Jane LarkindaleName: Jane Larkindale

Where do you work? I work for the Critical Path Institute, a nonprofit organization that works to develop tools to accelerate drug development and works towards regulatory acceptance of those tools (i.e., getting FDA, EMA and other worldwide regulatory authorities to accept them). We are based in Tucson, Arizona.

How long have you been working on FA and who was the first fellow FA researcher you met? I have been working on FA for about 12 years now, including time at FARA and at MDA, and now at C-Path. The first FA researcher I met was actually even earlier when a woman started in my lab and gave a talk about her previous work as a post-doc in a lab at the Mayo Clinic working on FA. Given I was working in a lab that focused on plant biochemistry, this is quite a coincidence! Her talk was really interesting, but it became much more interesting a couple of months later when I started looking at a job at MDA, and I realized FA was one of the diseases that MDA covered.

What got you interested in FA research? When I started at MDA my first project (given to me on day 1) was to look into a grant application to support a drug for FA. So, in order to understand whether the project should be funded, I had to learn about the disease, it's biology, what else was in development, how that proposed therapy worked, and much more. As I was a very enthusiastic new employee, I dug very deeply into the literature and what was known and became interested in the disease. To add to that, one of the first people I met at MDA outside of my department has FA (she was one of the writers for Quest magazine). Not long after that, I met FARA and started collaborations with them, and there was no going back after that. We collaborated on several projects, some of which are still going in one form or another, and when I left MDA moving to work at FARA was the best possible option for me.

Jane Larkindale 3What question or challenge were you setting out to address when you started this work? As I was working at a nonprofit, our goal was to get therapies for a number of different diseases to patients as fast as possible. So, we attacked it on several fronts - we funded preclinical work into a possible therapy (the first project I looked at), worked with FARA on FACOMS, the natural history study that is ongoing, so that we could understand how the disease progresses and funded several early academic studies into FA biology. I have never directly done research in FA, but I have touched on research all the way through the spectrum from very early-stage "blue sky" research, through developing tools (biomarkers, outcome assessments, etc.) and supported drug development into the clinic. It is fascinating to see how all the different areas of research inter-relate and all contribute to moving the science closer to treatments and cures.

What research topics or questions are you currently focused on? Right now, I am working with a nonprofit that is focused on accelerating how we develop drugs broadly across diseases. Specifically, I run projects focused on using existing patient data to improve drug development for rare diseases. We work very closely with industry and the FDA to develop tools to make clinical trials as informative as possible and help us (disease communities, and industry, and FDA) to agree on whether a drug will work or not as quickly as possible. I also have a project with FARA on the development of a database that includes data from FACOMS and data from past clinical trials that have been completed to develop a larger set of data for analysis. We want to use mathematical modeling to really understand how the disease progresses in groups of patients, any differences that are seen in groups taking part in clinical trials, how drug effects that are meaningful to patients can be measured, and how to optimize clinical trial design to tell us whether a drug works as quickly as possible and using as few patients as possible. Due to the diligence of FARA and the FA community, despite FA being a rare disease, there is sufficient data to do this! This work will help us understand what changes are meaningful to patients, and help the regulators (FDA and EMA) determine what drugs may really help patients - and help us get treatments to patients faster.

What do you hope to achieve or what excites you in FA research? We need treatments! The work I do won't directly give us treatments or cures, but I hope that it will help us get to a point that we can test new potential treatments and cures quickly and effectively, and help us as a community agree on what is likely to work and what we should stop wasting time on it. That will both encourage more companies to look at FA as an indication where they want to develop drugs and help us get answers faster - so we can focus on therapies that really have a chance of success.

Jane Larkindale 2If you have met someone living with FA, please tell us about that interaction. Did it have an impact on your work? Yes! Everyone I have met with FA has an impact. I could list 100 names of patients and family members I have met at meetings, bike rides, FARA events, etc. and every one of them has an impact. When we ran our Patient-Focused Drug Development meeting in 2017 we went out and talked to many different patients to try and make sure that we were covering the experience of as big a range of patients as possible, and I learned a huge amount about how people think and feel about the disease and their symptoms, how they handle the disease, what concerns them most and what worries them. This is incredibly impactful to me, and I have been recommending patient groups and companies do this kind of work ever since. However, the individual person who probably had the most influence on me was Kathy Wechsler. Just after lunch on my first day at MDA, a dog walked into my office and started nosing around the garbage. Moments later, Kathy followed the dog in and apologized for having a less-than-brilliantly trained service dog, and we got to talking. She was one of the first people I had met with any neuromuscular disease, and she helped me to understand that the conditions that I had studied affected actual people, and were not just an abstract scientific idea. She made FA (and all the other conditions that I have worked on) real for me.

You serve voluntarily on FARA's Scientific Advisory Board. Please tell us what you see as FARA's key role in the research process. FARA is absolutely critical as the glue that holds research together. While funding research is obviously important in keeping it going, what is even more important is FARA's ability to bring people together and focus them on how to move the research forward as quickly as possible, how to collaborate (and who to collaborate with), and bringing expert groups together to solve ongoing problems. FARA has always prized collaboration, and this is a key aspect of its success. It is easy as a researcher to get focused on what you are doing, and lose time by not realizing that someone else may have a solution to your problem - or you may have a solution to theirs. By bringing together everyone at all stages of research, FARA has successfully overcome many barriers to research. By including patients and families in the process, FARA also reminds everyone of the urgency and importance of their work, and makes sure that everyone remains focused on thing that matter to the FA community.

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