RE: 40119 Oct 2018 16:38
HardToHandle, difficult to explain in laymans terms, I can do my best to summarise various science and key trial requirements.
VAL401 is a re-purposed licensed drug (risperidone + fatty acid), in lab tests and animal testing, it demonstrated efficacy at slowing the rate of cancerous growth (note: technically not cure). It works by inhibiting HSD10, HSD10 is a natural body process that gives cells energy and tells them to grow (and not die). Valirx have proved that VAL401 inhibits HSD10, and should slow down the cancer growth (prolonging life and reducing pain). HSD10 is overexpressed by a factor of 10x in certain cancer types - so bringing it back down to normal stops the runaway cell growth.
Valirx wanted to push straight into a Phase 3 trial, the regulator apparently suggested a short Phase 2 trial to validate that the VAL401 re-formulation was safe and did not throw out any unusual surprises, so a phase 2b trial was arranged.
In summary for the results, the key findings are
1) No patients exploded, a key requirement.
2) I believe they confirmed that the drug entered the body in the assumed manner, and they had signs that it may be working
3) 50% of patients reported a reduction in pain, interesting, because the pain is typically caused by the tumour tearing through natural flesh
Take home indicators: Vague potential demonstrations of efficacy, possible cleared path to full phase 3 trials, potential indications of increased life expectancy and pain reduction, modest initial data on PK drug behaviour in humans.
VAL401 could be a relatively cheap helping drug in the fight against cancer, or it may join a hundred other failed cancer drug efforts. I personally suspect it may have a modest efficacy in certain patients - it will probably be useless in other types (for example sex-hormone driven cancers).