“What keeps me going is the memory of my dad and the thousands of people who are actively dealing with their worlds being rocked by neurodegenerative diseases.” — Amy Richards, Principal Research Associate, Neuroscience at Sanofi
For more than a decade, Amy Richards has worked to help discover new treatments and therapies for neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA). Today, as a principal research associate at Sanofi, Amy is part of a team of scientists studying a rare form of Parkinson’s disease known as GBA-related Parkinson’s disease.
The Driving Force
Amy’s lifelong interest in science was sparked by her father, who throughout his career worked as a clinical lab scientist, commander in the U.S. Navy Reserves and high school science teacher.
“I remember when I was in 5th grade, my dad helped me swab the desks of the boys and girls in my class to see who was dirtier for a science fair project,” she recalls. “After that I maintained a great interest in biology and the scientific method through high school, college and my career.”
Toward the end of his life, Amy’s father lived with ALS, a devastating neurological condition that leads to progressive paralysis, and ultimately, death. During that time, Amy moved home to help care for her father, an experience she says was extraordinarily challenging, but one that taught her resilience in the face of tough obstacles. To this day, she relies on the experience to push through frequent setbacks and failures that accompany the research and development of new medicines.
Challenges and Looking Ahead
Approximately 5,000 people in the U.S. are diagnosed with ALS each year—about 15 new cases each day. Currently, patients have few options, as there are no disease modifying therapies available. Amy and her team at Sanofi Genzyme are looking to change the trajectory of ALS by researching potential medicines, and even cures, for ALS and similar neurological conditions.
“It would be amazing for us to move the marker even by a small fraction, to understand the disease biology better,” she says. “The holy grail of research science is being able to get it to the clinic to deliver efficacious therapies to patients.”
According to Amy, gene therapies, which can allow researchers to deliver a health gene to patients through a modified virus, show great potential for treating diseases like ALS. In May 2019, the U.S. Food and Drug Administration approved the first gene therapy designed to treat young children with SMA. Like ALS, SMA is a neurodegenerative disease, as well as a leading genetic cause of infant mortality. Amy is hoping the learnings from research into treatments like these can spur a breakthrough in ALS.
“I’m very hopeful we will see progress in the next 10 years,” Amy says. “I know there are a lot of researchers out there who are working tirelessly on diseases like ALS, picking up after each failure and taking us closer to a new discovery with each option they try.”
As our payment system evolves to incorporate cutting-edge treatments like gene therapies that have the potential to treat or cure diseases like ALS, it’s crucial that innovative medicines are accessible and affordable for the patients who need them. To learn how America’s biopharmaceutical companies are exploring innovative payment mechanisms, such as outcomes-based contracts, visit innovation.org.
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