Treatment of Cystic Fibrosis with paullone derivatives

Treatment of Cystic Fibrosis with
paullone derivatives
CONTEXT
Our reference :
63960
Status des brevets
French priority patent
application n°
FR0410961 filed in
October 15, 2004, and
entitled: "Utilisation de
dérivés de paullones
pour la fabrication de
médicaments pour le
traitement de la
mucoviscidose et de
maladies liées à un
défaut d’adressage des
proteines dans les
cellules"
Cystic fibrosis (CF) is a major inherited disorder affecting most critically the lungs, but also
the pancreas, liver, and intestine. The disease affects arounf 70,000 people in the world,
mostly of european anscestry. Several treatment exists but the lifetime of the patients
remains shortened.
Cystic fibrosis (CF) involves abnormalities of fluid and chloride and sodium electrolytes
transport through epithelia due to mutation leading to abnormal function of cystic fibrosis
transmembrane conductance regulator (CFTR) protein.
Although there are over 1,000 mutations of CFTR proteins, the most common is delF508
that affects 70% of patients and causes the deletion of a phenylalanine at position 508.
This mutation alters the folding of the protein. Therefore, although the delF508 CFTR
could still be active as a chloride ion channel, it is no longer able to reach the membrane
and is rapidly degraded.
TECHNICAL DESCRIPTION
The invention relates to the use of paullone derivatives for the treatment of cystic fibrosis,
a family of compounds acting as CDK inhibitors and exhibiting antiproliferative activity.
The inventors have shown that some paullone derivatives, especially kenpaullones, could
act as activators of wild-type and mutant CFTR, and could induce the relocation of
delF508 mutated CFTR to the plasma membrane in CF epithelial cells, thereby restoring
its electrolyte transmembrane transport capacity.
DEVELOPMENT STAGE
Inventors
Frédéric BECQ
Laurent MEIJER
Conrad KUNICK
Status Commercial
Exclusive or nonexclusive licenses
Laboratoires
A in vitro screening of paullone derivatives is ongoing and retained candidates will be
validated on mice models of cystic fibrosis.
For further information, please contact us (Ref 63960)
Institut de physiologie et
biologie cellulaires
(IPBC, UMR6187),
Poitiers, France.
Keywords :
Cystic Fibrosis paullone
Powered by TCPDF (www.tcpdf.org)