Celebrating the Past, Shaping the Future: Our Three-Year Nonprofit Anniversary
Today is a special moment – it is exactly three years since our non-profit was founded! It’s time to celebrate but also to reflect on what we have achieved in the past three years. It was a tough start with many uncertainties but high ambitions… Developing a therapy for a genetic neurological disease so rare that only little knowledge was available and not even a name existed. Our initial strategy was to focus on three pillars – raising awareness for the disease, establishing a drug development plan in collaboration with dedicated research teams from the universities of Montréal, Strasbourg and Basel, and fundraising to financially support our mission of finding a therapy for children affected by MCOPS12.
With the great help of a small, but super motivated team, we shared as much information as possible – by creating a homepage and Wikipedia articles about MCOPS12, creating accounts on LinkedIn and Facebook, handing out flyers, having our story posted in newspapers, and connecting with rare disease patient organizations. Even more important, we could build a network of MCOP12 patient families and see our community grow: since last autumn a 2nd non-profit organization was founded in Australia named “A Cure for Sienna”, and a 3rd NPO will soon be created in the US😊.
We are proud to share that our achievements are starting to pay off – we have now outlined a path to treatment focusing on two cornerstones:
The first cornerstone is to alleviate the MCOPS12 related movement disorders and cognitive impairments with a so-called drug repurposing approach. Six drugs are being tested in a MCOPS12 mouse model at the IGBMC Strasbourg. Those drugs are either commercially available or in clinical trials for other indications – with the huge advantage that potential adverse effects are already known. In case of efficacy, we can then start a clinical trial for our loved ones – our MCOPS12 kids. Initial data shows very promising signs, and the final results can be published at the end of this year!
The second cornerstone focuses on the development of an Antisense Oligonucleotide therapy to tackle the genetic root cause of MCOPS12 – which is being developed by Everlum Bio and aims to stop the pathogenic impact of the gene mutation and improve the MCOPS12 related movement disorders.
In parallel, we have started a Natural History Study in close collaboration with the CHU Sainte-Justine research center in Montréal in spring 2023. The study will define and track the most relevant MCOPS12 disease symptoms over a long period of time with the goal to measure disease progression or improvement. In close alignment and combining efforts with the NPO “A Cure for Sienna” and the CHU Sainte-Justine institute we were able to co-finance this comprehensive and costly study.
Our third pillar is to collect money to financially support the research and drug development efforts of the academic teams and Everlum Bio. We were able to collect 140.000 € until now – from which the big majority was donated to research and drug development!
Let’s turn our vision into reality – Finding a Therapy for all MCOPS12 Patients!