Short Summary
Our new company OrganoTherapeutics uses human patient specific stem cell technology to generate 3D representations of the human brain, so called mini-brains. Particularly, we do that with stem cell from Patients suffering from Parkinson`s disease. We do that for two main purposes: 1. Find new medications for this devastating disease. 2. Demonstrate that such medications can be found without the use of animal models. Here you can see a bit more about our efforts to replace animal testing by alternative methods: http://organo-therapeutics.com/animal-alternati... This campaign is to collect funding to test two new small molecule compounds (drugs) of which we assume that they could protect the nerve cells in Parkinson`s disease patients to die, so called neuroprotective substances.
Our background
The two co-founders, Prof. Dr. Jens C. Schwamborn and Dr. Javier Jarazo have quite some track-record in the fields neuroscience, Parkinson`s disease, alternative methods and stem cell technologies. The company OrganoTherapeutics is a direct spin-off from our academic research lab at the Luxembourg Centre for Systems Biomedicine (http://schwambornlab.uni.lu). Additionally, we have an advisory team with an experienced medical doctor treating Parkinson`s disease and an experienced serial entrepreneur.
More about us at the bottom of this page: http://organo-therapeutics.com/about/
The campaign
We are working on two molecules, where we have evidences that they could protect the nerve cells that die in the brain of Parkinson’s disease patients. Normally, these compounds would be tested for this function in animal models (mice, rat or monkeys). However, in this new project we want to demonstrate that it is possible to further develop this compound by using advanced human stem cell technologies. For this approach, we take stem cells from Parkinson’s disease patients and differentiate them into 3D structures that resemble the human brain, so called minibrains. Particularly, we focus on the part of the brain that is mainly affected in the diseases, the midbrain.
We have pioneered this technology: https://www.cell.com/stem-cell-reports/fulltext...
The method itself is patented (https://patents.google.com/patent/WO2017060884A...) and our company has licensed this patent.
Importantly, already earlier this year we were the first world wide to shown that there are disease relevant alterations (e.g. reduced amount of nerve cells) in these mini-brains from Parkinson’s disease patients: https://www.nature.com/articles/s41531-019-0078-4
This is how a human mini-brain in the dish looks like:
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What we now want to do is take these patient specific mini-brains and treat them with the novel compounds. We want to find out whether this treatment can improve the situation in the mini-brains and hence whether these compounds can become new drugs for Parkinson’s disease. This project means a lot of work in the lab, we have to grow the stem cells and differentiate them into mini-brains, this differentiation can take 60 or even 90 days. At the same time we have to treat them with the compound. Afterwards the treated mini-brains will be analysed with high-end microscopy. Finally, the microscopy images will be analysed with computational methods, including artificial intelligence (AI) based tools.
The whole approach is described in an overview here: http://organo-therapeutics.com/our-approach-com...
What we need
We are here seeking a contribution of 200.000€. This money shall be used for paying the salary of scientists working on the project as well as for buying the necessary material (cell culture media, growth factors, staining kits etc.). Additionally, we would like to use part of the money for paying for the usage of instruments (e.g. microscopes), high-end computers and state of the art labs. Finally, some money shall be used for outreach and information work, e.g. for the website and for informing the public about our work. Your contribution will help us to further develop, in an animal-testing free manner, new compounds that might be used to treat Parkinson`s disease. You will not only make a contribution for this specific compounds and disease, but also for establishing this animal-testing free approach as a new standard for medication development. We would like to offer all contributors a naming on our website. The top 5 contributors shall be mentioned in the acknowledgements of our next scientific publication. Obviously, we will ask every contributor whether he or she wants to be named.
If you cannot help by contributing money
Please share the information that this campaign exists. Share it on Twitter, Instagram, Facebook or any social media that you might use. Make some noise about it :-)