When the ALS Therapy Development Institute (ALS TDI) launched its Precision Medicine Program (PMP) in the summer of 2014, we had a plan to enroll 25 people living with ALS. Thanks to unprecedented funds raised through the Ice Bucket Challenge and the commitment of so many ALS TDI donors over the past 3 years, that plan has expanded significantly. Today, 450 people are enrolled in the first, largest and most comprehensive precision medicine program for ALS in the world.

When a person signs up to the program they become a true partner in research, with access to their own live data through an online portal, making this a distinctively transparent approach to clinical research. This partnership between people living with ALS and researchers at ALS TDI has resulted in a database integrating genetics, voice recordings, lifestyle, demographics and accelerometer data. Using this information, we aim to identify subtypes of ALS and then screen drugs targeting these specific subtypes making clinical trials in ALS more effective and efficient.

We’re proud of the progress we’ve made to date with the Precision Medicine Program and excited to announce three new members of the Translational Research group, who will help us advance the Precision Medicine Program even further.

Erika Norabuena came on board as a Senior Associate Scientist with several years of experience in human stem cell biology and neural differentiation, most recently at the Harvard Department of Stem Cell and Regenerative Biology. She works with pluripotent cells and various cell types derived from them, using the cells for assay development and drug screening studies.

Erika decided to focus on ALS because of her prior research experience in motor neuron diseases and her admiration for Stephen Hawking. When explaining what she feels makes ALS TDI unique from her previous research experiences, she says, “Firstly, it has a Precision Medicine Program, allowing us to create an extensive and unique database helping identify the subtle differences found among ALS patients. This, in time, will make our drug screening process more accurate because we will be able to test compounds in participant cell lines, finding hits that will give more promising results in clinical trials. Working at ALS TDI gives me the opportunity to strengthen my commitment to finding possible treatments for ALS.”

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Aarthi Ravi joined ALS TDI as an Associate Scientist II following placements in Harvard Innovation Labs and HuaCells. She is working on cellular phenotype discovery and screening assay development. She brings knowledge in primary cell and stem cell culture, flow cytometry, and molecular biology techniques.

Aarthi is excited to work on the translational research team as she believes that “personalized medicine will be key to medical research in the future, not just for those with ALS, but across all diseases.” While here, Aarthi is looking forward to learning more about mutated cells: “I have worked with wild type cells in the past but never with mutant cells. They may behave in a different manner and we seek to identify and characterize such differences as potential basis for drug screening assays. I also hope to be part of the team that can have a real impact on people’s lives through precision medicine.”

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Haley Knight joined as an Associate Scientist I to support further development of the PMP patient-derived cell bank. Haley recently graduated from Wheaton College with a BA in Arts and Neuroscience/PreMed. After starting at ALS TDI, Haley shared what she’s most excited to work on here: “What I do may directly affect the lives of people living with ALS - working with their skin cells and IPSCs (Induced Pluripotent Stem Cells), seeing the cellular aspect and their data." Haley’s goals while here are to head her own project and learn more about “how ALS works.”

To learn more about Haley, Aarthi, Erika or any members of the ALS TDI team, click here.

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In addition to three researchers joining the PMP team, Dr. Steve Perrin, CEO and CSO of ALS TDI, will give a presentation at the world’s largest ALS/MND conference in December right here in Boston, MA where ALS TDI will be local co-hosts. He will speak about our progress with the Precision Medicine Program and the role that technologies, such as our accelerometers, will play in speeding up clinical development in ALS. Other members of the ALS TDI science team will present six posters on PMP data, drug screenings and ALS model development using cell based assays. We’re honored to co-host this International research symposium and to take a leadership role in the global conversation on ALS research. We will be offering a preview of the International ALS/MND Research Symposium via a webinar on November 15, for which you can register here.