ALS Organization Does CRO Work On The Side To Support Research
Executive Summary
To raise funds for its own ALS research, ALS Therapy Development Institute offers services in gene expression, disease modeling and drug screening for pharmas that think their candidate might work against the orphan neurodegenerative disease. It’s a win-win for ASLTDI, which also sees the service as a way of creating ALS champions in big pharma.
The ALS Therapy Development Institute is a novel, hands-on disease advocacy group that bills itself as a non-profit biotech focused solely on finding a cure for amyotrophic lateral sclerosis, also known as motor neuron or Lou Gehrig’s disease.
ALSTDI has engaged in discovery research from its start in 1999 and now has a facility in Cambridge, Mass., with 26,000 square feet of wet lab space and a vivarium with 4,500 animals. The organization has a staff of 52, 30 of them scientists, and will operate in 2013 with a budget of around $10.6 million.
With those funds, it will support a Phase IIa clinical trial testing Novartis AG’s multiple sclerosis drug Gilenya (fingolimod) in ALS. It will also pay for a high-content screen by the Gladstone Institute with a human model of ALS Gladstone created using induced pluripotent stem cells, and screen around 25 preclinical compounds in-house (Also see "Scientific Advances Increase Potential For Drugs To Treat ALS" - Pink Sheet, 25 Feb, 2013.).
“Our sweet spot really is up until IND-enabling studies, maybe Phase I,” Rob Goldstein, VP public affairs, explained. To go any further, “we really have to partner with somebody else. We kind of know our limits of what we do well,” he said.
One thing ALSTDI does well is play to its strengths, using the resources at hand to operate a contract research organization for pharma that offers services in gene expression, disease modeling and drug screening.
Someone in a big pharma will read a paper on ALS that identifies, for example, mitochondrial dysfunction as a target in ALS and realizes they have a couple of mitochondrial dysfunction drugs they should have screened, Goldstein said. They quickly realize they have no expertise in ALS, look around to find a CRO and find ALSTDI.
“We’ve raised between $1 million and $2 million a year on average over the last few years by working with pharma,” he said. “We probably run three to four a year, and they account for a substantial part of our budget.”
Goldstein calls the arrangement a win-win. “Our only goal is to see drugs get to the patients, so if we can help a pharma fill its need to determine whether it has an asset that helps patients, we’re doing our job.”
Ultimately, “the relationship creates expertise in their organization, and, hopefully, creates champions to keep the interest going.”