A new pitch to stop ALS

ALS Therapy Development Institute (ALS TDI) funds new $36 million, three-year study

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CAMBRIDGE, Mass.—Anew drug discovery program targeting amyotrophic lateral sclerosis (ALS) willreceive at least $36 million over three years to investigate potential ALStherapies for pharmaceutical companies to bring to market. Research will beconducted at the ALS Therapy Development Institute (ALS TDI) and funded by ALSTDI, together with the Augie's Quest fundraising initiative of the MuscularDystrophy Association (MDA).
The project will be an unprecedented integrated attempt tofind biological bases and, principals hope, therapies for ALS. "It's a veryambitious 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 ofprogress, 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 apharma" by exploring one indication from multiple approaches. ALS TDI'sdiscovery efforts will incorporate genomic and proteomic techniques, such asassaying tissues from a mouse model to investigate changes in messenger RNAover time. Researchers will have access to human tissue through MDA's clinicalnetwork, enabling data comparison on pathologies and pathways in mouse and man.Although Perrin expects to outsource genomic and some computational biologywork, 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 replicatepoorly because of faulty design, ALS TDI has screened 200 small molecules andestablished a tissue bank that will give the new project a jump. Perrin expectstarget discovery to last 12-18 months and hopes year two will see developmentof reagents and a strategy for delivery to the central nervous system.
Looking at funding, Sharon Hesterlee, vice president oftranslational research at MDA, says, "We think that $6 million [annually] isreally a minimum." She likens the money to venture capital and says high cashburn rates or successful fundraising could increase project financing. MDAdecided to support the work because most ALS-related clinical trials have shownlittle promise, says Hesterlee. "Industry doesn't stay with the ALS focus forlong enough beyond a Phase 1 or 2 clinical trial with a single product." Shesees the project as "our chance to take a step back and use some of these newtechnologies, in proteomics and genomics, without preconceived notions and, ina completely unbiased fashion, see what's there and see if we can find somethingnew."
ALS TDI's work is only one aspect of MDA's continuingcommitment to investigating ALS and supporting research infrastructure: in2006, MDA spent about $7 million funding academic ALS research plus $8 millionon services and clinics. Hesterlee says roughly 30,000 people in the United States have ALS, with incidences around 1-2per 100,000 annually. Despite those numbers, drug markets are not as smallas people may think: Perrin compares ALS incidences to multiple sclerosis,noting that ALS patients generally live for only three to five years afterdiagnosis. "It's actually a misconception by big pharma that the ALS market isnot a big market." Big pharma, Perrin believes, is savvy enough to recognizethat a validated molecule from the ALS TDI project could be inlicensed andbrought to market at minimal risk. Still, he says it is no coincidence thatthere are currently no good therapies for ALS: "Nobody has cracked thetherapeutic strategy yet."

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