ReviewNature reviews. Clinical oncology2012
Targeted therapy in rare cancers--adopting the orphans.
Review in Nature reviews. Clinical oncology, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
20 citing papers in PubMed, 46 citations in OpenAlex.
- Discover personalized drug opportunity and optimized tumor-infiltrating lymphocyte therapy for rare cancer: an organoid-based whole-journey clinical mapping study in a neuroendocrine cancer patient.Cellular oncology (Dordrecht, Netherlands) · 2026Article
- The complexity of finding fit-for-purpose real-world data for oncology patients with rareESMO real world data and digital oncology · 2025Article
- If it is a solid tumor target, then it may be a hematologic cancer target: Bridging the great divide.Med (New York, N.Y.) · 2025Review
- Failure to progress: breast and prostate cancer cell lines in developing targeted therapies.Cancer metastasis reviews · 2024Review
- Molecular Tumor Board for Unicorns: Outcomes for rare and ultra-rare cancers using an N-of-One personalized treatment strategy.iScience · 2024Article
- Images in Immunotherapy and Precision Oncology: A Case Report of Neurofibromatosis-1.Journal of immunotherapy and precision oncology · 2024Article
- RAPID framework for improved access to precision oncology for lethal disease: Results from a modified multi-round delphi study.Frontiers in health services · 2023Article
- Advances in the Treatment of Hairy Cell Leukemia Variant.Current treatment options in oncology · 2022Review
- Targeted and cellular therapies in lymphoma: Mechanisms of escape and innovative strategies.Frontiers in oncology · 2022Review
- Challenges and solutions to the study of rare childhood tumors.Current opinion in pediatrics · 2020Review
- Comprehensive Genomic Profiling of Rare Tumors: Routes to Targeted Therapies.Frontiers in oncology · 2020Article
- PI3K/mTOR Pathway Inhibition: Opportunities in Oncology and Rare Genetic Diseases.International journal of molecular sciences · 2019Review
- So Close, yet so Far: Discrepancies between Uveal and Other Melanomas. A Position Paper from UM Cure 2020.Cancers · 2019Article
- The crucial role of multiomic approach in cancer research and clinically relevant outcomes.The EPMA journal · 2018Review
- Rare Tumor Clinic: The University of California San Diego Moores Cancer Center Experience with a Precision Therapy Approach.The oncologist · 2018Article
- Article
- The PI3K Pathway in Human Disease.Cell · 2017Review
- Rare cancers: Challenges & issues.The Indian journal of medical research · 2017Review
- Identifying priority medicines policy issues for New Zealand: a general inductive study.BMJ open · 2014Article
- Integration of cancer genomics with treatment selection: from the genome to predictive biomarkers.Cancer · 2013Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Designation of a rare 'orphan' disease is usually conferred by a prevalence of one in 1,500 to 2,500 individuals. Increasingly, orphan diseases are also being defined by their molecular fingerprints. Rare diseases are uniquely challenging from a therapeutic standpoint; it is critical to modify clinical study design of treatments for orphan disorders as well as for the increasingly smaller molecular subsets within frequently occurring cancers. In spite of the immense challenges associated with developing a treatment for a rare disorder, some of the most groundbreaking therapeutic discoveries have been made in orphan malignancies. This situation may be because a limited number of driver molecular aberrations occur in rare disorders, which can be targeted by agents. Here, we describe drug-class examples of targeted therapies for orphan diseases, with particular emphasis on malignancies or tumour-prone nonmalignant conditions, as well as potential therapeutic strategies that can be adopted to treat these orphan conditions.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.