DeCure's autonomous Rare AI scientist is researching a drug-repurposing hypothesis for myxedema — screening already-approved drugs against its 41-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.
Disease moduleMyxedema maps to a 41-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
approvedLevothyroxineApproved drug
Structures already discussed alongside myxedema in the retrieved literature, rendered from public PubChem SMILES. Which drugs appear here reflects the evidence found, not a ranked prediction.
Molecular view
TRANSTHYRETIN THR119MET PROTEIN STABILISATION — Levothyroxine has a real, experimentally solved structure in complex with this target (PDB 1F86, 1.1 Å). This is the drug's own deposited structure, not a prediction, and confirms it is a structurally characterised molecule rather than an untested guess.
Loading structure…
helix sheet t44drag to rotate · scroll to zoom
RCSB Protein Data Bank · entry 1F86 · 1.1 Å · ligand Levothyroxine (T44). Experimental structure, not a prediction.
What the evidence adds up to
In 1924, the tendon reflex was described as a valuable aid in diagnosing myxedema, a constitutional disease in adults caused by decrease or absence of thyroid secretion (thyroxin) and characterised by a markedly decreased basal metabolic rate, myxedematous tissue, slowed mental condition, and typical facial expression. The authors noted that even then myxedema was frequently unrecognised, with patients going from place to place and often being treated for other conditions.
A 2020 case report describes a two-year-old boy with lissencephaly and developmental delay who presented with myxedema involving his face, arms, feet and legs, along with mild pericardial effusion and borderline QT prolongation. His thyroid function tests showed TSH of 562 mcu/ml and FT4 of 0.3 ng/dl; TPO and anti-thyroglobulin antibodies were negative, and thyroid ultrasound showed a small gland in the normal location. Newborn screen for congenital hypothyroidism had been normal. He was started on levothyroxine 50 mcg per day. The authors note the precise etiology and duration of his hypothyroidism, and its role in his developmental delay, remain unknown.
For pretibial myxedema specifically, a 1975 prospective study of nine patients treated with intralesional injections of triamcinolone acetonide reported complete resolution of myxedematous plaques in seven patients; the other two showed partial resolution but did not complete treatment. The most effective dosage was monthly injection of 8 ml or less of a 5 mg/ml solution. New nodules developed in some patients after initial therapy but responded to reinjection. A 1988 case report describes a 64-year-old man with severe pretibial myxedema in whom local fluocinolone acetonide was unsuccessful; plasmapheresis followed by azathioprine resulted in complete and long-lasting improvement of skin lesions.
What is still missing are controlled trials for any of these interventions in myxedema, particularly for the pretibial form, and any data on systemic myxedema treatment beyond thyroid hormone replacement in a single toddler. No trial has established optimal dosing, duration, or patient stratification for triamcinolone, plasmapheresis, or immunosuppression in this condition.
Evidence
Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.
JAMA · 1924 · 71 citations
TENDON REFLEXES IN MYXEDEMA: A VALUABLE AID IN DIAGNOSIS
AbstractSir William Gull,<sup>1</sup>in 1873, called attention to a disease with a distinctive group of symptoms which later became known as myxedema, and since then physicians' interest in this disease has steadily increased. Myxedema, according to Boothby's<sup>2</sup>definition, is "a constitutional disease, occurring in adults, due to decrease or absence of the secretion (thyroxin) of the thyroid gland as a result of its atrophy or removal, characterized by a markedly decreased basal metabolic rate, a myxedematous condition of the tissues, a slowed, impaired mental condition, a typical facial expression, and other secondary manifestations." Since the time of Gull, each year has brought forth an ever increasing amount of literature concerning this disease, especially about the diagnostic features. Even now, however, it is surprising how frequently myxedema is unrecognized, and the patient allowed to go from place to place in search of help, in many instances treated for such
Beneficial Effects of Plasmapheresis Followed by Immunosuppressive Therapy in Pretibial Myxedema
AbstractSummaryWe describe a 64-year-old man with severe pretibial myxedema in whom classical therapy with local applications of fluocinolone acetonide was u nsucccssful. Plasmapheresis followed by immunosuppressive therapy (azathioprinc) resulted in complete and long-lasting improvement of the skin lesions. This therapeutical regimen therefore may be of value in severe therapy-resistant pretibial myxedema.
