Myosotis Therapeutics

Quick Facts

Goal $4,500,000
Who Prof. Dr. Frank Heppner
What A drug currently used to treat Psoriasis can slow the progression of Alzheimer's by controlling inflammation in the brain.
Where Zurich, Switzerland and Berlin, Germany


A team of researchers at Myosotis Therapeutics AG in Zurich, Switzerland, and Berlin, Germany is studying Ustekinumab (trade name STELARA®), which is approved for the treatment of psoriasis.  Like psoriasis, Alzheimer’s Disease is believed to be driven by inflammation.  This drug inhibits important components of the inflammation process, helping to slow  Alzheimer’s progression or delay its onset. If successful, this trial could provide the basis for the rapid development of a new class of medications that not only treat symptoms but target the very cause of the disease itself.

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Researchers at Myosotis have shown that certain proteins secreted in the brain in the course of neurodegeneration cause inflammation (“neuro-inflammation”) which many scientists believe to be the cause of Alzheimer’s. The same proteins are also central to the skin disease, psoriasis, which is caused by inflammation facilitated by these same molecules. Animal studies indicate that several drugs that interfere with these proteins in the skin of psoriasis patients may be similarly effective in the brain of patients with Alzheimer’s.

This study is designed to demonstrate this effect and show that a decrease in these target proteins helps slow the onset and progression of Alzheimer’s Disease.

This is a Phase Ib Trial and will study 36 patients diagnosed with mild to moderate Alzheimer’s Disease (AD). Each patient will undergo a screening and treatment phase of approximately 6 months. The level of inflammation will be assessed throughout the trial through imaging and quantification of inflammatory markers. In total it is expected to last 2.5 years.

Many inflammatory diseases share underlying causes, and a variety of studies have shown that inhibiting specific molecules (such as those implicated in psoriasis,) may have a powerful effect in delaying or preventing the progression of Alzheimer’s Disease. Fortunately, several compounds are approved or in development which tackle these molecules and prevent them from triggering inflammation. By testing the most advanced of these (Ustekinumab) we hope to demonstrate the power of this approach in Alzheimer’s, opening the way for the development of a whole new class of medications and treatments. The data so far has been overwhelmingly powerful, but human trials are required to confirm initial findings.

Myosotis Therapeutics AG, founded in March 2014, is a privately-owned company set up by the University of Zurich and Charité - Universitätsmedizin Berlin. Myosotis Therapeutics is funded by private donors and its founding Universities. It was established by four founders, whose families have been impacted by Alzheimer’s Disease, to accelerate development of disease-modifying anti-inflammatory therapeutics.

The Study Director is Prof. Dr. Frank Heppner, Chairman Department of Neuropathology, Charité – Universitätsmedizin Berlin

Since 2007 Dr. Heppner has been a full professor of Neuropathology and Chairman of the Department of Neuropathology at the Charité – Universitätsmedizin Berlin, Germany, heading 50 clinical and research co-workers. Dr. Heppner continues to actively conduct clinical-diagnostic work in all areas of neuropathology (neurooncology, neuroinflammation, neurodegeneration, neuromuscular diseases, developmental disorders of the brain, CSF cytology). His research group aims to understand the impact of the immune system on the pathogenesis of neurological diseases such as neurodegenerative diseases.

What is the connection between plaque psoriasis and active psoriatic arthritis and Alzheimer’s Disease?

Many inflammatory conditions share common molecular mechanisms and there is evidence of an association between psoriasis and Alzheimer’s Disease.

If Ustekinumab is safe, why do we need a clinical trial? Why can’t my doctor just prescribe it right now?

Ustekinumab is FDA approved for the treatment of psoriasis. Repurposing drugs (using a known drug in the setting of a new disease) significantly accelerates development timelines but does not eliminate the need for clinical trials in the new disease area. Though the compound is safe in target populations, clinical trials are now required to determine the correct dosage, frequency, and most effective way to administer the drug, and are needed to show a positive effect in the new indication.

What are the side effects of Ustekinumab?

Ustekinumab has been shown to be safe and effective in the treatment of psoriasis. While one would expect that this may also apply for Alzheimer’s disease patients taking Ustekinumab, our study will also give insights into this question.

Why are you conducting this study and not the manufacturers of Ustekinumab?

The manufacturers of Ustekinumab are Janssen (Johnson and Johnson). Janssen has been approached to be involved in this study, but declined to participate at this time. Drugs are expensive to develop and it is not uncommon for companies to be nervous about new trials with approved drugs. There are many patients with Alzheimer’s and we desperately need a cure and therefore very much hope that following this study, armed with data specific to the impact of Ustekinumab on the Alzheimer’s Disease, Janssen will become involved in subsequent clinical development.