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- Question
- hello,
I wanted to find out the research against cystic fibrosis, which projects could emerge soon? and then at what stage is gene therapy?
thank you
- Answer
- Hello,
Here last news about clinical research in cystic fibrosis:
- Ataluren : ecorn-cf.eu/index.php?id=65&L=0&tx_expertadvice_pi1[showitem]=1586&tx_expertadvice_pi1[search]=Ataluren%20project
- VX770 and VX880 : ecorn-cf.eu/index.php?id=65&L=0&tx_expertadvice_pi1[showitem]=1361&tx_expertadvice_pi1[search]=Good%20news%20about
- regarding Miglustat: two clinical trials conducted by the pharmaceutical company Actelion (head office: Switzerland) took place first in Spain and Belgium. Unfortunately the results of these tests were not conclusive.
You can find information in French by clicking on this link:
www.vaincrelamuco.org/e_upload/pdf/message_jean_lafond_miglustat_et_cp_cnrs.pdf
A new phase II trial to be conducted only in France should be released in late 2011/early 2012.
- Bronchitol: the european marketing authorization will soon be obtained and the product marketed soon
- antibiotic: a phase III trial comparing the efficacy and safety of Aeroquin (inhaled levofloxacin) to those of the Tobramicine (inhalation solution) is underway in France
- gene therapy: This strategy aims to provide to the cells an 'healthy' CFTR gene, capable of the synthesis of a functional CFTR. The healthy gene is provided by a viral vector or a synthetic vector. Viral vectors have so far been unsatisfactory, but researchers have developed new synthetic vectors.
A pilot study supervised by the UK CF Gene Therapy Consortium with twenty patients in Britain has just ended. If the results are positive, a new study should raise this time one hundred patients.
Best regards
Anna Ronayette
- 02.01.2012
