A cancer drug has given hope to Parkinson’s patients, after a small trial showed it can improve cognitive and motor skills.
As researchers explain, nilotinib (Tasigna) is normally administered for blocking a protein that stimulates chronic myeloid leukemia. In addition, it appears it also helps deactivate another protein that negatively impacts lysosomes, which are virtually a waste disposal system allowing the removal of harmful proteins, viruses or bacteria.
Basically, nilotinib boosts the activity of lysosomes, and according to researchers, this process can assist in clearing toxic proteins that are associated with Parkinson’s disease.
Initially, the theory was proven through an animal experiment, since the drug succeeded in ‘rescuing’ mice which had been paralyzed due to Parkinson’s.
Afterwards, study authors conducted a small trial involving 12 volunteers suffering from advanced stages of Parkinson’s disease or a related disorder called dementia with Lewy bodies. The participants were administered daily doses of the drug for a period of 6 months.
It appears that Tasigna broke the blood-brain barrier, and reached the patients’ brain, as researchers detected it in the cerebrospinal fluid. Moreover, the drug was successful in stabilizing or even diminishing levels of tau, alpha-synuclein and amyloid beta proteins, while boosting dopamine levels.
As a result of this, despite the fact that the patients’ condition was very severe, the 11 people who completed the trial made significant progress, some actually showing signs of improvement as early as 3 weeks after the study began.
Clear headway was made when it came to motor skills, since subject who could barely move were eventually able to walk, sit or bend their legs. Tremor and “freezing” were greatly reduced, one patient was able to feed herself again, and another one no longer required a walker.
Moreover, there was discernible amelioration when it came to communication skills, as well as when it come to energy levels. Also, cognitive skills improved by approximately 5 points, on a scale of 30. Even more remarkably, 3 participants with severe speech impairment managed to regain their speaking skills as well, by the end of the trial.
“We’ve seen patients at end stages of the disease coming back to life”, explained study leader Charbel Moussa, of Georgetown University Medical Center in Washington D.C.
For example, one of the volunteers, Alan Hoffman, who had suffered from Parkinson’s since 1997, declared that thanks to the drug, he was finally able to dress himself, make the bed, do household chores or focus well enough in order to read a book.
The findings were presented at the Neuroscience 2015 meeting in Chicago, on October 17. Although nilotinib can normally cause unwanted side-effects when it comes to cancer patients (headaches, nausea, arrhythmia, fatigue), much lower doses were effective in this trial, and no adverse reactions were identified.
Therefore, it appears this drug gives hope that not only Parkinson’s disease symptoms can be alleviated, but also that the actual progression of the disease can be halted.
On the other hand, other researchers tend to be skeptical and call for a larger follow-up study in order to test the effectiveness of the drug against a placebo. These reservations are natural, especially given the fact that another compound called GDNF initially looked promising too, but its benefits couldn’t be replicated on a bigger scale.
Moreover, the downside is that even if Tasigna were proven potent enough, its effects only last for the duration of the treatment, and this normally costs a staggering $ 10,000 per month.
Image Source: Flickr