In our Beyond the Minor series, we’re continuing to share reflections from students and alumni who carry the lessons of Notre Dame’s Minor in Science and Patient Advocacy into their professional and personal lives. In the newest feature, Calvin Hawe (ND '24) reflects on how early exposure to rare disease patient stories shaped his passion for bridging science, medicine, and patient advocacy. From his time in the classroom and at the very first ND Patient Advocacy Summit, to his work in biotechnology, Calvin’s experiences highlight how listening to patients and engaging with their stories continues to guide his advocacy and future career path as a physician-scientist.
"When I first heard about CLN2—a subtype of Batten disease—I was shocked by its severity. Learning how this condition robs children of basic functions and ultimately their lives was deeply unsettling, especially since I was a child myself at the time. I also remember learning that there was this one hope—an investigational enzyme-replacement therapy—for this ultra-rare brain disorder that was going to begin human trials soon, and how this medicine would be administered by “drilling a hole through the patient’s skull.” But what truly kept me awake at night were the patient stories, such as Noah and Laine VanHoutan. It is one thing to know a disease’s symptoms; it is quite another to see a happy and seemingly-healthy child gradually losing vision, memory, and the ability to communicate and walk, eventually deteriorating into a seizure-ridden, incapacitated state dependent on feeding tubes and ventilators. This was when I first realized how heart-wrenching rare diseases are.
Years later, that investigational therapy—called cerliponase alfa—completed clinical trials and received FDA approval. By then I was in high school and better understood the impacts of a condition like CLN2, both on patients and their families. Moreover, I learned how this medicine—sold as Brineura—was brought to patients through the determined collaboration of scientists, clinicians, physician-scientists, and dedicated patient advocates. The researchers who used biotechnology to create a recombinant enzyme therapy amazed me, and I was deeply moved by the unwavering commitment of CLN2 parents to push this medicine to the finish line, even when it was too late for their own children. Equally inspiring were the physician-scientists of Brineura’s story, such as Dr. Angela Schulz, who dedicated her career to Batten diseases: caring compassionately for patients, working tirelessly to define the natural history of CLN2 (which laid the groundwork for clinical trials to be run solely against natural history), and ultimately leading the clinical investigations of Brineura. Together, these extraordinary individuals transformed a CLN2 diagnosis from a hopeless death sentence into a treatable condition—one where children born today can realistically envision living beyond their teenage years and even attending college, a milestone on my mind at the time. Brineura’s scientists, Dr. Schulz, and the patient families whose determination never wavered became my heroes, sparking a passion to dedicate my life to bridging science, medicine, and patient advocacy in rare diseases.
So when I arrived at Notre Dame and learned about the Patient Advocacy Initiative’s genuine commitment to rare disease patients, I was eager to join their mission and the Minor in Science and Patient Advocacy (MSPA). I vividly remember attending my first Rare Patient Advocacy Summit back in 2021 and witnessing Prof. Calhoun’s compassionate, patient-centered approach toward the individuals who were present and living with DMD and other inherited muscle diseases. She set a precedent for how I interact with and learn from rare disease patients. My path through the MSPA then began with a deep dive into Krabbe disease—a rare disease with similarities to CLN2 and marked by equally heart-wrenching stories, such as that of Judson Levasheff. Over the following years I had the opportunity to meet leaders in rare disease healthcare and biotechnology and, most importantly, hear directly from patients about living with rare diseases and how to make a meaningful difference in their lives. These experiences proved instrumental to the success of the partnership projects I’ve had with patient advocacy organizations—such as the Friedreich's Ataxia Research Alliance (FARA)—and helped prepare me to compassionately engage at patient conferences, including an FA research reception where I had the privilege of meeting inspiring patients and advocates. The most personally-meaningful collaboration I’ve had in the MSPA was working with a remarkable rare disease patient—Megan Crowley—to produce an Instagram Reel sharing her diagnostic journey, addressing misconceptions about Pompe disease, and highlighting the importance of social media within the rare disease community. This 90-second clip, while concise and engaging, captures just a small glimpse of Megan’s incredible story and awesome personality.
Now as an MSPA alumnus, I am continually building upon my experiences in rare disease advocacy and patient interactions. Back when I was seeking post-Notre Dame research opportunities, I felt particularly drawn to lysosomal storage disorders—an interest shaped by my exposure to CLN2, Krabbe, Pompe, Niemann-Pick Type C, and my undergraduate work synthesizing isotopically-labeled versions of GlcNAc-Asn, the diagnostic substrate biomarker for aspartylglucosaminuria. Equipped with that perspective and a desire to continue supporting rare disease patients in the lab, I joined a patient-centric biotechnology company immediately after graduating that is dedicated to discovering innovative therapies for lysosomal storage disorders and a specific form of retinitis pigmentosa. Although my primary responsibilities at Octant Bio are in the chemistry lab, I actively engage in patient advocacy as well. I've had the opportunity to become involved in advocacy for a new disease area—inherited retinal disorders—and to hear directly from patients, including powerful anecdotes from individuals who describe feeling trapped in their homes after dark, experiencing their world shrinking day by day. Moreover, knowing how devastating lysosomal storage disorders are—from watching children with CLN2 and Krabbe lose their sight to hearing young adults with Fabry disease describe relentless neuropathic pain—makes this post-MSPA research and advocacy deeply meaningful and profoundly motivating.
From my past year working in the biotechnology industry, directly researching and developing novel treatments for rare diseases, I cannot overstate how important it has been to have heard—and to continue to hear—patient stories in guiding my approach to patient-centered drug discovery and development. And as I aspire to become a physician-scientist, I fully expect to reflect and draw upon my experiences in the MSPA for the rest of my life." - Calvin Hawe, Class of 2024
Beyond the Minor is a guest-authored reflection series from the Patient Advocacy Initiative that highlights the experiences, growth, and impact of current students and alumni. From classroom insights to community engagement, these reflections capture the many ways students carry their advocacy forward - in their own voices.