Multiple System Atrophy Coalition Sponsors the 6th International MSA Congress and 1st Global MSA Advocacy Meeting

Multiple System Atrophy Coalition Sponsors the 6th International MSA Congress and 1st Global MSA Advocacy Meeting

The International Multiple System Atrophy Congress brings together top researchers, clinicians and pharmaceutical companies to stimulate global collaboration in the urgent search for better diagnostic tools and treatments for MSA, a rare and fatal neurodegenerative disease. The MSA Coalition is a proud sponsor of this year’s Congress, hosted by NYU Langone Medical Center. In addition, the MSA Coalition has initiated the First Global meeting of MSA Advocacy groups to be held in conjunction with the scientific meeting.

CHARLOTTE, NC, February 23, 2018 – The Multiple System Atrophy (MSA) Coalition today announced its platinum-level sponsorship of the 6th International MSA Congress and 1st Global MSA Advocacy Meeting, hosted by NYU Langone Medical Center in New York City. The only international scientific and educational event devoted entirely to multiple system atrophy, the Congress begins on March 1, 2018 — which also marks the start of Multiple System Atrophy Awareness Month — and runs through March 3. The world’s most prominent MSA researchers and advocacy groups will be attending with a goal of expanding collaboration aimed at improving patient support, diagnosis, and symptomatic treatment, while working toward a potential cure for this rare and always fatal disease.

The International MSA Congress will bring together scientists and clinicians to address new discoveries and to generate ideas for future research in this long-neglected field. Global specialists in autonomic and movement disorders, neurologists, and neuroscientists are expected to attend. The congress will feature a series of lectures given by invited, world-renowned international experts on MSA and will include a keynote lecture by Nobel Laureate Dr. Stanley Prusiner.

“The International MSA Congress is the single most important gathering of MSA researchers and advocacy groups,” said Horacio Kaufmann, M.D., Director of NYU Langone’s Dysautonomia Center. “We are very thankful to the MSA Coalition for its premier sponsorship of this Congress and NYU are proud to be the first ever US host of this exciting event. With a global gathering of the best MSA researchers and top advocates, real progress can be made in understanding all of the research that has recently been completed in many institutions and a collaborative roadmap can be completed. With limited research dollars available for MSA, a shared roadmap provides the best hope for making progress against this horrible disorder.”

Advocacy groups from around the world, concerned with awareness, support, and research for Multiple System Atrophy and related neurodegenerative disorders were invited to the 1st MSA Global Advocacy Meeting to be held in conjunction with the MSA Congress. The goal of this meeting is to bring together MSA and related charities to discuss key activities and challenges, and to develop coordinated plans for advancing the mission to support the MSA patient community.

The MSA Coalition is pleased to welcome these charity participants: MSA Trust (UK), MSA-AMS (Belgium), and U.S charites MSA NJ, Defeat MSA, the MSA Shoe, Cure MSA, the Rex Griswold Foundation, Blandford-Rees Foundation, Move Over MSA, the Cleveland Clinic Lou Ruvo Center for Brain Health, CurePSP, the Michael J. Fox Foundation, and the National Organization for Rare Disorders.

“MSA Trust is looking forward to a meeting of minds at the International MSA Congress and Global Advocacy Meeting,” said Karen Walker, Chief Executive of the MSA Trust. “We will renew our commitment to work collaboratively with our colleagues to develop shared goals for the advancement of knowledge around understanding MSA and supporting those living with MSA, their families, and care partners.”

The Global Advocacy Meeting agenda will include a presentation by Debbie Drell, Director of Membership, National Organization for Rare Disorders, who will speak about collaboration models and engaging stakeholders. Jamie Eberling, Ph.D., Director of Research Programs at the Michael J. Fox Foundation for Parkinson’s Research, will share best practices in developing research programs.

“The National Organization for Rare Disorders (NORD) supports the 6th International MSA Congress and 1st Global MSA Advocacy Meeting during this historic time for the multiple system atrophy community. This year, NORD celebrates 35 years as a hub and resource for rare disease advocacy organizations and will bring the experience of these decades to the advocacy meeting,” said Debbie Drell, NORD Director of Membership. “I’m proud to speak and support the dozens of organizations around the world who are present at this meeting, for a stronger and more unified MSA network.”

