Dr. Barnett-Tapia discusses how funding from the American Brain Foundation opened up new opportunities for patient-centered research.

The American Brain Foundation is dedicated to finding new treatments, better diagnosis methods, and cures for all brain diseases. With the help of the American Academy of Neurology, our Next Generation Research Grants provide support for a wide range of innovative research projects from early-career researchers.

In 2012, Carolina Barnett-Tapia, MD, PhD, associate professor of neurology at the University of Toronto, received a grant funded by the American Brain Foundation. Her research project worked to identify what people with myasthenia gravis found most important about their symptoms and resulted in the development of a new symptom evaluation tool that incorporates a patient’s own perspective into clinical assessments. 

We spoke with Dr. Barnett-Tapia about how her early American Brain Foundation-funded project launched her career as a researcher and provided crucial information on patient-centered care. Her research has provided valuable insights on why it’s important to include the patient’s voice in research and treatment plans, as well as unlock possibilities and new discoveries on future treatments.

Dr. Barnett-Tapia’s responses below have been condensed and edited for clarity.

Why were you inspired to study the brain?

I love neurology. I always thought the nervous system was fascinating and incredible. Our brain—and the spinal cord and nerves—allows us to move, to function, to think. It’s truly the core of what makes us, us. I work with nerves and muscles that relay information to and from the brain. I see how important it is for patients who have diseases that affect parts of the nervous system to have new treatments and new therapies that can make their lives much better, as well as relieve suffering and improve functionality.

What specific issue is your research trying to address for patients?

My research is focused on understanding what matters to patients the most, identifying those values and considerations, and making sure that new treatments are actually improving the important things for patients. A lot of my research is also focused on understanding what the most impactful symptoms are for patients in their daily lives. Asking patients what they consider important allows us to incorporate those aspects into research, new treatments, and clinical practices to improve patients’ lives.

Could you elaborate on the role of the patient’s perspective in your research? What kinds of insights did you discover?

We learned that clinicians and patients have different views. What clinicians find important and what patients find important come from different perspectives. But we’ve also learned that there are things we can do [to bridge this divide]. Every time we interview patients and do patient-centered research, we focus on the patient’s perspective and experience. We also now routinely ask patients in our clinic and studies if they’re satisfied with their treatment and symptom management. 

Even if we’re experts on a disease, no one is as much of an expert as the patients who live with it. Through this research, we learn about what patients find relevant that we hadn’t thought about. I find that so enriching as a clinician and a scientist—to be able to build on that knowledge from patients and try to bring it to the table in clinical trials or clinical practice.

What did your American Brain Foundation-funded Next Generation Research Grant enable you to work on?

This award allowed me to devote time to interviewing patients in order to gain knowledge about the impact of neurologic diseases on their lives. So I sat with patients and talked to them for an hour or two about their experience having this disease. It allowed me to take time off clinical work to really gain knowledge about the impact of brain disease in patients’ lives and come up with tangible ways to put numbers to that experience.

It’s hard to think about putting a number to something so personal, because everyone is different and has different experiences. But what we’ve tried to do is develop a tool to help us standardize [the patient care process], and now we’ve been able to use these insights in clinical practice. So when we see patients in the clinic, we can ask them the questions and measure the symptoms that we know matter to them.

How did this early American Brain Foundation-supported research open doors for additional research funding or future research projects? 

I cannot put into words how much the American Brain Foundation award changed my life. I do not know how successful I would have been without this award. I did not have a lot of research experience when I got the award; I was just learning. I was also not trained in North America, so I didn’t have a lot of that background that allows you to get more funding. 

This opportunity really opened doors for me, because it’s a big thing to be supported by the American Brain Foundation. After I got the award, I could see that people were noticing that my work had been recognized by the Foundation. I have been very lucky to be funded since and have been successful in other endeavors.

Additionally, the fellowship opened all these doors for me not just in terms of funding but also through a whole network of mentorship. With the American Brain Foundation, you are part of this cohort of amazing, talented clinicians and scientists, and this community really helps each other. Their goal is to help you succeed as a researcher. Even today, I correspond with some of the mentors in the program. They still invite me to events and offer advice if I need, and I think that’s really priceless.

How would discovering treatments or a cure for one brain disease impact other diseases?

