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White Paper Issue 13: NDR’s Roadmap to an ALS Cure

Updated: Mar 31, 2022

Finding a cure for ALS is a lofty goal shared by all those involved in the field. The differences between the organizations that support research and discovery are found in the roadmaps to that goal. NDR is historically similar to other not-for-profit ALS organizations, brought to the field by someone we know that is afflicted with disease. Our passion is personal.

Paraphrasing Isaac Asimov best explains our philosophy that science is arguably the greatest achievement of the human mind and the fact that progress has been made, mostly, by ordinarily clever people building step by step from the work of their predecessors makes the story remarkable. Anyone reading this, had they been in the right place at the right time, could have made the great discoveries we rely on today. And since science is progressive, you may yet be involved in the next step of the ALS story.

Reaching our goals supports our mission to benefit patients suffering from neurodegenerative diseases. Many decades of thinking and research have seemingly resulted in little in the way of treatments for ALS, but that isn’t true. Understanding the basic biology of ALS, diagnosis, genetics, animal modeling and identifying treatable targets have advanced and formed a foundation for additional discovery.

When the time is ripe, two or more individuals may make the next step independently of one another. It is the luck of the draw or nowadays, legal argument, whose name gets remembered as the discoverer of a new phenomenon. What is more important than the who is the how, the development of technology. With the best technology has to offer, connecting scientists is paramount. Finding researchers that are outside silos of information, fostered by paradigms imposed on funding institutions, is important to NDR. Innovative approaches to old information can fill in some missing pieces of ALS pathology.

The most pressing issue in ALS treatment is identifying the disease using biomarkers. Another large issue in ALS research is the discord between efficacy of treatments in in vitro ALS models and effectiveness in ALS patients. All will hail the person that solves this conundrum, and someone will. NDR funds new and enterprising out-of-the box thinkers that wouldn’t ordinarily receive traditional NIH funding. Our support helps build careers and product development. A little financial support can allow a new researcher to publish and that is instrumental in getting government funding. Research is expensive and traditional avenues are competitive. The same is true for product development. Companies want to see pre-clinical data for a product that may have utility in rare disease treatment. The pre-clinical stage is aptly called the Valley of Death for lead molecules and treatments.

Other important and difficult-to-find funding research areas are drug repurposing and drug synergy. Newer automated drug screening technologies for drugable targets opens the door of possibilities for finding therapies with a low bar for licensing an ALS treatment. Small safety trials in ALS patients become an opportunity for existing licensed drugs that can examine dose and effectiveness without using in vitro models. With a safe and possibly effective drug in hand, researchers can approach pharmaceutical companies that have deep pockets.

NDR impacts research in ALS by supporting new researchers, we find them through publications of novel data and encourage open sharing of data.

NDR supported research that led to successful grant applications ensuring funding in labs to continue the research and importantly, training new scientists in the field. We supported pre-clinical development that resulted in two safety trials for ALS patients. We hope to have a third this year. Safety trials get a treatment for patients and benefiting patients in the short term is especially important to us. There are other important considerations that keep us on our path to find a cure.

As is often said, it is all about the people. People that already contributed to the ALS field of knowledge have a leg up, those are scientists that have published in the last 4 years by their novel discoveries or building on the foundation that is already there. These people will train the next generation. NDR prioritizes funding to those that don’t have existing funding for a project, unless the project can benefit with a little more help. Again, the people with clarity of vision shown by specific aims, goals, and timelines can convey that novel idea or approach to us most quickly.

Science, unfortunately, is slow and the researcher with unified long term goals has an advantage. As said before, sharing ideas in an open forum can be synergistic, we fully support sharing resources and think this may ultimately be a key to success.

Our map to finding a cure for ALS is by funding and supporting research that investigates the basic biology of disease, stratifying patients by inventing accurate biomarkers of disease, and identifying how to treat ALS pathology that will slow the disease processes, until the cure is found.

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