The MSA Coalition continues to provide leadership and funding for global efforts to defeat MSA. Over the past 4 years, it has awarded $1.6 million to fund 36 research grants aimed at identifying diagnostic biomarkers and potential treatments. The Coalition has sponsored a past International MSA Congress, the American Autonomic Society Congress, and the European Federation of Autonomic Societies Congress, and has awarded travel grants to many young MSA investigators.

“The MSA Coalition believes it is critically important to support and collaborate on global efforts that can improve the outcomes for multiple system atrophy patients,” said Cyndi Roemer, MSA Coalition Chairperson. “We also are very excited to be hosting the 1st Global MSA Advocacy Meeting, bringing together MSA charities from around the nation and the world to collaborate on ways to best meet our shared goals to help MSA families through research, education, support services and increased awareness,” Roemer added.

“Our Global MSA Research Grant Program has injected new life into research efforts for MSA. Sponsoring the International MSA Congress and bringing so many amazing, dedicated researchers and advocates together helps to fuel enthusiasm and new ideas on how to collaborate which will speed progress towards improved treatments and hopefully a cure. Much of our revenue comes from grass-roots fundraisers, so these collaborative efforts are truly enabled by the patients themselves. The MSA community can be very proud of this sponsorship and it is a wonderful way to kick off MSA Awareness Month.”

During MSA Awareness Month in March, the MSA Coalition and other MSA charities will be conducting many awareness initiatives, including a nationwide campaign to have all US states declare March Multiple System Atrophy Awareness Month. Additionally, the MSA Coalition will be ringing the closing bell of the New York Stock Exchange on March 5, an event which will receive excellent media coverage and will be shared via NYSE social media channels, including Facebook, Twitter, and Instagram.

Giving Tuesday – A Critical Day for Multiple System Atrophy Research

Giving Tuesday – A Critical Day for Multiple System Atrophy Research

If you want to have the biggest impact possible in the quest to find better diagnostic tools, treatments and hopefully a cure for multiple system atrophy #GivingTuesday (November 28, 2017) is the most important day of the year!

100% of every dollar raised, matched, and won on #GivingTuesday will go directly into the MSA Coalition Research Grant Program! If you help us fundraise the total could reach $200,000 or more!

Don’t stay on the sidelines during this critical day!

Read on to learn more….

History of Giving Tuesday

#GivingTuesday has been celebrated each year since 2012, on the first Tuesday after Thanksgiving (in the U.S.). It symbolizes the start of the Holiday giving season, which for most charities represents the time of the year when the biggest portion of their donations are received. The MSA Coalition has participated each year through its partnership with the CrowdRise Holiday Challenge. Last year the MSA Coalition raised more than 10% of its total revenue for the year on #GivingTuesday.

The CrowdRise Holiday Challenge, A Great Event For MSA

This year’s CrowdRise Holiday Challenge runs from November 21 – January 3, 2018. It is being

The Multiple System Atrophy Coalition's 2017 Holiday Hope for a Cure fundraiser on Crowdrise

Join The MSA COalition’s 2017 Holiday Hope For A Cure Team

sponsored by the Newman’s Own Foundation and is offering $500,000 in prize money to the top charities. The winner of the 6-week challenge will receive $150,000 and the top 10 causes will receive prize money. There are also bonus prizes throughout the Challenge with another $100,000 being distributed. Plus, there are separate prizes for #GivingTuesday totaling another $100,000. The top 3 causes will receive grand prizes.

Who wants to see a significant portion of these awards coming into the MSA Coalition for research, education and awareness initiatives?

If you are saying “Me! Me! Me!” read on…

Why the MSA Coalition Needs You on Giving Tuesday

We all know multiple system atrophy is a very rare and little known disease. Because of this it is very difficult to motivate outsiders to give to the MSA cause, let alone to fundraise. Grass roots, at least for now, is our single most effective way to raise money. That means, we need YOU to participate.

Last year (2016), the MSA Coalition had 100 families actively participate in the CrowdRise Holiday Challenge. On Giving Tuesday we raised just over $90,000, which was good enough for third place for the day. Unfortunately, two other charities did better and the Coalition did not win any of the prize money for the day. On the bright side, The MSA Coalition maintained its third place status throughout the CrowdRise Holiday Challenge and in the end secured the Third Place prize of $25,000 which was added on to the Challenge total of over $300,000 raised for MSA.

That’s right, with just 100 families participating the MSA Coalition raised over $300,000 dollars in 6 weeks and added another $25,000 in prize money to that total. Imagine what we could do if 200 families participated! What about 300, 400 or even 500! This is why we need you!

