Our Research

OUR CURRENT PROJECT

DUE TO THE RARITY OF THE DISEASE, ONLY A MULTICENTER APPROACH IS FEASIBLE

David Liuzzo via Wikimedia Commons (CC 2.0)

10 different clinics participate in our project.

How should future therapies and clinical trials be designed?

Patients' attitudes

Our current study comprehensively surveys MSA- and Parkinson patients on how clinical trials and disease-modifying therapies for these diseases should be designed. In our questionnaire we are asking which side effects are particularly severe and which would be bearable.

Moreover, we are assessing the willingness of patients to take therapy-associated risks. We are specifically investigating whether the willingness to take risks correlates with the disease burden (assessed using the health-associated quality of life). Furthermore, our goal is to find out more about the general attitude of patients towards clinical trials and possible barriers that prevents them from participating.

How do MSA and M. Parkinson patients dream?

 

We are investigating potentially burdensome, but so far not assessed, symptoms linked to the contents of the patients' dreams. For that purpose, we use a 'dream questionnaire'. The majority of MSA patients suffer from a REM-Sleep-Behaviour-Disorder. Moreover, 50% of all Parkinson patients suffer from this disorder.

 

The disorder is characterised by a loss of muscle tension during the REM-sleep. This leads to the patients living out their often vivid and sometimes even violent dreams. We hypothesise that these brutal and possibly disturbing dreams increase the disease burden of MSA and M. Parkinson patients.

Elicitation of clinical data of MSA and Parkinson patients.

For the analysis we need a comprehensive picture of the health state of our participants. We are collecting some data retrospectively from the patients' medical records. Among other things, we are checking for additional diseases, such as terminal cancer, which could potentially increase the suffering and the patients' willingness to take risks. Moreover, it is invaluable for us to have information about the cognitive state of our patients and to know in which stage of the disease they are.

Patients in a later stage of the disease, which may already be tied to a wheelchair, suffer more than patients which aren't physically impaired and are still able to take care of themselves. Moreover, we are collecting information about symptoms specific to MSA and Parkinson to improve the characterisation of the disease burden, such as incontinence, impotence, orthostatic hypotension, REM sleep behavior disorder, slowing movement (bradykinesia), tremor and stiffness (rigor).

WHAT DO WE WANT TO ACCOMPLISH WITH OUR RESEARCH?

Collect the opinions of MSA and Parkinson patients about future clinical studies and therapies

We want to give patients the opportunity to articulate their views on how they think future therapies and clinical studies should be designed.  We are therefore assessing barriers and benefits for the participation in clinical studies. We are also surveying which side effects of drugs or surgery are tolerable. To this date, there is no scientific data on whether MSA-patients have a higher rate of acceptance for clinical studies, including procedures with average risk levels compared to Parkinson patients.

Develop synergistic approaches for future studies with MSA patients

With our study, we will create a new infrastructure connecting numerous clinics, from which future studies are able to benefit. Due to the rarity of MSA, one clinic alone wouldn't be able to reach adequate case numbers.  We will generate a cohort with approximately 120 patients, which would be one of the largest MSA-cohorts around the globe. We are convinced that research into possible therapies of MSA is key to fight M. Parkinson as well - both diseases share the alpha-synuclein pathology. Since MSA progresses much faster, it is easier to recognise clinical end points.

Aid the planning of future studies

We want to publish our work in peer-reviewed journals. We would also like to promote the results to a wider public audience to ensure that future clinical studies and therapies are adapted to the patients' needs. We are confident that our work offers valuable support in the design of future studies and improves their chances of receiving positive ethics approvals.

YAMSA e. V. PRESENTED THEIR PROJECT AT THE ALPHA-SYNUCLEIN-CONFERENCE IN ATHENS 2017

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YAMSA e.V. PRESENTED THEIR PROJECT AT THE KOLLOQUIUM NEUROLOGISCHE FORSCHUNG IN GÖTTINGEN 2019.

Facilitate the differential diagnosis between MSA and M. Parkinson

We want to survey the contents of the participants' dreams, since it is not known whether MSA patients dream differently than M. Parkinson patients. This could be important in face of the difficult differential diagnosis between MSA and M. Parkinson. In this event we are able to find disease-specific dream content for MSA and M. Parkinson, it may be possible to use this as another criterion to identify from which disease the patient is suffering. Our research therefore leads to an earlier and more  reliable diagnosis of MSA.

Create awareness for MSA

We would like to raise awareness among the scientific community by publishing our work in peer-reviewd journals and presenting it at conferences. Through our organisation, we want to raise public awareness of MSA and the people suffering from it.  It is important for us to convey information which is understandable to everyone and inform the public about current or planned studies.

OUR FUTURE PROJECTS

In accordance to our statues, we want to support and carry out additional multi-central MSA research projects - our current project sets the foundation and the starting point of this. With our current study we are already supporting  the clincis at the participating hospitals with the creation of a list of patients 

that show general interest in participating in future studies concerning MSA. This could lead to a possible follow-up project: A nationwide patient registry of German-speaking MSA  patients.

OUR PUBLICATIONS

We have started with the recruitment of MSA patients for our project in January 2019 in Marburg, Dresden and Beelitz. All other clinics will start by April 2019 as well. We are expecting first results in December 2019.

 

We presented part of our project at the  "6. International MSA Congress" in New York City in March 2018:

The abstract presented there was published in  Clinical Autonomic Research (2018) 28:142.

 

Tabea Barthel*, Alexander Maximilian Bernhardt*, Lioba Kahmann*, Emre Kocakavuk*, Eliana Nachman*, Marc Oeller*, Kevin Peikert*, Malte Roderigo*, Andreas Rossmann*, Jeremy Schmidt*, Isabel Weigelt*, Lea Olivia Wilhelm*, Armin Giese, Wolfgang Hermann Oertel (03/2018): Assessing the Willingness of Multiple System Atrophy Patients to Take Risks in Clinical Trials. 6th International MSA Congress, New York City, 1. – 2. März 2018.

 

* contributed equally

 

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