So even though as a clinician you may focus on a very specific number of diseases, from a scientific perspective, some of the methods and approaches we use can be applied to other diseases. My focus has been on understanding patients’ views and preferences, as well as developing measures and outcomes—things we can actually use in clinical trials to see if medications are working. That translates to any other disease. 

The methods and the way we come to these conclusions can certainly be used for different diseases. If we do well in one field, some of our colleagues from other fields may say, “Oh, that’s a really interesting approach. Can we use this approach for this different disease?” This really helps everyone because in science, as we move forward, it’s like stepping stones. One discovery helps everyone else get new ideas and bring things that have been working for one disease to help battle another one.

What do you hope your research will help accomplish for patients? 

My hope is that my research will help bring patients’ views and perspectives to the table so that the treatments we’re developing and using are those that really have value for patients and are really improving the things that are meaningful for patients. For instance, sometimes as clinicians, we are focused on something in our exam or testing, but often patients don’t care about those things. Patients want to be able to spend time with their families, they want to be able to work, they want to be able to do their hobbies—they want to be able to have a normal life.

The other thing is that by learning more about what patients value and what’s important to them, we can use that information for other parts of the research and treatment process. For instance, we can help make sure that the voice of the patient is included in conversations about what medications are funded and approved. So that would be my goal—keeping the patient perspective in mind across the development and approval of new therapeutics.

How far has research come since you began your career, and what are the most exciting discoveries and developments you’ve seen since you entered the field?

There are too many to count. We are treating things today we couldn’t treat before. In my specific field, we are treating diseases—like genetic disorders—that were untreatable even a few years ago. It’s really amazing what we have accomplished. The research across all fields of neurology is just amazing, and this is why we need more research—to keep going and really expand beyond the diseases that we are currently able to treat.

In the past seven years, we’ve had around 15 drugs or interventions approved to treat neuromuscular disorders. Between 1955 and 2010, we had less than 10. So in seven years, we’ve had multiple times the number of medications approved specifically for nerve and muscle disorders than we had in the past 60 years. We’re seeing three to four new drugs approved every year. That is truly amazing. 

One of the biggest examples is treatment for spinal muscular atrophy (SMA). This is a genetic disease that affects the nerves, and it can affect babies from birth and young children, most commonly, although sometimes it presents later. These were children that used to not be able to walk or sit, many of them with very early death—it’s a devastating genetic disease that had no treatment. Now we have treatments that, especially if they’re given early while someone is a baby, they can [restore movement] and achieve the standard milestones of a healthy child without SMA that was unthinkable years ago. That’s research at work.

Why is it so important to fund research?

Research is a massive undertaking, and it cannot happen without funding. To produce good research, we need funding, and funding through usual sources [like the NIH] is very limited. Unfortunately, we always need more funding for research—the only way research has advanced so much [in recent decades] is through philanthropy and donations.

Also, there are many rare neurologic diseases that affect a very small number of people but are quite severe. For those diseases, we probably know 10% of what we need to know to cure them, but we’re way above where we were in the past—even just 5 years ago. It’s been so accelerated, and as we continue with our research, we’re going to come closer to curing all brain diseases.

I got my award over 10 years ago. We have to think about the next generation of people who will take over and bring in new ideas—so funding is also about training new people, and about getting new people excited about the questions that we’re pursuing in research. That won’t happen without these types of programs that really help researchers who are just starting out.

Why are organizations like the American Brain Foundation so important? 

The American Brain Foundation is important because it is dedicated specifically to research that will help us develop treatments for people with brain and nervous system diseases. The other reason the Foundation is so important is that it’s committed to training new generations of clinicians and scientists, as well as building a network so that this work is sustainable in the future. The way I see it, the American Brain Foundation is a community, and the goal is to maintain and grow this community for the benefit of patients and their families. I think that’s pretty unique. 

The American Brain Foundation is invested in people, and at the end of the day, what research needs is people—it’s people who do the work and drive research, and it’s people we’re treating. This investment in people whose lives are devoted to brain and nervous system diseases is really crucial.

Research is what allows for the discovery of cures for all brain diseases. Investing in the people behind these studies and programs is what allows people living with brain disease to receive the treatment they need and deserve. Through research and understanding the patient’s perspective, we can fulfill the American Brain Foundation’s mission of discovering new treatments and cures for all brain diseases.

The American Brain Foundation is committed to supporting the next generation of brain disease researchers. By donating today you can help us achieve our vision of life without brain disease.