Hi Olivia,
The potential use of a tki such as nilotinib for purposes other than CML refers to some research work being undertaken at Georgetown Medical Centre in USA .There was a small sample of 12 patients enrolled in a clinical trial and low dose nilotinib was administered daily for six months and the conclusion was that there was proof of evidence that the treatment significantly increased brain dopamine and reduced toxic proteins linked to disease progression in patients with Parkinsons disease or DLB-dementia Lewy bodies.Dopamine is the brain chemical or neurotransmitter lost as a result of death of dopamine producing neurons in a range of neurodegenerative diseases.The researchers examined biomarkers in the blood and cerebral spinal fluid as well as cognitive measures and indicators of motor and non- motor improvement.There was evidence of significant improvement as a result of the nilotinib therapy but 3 months after withdrawal from the drug sadly participants reverted back to the same reduced cognitive and motor state they had prior to the beginning of the study.The same Centre will undertake larger studies and trials in the future: the sample will be larger and with some randomised controlled groups taking a placebo.
It is interesting that there might be some cross over benefits from tkis that were invented for the purposes of treating CML and GIST.At the moment nilotinib is so called "off label" for the treatment of neurodegenerative diseases and is not licensed for that purpose -it would not be possible to engage a qualified neurologist to prescribe even if the drug were purchased privately.Residents of US who are near to Mexico can hop across the border and secure supplies or fly to Panama to be treated-apparently it is happening.I am not aware of any generics of nilotinib that are yet available.
CML was once a fatal disease but thanks to tkis is for some now a managed condition;as we know it is not cured but if one is able to maintain a bcr/abl score below 0.1 then according to most clinicians one is in safe territory.Dementias are terminal conditions and so far therapies slow down or modify the degeneration of the brain and just pospone the end of life. Of course not all, like many of us on this forum, are able to manage their CML and some succumb to the accelerated and blast phases.
Olivia your comment that those of us on tkis are relatively fortunate and symptom and side effect free is very true;I find it relatively easy to manage my own CML compared to that of my partner who exhibits substantial symptoms of DLB which are very difficult to manage.
With best wishes
John