1: Sign Up to Help Us Raise and Win Money This Holiday Season

Joining our team is easy and will only take a few minutes

MSA Coalition 2017 Holiday Hope For A Cure Team Members

2017 Holiday Hope for a Cure Team Members

Join the MSA Coalition’s 2017 Holiday Hope for a Cure Team

2: Like and Follow The MSA Coalition’s Facebook Page for Volunteers

Don’t fundraise alone! Become a part of the MSA Coalition team of fundraising volunteers. We

Diane & Elaine Offer Hope & Advocacy for those with MSA

will post lots of great tips, updates and key dates to help make your fundraiser a success! Likewise, share your ideas, questions and success stories to help the whole team become better!

Like The MSA Coalition Volunteers Facebook Page

3: Sign Up for Special Giving Tuesday Newsletters

  • How to Set Up a CrowdRise Holiday Challenge Fundraiser
  • Tips on Fundraising Easily and Successfully
  • Success Stories From Other MSA fundraisers
  • Alerts About Key Fundraising Dates and Bonus Challenges
  • Fundraising Updates

If you are on the fence or nervous about fundraising, sign up for our newsletter and select the Holiday Challenge under fundraising as an interest. It is not a commitment to fundraise, it is only a commitment to learn more!

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Giving Tuesday – A Critical Day for Multiple System Atrophy Research

The Importance of Supporting Young MSA Investigators

A Report on the Movement Disorder Society Congress in Vancouver

June 4-8, 2017

By Roberto De Marzi

Since I started working as a research fellow in Innsbruck 2 years ago, I focused on new methods for the early diagnosis of Parkinson’s Disease (PD) and Multiple System Atrophy (MSA). In MSA an early diagnosis is of particular interest, since it would enable a more specialised patient’s care and a better selection of patients for clinical trials testing new therapies. Unfortunately the early diagnosis is still a big challenge for the clinicians, since the clinical overlap with PD doesn’t always allow a correct classification.

Roberto DeMarzi, a multiple system atrophy researcher

Roberto De Marzi
2017 MSA Coalition Travel Award Winner

In the project I presented at the Movement Disorders Society Congress in Vancouver, my colleagues and I tried to find early changes in the magnetic resonance imaging (MRI) of MSA patients. We tested six different approaches in selecting the areas of the brain to be analysed with MRI on 115 subjects. 61 PD patients, 24 MSA patients and 30 healthy controls were evaluated, and the regions of the brain to be analyzed had to be manually selected for each of the six approaches. That means that 690 brain scans were manually processed by our team! The results of the study were really interesting, showing that, using 3 of the methods that we tested, we were able to differentiate MSA patients from PD with a very high accuracy. This method could be used in the clinical routine to better individuate patients with MSA even in an early stage.

This study was really time-consuming and it was a great honor and a privilege to be awarded with the travel grant of the MSA Coalition in order to present my project with a poster at the Vancouver MDS Congress. The funding for clinical research in the academic field is often lacking, especially for rare diseases like MSA. Therefore it is a big help for us researchers to have charitable organizations like the MSA coalition providing for additional funding, even more to take part to a congress on the other side of the world.

The MDS congress is the most important congress for researchers in the field of movement disorders. It was only my second participation and on my arrival to the conference hall in Vancouver I was once again amazed by how big the congress is. The movement disorders field is not a big field of research and I work in the university hospital of a medium-sized city, therefore I was also amazed when I saw thousands of other movement disorders specialists taking part to the congress.

On the first day of the congress I attended a very exciting poster session regarding atypical Parkinsonism. It was interesting to see in which directions the researchers are moving. I received a lot of inputs on which topics could be part of my future projects. Especially interesting was a poster by Y. Compta and colleagues, from Barcelona, who detected a decrease in coenzyme Q10 levels in the cerebrospinal fluid of MSA patients. The role of coenzyme Q10 in MSA is still controversial, therefore this study could probably shed some light on the role of coenzyme Q10 in the pathogenesis of MSA or help in the early diagnose of the disease.

The day of my poster presentation was the most exciting. I presented my project in front of the guided poster tour and I received positive feed backs and comments from many experts of the field. There were also some advices on how to better conduct similar studies in the future. I will surely take these advices into account once I further develop my project.

