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2021 ALS TDI Summit Presents Potential Treatments and Innovative New Technology

Posted: November 17, 2021 in Other News

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WATERTOWN, MA – Every year, the ALS Therapy Development Institute (ALS TDI) hosts the ALS TDI Summit as a way to update its community on the state of ALS research and drug discovery at ALS TDI. This year, for the safety of their community, ALS TDI streamed the Summit virtually instead of hosting a large in-person event. Broadcasting from their new laboratory in Watertown, MA, they were joined by a small in-person audience as well as hundreds of online attendees.

The Summit program included updates about drug discovery research programs in various stages of development at ALS TDI, as well as an extensive update on Project Euphonia, an initiative from Google to develop communication tools for people with conditions that affect their speech like ALS that was launched in collaboration with ALS TDI. ALS TDI also paid tribute to outstanding members of the ALS community with the annual Leadership Awards.

A Reintroduction to ALS TDI: The Drug Discovery Engine for ALS

For the day’s first presentation, CEO and Chief Scientific Officer, Dr. Fernando Vieira, M.D., presented an overview of ALS TDI’s research and its role in the overall landscape of ALS drug development. Dr. Vieira explained why we talk about ALS TDI as the Drug Discovery Engine for ALS – a description that encapsulates our role of working to advance many ALS drugs into human trials, until everyone with ALS has effective treatments. He further emphasized why this approach – doing the preclinical drug discovery that is needed to keep feeding the clinical pipeline with many treatments – is essential to addressing the extremely complex and diverse nature of ALS.

Dr. Vieira also explained the structure of our in-house preclinical research program, and described when and how we work with partners to advance discoveries into the clinic. This provided context for the presentations to follow, which would cover promising new discoveries making their way through ALS TDI’s research pipeline, as well as our collaboration with Google which helped to catalyze the development of communication tools for people living with ALS.

New Drug Hits Work Their Way Through the ALS TDI Pipeline Toward Clinical Testing

The next two presentations, from ALS TDI scientists focused on the discovery of a new class of potential therapies in development at ALS TDI, Redox Metabolism Modulators. Dr. Kyle Denton, ALS TDI’s director of Cell Biology and Dr. Theo Hatzipetros, the Director of Pharmacology, each provided a summary of how their respective teams have worked to move this discovery through our pipeline and closer to the clinic.

Dr. Denton’s presentation detailed how ALS TDI’s cell biology team uses patient-derived induced pluripotent stem cells (iPSCs) to inform the discovery of a new potential treatments. These iPSCs are often cells donated by people living with ALS who are enrolled in our Precision Medicine Program (PMP). The cells are coaxed to develop into motor neurons – cells that are affected by ALS. Dr. Denton described the process of working to develop and improve these models to reflect the biology of common ALS-related genetic mutations such as SOD1 and C9orf72. Using these cellular models of ALS allows Dr. Denton and his team to rapidly screen many potential compounds for potential effectiveness and also for overt toxicity.

Dr. Denton went on to describe a class of test molecules that showed promise in this model, molecules that ALS TDI is referring to as “redox metabolism modulators.” After seeing promise in the cell-based model and performing a number of experiments to identify the most promising compounds, or “hits”, have been passed on to Dr. Hatzipetros’ pharmacology team for testing in an animal model of ALS.

Dr. Hatzipetros’ presentation followed Dr. Denton’s, detailing ALS TDI’s In Vivo, or animal-based, drug testing program. He began by describing our primary animal model, the SOD1 G93A mouse model of ALS, and why this animal has been so essential to testing and validating ALS drugs over the years. He then addressed the process of further validating the discovery of the cell biology team in the pharmacology team’s mouse model of ALS. He described how these in vivo, or in animal, experiments led to validation of multiple hits in the complex SOD1 G93A Mouse model. These molecules, including one called TDI-1831, are currently being optimized in more animal experiments to identify the best lead candidate for clinical testing for ALS.

Updates on Type-I PRMTs as an ALS Drug Target

Anna Gill and Alan Premasiri, both Associate Scientists, followed Dr. Denton and Dr. Hatzipetros with a presentation about another promising discovery at ALS TDI. They discussed their work on the discovery of a new drug target for C9orf72-related ALS, Type I PRMTs, and the current effort to develop treatments that address this target.

They described how the discovery of this target emerged from ALS TDI’s fundamental research efforts, in which our scientists work to better understand the complex ways ALS affects the body. They also delved into our current understanding of the biological reasons that inhibiting these Type-I PRMT proteins may be protective for motor neurons affected by ALS. Finally, they covered the state of our efforts to find a lead drug candidate for this target, which include testing potential compounds identified in an in-silico, or computer-based screen.

Google Collaborates with ALS TDI to Improve Communication for People with ALS

The final science presentation of the day highlighted an ongoing collaboration between people with ALS and Google artificial intelligence researchers, known as Project Euphonia. The mission of Project Euphonia is to leverage research initiatives at Google to help people who have difficulty speaking because of neurologic conditions, like ALS, communicate and gain independence. The early stages of the program took advantage of voice recordings from ALS TDI’s PMP, which Google researchers used to teach the artificial intelligence algorithms that power devices such as Google assistant to better recognize the speech patterns of people with dysarthria.

Google team members Pan-Pan Jiang, Julie Cattiau, and Lissie Lillianfield presented updates on this program’s progress since the last time they addressed the ALS TDI Summit in 2019. They detailed how PMP participants have continued to be essential to the program, participating in the ongoing effort to collect voice recordings and also volunteering to help test adaptive apps developed using Euphonia’s speech recognition technology.

Google recently announced an expanded testing project for one of these apps, Project Relate, which they discussed in their presentation. Project Relate is an app for android phones that transcribes speech by people with dysarthria in order to fulfill a number of different communications needs. The app can transcribe speech and copy it into other apps on a device, read the transcription out loud in order to help the user communicate with others more clearly, or even recognize commands to operate other smart devices such as turning on lights or playing a song.

2021 Leadership Award Winners

To conclude the ALS TDI Summit, ALS TDI’s Senior Director of Development, Carol Hamilton, honored the individuals that were chosen by the community to be our 2021 Leadership Award winners. We were pleased to present these awards the individuals below.

Fran Delaney Challenge & Respect Award: Mac and Bailey Brown

Mary Lou Krauseneck Courage & Love Award: Patrick Liam Quinn, Katie Bauer

Stephen Milne Adventurous Spirit Award: Teresa Thurtle

Stephen Heywood Patients Today Award Winners: Rahul Desikan M.D., Ph.D., David & Scott Lloyd

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