Buoyed by the extraordinary progress being made by the ALS Therapy Development Institute, the Muscular Dystrophy Association today announced a new milestone-driven grant of $2.5 million, adding to the $18 million MDA already has invested with ALS TDI -- the world's only non-profit research center focused exclusively on developing treatments for amyotrophic lateral sclerosis (ALS).
The funding comes through MDA's fast-track ALS research fundraising initiative, Augie's Quest, which already has granted $21.7 million for promising ALS work since June 2006.
"MDA has seen an enormous return on its investment in ALS TDI during the past three years. Thanks in part to this strategic research partnership, scientists are accelerating knowledge about amyotrophic lateral sclerosis while zeroing in on a variety of promising therapeutic approaches. We're excited about the rapid progress ALS TDI has made on bringing several promising drug candidates to the most crucial stages of preclinical development," said R. Rodney Howell, M.D., Chairman of the MDA Board of Directors.
Amyotrophic Lateral Sclerosis (ALS, Lou Gehrig's disease) is a progressive, fatal neurodegenerative disease affecting approximately 30,000 people in the United States at any given time. The average survival following diagnosis is only about 36 months, and onset can occur in teenage years plus every decade of adulthood. ALS usually progresses rapidly, with patients losing their ability to move, speak, swallow, and eventually breathe on their own. Currently, there is neither a cure nor an effective treatment for ALS.
"The support and guidance that the MDA has provided ALS TDI researchers has led to some very exciting outcomes. With this additional investment, we hope to turn the tide on this disease and deliver a variety of well validated therapeutic options for clinical trial," said Steven Perrin, Ph.D., chief executive officer and chief scientific officer of ALS TDI.
When it received its first MDA Augie's Quest installment in late 2006, ALS TDI quickly identified and filled critical technology and talent gaps at its state-of-the-art research center, located in the Cambridge biotech hub of Kendall Square. ALS TDI now boasts an industrial scale drug development pipeline that integrates unbiased discovery technologies with rigorous drug development capabilities. The progress of the first phase of the collaboration exceeded all expectations, delivering the first validated therapeutic that ALS TDI has ever tested. This drug, known as ALS TDI 00846, is a blocking antibody to CD40L which ALS TDI is now in the process of partnering for clinical development.
The two organizations began this historic collaboration three years ago when fitness pioneer Augie Nieto, diagnosed with ALS in 2005, partnered with MDA to raise funds for ALS research and therapeutic development. The result, MDA's Augie's Quest, was created as a fast-track ALS research funding initiative aimed at filling the drug development gap for ALS. Over the past four years, Augie's Quest has raised $21.7 million for MDA-funded translational research seeking effective treatments for ALS. Notable scientific projects previously funded by Augie's Quest include: work at the University of California at Irvine ($500,000); at Targeted Genetics Corporation in Phoenix ($652,000); and by ALS TDI ($18 million investment 2007-2009).
"My entire life I spent teaching people the importance of physical fitness. But I didn't just preach it. I lived it and led by example. When I was told I had this disease, I knew I needed to do the same thing and make a difference. We have surpassed the original fundraising goal through Augie's Quest. But we aren't stopping there, and we will not stop until ALS is stopped," said Augie Nieto, chairman of ALS TDI, chief inspirational officer of Augie's Quest and national co-chairman, with his wife Lynne, of the MDA'S ALS Division.
The renewed contract announced today includes an initial milestone payment of $2.5 million with mechanisms to add additional milestones and associated funding at the discretion of the MDA steering committee. Progress towards meeting milestones will be assessed quarterly by a review board comprised by representatives from both organizations and non-affiliated, outside researchers recognized for their expertise in ALS research.
Unraveling the complexities of muscle repair in diabetes: A call for targeted research and therapies