Read Roberto De Marzi’s MDS Poster

I also had the chance to talk to many other young researchers from the entire world (UK, Italy, USA, Germany and many other countries) which are active in the MSA research and who also are investigating MRI in MSA patients. While exchanging ideas and experiences I talked to them about the MSA coalition and its initiatives. We also realized how important it would be to have more networks in the MSA research in order to increase the number of patients studied and to reproduce the findings of our research in different centers. I was really motivated by these talks and we exchanged contacts in order to try to start collaboration within our groups.

During the congress I had the privilege to hear some outstanding lectures by the world’s most renowned experts in the movement disorders field and to personally meet some of them. It was really interesting to finally connect a face to many specialists that I knew because of their scientific publications. Particularly stimulating was a lecture by Prof. Kordower about immunotherapies in PD and MSA. In Innsbruck we conducted a study with one of these therapies and it was an honor to see our study being cited in one of the plenary lectures. The last day I attended a special session about atypical forms of atypical parkinsonism, which saw Prof. Wenning as chairman and presenter. During this lecture many videos of the different clinical presentation of MSA were shown and I learn a great deal about the different phenotype of the disease. It was an exciting presentation and I’m sure it will improve my ability to recognize atypical cases of MSA in the clinical setting.

In summary, attending the MDS congress in Vancouver was a very enriching experience. In a few days I learned a lot of things about the latest discoveries in the field of movement disorders and I had the chance to know famous experts and to network with other young researchers. All these inputs gave me new motivations and ideas to further develop my projects in this field.

 

Giving Tuesday – A Critical Day for Multiple System Atrophy Research

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 https://www.multiplesystematrophy.org/msa-donation

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”).

https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse

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:

https://www.clinicaltrials.gov

https://foxtrialfinder.michaeljfox.org

https://www.clinicaltrialsregister.eu

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.

https://natural-history-synucleinopathies.com/glomsar

Giving Tuesday – A Critical Day for Multiple System Atrophy Research

Ambiguity in MSA Clinical Trials

Science is the discipline used by researchers to answer questions and/or test hypotheses. The results of a scientific investigation may be for a specific subset or can be generalized. If, for example, a small sample size yields a particular result it in no way means that same result will fit with a larger sample size. The result has greater meaning if there is a large sample size with similar results. Since it is so difficult to get large sample sizes when the subject of study is a rare disease like Multiple System Atrophy it is difficult to confirm findings.

Scientists can get around this problem by using alternative resources as subjects for testing. One alternative resource might be mice models while another might be petri dishes and a specific media. Another might be computer modeling. Yet even these substitutes don’t always yield useful results. Scientists are then forced to ask different questions or pursue a different hypothesis.

There is a current issue being discussed in an MSA support group that is a good example of the vagaries of science. It involves physical testing and a recent science “claim.” In a short synopsis of the situation, a patient apparently diagnosed with MSA was asked to report to the doctor to have a skin biopsy. She put out an email questioning the procedure.

A recently published article abstract described the results of an MSA clinical trial examining sudomotor nerve density (total length of nerve per volume of glandular tissue) from three different body areas. Results were consistently less dense in test patients (patients with MSA) than in the controls. The conclusion of the authors was “Our data support the hypothesis that postganglionic impairment occurs in MSA …”

This author wrote the patient and explained the testing her doctor was performing may be based upon the results of this study. The study reports results based upon observations of human tissue that lead to a conclusion. The patient’s doctor took her biopsies from similar body sites thus raising the possibility the test will yield similar results. But the results the patient received were labeled “normal” by the doctor. That seems counter to the results of the above study. Thus is there anything the patient can take away from this?

From a science perspective there isn’t much. There were 29 MSA patients in the sample size with results significantly different from the control group for each of the three areas – a P value of .0001. That one patient did not have similar results is not unusual. Why? The sample size is small. The MSA patient may be mis-diagnosed and these are the correct results. The accuracy of the measurement tools may be off. The patient may have the correct diagnosis but is earlier in the disease process than the 29 subjects. It is also possible her doctor followed a different hypothesis or asked a different question and “normal” has a different meaning to him.

Science is not clean. It is cleaner when the questions are focused, the technology matches the tests being done, the observations are real-world, the sample size is large enough and the tests can be replicated. You can see the researcher has control over three of the criteria but not over the sample size. Nature decides the sample size when studying rare diseases. Until patients can be brought together in large enough groups or the percentage of MSA patients to the overall population increases – God forbid – ambiguity will continue. That is not what patients and caregivers want to hear.

Author: Larry Kellerman, PhD; Caregiver Representative, The MSA Coalition Board of Directors