Leukemia Drug May Stop Dementia Related Plaque Buildup

Caregiver, patient expert

An existing drug already used to treat leukemia has shown promise as a treatment for neurological diseases such as Lewy body dementia, Alzheimer's disease and Parkinson's disease. Researchers from Georgetown University successfully used small doses of the drug nilotinibin in experiments using mice.   This drug, which is used to treat chronic myelogenous leukemia (CML), appears to eliminate abnormal protein build-up in the brain.

An article on FoxNews.com reported that the researchers targeted the alpha-Synuclein and tau proteins, which have been previously implicated in the development of Parkinson's disease, Alzheimer's disease, amyotrophic lateral sclerosis (ALS), Huntington's disease, Lewy body dementia and other neurodegenerative conditions.

Lead study author Dr. Charbel E-H Moussa, head of the neuroscience department at Georgetown University, explained, "In degenerative diseases these proteins accumulate and kill the cell. The best strategy to make the cell survive is to clean the debris."

The researchers estimate that humans would only need one percent of the dose generally used in chemotherapy in order to see neurological benefits. This could potentially decrease the already minimal side-effects of nilotinib. Moussa hopes that the drug will quickly enter phase two clinical trials, but that depends on funding. "These drugs are FDA approved already, so we know they are well-tolerated in humans. All the toxicity data - it's all available," Moussa said.

Lewy body dementia is the first type of dementia that Moussa and his colleagues would like to target with the drug as they go into clinical trials. They hope to expand the clinical trials to include Alzheimer's and Parkinson's patients. Their research will be published online May 10 in the journal Human Molecular Genetics.

Other existing drugs being tested for effectiveness against dementia

Last February, I wrote about the psoriasis drug ustekinumab. A group of German and Swiss researchers referred to medications tested in psoriasis and other autoimmune diseases such as Crohn's disease and multiple sclerosis as the "ideal candidate for the initiation of clinical trials for Alzheimer's." The new study was published in a recent edition of the journal Nature Medicine.

In October of 2012, I wrote about the anti-cancer drug Gleevec. Gleevec has been shown to disable a newly discovered key protein linked to the development of Alzheimer's. The protein, gSAP, stimulates production of toxic beta-amyloid which is linked to the development of plaques in the brain that are typically associated with Alzheimer's disease.

As I've mentioned in the above posts on existing drugs, I find these studies particularly hopeful in that they involve drugs already in use, therefore they are deemed safe for humans.That fact should speed up FDA approval for fighting other diseases.

I'd like to see funding for research on these existing drugs fast-tracked so that those already affected by AD can potentially have a chance to improve their condition. Anything that can speed up access to drugs that may help people with dementia needs to be done. People with Alzheimer's don't have many other options.

For more information about Carol visit  www.mindingourelders.com  or www.mindingoureldersblogs.com.


Woerner, A. (2013, May 10) Potential treatment for Parkinson's, Alzheimer's, dementia discovered. Fox News online. Retrieved from http://www.foxnews.com/health/2013/05/09/potential-treatment-for-parkinsons-alzheimers-dementia-discovered/

Healy, M. (2012, November 27) Psoriasis drug may halt or reverse Alzheimer's disease. LA Times. Retrieved from http://www.latimes.com/health/boostershots/la-heb-alzheimers-autoimmune-reverse-mental-decline-20121127,0,3568193.story

Kolata, G. (2012, November 14)   Alzheimer's Tied to Mutation Harming Immune Response. New York Times.   Retrieved from http://www.nytimes.com/2012/11/15/health/gene-mutation-that-hobbles-immune-response-is-linked-to-alzheimers.html?_r=0Alzinfo.org.gSAP: A Key Protein in Plaque Formation Retrieved from http://www.alzinfo.org/07/alz-guide/gsap-key-protein