On October 14, 2022, ALS Therapy Development Institute (ALS TDI) scientists and guest presenters from across the ALS space gathered at the ALS TDI Summit in Boston, to provide the community with updates on research from the past year. They were joined by an audience from across the ALS community, including people with ALS, families, advocates, researchers, pharmaceutical industry professionals, and many more who streamed the event online.

Research Updates from ALS TDI

The conference began with a welcoming address from Dr. Fernando Vieira, ALS TDI’s CEO and Chief Scientific Officer. Dr. Vieira offered an overview of the coming topics for the day and an assessment of the current state of ALS research at ALS TDI and across the industry.

Dr. Vieira emphasized how far we have come in ALS research – with more papers published about ALS in the past 9 years than in all of previous history and the recent approval of a new treatment for the disease. However, he also highlighted how little we still understand about ALS – and the need for more research and more effective treatments. To this end, he reiterated ALS TDI’s commitment to continuing our efforts until there are treatments for every person living with ALS.

Dr. Viera’s presentation was followed by several presentations from ALS TDI’s researchers, demonstrating the institute’s latest and leadership in the ALS space.

Dr. Theo Hatzipetros, ALS TDI’s Director of Pharmacology, went first with a summary of ALS TDI’s animal model program. He covered our industry-leading expertise in the use of the SOD1 G93A mouse model for testing potential ALS treatments, as well as our current efforts to develop new models of the disease. Potential models he discussed included Profilin1 and TDP-43 mice, and a C9orf72 Zebrafish model.

Continuing on the theme of exploring models used for drug testing at ALS TDI, Anna Gill, Associate Scientist III and Program Manager, gave an overview of ALS TDI’s use of cellular models of ALS. She focused in particular on how induced Pluripotent Stem Cells (iPSCs) were used in the discovery of a new drug target for C9orf72 ALS, Type-I PRMTs.

Dr. Kyle Denton, ALS TDI’s director of Cell Biology, followed with more about our cellular models. His presentation explained the process of using iPSC models of ALS to test thousands of compounds to find those that could show promise as potential ALS treatments.

Finally, Alan Premasiri, Manager of Clinical Operations and Associate Scientist II provided an explanation of ALS TDI’s Precision Medicine Program (PMP). He provided an overview of how this partnership with people with ALS provides data that helps us learn more about ALS and supports our drug discovery efforts. He also covered how ALS TDI is working to share this data with people living with ALS in the program, as well as other researchers in the ALS space.

Guest Presentations from Other ALS Leaders

Following the presentations from the ALS TDI science team, we were joined by several other leaders in the ALS space.

Dr. Merit Cudcowicz, Chief of Neurology at Mass General Hospital and Director of the Sean M. Healey & AMG Center for ALS, gave an overview of the wider state of ALS research across the industry. She addressed how a better understanding of disease biology has led to more therapies being in development for ALS than ever before. She also reviewed some of the current approaches being pursued in preclinical research and clinical trials to prevent onset, slow progression, and repair function in ALS.

Dr. Lyle Ostrow, an ALS Clinician and Scientist at Temple University, delivered a presentation surveying the ways people with ALS, scientists, advocates, clinicians, and pharmaceutical industry professionals are working together to create a better future for ALS therapy development. He spoke to the collaborative efforts of many organizations, groups, and individuals to accelerate ALS research and drug development, as well as improve access to promising therapies for people with ALS.

The final two science presentations of the day both focused on ALS therapies. The first, from Josh Cohen and Justin Klee, co-founders and co-CEOs of Amylyx Pharmaceuticals, covered their treatment Relyvrio. They described the long process of developing the drug, from first deciding to pursue the combination of two previously tested ALS therapies to, nearly a decade later, securing approval from the FDA.

The last presentation by Dr. Steven Perrin, Chief Scientific Officer of Eledon Pharmaceuticals, provided an in-depth overview of the history and current state of tegoprubart (AT-1501). As the former CEO of ALS TDI, Dr. Perrin was able to describe the story of the potential treatment from its early development at ALS TDI, through its recent successful Phase 2a study conducted by its current sponsor, Eledon.

Honoring the ALS Community

Following the day’s scientific presentations, ALS TDI’s Vice President of Development Carol Hamilton took the podium to announce this year’s Leadership Awards. These annual awards honor members of the ALS community who have gone above and beyond to support research and advocate for the needs of people with the disease. This year, we were lucky to be joined by all of the award winners, all of whom were, for the first time, women. Each delivered a touching acceptance speech that ended the summit on an inspiring note.

This year’s Leadership Award winners included:

  • Stephen Heywood Patients Today Award—Rebecca Mourey, in recognition of advocacy on behalf of her fellow people ALS, including contributing to the passage of the Act for ALS and securing equitable patient access for Relyvrio.
  • Stephen Milne Adventurous Spirit Award—Her ALS Story, the first time any of these awards were presented to a group, for their efforts to raise awareness and change the stereotypes of who is affected by ALS.
  • Mary Lou Krauseneck Courage & Love Award—Sandra Marlowe, to honor her unwavering courage and optimism in the face of ALS, communicated to the community through her inspiring and informative social media presence.
  • Fran Delaney Challenge & Respect Award—Lori Larson Heller, in recognition of her efforts to support and raise awareness of the critical research to find effective treatments for ALS at ALS TDI.

Following the awards presentation, guests who attended the Summit in-person were invited to join ALS TDI for “Cocktails and Conversations”, where they had the opportunity to view research posters from ALS TDI scientists and talk to them in more detail about their work.

ALS TDI hopes that the ALS Summit provided valuable information and helped to inform the community about our current work.

For those who want to learn more about ALS TDI’s research, you can register to join our Community Conversations on October 25th at www.als.net/als-tdi-community-conversations/.

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