Intralesional triamcinolone therapy for pretibial myxedema
AbstractIn a prospective study, nine patients with pretibial myxedema were treated with intralesional injections of triamcinolone acetonide. Complete resolution of the myxedematous plaques was obtained in seven of the nine patients. The other two patients failed to complete their treatment programs but did show partial resolution. For most patients the monthly injection of 8 ml or less of a solution containing 5 mg/ml of triamcinolone proved to be the most effective dosage schedule. No serious side effects were encountered. New nodules of myxedema developed in some patients after the initial completion of therapy; these nodules responded to reinjection using the same dosage schedule.
AbstractIn Response: It is exceedingly rare for a patient to present for elective surgery with myxedema coma. There are no large randomized clinical trials that demonstrate an improvement in outcome with the use of a particular regimen of thyroid hormone supplementation. Therefore, the decision to use levothyroxine, triiodothyronine, or a combination of these agents must largely be made on a theoretical basis. Dr. Mathes is correct in suggesting that a combination of levothyroxine and triiodothyronine may offer some advantages. Elliott Bennett-Guerrero, MD Department of Anesthesiology; The Mount Sinai Medical Center; New York, NY 10029 Debra A. Schwinn, MD Department of Anesthesiology; Duke University Medical Center; Durham, NC 27710
Journal of the Endocrine Society · 2020 · 0 citations · open access
SAT-LB82 Myxedema as Presenting Feature of Profound Primary Hypothyroidism in a Toddler
AbstractAbstract Background: Myxedema is a rare presenting feature of profound primary hypothyroidism that results from disordered accumulation of glycosaminoglycan in the skin and soft tissue. Very few cases of myxedema have been reported in children during the first few years of life. Clinical Case: A 2-year-old boy with a history of lissencephaly and developmental delay was sent to the emergency room by his primary care physician for worsening of edema. It was noticed by his mother six weeks previously and involved his face, arms, feet and legs. He had no history of cold intolerance, dry skin, hair loss, constipation, or excessive sleep. On exam, he had normal vital signs with no bradycardia and normal blood pressure. Physical exam was remarkable for a sallow complexion, coarse facial features, abdominal distention and non-pitting edema of the face, limbs, hands and feet. A cardiac echo revealed mild pericardial effusion and borderline QT prolongation was noted on EKG. Chest x-ray and abdominal x-ray were unremarkable. Metabolic profile showed normal electrolytes, a mildly low albumin of 2.8 gm/dl and normal renal and liver function. Thyroid function tests were remarkable for an elevated TSH of 562 mcu/ml and a low FT4 of 0.3 ng/dl. TPO and anti-thyroglobulin antibodies were negative. Thyroid ultrasound demonstrated a small thyroid gland in the normal location. Growth data revealed a normal linear growth velocity and weight persistently above the 25th percentile. The child’s newborn screen for congenital hypothyroidism had been normal. He was started on levothyroxine 50 mcg PO per day. Conclusion: We report an extremely atypical presentation of primary hypothyroidism in a two-year-old boy. He presented with myxedema, pericardial effusions, severe biochemical hypothyroidism and negative thyroid antibodies. The precise etiology and duration of his hypothyroidism, as well as its role in his developmental delay, remain enigmatic. Awareness of unusual presenting features in very young children with hypothyroidism is essential in order to achieve expedient treatment and optimal neurocognitive outcomes.
AbstractIn Response: It is exceedingly rare for a patient to present for elective surgery with myxedema coma. There are no large randomized clinical trials that demonstrate an improvement in outcome with the use of a particular regimen of thyroid hormone supplementation. Therefore, the decision to use levothyroxine, triiodothyronine, or a combination of these agents must largely be made on a theoretical basis. Dr. Mathes is correct in suggesting that a combination of levothyroxine and triiodothyronine may offer some advantages. Elliott Bennett-Guerrero, MD Department of Anesthesiology; The Mount Sinai Medical Center; New York, NY 10029 Debra A. Schwinn, MD Department of Anesthesiology; Duke University Medical Center; Durham, NC 27710
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.
