The discoveries follow complicated paths and appear a posteriori to be self-evident. We have been working for years on a co-transporter (NKCC1) which plays a major role in maintaining ionic balances in almost all cells. Bumetanide has shown promise in treating a number of brain diseases, including autism (see my blog ). I put forward a seemingly far-fetched hypothesis, namely that brain tumor metastases depended on ionic modifications involving NKCC1, which also regulates cell volumes. A bibliographic study confirms as usual that the idea is not new and numerous experimental data confirm the very promising actions of Bumetanide for treating Glioblastomas – a particularly severe form that is completely drug-resistant. But, to put the odds in our favor, it seemed to me that in order to migrate and metastasize, the cells needed to flatten given the pressure of the tumor and the restricted volume around them. And Bingo, a molecule commonly used to treat intestinal worms (taenia tecta) acts on the cellular cytoskeleton. And here too, I didn’t invent anything, the teams from the University of Baltimore (USA) showed experimental data and pilot trials that Mebendazole, which acts on the cytoskeleton, had a beneficial action on Glioblastoma.
The next step was obvious, to show on human Glioblastoma cell lines that the 2 molecules act and even better that they have synergistic cations. and as these generic molecules are repositioned, we were able to test them on 2 patients in the terminal phase of brain tumors, with a spectacular effect on one of the 2 patients with a survival of almost 10 months while the outcome being given the severity of the tumor was imminent. We have patented this dual approach in Europe and the US.
The rest…as usual, carry out larger trials and above all raise funds and with this aim hoping to convince a pharma of the interest of our approach.
The moral of this brief story is that thinking outside the box always has an advantage, allowing discoveries to be made, it remains to materialize it and provide treatment for the patients
