The ALS Therapy Development Institute (ALS TDI), an MDA-supported, nonprofit biotechnology organization dedicated to developing effective treatments for amyotrophic lateral sclerosis (ALS), will collaborate with two major biotechnology companies to investigate a potential treatment that modulates the immune system.
The experimental treatment is an antibody (immune system protein) fragment dubbed CDP7657. It will be tested in mice with a mutation in the SOD1 gene, one of the causes of familial ALS in humans and of an ALS-like disease in mice.
The two multinational biotechnology companies are Biogen Idec, headquartered in Weston, Mass., and UCB, based in Brussels, Belgium. Cambridge, Mass.-based ALS TDI announced the research agreement Dec. 19, 2011.
Sanjay Bidichandani, MDA's vice president of research, expressed great enthusiasm about the new collaboration.
"This is exactly what MDA's translational research program was designed to do — to turn promising laboratory results into drug development projects that attract support from biotechnology and pharmaceutical companies," he said.
The mouse version of CDP7657 that will be used in these experiments targets a protein called CD40L, which is on the surface of immune cells known as T cells. The compound interferes with the ability of the CD40L protein to engage with other proteins and launch a full-blown immune response.
A human version of CDP7657 is being tested in a phase 1 clinical trial for lupus, a disease in which the immune system is misdirected and attacks the body's own tissues. Mounting evidence suggests that a renegade immune system plays a role in ALS as well.
An earlier blocker of CD40L was known as ALSTDI-00846. In March 2010, an MDA-supported study conducted by ALS TDI scientists showed that blocking the CD40L pathway with this compound delayed disease onset and extended survival in SOD1 mice.
The 2010 study identified CD40L as "a relevant target in ALS," said Rob Goldstein, vice president of communications and public relations at ALS TDI.
A summary of the scientific paper, published online March 28, 2010, in Nature Genetics, can be read on the journal's website.
Goldstein said ALS TDI will complete some additional laboratory experiments, and then Biogen Idec and UCB will have the option to license global rights to develop and commercialize compounds targeting the CD40L pathway in ALS.
MDA, through its Augie's Quest initiative, has awarded more than $23 million to ALS TDI, including support of much of the research upon which development of CDP7657 is based.
The MDA-ALS TDI partnership, which began in 2007, has resulted in the achievement of several key milestones in ALS research, such as analysis of hundreds of blood samples from ALS patients and ALS mice to identify targets related to disease onset and progression, and screening of more than a dozen compounds against those targets.
"This agreement validates the historic investment and incredible trust of MDA in our research program," said Steve Perrin, CEO and chief scientific officer at ALS TDI.
"This project took a great deal of effort to get to this stage, and without the support of MDA's Augie's Quest, it likely would have taken much longer. This is a great accomplishment of our collaboration, and we look forward to continuing to push exciting potential treatments for ALS forward with the help of MDA."
A webinar discussion of the ALS TDI-Biogen Idec-UCB research agreement will take place Jan. 5, 2012, at 1 p.m. ET. Anyone can participate, but registration is required.