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A new pitch to stop ALS
CAMBRIDGE, Mass.—A new drug discovery program targeting amyotrophic lateral sclerosis (ALS) will receive at least $36 million over three years to investigate potential ALS therapies for pharmaceutical companies to bring to market. Research will be conducted at the ALS Therapy Development Institute (ALS TDI) and funded by ALS TDI, together with the Augie's Quest fundraising initiative of the Muscular Dystrophy Association (MDA).
The project will be an unprecedented integrated attempt to find biological bases and, principals hope, therapies for ALS. "It's a very ambitious program," says Steve Perrin, chief scientific officer at ALS TDI, noting that the entire institute is working under one mission: finding ALS therapies. "The unique thing here is we're focusing on one indication… because of that, you can get a team working in a unified fashion and really get a lot of progress, which is very exciting."
Perrin, a veteran of Aventis and Biogen Idec, calls ALS TDI "a nonprofit biotech that has the funding and infrastructure to behave like a pharma" by exploring one indication from multiple approaches. ALS TDI's discovery efforts will incorporate genomic and proteomic techniques, such as assaying tissues from a mouse model to investigate changes in messenger RNA over time. Researchers will have access to human tissue through MDA's clinical network, enabling data comparison on pathologies and pathways in mouse and man. Although Perrin expects to outsource genomic and some computational biology work, ALS TDI aims to hire about 10 additional people in 2007.
ALS TDI's ongoing research has primarily mined literature, says Perrin, repeating studies in journals. Although most studies replicate poorly because of faulty design, ALS TDI has screened 200 small molecules and established a tissue bank that will give the new project a jump. Perrin expects target discovery to last 12-18 months and hopes year two will see development of reagents and a strategy for delivery to the central nervous system.
Looking at funding, Sharon Hesterlee, vice president of translational research at MDA, says, "We think that $6 million [annually] is really a minimum." She likens the money to venture capital and says high cash burn rates or successful fundraising could increase project financing. MDA decided to support the work because most ALS-related clinical trials have shown little promise, says Hesterlee. "Industry doesn't stay with the ALS focus for long enough beyond a Phase 1 or 2 clinical trial with a single product." She sees the project as "our chance to take a step back and use some of these new technologies, in proteomics and genomics, without preconceived notions and, in a completely unbiased fashion, see what's there and see if we can find something new."
ALS TDI's work is only one aspect of MDA's continuing commitment to investigating ALS and supporting research infrastructure: in 2006, MDA spent about $7 million funding academic ALS research plus $8 million on services and clinics. Hesterlee says roughly 30,000 people in the United States have ALS, with incidences around 1-2 per 100,000 annually. Despite those numbers, drug markets are not as small as people may think: Perrin compares ALS incidences to multiple sclerosis, noting that ALS patients generally live for only three to five years after diagnosis. "It's actually a misconception by big pharma that the ALS market is not a big market." Big pharma, Perrin believes, is savvy enough to recognize that a validated molecule from the ALS TDI project could be inlicensed and brought to market at minimal risk. Still, he says it is no coincidence that there are currently no good therapies for ALS: "Nobody has cracked the therapeutic strategy yet."