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Report on MSA Coalition Funded Research: Exendin-4

Report on MSA Coalition Funded Research: Exendin-4

March 21, 2017

A new paper published online this month in the journal Brain “Insulin Resistance and Exendin-4 Treatment for Multiple System Atrophy” has added to a growing body of evidence that drugs designed to treat type 2 diabetes may have potential to treat multiple system atrophy and other neurodegenerative diseases. In this innovative work, partially funded with a seed grant from the MSA Coalition in 2015, Wassilios Meissner and his team at the University of Bordeaux, France describe results that suggest the anti-diabetic drug exendin-4 may be neuroprotective in a mouse model of multiple system atrophy.

In the experiment, MSA mice were split into three groups, one group received a placebo and each of the other two

University of Bordeaux MSA Research Team L to R: Wassilios Meissner, MD, PhD; Pierre-Olivier Fernagut, PhD; Erwan Bezard, PhD

University of Bordeaux MSA Research Team
L to R: Wassilios Meissner, MD, PhD; Pierre-Olivier Fernagut, PhD; Erwan Bezard, PhD

groups received different doses of exendin-4 over several months. Those mice treated with the drug showed more preserved dopamine neurons and a reduced accumulation of alpha-synuclein, the same protein that accumulates in the brains of MSA patients.

The Importance of Animal Model Experiments

Animal model experiments (known as pre-clinical trials) are an important early step in the process of determining if a drug is a good candidate to treat MSA. Although these results seem positive, it is not yet possible to know if this or similar drugs will work for MSA patients. Dr. Meissner states, ”Preclinical models can at best illustrate the biological engagement of a compound and make us confident that the drug will do what it is supposed to, while the real test will always remain the evaluation in humans.“ Indeed, gathering this preliminary evidence aids in building a stronger rationale for moving on to clinical studies of a drug in patients.

Mounting Evidence from Other Neurodegenerative Disease Research

In recent years, exendin-4 as well as another FDA approved anti-diabetic drugs liraglutide have both shown positive effects in animal models of Alzheimer’s Disease (AD) and Parkinson’s Disease (PD). Early phase clinical studies of these drugs are in progress now with AD and PD patients with results expected to be reported soon.

What is the Next Step for Multiple System Atrophy?

The pharmaceutical industry drug owners are already starting to take an interest in repurposing this class of type 2 diabetes drugs, known as GLP-1 analogues, for neurodegenerative diseases. MSA researchers are already engaged in discussions with industry about the future possibility of more drug trials with derivatives of GLP-1 analogues. It is hoped these trials might include MSA patients.

About the MSA Coalition Research Grant Program

The MSA Coalition takes a three pronged approach to investments in research:

  1. Fund studies looking for clues to the underlying cause (pathogenesis) of MSA.
  2. Fund research to develop a definitive biomarker to achieve an accurate early diagnosis.
  3. Fund work to evaluate and develop potential new therapies.

Up to now the MSA Coalition’s investments in research have taken the form of small grants of up to $50,000 each. These are offered to research groups around the globe looking to test new and innovative ideas. 27 such grants have been funded since 2013 for a total investment of $1.25 Million.

In January 2017 the MSA Coalition published a request for research proposals and received back 51 applications requesting a total of $2.3 million in funding. Peer review of these projects has begun in order to pare down the number of applications to fund only the best of the best within our limited research budget. The top projects will be awarded funds from the 2017 research budget amounting to $525,000.

The MSA Coalition thanks our generous donors for their support without which our work would not be possible. Donations are gratefully accepted at

Frequently Asked Questions

Q. Can I ask my doctor to prescribe Exendin-4 (Byetta or Bydureon) “off label”?

A. There is no clear evidence available that this drug will help MSA patients. In the event an off label prescription is obtained, the cost would likely not be covered by health insurance even though this is an FDA approved drug, because it is not currently indicated for use in MSA.

Q. Can I get Exendin-4 (Byetta or Bydureon) prescribed through a “compassionate use” program?

A. Your doctor would need to apply to the drug company (Astra Zeneca) and also to the FDA to request this. This information from the FDA describes the Compassionate Use Program (also known as “Expanded Access”).

Q. When and where will a clinical trial for Exendin-4 be available to MSA patients?

A. This is unknown at this time. The MSA Coalition is currently assessing the situation and getting scientific opinions. It may be that more preliminary work is needed in animal models before this could realistically be tried in human MSA patients.

Q. Where is the best source for MSA patients to track clinical trials?

A. You can search for clinical trials at the following websites:

Q. How can MSA patients be included in future clinical trials?

A. MSA patients in every country are encouraged to register in the Global MSA Registry (GLOMSAR) so that they may be notified of future clinical trials and participate in research surveys. Note that a caregiver or family member may enter the information on behalf of the patient.