Colchicin

Question

Hello,
I read an article (relatively old) that speaks of colchicine, an anti-cancer molecule which could have beneficial effects on CF. Could you please give me some news about this molecule?

Answer

Hello,
First, let me recall a fact that has raised a hope toward colchicin: a cystic fibrosis patient treated with chemotherapy for cancer has seen his respiratory function improved significantly. (cases reported in the Lancet).
It is known that anticancer treatments can induce the production of proteins of the same family as the CFTR protein. The hypothesis was that these proteins induced by chemotherapy, could replace the CFTR protein whose function is abnormal in cystic fibrosis.

Then a medical team conducted a preliminary study in 1997 on a small number of patients (8) with colchicin (which has an effect on cell proliferation but also anti-inflammatory effects and is less toxic than chemotherapy) to examine whether this molecule could have a beneficial effect on the health of CF patients. The results of this first pilot study were encouraging (see reference section) and justified the initiation of a clinical trial of colchicin compared to placebo.

A phase II trial was conducted from April 1999 to October 2000: all patients received N-acetyl-cysteine with or without colchicin. The methodology of this trial was controversial and the results were not published in any scientific paper. To date no other studies regarding colchicin and cystic fibrosis have been performed.

In clinical research, for 100 promising compounds, only a few come to pass all the steps and become an effective drug with fewer side effects. Several molecules whose mechanism is to correct the function of the protein are currently in advanced stages, see the reply on the forum ECORN: ecorn-cf.eu/index.php?id=65&L=0&tx_expertadvice_pi1[showitem]=1187&tx_expertadvice_pi1[search]=


Hope this provides some answers

Anna Ronayette (Head of clinical research programs of Vaincre la mucoviscidose)

Références:
Lancet. 1997 Sep 6;350(9079):711-2.
Mediators Inflamm. 1999;8(1):13-5: downloads.hindawi.com/journals/mi/1999/395129.pdf

05.05.2011