Through our fundraising efforts, we will ensure that 100% of the money we raise will go directly to research and treatment to find a cure. We’ve done a survey of existing ALS research and we’ve identified and are working with the two organizations leading the charge on the fight to cure ALS. We have outlined the efforts so you can see how our fundraising efforts will help lead to a cure.
ALS Therapy Development Institute
ALS TDI, has one urgently important goal: to end ALS. They envision a future where patients no longer die from ALS and where today’s patients are alive, well, and enjoying the company of their children and grandchildren.
What makes ALS TDI so different from other biotech companies and research labs?
ALS TDI unique and comprehensive approach leverages more than a decade of treatment-focused ALS research and applies it on an industrial scale to get therapeutics to today’s ALS patients as quickly, efficiently, and cost-effectively as possible. Our research team evaluates the most promising therapeutic hypotheses through the lenses of the richest ALS research databases in the world. Treatments of all kinds are considered and tested rigorously and rapidly. Dr. Steve Perrin, CEO frequently speaks about their research plan to patients, families, industry executives and academics worldwide.
Houston Methodist Neurological Institute – Dr. Stanley Appel MD
Houston Methodist has established translational research as a top priority to meet this challenge and to bring new therapies, medical devices and other high impact medical applications to patients faster and more economically – within years rather than decades.
To date, Dr. Appel’s research team has successfully confirmed the proinflammatory response of peripheral blood monocytes in ALS patients, as well as confirmed that patients with ALS have impaired ability to suppress the proinflammatory activities of T effector cells. These important findings will inform the direction of studies toward new, more effective therapies for ALS aimed at addressing these factors. Funds raised can expedite the translation of these findings into human clinical trials.
While medical discoveries have led to a much better understanding of human health and disease, very few of these discoveries have made their way into improved diagnostics or clinical treatments. It is estimated that only one in 10,000 new therapies actually make it to the market for human use.
Researchers often refer to this gap as the ‘valley of death,’ a void in which promising research projects languish and disappear, preventing new laboratory discoveries from being translated into a therapy or device that can improve human health. The valley of death is exacerbated by increased regulatory oversight and decreased funding – from both the federal government and from pharmaceutical companies – for early stage medical discoveries.
The challenge, then, is to find a way to leap this void and move medical discoveries from the laboratory to patients’ bedsides – to ‘translate’ the discovery to the world of patient care. Houston Methodist has made a huge investment in facilities that meet preclinical trial requirements for Good Laboratory Practice (GLP) studies to FDA-certified Good Manufacturing Practices (GMP) which allows us to manufacture our own compounds for clinical use. Houston Methodist has all this capability in one building – few facilities have this capability and even fewer have it in one location.