Welcome to Chemren.com!Help Center 中文 Sign upLogin
Post Information
Products Suppliers Technologies Group Buying Surplus
Organic Raw Materials
Hydrocarbon Compounds Alcohols、Phenols、Ethers Aldehydes、Ketones、Quinones Esters Carboxylic Acids Halides Aromatic Compounds Heterocyclic Compounds Amino Acids、Proteins、Nucleic Acids Nitrogenous Organic Compounds Nitrogenous Organic Compounds Phosphorated Organic Compounds Sulfurated Organic Compounds Other Elemental Organic Compounds Organic Salts Carbohydrates Others
Organic Intermediates
Synthetic Intermediates Other Intermediates
Biochemicals and Pharmaceutical Chemicals
APIs Pharmaceutical Intermediates Biological Products Pharmaceutical Excipients Veterinary Drugs Finished Dosage Forms Pharmaceutical Impurities
Pesticides Fertilizers Pesticide Intermediates Others
Food and Feed Additives
Food Additives Feed Additives
Flavors and Fragrances
Flavors and Essences Spices Flavor and Fragrance Intermediates
Dyes and Pigments
Dyes Pigments Dye Intermediates
Catalysts and Additives
Additives and Auxiliaries Catalysts
Coatings and Paints
Coatings Paints Inks Others
Organic Adhesives Inorganic Adhesives
Inorganic Chemicals
Inorganic Salts Inorganic Acids Inorganic Alkalines Inorganic Oxides Elements Halides Others
Natural Products and Extracts
Plant Extracts Herbal Extracts Natural Products
Material Chemicals
Polymer Materials Metal and Ceramic Materials Nanomaterials
Electronic Chemicals
OFET Materials Electrode Materials Liquid Crystals
Household Chemicals
Cosmetics Oral Care Chemicals Skin Care Chemicals Household Insecticides Detergent Chemicals
Heat Exchange Equipment Reaction Equipment Crushing Equipment Mixing Equipment Separation Equipment Pressure Vessel Refrigeration Equipment Mass Transfer Equipment Molding Equipment Storage/Transport Equipment Drying Equipment Green Facilities Pump/Valve Equipment Conveying Equipment Instrumentation Auxiliary Equipment Crystallization Equipment Seal Machinery Heating Equipment Other Chemical Equipment
Other Chemicals
Other Chemicals
Location:Home > News Detail

New insights emerge in an effort to repurpose Novartis’ cancer drug Tasigna in Parkinson’s disease

Last Update:2019-03-14   |   Comments:0 A+ A-

Brief:A research team at Georgetown University Medical Center previously repurposed Novartis’ blood cancer drug Tasigna for Parkinson’s disease, and turned up promising results in a preclinical mouse study and in a small group of patients. Now, they have return

A Georgetown University Medical Center research team showed Novartis' cancer drug Tasigna could target a misfolded protein implicated in Parkinson's. (alex-mit/iStock/Getty Images Plus)

A research team at Georgetown University Medical Center previously repurposed Novartis’ blood cancer drug Tasigna for Parkinson’s disease, and turned up promising results in a preclinical mouse study and in a small group of patients. Now, they have returned with further insight into how the drug works in people with Parkinson’s.


A pharmacology analysis of 75 Parkinson’s patients who are currently participating in a phase 2 study showed that Tasigna can reduce levels of toxic alpha-synuclein protein clumps that are the hallmark of the disease. That increases the levels of dopamine in the patients’ brains. The Georgetown team reported their findings in the journal Pharmacology Research & Perspectives.


Tasigna (nilotinib) is approved by the FDA as a treatment for chronic myeloid leukemia. It is meant to be given twice daily at a recommended dose of as much as 400 mg for adults. In the Parkinson's study, the Georgetown researchers found that the drug could improve Parkinson’s biomarkers at a lower dose.


The team, led by Charbel Moussa, first  demonstrated Tasigna’s anti-neurodegeneration potential in 2013, testing the drug in mice that over-express alpha-synuclein. When the protein folds in the wrong way, it can block the brain from releasing neurotransmitters such as dopamine from small storage vesicles. That loss of dopamine can lead to motor symptoms like the impaired coordination that's commonly seen in Parkinson’s.


After the mouse trial showed that Tasigna could help clear the harmful accumulation of alpha-synuclein and improve movement, Moussa and colleagues moved to a small trial of 12 people with either advanced Parkinson’s or dementia with Lewy bodies. The observed improvements in motor functioning and cognitive outcomes, according to a 2016 report in the Journal of Parkinson’s Disease.  But in an editorial that ran alongside it, critics cautioned that the small sample size and lack of a control group made it impossible to rule out a placebo effect.

RELATED: Novartis cancer drug to be tested for Parkinson’s disease


In collaboration with the Michael J. Fox Foundation and others, the Georgetown researchers started the larger phase 2 trial. While the primary goal was to determine the safety of Tasigna, the researchers also searched patients’ cerebral spinal fluid (CSF) for traces of Parkinson’s biomarkers. They measured levels of 3,4-Dihydroxyphenylacetic acid (DOPAC) and homovanillic acid (HVA) in the CSF. Because these molecules are produced when dopamine is metabolized, they could indicate how much dopamine was being used in the brain.


The patients were divided into five groups that received Tasigna at 150 mg, 200 mg, 300 mg or 400 mg or a placebo. After a single dose of Tasigna, the researchers found higher levels of DOPAC and HVA than were seen in the control group.


“When the drug is used, levels of these breakdown molecules quickly rise,” Moussa said in a statement. “This is exciting because this kind of potential treatment for Parkinson’s could increase use of a patient's own dopamine instead of using or periodically increasing drugs that mimic dopamine.”


Furthermore, Tasigna at 200 mg—which the team found to be the optimal dose for elevating DOPAC and HVA—also significantly increased the level of TREM-2 in the central nervous system. Because of TREM2’s anti-inflammatory role in the brain, some have suggested it could target misfolded alpha-synuclein and other harmful plaques in neurodegenerative disorders.


Other scientists are also investigating potential new uses of approved drugs in neurodegeneration. A team at University College London, for example, previously showed that GLP-1 receptor agonist exenatide, which is the active ingredient in AstraZeneca’s Byetta and Bydureon for Type 2 diabetes, also improved motor functions in some Parkinson’s patients.


Fernando Pagan, principal investigator of the Tasigna phase 2 trial and first author of the paper said the findings suggest the Novartis drug can reduce protect dopamine-secreting neurons. But how that can be translated into clinical outcomes remains to be seen. The trial is expected to be completed in mid-2020, according to a listing on ClinicalTrials.gov.




About Us
Contact Us
User Agreement
Privacy Policy
Resource Docking
Offline Activities
Technical Services
Sales Promotion
Update Info
Retrieve Password
Sign Up
User Center
Product Presales
Technology Purchase
Technology Transfer
Sell Products