Guiding the Next Generation of ALS Researchers

Answer ALS holds the largest ALS patient data resource in the world, and it is using that resource to help young scientists get their start. Students come for the science, and sometimes for personal reasons. Answer ALS gives them mentorship, scientific guidance, and the data they need to take on their first research on the disease.

Kelsey Valentine, a data scientist at Answer ALS, handles much of that work. She fields questions and access requests, and more of those requests now come from university students, high schoolers, and at least one middle schooler. Many are teaching themselves with free online tools for coding and research. Valentine says that self-teaching is part of why more young people are reaching out, and that their starting points and ideas are often further along than expected.

Getting clinical data still requires two things: a secure place to store and analyze it, and an institution or legal representative who can vouch for the project and confirm the data will be handled responsibly. High school students rarely have either. A teacher can sometimes help, but most students have no place to work with the data and no one who can serve as a principal investigator or business official.

Valentine knows that position. She took the conventional route, spending years in a lab before she could access this kind of information. She studied bioengineering at UC Irvine and added a computer science focus late in the degree, which led her into bioinformatics and genetics. She then worked for more than a year as a research assistant and lab technician in the Fraenkel Lab at MIT, which produces epigenetic data for the Answer ALS portal. She had planned to apply to PhD programs, then realized she was more interested in the clinical side: working with the people the research is meant to help, and seeing how findings become treatment. That led her to medical school. She completed a master’s at Boston University and started medical school at Virginia Tech this past July. She had a university and a lab behind her. Until recently, a student without that backing could not get the data or the institutional sponsor an ALS research project requires.

Answer ALS now removes that barrier for high school students and others working independently. For approved projects, Answer ALS itself serves as the sponsoring institution, so students do not need to find a principal investigator or institutional sponsor. They work in a secure online research workspace provided by the Alzheimer’s Disease Data Initiative. That lets them analyze de-identified clinical data without downloading it or handling sensitive participant information directly.

“A lot of times they didn’t have a person who could really serve as the PI or business official, but they had really great ideas, they were really impressive, and so we wanted to make it possible for them to pursue these projects,” Valentine said.

The dataset is large enough to overwhelm a student seeing it for the first time. Valentine starts with a call to explain what the data is and to learn what the student wants to study. She then helps them choose only the files that fit the project, so they are not buried in data they will never use. After that she holds regular check-ins and works through whatever they are stuck on.

Projects vary. Many students want clinical data on disease progression, which Answer ALS has. Some work with omics data. One student, partnered with a lab, wanted to grow iPSC lines. More often the questions are basic: what is this data, what do I need from it, and what is the difference between genomics, epigenomics, and transcriptomics.

To meet those questions, Valentine is recording short YouTube videos on the foundational concepts, planning workshop-style sessions on the technical side of analysis, and building a mentorship program that connects students with researchers in their area. A website is also in progress so students can find the program and see how to start a project.

Individual labs cannot always provide this support. Sponsoring a student and taking on a new project takes capacity that many labs do not have. Instead of asking laboratories to formally take on more trainees, Answer ALS uses its network of ALS researchers to match students with scientists whose expertise fits the project. Those researchers mentor students through the scientific process and help them shape data-driven questions. Answer ALS adds educational videos, technical workshops, and online resources that introduce ALS research and data analysis.

The team works with researchers in academia, industry, and nonprofit organizations, and is still building the mentorship program. Several mentors are already in place, and Answer ALS is looking for more. The aim is to offer support that no single laboratory could provide on its own.

“Having someone who can give you information and guidance is a real game changer when you’re just starting research, and you just don’t know what you don’t know,” Valentine said. “The students who come to our site are so bright. I feel like we really helped to foster their projects and give them the space to explore, but their research is really impressive, and I don’t always have all the answers for them, which is exactly why we are working so hard to find them dedicated mentors.”

Students who want to learn more about Answer ALS’s mentorship program or start a project can reach out to Kelsey Valentine at Answer ALS at kelsey@onpointsci.com