Recherche sur les cancers rares : Ouvrir la voie au développement de nouvelles thérapies contre le cancer
Any cancer diagnosis is a difficult one; however, being diagnosed with a rare cancer can be particularly challenging as most cancer research focuses on the four major malignancies: prostate, lung, breast, and colon cancer. When patients with rare cancers wish to participate in clinical trials, there are typically fewer choices and the research sites may be at a few select, high-volume centers. However, research into rare cancers, like other rare diseases, can have a larger impact on the development and delivery of new cancer therapies to patients.
Rare cancers individually affect fewer than 6 in 100,000 people a year in the US (13% of all cancers diagnosed in adults), and in the EU, around 640,000 people are diagnosed with rare cancers (24% of all cancers diagnosed in the EU). Rare cancers can occur throughout the body, and some common locations are the oral cavity and pharynx, digestive system, and locations within the respiratory system.
Les cancers rares, comme d'autres maladies rares, peuvent souvent être caractérisés par :
Diagnostic tardif ou incorrect
Le manque d'accès à l'expertise clinique sur la maladie
Limited availability of clinical trials, a small number of sites for patient participation
Few available registries and tissue banks for future learning
Cependant, de nombreuses thérapies actuelles contre le cancer ont été initialement étudiées dans le cadre d'études sur des cancers rares. Dans une analyse des nouveaux médicaments anticancéreux approuvés par la FDA de 1995 à 2005, 12 des 51 médicaments ont été approuvés pour les quatre principaux cancers, tandis que la majorité l'ont été pour des cancers rares. En voici quelques exemples :
· 10 years ago, researchers studying rare Fanconi anemia gained a greater understanding of bone marrow failure, cancer, and the resistance to chemotherapy. (N Engl J Med. 2010 May 20;362(20):1909-19.)
- Les chercheurs qui étudient la tumeur de Wilms, un cancer rare chez les enfants, ont mis au point un "modèle" pour l'exploration de la génétique et de la biologie cellulaire, qui a ensuite été appliqué aux cancers généraux chez les enfants et les adultes.(Methods Mol Biol. 2003;222:239-48.)
· When researchers were studying an investigational medication for the treatment of chronic myeloid leukemia (CML), seminoma, and gastrointestinal stromal tumor (GIST) it opened the door for the development of “molecularly targeted therapies” and led to a whole new category of cancer therapies, tyrosine kinase inhibitors. (Curr Opin Cell Biol. 2009 Apr;21(2):288-95. Epub 2009 Feb 11.) (Curr Pharm Des. 2009;15(2):120-33.)
Exploring rare cancer has led to research into the triggers of the growth and spread of tumors, and potentially how to target them with innovative therapies.
Sources :
1. https://www.rarecancerseurope.org/About-Rare-Cancers/The-Added-Value-of-Research-on-Rare-Cancers. Updated 2019. Accessed November 5, 2020
2. Joint Action for Rare Cancers Europe Brochure: www.jointactionrarecancers.eu. Accessed November 5,2020.
3. Braiteh F, and Kurzrock R. Uncommon tumors and exceptional therapies: paradox or paradigm? Mol Cancer Ther 2007;6(4).
Parrainé par Boehringer Ingelheim
