in those with early manifest symptoms of SCA 1, 2, 3, and 6. METHODS: We studied 53 patients with early stage SCA1-3 and SCA6 from The Instrumented Data Exchange for Ataxia Study and 24 age-matched healthy controls. Participants were seen every 6 months for 2 years. Mixed models were used to estimate change over 12- and 24-months of follow-up. Changes on the FARS-FS and PGI-C were used as anchors to estimate meaningful changes. RESULTS: Among persons with SCA, mean age was 48.7 years and mean SARA score was 9.3. Few measures showed statistically significant changes at 12 months. At 24-months, the FARS-ADL, PROM-Ataxia total, PROM-Ataxia physical, and PROM-Ataxia ADL scores showed the strongest associations of change. CONCLUSIONS: Patient reported or derived outcome measures, such as FARS-ADL and ADL sub domain of the PROM-Ataxia, can capture longitudinal change in patients' symptom experience over a 2-year period and its impact on daily activities, even in those with early disease. More work is needed to identify outcomes that reliably capture change earlier.
Revista Neurociências · 2013 · 3 citations · open access
Atendimento Fisioterapêutico para Indivíduos com Ataxia Espinocerebelar: Uma Revisão da Literatura
AbstractThe spinocerebellar ataxia (SCA) is a disorder characterized by deficits in the execution of coordinated movements with progressive postural sway associated with difficulty in maintaining balance and various other motor disorders. The gait may be ataxic, with broadening the base of support, instability, irregular steps and slow, lateropulsion and trembling in range of motion, so that physical therapy is an important alternative for the improvement of the disorders of this pathology. Objective. Make, based on scientific literature, a review of physical therapy strategies in the treatment of spinocerebellar ataxia. Method. The study researches the databases Medline and SciELO from 2001 to 2011, considering the following keywords: ataxia espinocerebelar, Fisioterapia, tratamento, reabilitação and its correlates in English. Results. We found 33 studies that had as its main theme ataxia, 20 articles were excluded because they did not report the physical therapy approach for this type of pathology. After review, 13 references were used. Conclusions. After this study, the importance of physical therapy in the treatment of patients with SCA becomes obvious, according to the benefits promoted, as all studies found an improvement of symptoms of this pathology. Methodological limitations observed suggest the need for greater rigor in future research.
Long-Term Follow-Up before and during Riluzole Treatment in Six Patients from Two Families with Spinocerebellar Ataxia Type 7
AbstractBACKGROUND: Currently no curative treatment exists for spinocerebellar ataxias (SCAs). Riluzole repurposing was proposed as a symptomatic treatment in different types of cerebellar ataxia. We report a long-term-follow up under riluzole treatment in SCA type 7. METHODS: Six patients received Riluzole 50 mg twice daily on a compassionate use program for a mean of 4.8 years (range 3.5-9). We measured ataxia onset and progression through the Scale for the Assessment and Rating of Ataxia (SARA), and collected extensive ophthalmological data before and after Riluzole treatment. Electrocardiogram and laboratory profile for drug safety were performed every six months. RESULTS: Riluzole treatment showed no effect on visual function in two patients with an advanced retinal damage. Improvements of visual function occurred in four patients followed by ophthalmologic stability up to 5 years after starting treatment. Two patients had a less steep deterioration of ataxia after treatment compared to pre-treatment, during the first 2,5 years of therapy. One showed soon after therapy an improvement of the SARA score, and then overall stability lasting 3,5 years, followed by ataxia worsening. One visually impaired patient without neurological impairment did not worse until the last visit after 3,5 years of follow-up. The remaining 2 patients showed an improvement of SARA scores soon after therapy, and an overall stability lasting respectively 5 and 3 years. No adverse event was registered during the observation period. DISCUSSION: This study suggests a possible beneficial action of Riluzole in SCA7 and provides a detailed description of the ophthalmologic profile of these patients.
AbstractBettencourt and colleagues address the possible relationships among known spinocerebellar ataxia (SCA) genes, predict their functions, identify overlapping pathways, and provide a framework for candidate gene discovery using whole-transcriptome expression data. They identified significant cell types and pathways in SCA pathogenesis.
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.
