DeCure's autonomous Neuro AI scientist is researching a drug-repurposing hypothesis for amyotrophic lateral sclerosis type 11 — 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 moduleAmyotrophic lateral sclerosis type 11 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 amyotrophic lateral sclerosis type 11 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
FIG4 phosphoinositide 5-phosphatase (FIG4) — FIG4 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 7K1W · 5.1 Å · ligand none (apo structure). Experimental structure, not a prediction.
What the evidence adds up to
Amyotrophic lateral sclerosis type 11 is a fatal motor neuron disorder for which riluzole remains the only therapy approved by both the FDA and EMA as of 2022, and its effect on survival is described as modest or marginal. Two other drugs, edaravone, have been approved by the FDA, but these also provide only modest benefits in mortality or function. Most patients die within 2 to 5 years of diagnosis. No abstract specifies results for amyotrophic lateral sclerosis type 11 separately from the broader disease.
A 2005 review notes that beta-lactam antibiotics and minocycline were being re-investigated as neuroprotective agents, and that heat-shock protein co-inducers were a novel class of compounds under study. By 2007, no effective therapies beyond riluzole had emerged in the decade since its approval, though drugs were in Phase I, II and III trials. A 2022 review mentions the recent approval of two new drugs in some countries and the development of targeted gene therapies, but does not name them or give survival or response rates. The same review states that despite impressive advances in understanding the disease's mechanisms, the only drug approved by both the FDA and EMA remains riluzole.
A 2023 paper highlights that tracking disease progression remains a major challenge, and that flawed outcome measures can miss real treatment effects or falsely suggest positive effects for ineffective therapies. The 2005 review warns of the long-standing failure to transfer therapeutic compounds to the clinic. No abstract reports a completed trial showing improved survival or function for any drug in amyotrophic lateral sclerosis type 11 specifically.
What is still missing are clinically relevant preclinical models that translate to human trials, reliable and responsive outcome measures that can detect modest treatment effects in a heterogeneous patient population, and trial designs that avoid exposing patients to ineffective therapies. The socioeconomic and ethical issues of patients' need for rapid access to new treatments remain unresolved.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
Current Opinion in Neurology · 2005 · 62 citations
Amyotrophic lateral sclerosis: recent advances and future therapies
AbstractPURPOSE OF REVIEW: Amyotrophic lateral sclerosis is a rare but fatal motoneuron disorder. Despite intensive research riluzole remains the only available therapy, with only marginal effects on survival. Here we review some of the recent advances in the search for a disease-modifying therapy for amyotrophic lateral sclerosis. RECENT FINDINGS: A number of established agents have recently been re-investigated for their potential as neuroprotective agents, including beta-lactam antibiotics and minocycline. Progress has also been made in exploiting growth factors for the treatment of amyotrophic lateral sclerosis, partly due to advances in developing effective delivery systems to the central nervous system. A number of new therapies have also been identified, including a novel class of compounds, heat-shock protein co-inducers, which upregulate cell stress responses thereby mediating neuroprotection. Non-drug-based therapies are also under development, with progress in gene-silencing and stem cell therapies. SUMMARY: In the past few years, significant advances have been made in both our understanding of amyotrophic lateral sclerosis pathogenesis and the development of new therapeutic approaches. However, caution must be exercised in view of the long-standing failure to successfully transfer therapeutic compounds to the clinic. A deeper awareness in the research community of the need for clinically relevant preclinical studies, coupled with a better understanding of the issues surrounding clinical trial design for amyotrophic lateral sclerosis, offers hope that the growing list of validated preclinical therapeutics can finally yield an effective disease-modifying treatment.
Expert Opinion on Investigational Drugs · 2007 · 33 citations
Current clinical trials in amyotrophic lateral sclerosis
AbstractAmyotrophic lateral sclerosis is caused by selective degeneration of motor neurons in the brain and spinal cord. There are still no other effective therapies 10 years after the approval of riluzole for the treatment of amyotrophic lateral sclerosis, but advances in drug development and screening are substantially increasing the number of potential therapeutic agents. This review provides an overview of clinical trial methodology in amyotrophic lateral sclerosis followed by a systematic evaluation of drugs that are presently in Phase I, II and III clinical trials. There is an emphasis on the scientific evidence supporting the selection of each drug being tested, as well as on trial design.
Journal of Personalized Medicine · 2022 · 29 citations · open access
Status of ALS Treatment, Insights into Therapeutic Challenges and Dilemmas
AbstractAmyotrophic lateral sclerosis (ALS) is an extremely heterogeneous disease of motor neurons that eventually leads to death. Despite impressive advances in understanding the genetic, molecular, and pathological mechanisms of the disease, the only drug approved to date by both the FDA and EMA is riluzole, with a modest effect on survival. In this opinion view paper, we will discuss how to address some challenges for drug development in ALS at the conceptual, technological, and methodological levels. In addition, socioeconomic and ethical issues related to the legitimate need of patients to benefit quickly from new treatments will also be addressed. In conclusion, this brief review takes a more optimistic view, given the recent approval of two new drugs in some countries and the development of targeted gene therapies.
The Journal of Clinical Pharmacology · 2005 · 15 citations
Clinical Trials in Amyotrophic Lateral Sclerosis: The Tenuous Past and the Promising Future
AbstractThe past decade of research in amyotrophic lateral sclerosis has contributed to a greater understanding of the disease process, the development of relevant animal models, and the identification of several therapeutic approaches that may delay disease progression. Completed and ongoing clinical trials and the process of selecting drugs for clinical trials are presented.
AIMing for Better Tools to Track Disease Progression
AbstractDespite recent progress in amyotrophic lateral sclerosis (ALS) therapeutic development, accurately tracking disease progression remains an ongoing challenge for research studies and in the clinic. The need for more reliable and responsive outcome measures is particularly important in the setting of ALS clinical trials, where modest treatment effects are evaluated in patient populations with heterogenous phenotypes and rates of decline. At a time where underlying causes of disease are still not understood, yet stepwise progress toward new treatments is expected, flawed outcome measures have the ability to miss real treatment effects when they are actually present or can falsely suggest the presence of positive treatment effects for ineffective therapies, potentially exposing patients to unnecessary costs and risks.
Molecular Medicine Communications · 2022 · 0 citations · open access
Emerging Therapies in Amyotrophic Lateral Sclerosis
AbstractAmyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder characterized by the loss of cortical and spinal motor neurons, leading to weakness, muscle atrophy, and, in a substantial number of patients, cognitive impairment. Most patients die within 2 to 5 years of diagnosis. The disease initiates from the death of upper and lower motor neurons leading to a degeneration of motor pathways and the paralytic effects of the disease. The disease has huge economic costs as well. FDA has approved two drugs, riluzole, and edaravone for the treatment of ALS. However, these drugs provide modest benefits in mortality and/or function. Recent developments in the understanding of the underlying pathophysiologic processes that contribute to ALS have led to the development of numerous investigational therapies, with several now in phase 3 trials. This article highlights the epidemiology, pathophysiology, and several current and emerging treatment options for ALS including stem cell therapy.
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.
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