There is no cure for Megalencephalic Leukoencephalopathy with subcortical cysts (MLC). This is a degenerative rare disease, which, unlike the majority of other leukodystrophies, may have the potential to reverse some of its symptoms and improve the quality of life of our kids. But time is not on our side. Current treatment strategies are supportive and, at best, aimed at improving the quality of life and managing common symptoms. There is an urgency to accelerate the research, move to clinical trials, and develop a cure for this rare disorder.
What needs to be done?
There are three processes that must occur before a treatment option can be licensed for use in our kids:
- Understand the disease
- Develop and test therapies
- Create a Registry and Natural History Study
Fortunately, we are already well on our way to accomplishing stage one and have begun to embark on the second stage. There are now several researchers around the world in the early stages of preclinical trials pursuing promising treatment options. This is extremely exciting and brings with it, for the first time, a real sense of hope.
What is a Registry?
A registry is a secure international database that contains all the relevant demographic, genetic and disease specific information of those affected by this disease. Our MLC Registry will be free to use and accessible to MLC researchers, physicians and pharmaceutical companies.
Why is it important?
The registry provides researchers, doctors and pharmaceutical companies with the comprehensive information to better understand and more easily study, treat and reach affected individuals worldwide. Without this registry, MLC researchers cannot move to clinical trials which help find a treatment for our kids.
YOUR DONATION SUPPORTS OUR RESEARCH
How is it done?
The international MLC Registry will be created by a PhD student under the guidance of Dr. Van der Knaap and in coordination with an international Medical Consortium. The four-year PhD program will include the creation of the MLC registry, the natural history study on how the disease progresses, and a survey of the disease’s impact on the affected individuals, their families and the society.
When will this project begin?
In early 2024.
What can we expect from this project?
The MLC Registry will help accelerate research as it will provide researchers with important information on the disease and affected patients. It will allow them to contact the families when they move to clinical trials. Without a registry we cannot proceed to clinical trials. Neither will a treatment be approved, no matter how spectacular the lab results are.
If you would like to participate in the MLC Registry, please contact Christina Marouda at christina.marouda@AllianceMLC.org.
Who will pay for it?
This is a €400,000 project. In collaboration with Dr. Van der Knaap who has recently secured a critical grant, along with initial contributions from families and their network, we have already secured €222,500! It is the responsibility of all of us, the affected families, our friends and colleagues to raise the remaining funds.
How should we, the families, fund this project?
There is finally hope for MLC families. Collectively, we can fund this together and give our kids, and future generations, a chance to live full, healthy lives. Every little bit helps so please consider making a donation. Ask relatives, friends and colleagues to pitch in, organize a walk or a Facebook fundraiser. Please give generously. Our kids are depending upon us.
We can help you plan and execute any fundraising ideas you have. Contact us and we will get started!
WE NEED YOUR HELP
Participate in the Patients Registry.
Make a donation to help us fund this project.
If we do not do it, no one else will!
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