ALS Therapy Development Institute: Looking Ahead to 2011

New space for research, new mouse models of ALS and new technology to screen therapeutic agents all will play a role in 2011 at the ALS Therapy Development Institute (ALS TDI), said Steve Perrin, ALS TDI chief scientific officer and CEO, in the biotech's monthly webinar in January.

Accelerate ALS research

Perrin listed a number of specific milestones the Institute hopes to reach in 2011.

One of the most exciting goals, reflective of ALS TDI's progress and subsequent increasing workload, is to significantly expand its core research facility. More space means increased capability to accelerate research toward the development of effective treatments for ALS. The expanded space will enable the Institute to hire two or three additional scientists.

Also high on the list is the Institute's plan to get its TDP43 ALS research mouse colony up and running. This includes completion by TDI researchers of a full characterization of the new mouse that will help scientists design the best possible trials for its use in drug screening. The mouse, which has a mutation in the TDP43 gene (associated with some cases of human ALS) mimics aspects of neurodegeneration associated not only with ALS, but also with other neurological conditions such as Alzheimer's and Parkinson's diseases. (See New ALS Mouse.)

ALS TDI recently acquired an Affymetrix GeneTitan, an automated device that can monitor gene activity (also known as gene "expression") and search for DNA mutations via a process called "genome-wide SNP genotyping." This crucial tool will be used to generate a "pharmacogenomic" profile of multiple therapeutic agents being tested for efficacy in the SOD1 research mouse.

The addition of the GeneTitan is part of ALS TDI's implementation of a multi-therapeutic screening program that will allow researchers to study the pharmacodynamics (the way a drug behaves in the body) of candidate therapeutic agents, and global changes in biological pathways relevant to ALS.

In addition, ALS TDI plans to screen between 25 and 30 new potential therapeutic agents for ALS in the preclinical model. Part of this process includes determining the correct dose of a drug needed to obtain a desired effect without causing unwanted or harmful side effects.

2010 highlights at ALS TDI

ALS TDI highlights over the pastyear cover a great deal of ground, Perrin noted, including the acquisition of sophisticated new equipment; new and renewed partnerships; publication in a prestigious journal; participation and presentations at major ALS-related meetings; and the Institute's annual ALS TDI Leadership Summit:

  • The GeneTitan, developed over the last decade, allows researchers to look at changes (mutations) that occur in a complete set of DNA, or "genome," in mice, rats and humans. The Titan can run more than 400 DNA samples a week and will help speed ALS TDI's research into the ALS disease process and preclinical therapeutic development.
  • ALS TDI scientists participated in and presented at a number of major ALS-related meetings, including the Society for Neuroscience, Nov. 13-17, 2010, in San Diego, Calif.; and the International Alliance of ALS/MND Associations, held in conjunction with the International Symposium on ALS/MND, Dec. 7-13, 2010, in Orlando, Fla.
  • For the first time, ALS TDI invited outside experts to present at its annual Leadership Summit. The two-day event highlighted both successes and challenges in the field of ALS research and touched on a number of themes dominating the research landscape today, including ALS genetics, clinical trials, the role of the immune system in disease onset and progression, biomarkers and the use of stem cells in ALS therapeutics. (To read more: ALS TDI Conference Summarizes ALS Research Progress.)  

Next up

ALS TDI's next monthly webinar is scheduled for Feb. 17, 2011, at 12 p.m. EST.

The Jan. 19 webcast has been archived on the Institute's website. (Note: You must register to view the webcast.)

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