DeCure's autonomous Neuro AI scientist is researching a drug-repurposing hypothesis for spinocerebellar ataxia type 36 — screening already-approved drugs against its 2-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 36 maps to a 2-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 36 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.
What the evidence adds up to
Spinocerebellar ataxia type 36 is one of more than 30 spinocerebellar ataxias for which causative mutations have been identified. The past 25 years have seen the cloning of genes for the common spinocerebellar ataxias, and recurrent pathophysiological themes across these disorders include protein aggregation, failure of protein homeostasis, ion channel dysfunction, DNA repair defects, and mitochondrial dysfunction. Effective therapies for ataxias are still lacking, though novel drug targets are under investigation and an increasing number of therapeutic trials are expected.
A 2013 review of physical therapy strategies for spinocerebellar ataxia found 13 relevant studies from 2001 to 2011. All studies reported improvement of symptoms, but the authors noted methodological limitations and called for greater rigour in future research. No specific numbers for improvement or sample sizes were given in that review.
A 2023 review states that no disease-modifying therapy has been established for spinocerebellar degeneration. Only symptomatic therapy is currently available. Taltirelin and protirelin are drugs covered by health insurance for cerebellar ataxia symptoms in Japan and are expected to suppress symptom progression. Muscle relaxants are used for spasticity. The authors state that a new therapeutic agent with a different mechanism of action, aimed specifically at modifying disease progression, is needed.
A 2025 review confirms that autosomal-dominant spinocerebellar ataxias remain incurable. Certain medications and physical therapy can alleviate symptoms of cerebellar ataxia. The review expresses high hopes that gene therapy methods will be developed to slow or stop disease progression, but no such therapy is yet available. What is still missing for spinocerebellar ataxia type 36 specifically are dedicated clinical trials, patient stratification by genetic subtype, and funding for the development of disease-modifying or gene-based treatments.
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 · 2011 · 94 citations · open access
Milestones in ataxia
AbstractThe past 25 years have seen enormous progress in the deciphering of the genetic and molecular basis of ataxias, resulting in improved understanding of their pathogenesis. The most significant milestones during this period were the cloning of the genes associated with the common spinocerebellar ataxias, ataxia telangiectasia, and Friedreich ataxia. To date, the causative mutations of more than 30 spinocerebellar ataxias and 20 recessive ataxias have been identified. In addition, there are numerous acquired ataxias with defined molecular causes, so that the entire number of distinct ataxia disorders exceeds 50 and possibly approaches 100. Despite this enormous heterogeneity, a few recurrent pathophysiological themes stand out. These include protein aggregation, failure of protein homeostasis, perturbations in ion channel function, defects in DNA repair, and mitochondrial dysfunction. The clinical phenotypes of the most common ataxia disorders have been firmly established, and their natural history is being studied in ongoing large observational trials. Effective therapies for ataxias are still lacking. However, novel drug targets are under investigation, and it is expected that there will be an increasing number of therapeutic trials in ataxia.
Journal of Clinical Case Reports · 2015 · 12 citations · open access
Human Embryonic Stem Cells in the Treatment of Spinocerebellar Ataxia: A Case Series
AbstractSpinocerebellar ataxias, dominantly inherited ataxias, constitute a large heterogenous group of progressive neurodegenerative disorders that commonly affects the cerebellum and its afferent and efferent pathways. No pharmacological treatment has been found to be effective in the treatment of spinocerebellar ataxias. Stem cellbased therapy is emerging as a promising therapeutic option for the treatment of Spinocerebellar ataxias. In this case report, three patients with spinocerebellar ataxias were treated with human embryonic stem cells. Following the treatment, all patient showed noticeable changes in their health such improvement in hand eye coordination, gait pattern, ability to stand without support, muscle strength in all the limbs, ability to walk and turn while standing without support, clearance in speech, good energy levels, reduction in twitching of cheek muscle, stamina, endurance and coordination.
Ataxia espinocerebelosa tipo 2: una experiencia en la rehabilitación psicológica
AbstractINTRODUCTION: Psychological rehabilitation in patients with neurodegenerative disorders helps to improve their quality of life and provide the most suitable approach to their disease. There are no records of this type of treatment being used in type 11 spinocerebellar ataxia. OBJECTIVES: To evaluate the efficacy of group therapy within the framework of psychological rehabilitation and determine the most favored psychological function markers. PATIENTS AND METHODS: This study is a quasi experimental study of 24 patients with type 2 spinocerebellar ataxia rehabilitated in the CIRAH (Cuba). The procedure involved psychological assessment before and after the strategy for intervention, which consisted of 15 sessions of group therapy. RESULTS: The pathological levels of anxiety were reduced in 50.1% of the cases, and 31.4% of the patients with depression improved. The self assessment markers of a taking step forward were happiness and worry level. The disorder affected all aspects of the life of the patients studied, particularly their interests, family, self esteem and work. CONCLUSIONS: It is possible to improve the attitude of the patient to his disease and his psychological function by means of group therapy during the process of psychological rehabilitation of patients with type 2 spinocerebellar ataxia.
Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques · 1976 · 4 citations · open access
Design of the Investigation
AbstractThe general outline of the complete prospective study of 50 cases of spino-cerebellar degeneration is given. The general protocol followed, the criteria for inclusion and the mode of analysis are described. The aim of this study was to establish a base of clinical, physiological and biochemical facts upon which a logical and systematic approach to pathogenesis and treatment of Friedreich's ataxia could be attempted.
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.
AbstractNo disease-modifying therapy has been established for spinocerebellar degeneration and multiple system atrophy, and only symptomatic therapy is currently available. Taltirelin and protirelin are drugs covered by health insurance for cerebellar ataxia symptoms, and are expected to suppress the progression of symptoms. Muscle relaxants are used for spasticity associated with spinocerebellar degeneration, and vasopressors and therapeutic agents for dysuria are used for autonomic symptoms of multiple system atrophy. It is necessary to develop a new therapeutic agent with a different mechanism of action, aimed specifically at modifying the disease progression in patients with spinocerebellar degeneration and multiple system atrophy.
Yakut Medical Journal · 2025 · 0 citations · open access
Approaches to the treatment of autosomal-dominant spinocerebellar ataxias
AbstractThe article is devoted to the prospects for the treatment of neurodegenerative diseases with dynamic mutations based on published studies of the search for approaches to the treatment of spinocerebellar ataxia. Although these diseases are incurable, research results show that certain medications and physical therapy can alleviate the symptoms of cerebellar ataxia. Due to the progress made in the study of spinocerebellar ataxia in recent years, there are high hopes that it will be possible to develop gene therapy methods that will slow down the progression of the disease or even stop its development.
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.
