DeCure for Adult-onset proximal spinal muscular atrophy, autosomal dominant
DeCure's autonomous Neuro AI scientist is researching a drug-repurposing hypothesis for adult-onset proximal spinal muscular atrophy, autosomal dominant — 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 moduleAdult-onset proximal spinal muscular atrophy, autosomal dominant 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 adult-onset proximal spinal muscular atrophy, autosomal dominant 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
VAMP associated protein B and C (VAPB) — VAPB 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 apo structuredrag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 3IKK · 2.5 Å · ligand none (apo structure). Experimental structure, not a prediction.
What the evidence adds up to
The 2019 review notes that spinal muscular atrophy (SMA) type 1, the most severe early-onset form, has shifted from a rapidly lethal disease to one where long-term event-free survival and acquisition of important motor milestones are likely. For SMA type 2, prognosis has shifted from slow and progressive deterioration to long-term stability. However, the review emphasises a large heterogeneity in clinical response to currently available treatments, ranging from absence of response to impressive improvement. The only factor identified as predictive of treatment success is the age of the patient at initiation of treatment, closely related to disease duration.
A 2024 review on repurposed drugs for SMA lists branaplam, riluzole, olesoxime, harmine, and prednisolone as agents that have shown some improvement in treating the condition. The same review states that despite comprehensive investigations, effective treatments or interventions remain elusive. It criticises the current strategy for medication repurposing as lacking systematicity, frequently depending more on serendipitous discoveries than on organised approaches, and calls for a methodical approach targeting the molecular origins of SMA.
Two 2025 clinical practice guidelines for adolescent and adult SMA patients, developed by multidisciplinary experts from Chinese tertiary medical centres, acknowledge progress in multidisciplinary care and disease-modifying therapies, with significantly improved survival and quality of life. They note that no prior clinical practice guidelines existed for the management of SMA in adult and adolescent patients. These guidelines serve as an instrumental reference for standardised care of Chinese SMA patients.
What is still missing is a systematic, methodical approach to drug repurposing that targets the molecular origins of SMA, rather than relying on serendipity. The predictive factor of early treatment age remains the only identified determinant of success, and the heterogeneity of response is unexplained. No trial design or patient stratification strategy is offered that accounts for this variability, and the guidelines are limited to Chinese patients, leaving generalisability unaddressed.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Therapeutics and Clinical Risk Management · 2019 · 200 citations · open access
<p>Clinical Evidence Supporting Early Treatment Of Patients With Spinal Muscular Atrophy: Current Perspectives</p>
AbstractRecent advances in the treatment of spinal muscular atrophy (SMA) have dramatically altered prognosis. Rather than a rapidly lethal disease, SMA type 1, the most severe form with the earliest onset of SMA, has become a disease in which long-term event-free survival with the acquisition of important motor milestones is likely. Prognosis for patients with SMA type 2 has shifted from slow and progressive deterioration to long-term stability. Nevertheless, there is a large heterogeneity in terms of clinical response to currently available treatments, ranging from absence of response to impressive improvement. The only factor identified that is predictive of treatment success is the age of the patient at the initiation of treatment, which is closely related to disease duration. The aim of this paper is to review available evidence that support early intervention using currently available treatment approaches.
Cellular and Molecular Neurobiology · 2024 · 5 citations · open access
Spinal Muscular Atrophy: Current Medications and Re-purposed Drugs
AbstractSpinal muscular atrophy (SMA) is an autosomal recessive genetic neuromuscular disorder that is characterized by gradual muscle weakness and atrophy due to the degeneration of alpha motor neurons that are present on the anterior horn of the spinal cord. Despite the comprehensive investigations conducted by global scientists, effective treatments or interventions remain elusive. The time- and resource-intensive nature of the initial stages of drug research underscores the need for alternate strategies like drug repurposing. This review explores the repurposed drugs that have shown some improvement in treating SMA, including branaplam, riluzole, olesoxime, harmine, and prednisolone. The current strategy for medication repurposing, however, lacks systematicity and frequently depends more on serendipitous discoveries than on organized approaches. To speed up the development of successful therapeutic interventions, it is apparent that a methodical approach targeting the molecular origins of SMA is strictly required.
Clinical Practice Guideline for Adolescent and Adult Patients with Spinal Muscular Atrophy – Part 3
AbstractIn recent years, the field of spinal muscular atrophy (SMA) has made progress in multidisciplinary care and disease-modifying therapies. Survival and the quality of life of patients have significantly improved. However, no clinical practice guidelines exist for the management of SMA in adult and adolescent patients. Multidisciplinary experts from a number of tertiary medical centers in China, specializing in the diagnosis and treatment of SMA, came together to remedy this using evidence-based medicine. This guideline serves as an instrumental reference for the standardized care of Chinese SMA patients.
Clinical Practice Guideline for Adolescent and Adult Patients with Spinal Muscular Atrophy – Part 2
AbstractIn recent years, the field of spinal muscular atrophy (SMA) has made progress in multidisciplinary care and disease-modifying therapies. Survival and the quality of life of patients have significantly improved. However, no clinical practice guidelines exist for the management of SMA in adult and adolescent patients. Multidisciplinary experts from a number of tertiary medical centers in China, specializing in the diagnosis and treatment of SMA, came together to remedy this using evidence-based medicine. This guideline serves as an instrumental reference for the standardized care of Chinese SMA patients.
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.
