Preliminary findings from a small, proof of concept pilot study using the repurposed cancer drug Nilotinib
Why is it important?
- Novelty 80% 80%
- Proximity 40% 40%
- Deliverability 40% 40%
Nilotinib is a cancer drug which does that, in addition to other functions. Evidence from animal studies has shown that it can reduce the accumulation of a-synuclein, protect dopamine neurons and increase dopamine.
At baseline, and at 2 and 6 months, blood samples were taken and lumbar puncture was performed to check the concentration of the drug and c-Abl, dopamine metabolites and a-synuclein in blood and cerebrospinal fluid which is contained in the inner chambers of the brain and bathes the spinal cord (CSF).
Within the 6 month period, some adverse events occurred: one out of the 12 patients taking part had a heart attack, which the authors attributed to an undetected abnormality prior to commencing the study, while another 2 patients showed evidence of adverse changes in heart function. Since Nilotinib can sometimes lead to fatal heart problems in patients with leukemia, more rigorous testing is required. Less serious adverse events included a urinary tract infection and pneumonia requiring hospitalization but which are not uncommon in patients at later stages. Preliminary changes in certain markers of harmful oxidative stress, amyloid and dopamine degradation products, and some improvement in cognition and movement following Nilotinib treatment were observed.
However, since this small study was conducted in very few patients, and importantly, there was no placebo group, or a blinding process, it is difficult to interpret. Since the placebo effect is pronounced in Parkinson’s it is impossible to tell whether the suggested increases in dopamine, cognition and movement were caused by Nilotinib, or the expectation of improvement (placebo effect).