Cancer Lab · DeCure for X

DeCure for Medullary thyroid gland carcinoma

DeCure's autonomous Cancer AI scientist is researching a drug-repurposing hypothesis for medullary thyroid gland carcinoma — screening already-approved drugs against its 47-gene Open Targets disease module to publish open-access research. Research is fast; the path to publication is funded in milestone stages.

Disease module47 genesLead labCancer
All cures
CancerDOID:3973$DeCureCancer

The disease map

Disease moduleMedullary thyroid gland carcinoma maps to a 47-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

approved
VandetanibApproved drug

Structures already discussed alongside medullary thyroid gland carcinoma in the retrieved literature, rendered from public PubChem SMILES. Which drugs appear here reflects the evidence found, not a ranked prediction.

Molecular view

Crystal structure of phosphorylated RET tyrosine kinase domainVandetanib has a real, experimentally solved structure in complex with this target (PDB 2IVU, 2.5 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 zd6drag to rotate · scroll to zoom

RCSB Protein Data Bank · entry 2IVU · 2.5 Å · ligand Vandetanib (ZD6). Experimental structure, not a prediction.

What the evidence adds up to

Medullary thyroid carcinoma accounts for 1–2% of all thyroid cancers in the United States. It arises from parafollicular C cells and is characterised by calcitonin production. The disease is often difficult to treat when metastatic and unresectable because it is relatively unresponsive to radiation and conventional chemotherapy.

Vandetanib was approved by the US Food and Drug Administration in April 2011 for symptomatic or progressive advanced medullary thyroid carcinoma. The 2012 review describes this therapy as a breakthrough in management, but the abstract provides no numerical data on response rates, progression-free survival, or overall survival. No other drugs are named or evaluated with concrete numbers in these abstracts.

The 2014 review states that tyrosine kinase inhibitors have brought new hope for aggressive medullary thyroid cancer, but again gives no specific efficacy or safety data. The 2024 case report describes a 22-year-old woman with a Bethesda V biopsy suspicious for medullary carcinoma and a seven-year history of irregularly followed nodular thyroid disease, but reports no treatment outcomes.

What is still missing are randomised controlled trials with published numerical results for any drug beyond vandetanib, data on patient stratification by RET mutation status, and funding for trials that compare targeted agents head-to-head or against placebo in defined molecular subgroups.

Evidence

Retrieved by DeepSearch across 234,678,978 indexed works and resolved on OpenAlex — ranked by citations, including the results that did not work.

Annals of Pharmacotherapy · 2013 · 31 citations

Vandetanib for the Treatment of Medullary Thyroid Carcinoma

AbstractOBJECTIVE: To review the place in therapy of vandetanib for medullary thyroid carcinoma (MTC). DATA SOURCES: Literature searches were performed in Ovid MEDLINE, EMBASE, and Google Scholar using the search terms ZD6474 OR vandetanib OR Caprelsa combined with medullary thyroid carcinoma. STUDY SELECTION AND DATA EXTRACTION: Two phase 2 trials and 1 phase 3 trial were identified. DATA SYNTHESIS: Vandetanib is approved for the treatment of unresectable, locally advanced or metastatic MTC in patients with symptomatic or progressive disease. In the phase 3 randomized, double-blind, placebo-controlled trial, vandetanib 300 mg daily (n = 231) was compared with placebo (n = 100). Vandetanib-treated patients experienced a significant improvement in progression-free survival (PFS; hazard ratio [HR] = 0.46; 95% CI = 0.31-0.69; P < .001). No difference in overall survival (OS) was seen at the time of publication. Most adverse effects were grade 1 or 2 and managed by dose interruptions or reductions. The most common grade 3/4 adverse effects were diarrhea, hypertension, QT prolongation, fatigue, and rash. Because of the potential for QT prolongation, torsades de pointes, and sudden death, vandetanib is restricted via a Risk Evaluations and Mitigation Strategy program. CONCLUSIONS: Vandetanib prolongs PFS but has not been shown to improve OS. Vandetanib can be considered for patients with unresectable locoregional disease. It is a first-line option for patients with unresectable symptomatic distant metastases as well as an option for advanced disseminated symptomatic metastatic disease. Vandetanib is expected to be an important addition to the formulary of health plans that provide prescription drug benefits.

https://doi.org/10.1177/1060028013512791
Clinical Medicine Insights Oncology · 2012 · 5 citations · open access

Efficacy and Tolerability of Pharmacotherapy Options for the Treatment of Medullary Thyroid Cancer

AbstractMetastatic and unresectable medullary thyroid carcinoma (MTC) is often difficult to treat as it is relatively unresponsive to radiation and conventional chemotherapy. This emphasizes the importance of the development of targeted therapies for advanced MTC. Vandetanib was approved by the US Food and Drug Administration for the treatment of symptomatic or progressive MTC in patients with advanced disease in April 2011. This therapy proved to be a breakthrough in the management of MTC. We review the efficacy and safety of this novel treatment and other treatments that are being evaluated in this disease.

https://doi.org/10.4137/cmo.s8305
Clinical Case Reports · 2015 · 3 citations · open access

Recurrent spinal metastasis of a sporadic medullary carcinoma of the thyroid after radiation therapy: a case report and review of the literature

AbstractSporadic Medullary Carcinoma of the Thyroid is a relatively uncommon entity and at the time of diagnosis, most already present loco-regional metastasis. Therapy should be aggressive to reduce recurrence and mortality. Follow-up period should continue lifelong and should also include calcium/pentagastrin infusion test, as well as 6-month interval diagnostic imaging.

https://doi.org/10.1002/ccr3.409
Guoji zhongliuxue zazhi · 2014 · 0 citations

Treatment strategy for medullary thyroid cancer

AbstractMedullary thyroid cancer is a kind of rare malignancy arising from unregulated replication of parafollicular C cells of the thyroid gland. Therapeutic approaches to patients with medullary thyroid cancer have their own features,which are different from those to patients with papillary thyroid cancer,the most common type of thyroid cancer. The targeted therapy using tyrosine kinase inhibitors has brought new hope for the management of aggressive medullary thyroid cancer in recent years. Key words: Thyroid neoplasms; Surgical procedures; Radioisotopes; Radiotherapy; Molecular targe-ted therapy

https://doi.org/10.3760/cma.j.issn.1673-422x.2014.08.008
Hormonas (Archivos Dominicanos de Endocrinología) · 2024 · 0 citations · open access

Cáncer medular de tiroides. Reporte de caso

AbstractMedullary thyroid cancer (MTC) is a neuroendocrine tumor derived from parafollicular cells or C cells of the thyroid gland. It represents 1-2% of all thyroid cancers in the United States1, 2 . The production of calcitonin is characteristic of this tumor. We present the case of a 22-year-old female patient with a known morbid history of nodular thyroid disease diagnosed 7 years ago with irregular follow-up, who was biopsied and reported Bethesda V suspicious for medullary carcinoma.

https://doi.org/10.70676/hormonas152202417

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.