The 2021 virtual American Academy of Neurology (AAN) Annual Meeting is taking place from April 17 through April 22, 2021. During the conference, experts and researchers are offering a wealth of knowledge and education into topics surrounding neurology. According to Parkinson’s News Today, one poster presentation covers the beneficial effects of Tasigna (nilotinib) for patients with Parkinson’s disease. Check out an abstract from the presentation here.
Nilotinib
Historically, nilotinib has been used as a treatment for patients with chronic myelogenous leukemia (CML). However, researchers from Georgetown University explored Nilotinib as a potential therapy for patients with Parkinson’s disease. This is because much like nilotinib inhibits BCR-ABL in CML, BCR-ABL also plays a role in cell death and oxidative stress. Thus, researchers hypothesized that nilotinib could have a helpful effect for Parkinson’s.
In a Phase 2 clinical trial, 63 patients enrolled. During the trial, patients received either 150mg nilotinib, 300mg nilotinib, or a placebo daily for a 12 month (1 year) period. Following the trial, patient outcomes were explored in a 15-month extension study.
In initial data, reported in 2019, researchers determined that nilotinib was safe, well-tolerated, and effective. The drug helped to raise dopamine levels in the brains of patients with Parkinson’s disease. Since Parkinson’s is characterized by dopaminergic (dopamine-producing) neuron death, being able to raise dopamine levels is crucial in preventing disease progression.
Now, researchers are presenting extension data from the 15-month extension trial. Some of the findings include:
- Both 150mg and 300mg doses were safe and well-tolerated.
- Patients receiving 300mg nilotinib experienced a better quality of life (QOL) than the lower dose group.
- Unfortunately, the lower dose group experienced a QOL decline according to data measurements from the Parkinson’s Disease Questionnaire. However, patients receiving 300mg nilotinib did not see any worsening of their motor or non-motor skills following the 15-month treatment period.
As a result, researchers believe further trials should be performed exploring 300mg nilotinib as a possible treatment option for patients with Parkinson’s disease.
Parkinson’s disease (PD)
Overall, dopaminergic neuron death causes Parkinson’s disease, a progressive central nervous system (CNS) disorder. Normally, dopamine helps translate messages between the brain and muscles. But when dopamine-producing neurons degenerate and die, those messages are inhibited. Doctors believe that genetics, Lewy bodies, or environmental factors could play a role in developing Parkinson’s disease. Altogether, Parkinson’s occurs in five stages, characterized by worsening motor symptoms. In stage one, most patients experience only light tremors, typically on one side of the body. However, symptoms become increasingly noticeable through the second and third stages. These stages are characterized by muscle rigidity and tremors on both sides of the body, as well as changes in movement and balance.
By the fourth stage, many patients with Parkinson’s disease are unable to live independently. Finally, in stage five, many patients are no longer able to stand or walk. At this stage, patients may also experience neurological symptoms, such as hallucinations or delusions. Typically, Parkinson’s disease symptoms appear after 50 years old. However, they can appear earlier. Symptoms include:
- Muscle rigidity
- Slowed movement
- Tremors in one or both hands
- Note: A common tremor experienced by patients is called a “pill-rolling” tremor, and consists of the repeated rubbing of the thumb and index finger.
- Dementia or hallucinations
- Changes in speech, such as slurring or stuttering
- Difficulty standing or walking
- Impaired posture and balance
- Incontinence
- Loss of smell
