Target ALS

For #WomensHistoryMonth, we’re highlighting remarkable women who provide meaningful care for people living with #ALS. Dr. Ileana Howard, MD is a clinician and professor of rehabilitation medicine at the University of Washington as well as the associate director for ALS in the national program office of neurology at the U.S. Department of Veterans Affairs. In her work, Dr. Howard focuses on improving and standardizing ALS care for veterans across the U.S. She’s made lasting contributions to enhance treatment capability and care for veterans with ALS and their families. As a woman in science, she aims to follow in the footsteps of the trailblazing women who came before her. When reflecting on the contributions of Dr. Lisa Krivickas, who paved the way for Dr. Howard’s own work in rehabilitation medicine, she shared this: “I hope to follow her example by not only meeting the needs of my patients and families today, but also mentoring the next generation of ALS rehabilitation physicians.” Dr. Howard is one of the many compassionate and brilliant women who are building on years of progress to improve life with ALS today and in the future.

“Being part of a team working toward a greater cause means having a purpose-driven career where every action I take can directly contribute to making a meaningful difference in the lives of others.” – Ruby Hoglund Mission-driven work is more than a profession—it is a commitment to making a lasting impact. Across industries, those dedicated to advancing research, driving innovation, and advocating for change understand the fulfillment that comes from working toward a shared vision. At Target ALS, we are united by the belief that we will build a world where #EveryoneLives. Our collective efforts break down barriers, accelerate scientific progress, and bring us closer to a future where effective treatments for #ALS are a reality. When each contribution, no matter how small, is part of something greater, the potential for change is limitless. #ALSResearch #BreakingBarriers #WomensHistoryMonth

At Target ALS, we believe that progress is driven by the people behind the mission. In honor of #WomensHistoryMonth, we asked our team to reflect on their work and what it means to be part of a team dedicated to a greater cause. Courtney Christa shared her perspective: "Working together toward a greater cause means collaborating with passionate individuals who share a common vision. At Target ALS, teamwork not only amplifies the impact of research but also fosters a dedicated community striving to accelerate progress towards effective treatments.” Her words speak to the heart of what we do—bringing together multidisciplinary teams and uniting leaders across pharma, biotech, academia, venture capital, government, and related nonprofits. Every discovery, every breakthrough, and every step forward is made possible by the collective effort of those who share our vision of building a world where #EveryoneLives #ALS #ALSResearch

Dr. Morwena Latouche never had just one career path in mind—first, she wanted to be a science journalist, then a geneticist. But her curiosity led her to the field of neuroscience, where she now tackles one of the biggest mysteries in #ALS and Frontotemporal Dementia (#FTD): TDP-43 toxicity. As a Target ALS Basic Biology Consortia Grant recipient (2024) and researcher at the Paris Brain Institute, Dr. Latouche is taking a new approach to studying ALS. Instead of traditional drug screening, her team is using advanced computer-based design to develop small molecules that could target and potentially regulate TDP-43, a protein that plays a major role in ALS. These molecules could change how we diagnose and treat the disease, offering new hope for patients. But ALS research is full of challenges. Scientists are still learning how TDP-43 behaves in different patients, making it difficult to design precise treatments. That’s why #collaboration is key. Dr. Latouche is working with chemists and computational biologists, combining different areas of expertise to move research forward faster than any lab could alone. Her journey hasn’t been without obstacles. During her PhD, she encountered gender bias in science for the first time. “I had never thought about being a woman as part of the equation until I realized others did,” she recalls. But she refused to let it hold her back. Her message to young women in STEM? “Go for it. And if something has to stop you, don’t ever let it be the fact that you’re a woman. That’s nonsense.” Read her full story and groundbreaking research here: https://bit.ly/4imr3li #WomensHistoryMonth

Target ALS has reached a significant milestone—200 participants are now enrolled in our Global Natural History Study, a key component within our ALS Global Research Initiative (AGRI). This study is designed to enroll 1,000 participants—800 individuals with #ALS and 200 healthy controls—to advance the understanding of ALS and accelerate the development of new treatments. This effort enables participants to donate blood, cerebrospinal fluid (CSF), and other biofluids at 14 international sites (and growing). By building a comprehensive collection of samples and clinical data, we are equipping the scientific community with the resources needed to develop biomarkers, identify risk factors, and drive therapeutic breakthroughs. The study follows participants for 12 to 16 months, with ALS participants visiting the clinic every four months and healthy controls visiting twice. During these visits, biofluid samples are collected every three to four months, while optional at-home digital assessments of speech, respiratory function, and cognition take place every two weeks. These data points allow researchers to track disease progression in real-time, identify potential biomarkers, and develop tools for earlier detection and targeted treatments. Beyond this study, the long-term biobank established through this effort will continue fueling #ALSResearch for years to come. With every new participant, we move closer to a future where ALS is diagnosed earlier, understood more deeply, and treated more effectively. Through collaboration and innovation, we are breaking down barriers in ALS research and accelerating progress toward a world where #EveryoneLives

