MDA and ALS TDI: Partnership Extended Through 2013

MDA and the nonprofit biotech ALS Therapy Development Institute (ALS TDI) have extended their strategic research partnership through 2013. With the extension comes a $3.2 million MDA grant to help support the nonprofit biotech's continued progress toward developing treatments for amyotrophic lateral sclerosis (ALS).

The extension of the partnership reflects the continued success of the relationship between the two organizations, as MDA support helps ALS TDI move experimental compounds forward through the drug development process.

"MDA is happy with the progress that's been made so far as we work together with ALS TDI to find therapies for ALS as quickly as possible," said MDA Vice President of Research Jane Larkindale. "ALS TDI is in the process of bringing its first lead compounds into the clinic, and meanwhile is also covering all the bases with a rich and varied pipeline of therapeutic strategies under investigation.

“We're pleased to support their efforts as we take our partnership to the next level."

Research in the works

ALS TDI, located in Cambridge, Mass., screens at least 25 potential ALS therapeutics every year.

“The reality of ALS demands that we have a sense of urgency as scientists committed to the discovery and development of effective treatments for patients today,” said Steve Perrin, ALS TDI CEO and chief scientific officer.

“We feel this sense of urgency every day at the ALS Therapy Development Institute. MDA has been, and continues to be a key partner in providing us the resources needed to move more ideas and projects forward faster than before. MDA support has enabled many discoveries and advancements which hold great potential for people diagnosed with ALS.”

Candidate therapies in the ALS TDI pipeline address a number of different pathways or events thought to play a part in ALS, including:

  • problems in energy production, or metabolic dysfunction;
  • the unfolded protein response, a cellular defense mechanism;
  • oxidative stress, a cell-damaging process that occurs during energy production;
  • modulation of the immune system, or immunomodulation;
  • the process of generating myelin, an insulating sheath that surrounds nerve fibers and speeds the transmission of nerve signals;
  • the cellular breakdown and disposal system called autophagy;
  • proteins called trophins that help motor neurons survive;
  • the neuromuscular junction (the place where communication occurs between muscle and nerve); and
  • defects in RNA metabolism.

In addition, ALS TDI is focused heavily on creating badly needed new models of neurodegeneration based on recently discovered genetic mutations. Because of its industrial approach to drug development, the Institute also is able to apply much-needed resources toward the identification of biomarkers of drug response or even biomarkers of disease progression. Discovery of such biomarkers may drastically speed up clinical trials of potential treatments in ALS. 

ALS TDI also has plans to launch its highly anticipated phase 2 clinical trial of Gilenya, a drug currently approved by the U.S. Food and Drug Administration to treat multiple sclerosis. The trial in people with ALS will assess safety and tolerability of the drug, which works by modulating the immune system.

Historic drug search

MDA and ALS TDI forged a historic partnership in January 2007 when they launched a three-year, $36 million collaboration — the largest to date in ALS — focused on identifying biological components and pathways in ALS and finding drugs that target them. (MDA's initial $18 million investment was matched by ALS TDI.)

In 2010, MDA renewed its partnership with ALS TDI with a grant totaling $2.5 million. The Association awarded a supplemental grant of $855,000 in December of that same year, based on the biotech's extraordinary progress.

The partnership continued in 2011 with grants totaling $3.2 million, which helped ALS TDI scientists complete preclinical testing of several different experimental therapies, including the mouse version of a drug approved for use in humans to treat rheumatoid arthritis, in the SOD1 research mouse model of ALS. MDA support also helped expand ALS TDI's research program to include a TDP43 mouse model of the disease.

In 2012, MDA awarded grants totaling $4.2 million to ALS TDI. The nonprofit biotech announced in February of that year that it planned to initiate a phase 2 clinical trial in ALS of a drug currently approved for the treatment of multiple sclerosis. The U.S. Food and Drug Administration has since given ALS TDI the go-ahead to conduct its trial of the drug (Gilenya).

MDA support of ALS TDI has been awarded through MDA's ALS fundraising initiative Augie's Quest, an aggressive, cure-driven effort focused on finding treatments and cures for ALS. The $3.2 million grant for 2013 brings the total amount MDA’s Augie's Quest has awarded ALS TDI to more than $31 million since 2007.

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