MADISON, Wis., Nov. 17, 2014 /PRNewswire-USNewswire/ -- ALS Worldwide, a nonprofit organization dedicated to advancing promising research and providing support to ALS patients and their loved ones, launched an Indiegogo crowdfunding campaign to raise the funds for preclinical testing of a new compound for the treatment of ALS (Amyotrophic Lateral Sclerosis, or Lou Gehrig's disease).
MicroNeurotrophins, a new treatment developed by Dr. Achilleas Gravanis, Professor of Pharmacology at University of Crete's School of Medicine, mimics the beneficial properties of neurotrophins, naturally-occurring proteins that support healthy motor neuron function. By contrast, ALS causes motor neuron death preceded by a profound loss of neurotrophins as the disease progresses.
ALS Worldwide has organized The ALS MicroNeurotrophin Research Consortium, which includes research teams from Harvard University/Massachusetts General Hospital; Virginia Commonwealth University; Penn State Hershey; The University of Sheffield's Institute for Translational Neuroscience (SITraN) UK and the University of Crete, to conduct the preclinical studies needed to begin testing the compound in humans in a Phase 1 Clinical Trial in the next 12-18 months. ALS Worldwide is kicking off the "Crowdfunding Campaign for a New ALS Medication" with a goal to raise $1.4 million over the next two months to support the consortium's research.
"The Ice Bucket Challenge showed us that people want to contribute directly to research," said Stephen Byer, ALS Worldwide's Co-Executive Director. "They want to see their dollars actually making a difference in treatments available to patients. There's only one FDA-approved option right now, and it only extends life by a couple of months. The crowdfunding campaign is a way for people to contribute directly to bring this specific treatment—vetted by experts and supported by a consortium of research teams—into a clinical trial. This is a concrete way to make a difference."
It will cost roughly $4.5 million to bring MicroNeurotrophins to a Phase 1 Clinical Trial. ALS Worldwide has already raised $600,000 and is leading the fundraising effort to raise the additional $3.9 million dollars needed for the FDA-required preclinical studies. Through the crowdfunding campaign, the organization is attempting to raise a little more than one-third of what's needed. ALS Worldwide is also pursuing traditional philanthropy and institutional grants to reach their overall funding goal. 100% of all donations will go directly to the efforts of the ALS MicroNeurotrophin Research Consortium.
"After rigorous testing in my laboratory, I discovered that MicroNeurotrophins have great potential for treating ALS because they target the multiple pathways of motor neuron degeneration that are characteristic of the disease. However, without scientific collaborators with expertise in ALS, I knew that my discovery would never advance to a clinical trial. That's why I am grateful to ALS Worldwide for assembling the consortium and leading the fundraising effort to underwrite our collective research," said Dr. Achilleas Gravanis.
Many promising drugs never make it to clinical trial due to lack of funding for this type of translational research. Additionally, researchers often work in isolation or in competition with one another, wasting time, resources and even discoveries without producing results. In contrast, ALS Worldwide made the connections among researchers that brought the Consortium together.
"As an ALS patient diagnosed in December 2010, I think this is one of the best examples of true collaboration that I have seen in the ALS community over the last four years. We have several of the world's top ALS research organizations working together. The best part is that the common objective is not a research paper—the consortium is focused on quickly bringing an innovative ALS treatment to a phase 1 clinical trial," said Tom Murphy, ALS patient and noted ALS activist from Gainesville, Virginia.
SOURCE ALS Worldwide