On Rare Disease Day, Target ALS held a community-based pop-up clinic in Phoenix, Arizona to address a critical gap in ALS research—diversity. Historically, 94% of ALS research participants have been Caucasian, leaving entire communities underrepresented in the search for effective treatments. This study was designed to change that. By bringing research directly to communities, we’re eliminating barriers for individuals with ALS and healthy volunteers to contribute vital data that could shape the future of ALS treatment. At these events, participants not only provide blood and saliva samples and complete environmental questionnaires, they also receive important information about resources available to them and have the opportunity to sign up for additional research studies. This event marked a significant step forward in our nationwide effort to make ALS research more inclusive. So far, we have enrolled 43% of participants from Hispanic, African American, Asian, and Indian backgrounds, a crucial milestone to ensure that ALS studies reflect the full spectrum of those affected. With a goal of reaching 300 participants by the end of the year, we are rapidly expanding our understanding of genetic and environmental risk factors for the disease. By April, the first dataset—including long-read sequencing and environmental questionnaire data—will be available to researchers through the Target ALS Data Engine, accelerating the development of treatments that work for everyone. This initiative is about more than data collection. It is about breaking down barriers to participation, empowering individuals with ALS and their families, and ensuring that the future of ALS research includes treatments for all communities. Events like this are proving that meaningful change is possible—one step, one sample, and one conversation at a time. Read more from Laura Dugom: https://bit.ly/4c1w0NN #ALSResearch #Diversity #EveryoneLives

This #WomensHistoryMonth, we recognize the groundbreaking work of Dr. Martha Pe?a, a neurologist and lead investigator in the Target ALS Global Natural History Study at our site in Bogotá, Colombia. Her research is uncovering critical insights into #ALS by studying Colombia’s unique genetic diversity—an approach that could reshape our understanding of disease progression and treatment. For Dr. Pe?a, this work is more than scientific discovery; it is a deep, personal commitment to the patients and families she serves. “People with ALS have taught me a lot about the disease, about medicine, about neurology, but also about life. So, for me, it’s a privilege—a privilege that comes with suffering and demands, but it’s a privilege, just like doctors who are dedicated to delivering babies consider it a privilege. I consider it a privilege to work with people with fatal diseases like ALS.” In regions like Huila, where ultra-rare genetic mutations have been passed down through generations, her team is bridging the gap between research and care. By integrating genetic, environmental, and cultural factors, her work provides a more comprehensive understanding of ALS—one that reflects the #diversity of those affected. Read more about her research and its impact on the future of ALS treatment: https://bit.ly/4bHMiLO

Scientific progress is about more than just solving a problem—it’s about understanding its root causes to create lasting change. In honor of #WomensHistoryMonth, we asked our team what inspires them most about working in a field dedicated to advancing science. Tammy Sutherns shared her perspective: "The radical collaboration approach by Target ALS resonates deeply with me. My MPH focused on health systems, where I learned the power of collective action. Addressing root causes has a far greater impact than solutions applied too late—what I call the 'band-aid approach.' I truly believe in Target ALS’s message that research is the highest form of care. With so many brilliant minds, including incredible women, coming together to understand ALS at its root, we have a real chance to ensure that effective treatments exist for all those affected. I look forward to being part of that story very soon." Progress in ALS research demands more than short-term solutions—it requires a commitment to uncovering the fundamental mechanisms of the disease and ensuring treatments reach those who need them most. Science is not just about discovery, but about impact. #ALS #ALSResearch #RadicalCollaboration #EveryoneLives

A Visionary Leader. A Champion for ALS. Dan Doctoroff’s life has been defined by bold ideas and extraordinary impact. As Deputy Mayor of New York City, he reimagined what was possible—driving the redevelopment of the World Trade Center site, Hudson Yards, the High Line, and Brooklyn Bridge Park. His leadership reshaped a city. But his greatest challenge would come not from the city, but from a diagnosis. ALS—a rare disease that took his father and uncle—became his reality. When symptoms first appeared, they disrupted a life built on momentum. But Dan’s response was not anger or despair. It was action. He founded Target ALS, not just to fight his own battle, but to confront a rare disease that devastates too many families. With the same relentless drive that built iconic landmarks, he is breaking down barriers to accelerate ALS research—driving collaboration, innovation, relentlessly in pursuit of effective treatments. Video courtesy of ALS United Greater New York #ALS #ALSResearch #EveryoneLives #NYC

Scientific innovation is driven by those who refuse to accept limitations. In Puerto Rico, Dr. Wojna Valerie, Dr. Brenda Deliz, and Frances Aponte Caraballo are pioneering efforts to decode the complexities of ALS within the island’s diverse population. Through the Target ALS Global Natural History Study, their research is identifying unique disease patterns, tracing biomarkers, and improving diagnostic precision—all critical steps toward personalized treatments. Their findings not only contribute to a global understanding of ALS but also highlight the necessity of inclusive research that represents the full spectrum of disease progression. But their impact goes beyond the lab. They are building infrastructure where none existed before, creating ALS education programs, and empowering future neuroscientists. Just two years ago, an ALS clinical trials unit in Puerto Rico was only a vision—today, thanks to their dedication, it is a reality. Those living with ALS who once faced isolation now have access to specialized care, cutting-edge research, and a support system that sees them, hears them, and fights for them. This #WomensHistoryMonth, we recognize these incredible women who are rewriting the future of ALS in Puerto Rico. Their passion, persistence, and belief in a better tomorrow are proof that change is possible—and that every voice, every discovery, and every step forward brings us closer to a world where #EveryoneLives Read More: https://bit.ly/3DyvZnM University of Puerto Rico Medical Sciences Campus #ALSResearch #ALS #BreakingBarriers #GlobalNaturalHistoryStudy #PuertoRico