LuMind’s Laser Focus on Research
Hillerstrom knows that given the range of symptoms of those with Down syndrome (Ds), a one-size-fits-all approach does not work for early intervention, inclusion, or research.
“In theory, current cutting-edge research can help overcome independence obstacles, either symptomatically, or by going after the root cause. I believe that it is important to strategically design future research based on what we have learned so far,” said Hillerstrom.
Working with team members, the board, funded researchers, and other scientists, LuMind RDS has defined four scientific research categories, which will guide future grant decisions.
The categories are:
1. Preventing Alzheimer’s
2. Improving cognition
3. Developing gene therapies
4. Advancing understanding
In terms of preventing Alzheimer’s, Hillerstrom believes that LuMind RDS can lay the groundwork for successful clinical trials in Alzheimer’s for individuals with Ds. This would involve: developing assessment scales, accounting for the variation in symptoms; supporting the establishment of a clinical trial network of clinicians; and establishing a registry for rapid enrollment in trials.
Without these necessary resources, Pharma will not be able to measure how well any therapies work for people with Down syndrome, which will delay launching late stage clinical trials of promising Alzheimer’s therapies in in Ds population.
For improving cognition, LuMind RDS will be evaluating the best way to support research on several promising targets that have the potential to improve independence. Other studies will be focusing on specific genetic targets (e.g. those found in green tea), cerebellum targeting molecules, and anti-interferon approaches. Gaining a better understanding of unique sleep patterns in people with Down syndrome will also be investigated.
Gene therapy is also an exciting avenue for possible treatment. The second X chromosome in females is naturally silenced throughout life. The gene that causes this silencing could be applied to silence the extra chromosome 21.
There is also promising research into genes that could possibly be modulated with gene therapy or with other therapeutic modalities. These genes found on chromosome 21 include Dyrk1A (also found in green tea), and USP16. Each of those genes could possibly be modulated with gene therapy or with other therapeutic modalities.
As for advancing understanding, LuMind RDS will support using stem cells and other research methods to help narrow down those genes or gene regions are most important in Ds.
LuMind RDS has also supported resources for the Ds research community including a database and repository. The large database plans to include the genomes, cognitive abilities, and other characteristics in over a thousand individuals with Ds. The repository will contain biological material for research to study underlying features of Ds, and potential new therapies. LuMind RDS plans to explore and grow these efforts in collaboration with other clinics, researchers, and foundations across the globe.
LuMind RDS wants to explore and increase efforts to collaborate with other clinics, researchers, and foundations across the globe.
Hillerstrom said, “There is tremendous potential to accelerate research by utilizing important resources, and cross-pollinating ideas among researchers. Pushing the frontiers of understanding Down syndrome, research can help bring forward better clarity on other conditions, including cancer and Alzheimer’s disease.”
He added, “My own disability has taught me that everything is possible if you put your mind to it… I needed to apply the best technology to achieve my own independence goals.”
He feels exactly the same way about Ds. “I’m applying that same spirit to improving the outcomes for people with Down syndrome enabling them to independently achieve their goals.”
“I truly believe that we are on the cusp of research breakthroughs that will result in improved quality of life and independence for our loved ones with Ds. That’s why I’m here at LuMind RDS. I hope that you’ll join me by supporting our foundation,” said Hillerstrom.
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