MSA COALITION FUNDED EXPERIMENTAL LABORATORY STUDIES OF POTENTIAL DISEASE-MODIFYING TREATMENTS FOR MSA

These compounds have all been studied in experimental models of MSA in the laboratory. The MSA Coalition provided the seed funding to allow researchers to carry out this critically important work. Before a compound can be approved by the FDA, this early laboratory work is required in order to build up enough evidence to propose a solid rationale for their use as a therapy for MSA patients.

Formal clinical trials using these compounds will need to be further developed for MSA patients. The MSA Coalition has funded the below early stage laboratory work – also known as “translational research” – which has served to lay the groundwork that may yet hold promise for the future.

The MSA Coalition is tremendously grateful to our community who have donated or raised critically needed funds that were directed towards these studies!

**NEW** April 14, 2020: Nilotinib Fails to Prevent Synucleinopathy and Cell Loss in a Mouse Model of Multiple System Atrophy.

December 18, 2019: Nilotinib Study Results Show People with Parkinson’s Disease Should Pass on This Drug

November 18, 2019: FTY720-Mitoxy reduces synucleinopathy and neuroinflammation, restores behavior and mitochondria function, and increases GDNF expression in Multiple System Atrophy mouse models. (Fingolimod)

November 4, 2019: Researchers begin trial of drug to slow progression of neurodegenerative condition Multiple System Atrophy (Exendin-4)

July 15, 2019: The molecular tweezer CLR01 reduces aggregated, pathologic, and seeding-competent α-synuclein in experimental multiple system atrophy.

Click on each compound to learn more details about the role of the MSA Coalition in funding these laboratory studies and what we have learned so far.

EXENDIN-4(EXENATIDE) - Phase 2 Clinical Trial in Progress *

** MSA COALITION FUNDED ** Laboratory work to test Exendin-4 in MSA experimental mouse models was previously carried out at Bordeaux University, France. This project was funded by the MSA Coalition in 2015-2016 and showed positive results, paving the way for MSA patient trials.

Exendin-4 (Exenatide) is an FDA approved drug for treating Diabetes.

A Phase 2 pilot study to test Exendin-4 in MSA patients is beginning soon at the University College London.

https://clinicaltrials.gov/ct2/show/NCT04431713

Related Articles:

November 2019: Researchers begin trial of drug to slow progression of neurodegenerative condition Multiple System Atrophy

May 2017: New hope for devastating neurodegenerative disease

May 2017: Insulin resistance and exendin-4 treatment for multiple system atrophy

March 2017: Report on MSA Coalition funded research Exendin-4

June 2016: Glucagon-like peptide 1 analogues for treating multiple system atrophy: A translational study


NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

CLR-01

** MSA COALITION FUNDED **

Known as “molecular tweezers”, CLR-01 is being tested in MSA experimental mouse models through a joint collaborative project between University of California Los Angeles and Innsbruck Medical University.

This project was funded by the MSA Coalition in 2015-2017.

Related Presentations:

January 2019: Promising results in a new proof-of-concept preclinical study of the drug candidate CLR01

Related Articles:

July 2019: The molecular tweezer CLR01 reduces aggregated, pathologic, and seeding-competent α-synuclein in experimental multiple system atrophy.

March 2017: Targeting Alpha-Synuclein Pathology with the Molecular Tweezer CLR01 in Multiple System Atrophy


NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

FINGOLIMOD

** MSA COALITION FUNDED **

An FDA approved drug for treating Multiple Sclerosis is being tested in MSA experimental mouse models at Texas Tech University Health Sciences Center.

This project was funded by the MSA Coalition in 2016.

Dr. Ruth Perez presented preliminary results of this work at the MSA Coalition’s annual conference in 2016.
(Video link coming soon)

Related Articles:

November 2019: FTY720-Mitoxy reduces synucleinopathy and neuroinflammation, restores behavior and mitochondria function, and increases GDNF expression in Multiple System Atrophy mouse models.


NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

LENALIDOMIDE

** MSA COALITION FUNDED **

The anti-inflammatory drug Lenalidomide is being tested in MSA experimental mouse models at the University of California San Diego.

This project was funded by the MSA Coalition in 2015-2016.

Dr. Eliezer Masliah presented preliminary results of this work at the MSA Coalition’s annual conference in 2015.

(Video link coming soon)

Related Articles:

January 2017: Combination of alpha-synuclein immunotherapy with anti-inflammatory treatment in a transgenic mouse model of multiple system atrophy.


NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

KALLIKREIN-6

** MSA COALITION FUNDED **

Kallikrein-6 is being tested in MSA experimental mouse models at the University of California San Diego.

This project was funded by the MSA Coalition in 2013-2014.

Dr. Eliezer Masliah presented preliminary results of this work at the MSA Coalition’s annual conference in 2015.

(Video link coming soon)

Related Articles:

September 2015: A brain-targeted, modified neurosin (kallikrein-6) reduces α-synuclein accumulation in a mouse model of multiple system atrophy.


NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

NILOTINIB

** MSA COALITION FUNDED **

An FDA approved chemotherapy drug, Nilotinib was recently explored in a Phase 2A trial in Parkinson’s disease patients.

See results here: https://clinicaltrials.gov/ct2/show/results/NCT03205488

In 2016-2017, the MSA Coalition funded a pre-clinical study to assess this drug in MSA experimental animal models.
Results of this study were presented at the Movement Disorder Society Congress in 2018 and showed that Nilotinib failed to demonstrate positive effects in the experimental MSA mouse models. See the abstract here: Nilotinib for treating MSA: A preclinical proof of concept study

Related Articles:

April 2020: Nilotinib Fails to Prevent Synucleinopathy and Cell Loss in a Mouse Model of Multiple System Atrophy.

December 2019: Nilotinib Study Results Show People with Parkinson’s Disease Should Pass on This Drug

October 2018: Nilotinib for treating MSA: A preclinical proof of concept study

October 2018: A Phase 2A Study of Nilotinib in Patients with Advanced and Early Parkinson’s disease: Study Design

July 2016: Nilotinib Update: Where We Stand with a Cancer Drug for Parkinson’s

June 2016: Nilotinib significantly alters CSF biomarkers and increases endogenous dopamine in open-label phase I clinical trial in Parkinson’s disease with dementia and Lewy body dementia


NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

MPH (Ritalin)

** MSA COALITION FUNDED **

This project was funded by the MSA Coalition in 2020. More details coming soon.

Related Presentations:

Related Articles:


NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

PLX5622

** MSA COALITION FUNDED **

This project was funded by the MSA Coalition in 2020. More details coming soon.

Related Presentations:

Related Articles:


NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

More MSA Treatment Pipeline Categories

Please be sure to check back regularly to find out what’s new in the Multiple System Atrophy Treatment Pipeline and be sure to Sign up for our newsletter mailing list to stay informed of the latest updates.

Not sure what to look for in a clinical trial? Watch this informative video: Helping you understand clinical trials

Are you interested in participating in a clinical trial? Visit www.clinicaltrials.gov, enter the name of the disease and you’ll be able to stay abreast of the latest clinical trial news. You can also enroll in the Fox Trial Finder which not only allows you to share your information for future research studies but will also inform you of any research study or clinical trial for which you qualify.

© Copyright The Multiple System Atrophy Coalition 1989-2021. All Rights Reserved.