advanced research

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