The Hermstad Legacy: Advances in Treatments for ALS

A Rare Disease Trailblazer Story

In Jaci’s honor, we gathered to share the challenges and triumphs of rare disease drug discovery. Listen to the recordings below that describe the collaboration that led to the development of the novel antisense therapy Jacifusin and how the work continues.

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Jaci Hermstad feeding a horse

Alex and Jaci Hermstad

Alex Hermstad lost her battle with a rare form of amyotrophic lateral sclerosis (ALS) at age 17. When her twin sister, Jaci, was diagnosed with the same genetic mutation 8 years later, she refused to take the news lying down. Together with her family, she mobilized a village of supporters on a quest to conquer her rare disease.

“What that young girl did and what we all did as a committed team in her honor will serve as a template for accelerating treatment for the future” – Lauren Black, Distinguished Scientist
 

Wondering how collaboration can help you achieve your rare disease drug development goals?

 

 

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Just press play hear from our host, Ben Stiller, watch a moving documentary narrated by Julianna Margulies, and learn from Project ALS, physicians involved in Jaci’s care, patient advocates, and leading researchers from academia and industry.

Welcome
Special guest, Ben Stiller

Documentary A Cowgirl's Courage: The Jaci Hermstad Story
Narrated by award-winning actress, Julianna Margulies 

The History of ALS
Bob Brown, MD, PhD, Professor of Neurology and Director of the Program in Neurotherapeutics, UMMSP

ALS Treatment: A Personalized Approach
Neil Shneider, MD, PhD, Director of the Eleanor and Lou Gehrig ALS Center

Panel Discussion: The Power of Collaboration 1
Moderators: Valerie Estess & Erin Flemming 

Animated Film: Antisense Technology
Ionis Pharmaceuticals

Antisense Technology Today
Frank Bennett, PhD, Executive Vice President, CSO, Ionis

Transformative Therapies for ALS
Toby Ferguson, MD, PhD, VP, Head Neuromuscular Development Unit, Biogen

Clinical Applications
Jinsy Andrews, M.D., M.Sc., Associate Professor of Neurology and Director of Neuromuscular Clinical Trials, Columbia University

Panel Discussion: The Power of Collaboration 2
Moderator: David Fischer

Advocacy & How You Can Connect
Kristina Bowyer, Vice President, Patient Advocacy, Ionis 

Headshot of Ben Stiller
Ben Stiller
Actor, Director, Writer, Producer, Friend of Project ALS

 

Ben Stiller is one of Hollywood's most successful and acclaimed multi-hyphenates, with a career spanning three decades across film, stage, and television. His versatility as an actor, writer, director and producer across genres is unparalleled, effortlessly moving between major franchises, arthouse films, blockbuster comedies, animated features, and dynamic dramas, many produced under his production company, Red Hour Films. He is the recipient of several of the industry's highest honors, including the Emmy Award, Directors Guild of America Award, BAFTA’s Charlie Chaplin Britannia Award for Excellence in Comedy, an American Cinematheque Tribute, and has been named to Time Magazine's TIME 100, a list of the world's most influential people. Among his numerous charitable and humanitarian endeavors, he currently serves as a Goodwill Ambassador to UNHCR, the UN Refugee Agency.


Headshot of Julianna Margulies
Julianna Margulies
Actor, Writer, Producer, Friend of Project ALS

 

As an Emmy, Golden Globe, and Screen Actors Guild Award winner, Julianna Margulies has achieved success in television, theatre, and film. Margulies starred as Alicia Florrick on the long-running hit CBS show The Good Wife, which she also produced, and is also well known for her role as one of the original cast members of ER. More recently, Margulies has starred on critically acclaimed series including Billions and The Hot Zone and she will soon be seen on the sophomore season of Apple TV+'s The Morning Show. Julianna will add author to her list of credits this May with the release of her upcoming memoir, Sunshine Girl: An Unexpected Life. She has been involved with Project ALS and Erin's Law and is also a board member of the New York City-based MCC Theater company. She resides in New York City with her husband and son.

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Frank Bennett
Executive Vice President, Chief Scientific Officer, Ionis

 

Dr. Bennett is the executive vice president and chief scientific officer at Ionis Pharmaceuticals and one of the founding members of the company. He is responsible for continuing to advance antisense technology and expanding Ionis drug discovery platform. Dr. Bennett is also the franchise leader for neurological programs at Ionis. He has been involved in the development of antisense oligonucleotides as therapeutic agents, including research on the application of oligonucleotides for inflammatory, neurodegenerative diseases and cancer, oligonucleotide delivery, pharmacokinetics and medicinal chemistry.

Dr. Bennet was awarded the 2020 Lifetime Achievement Award by the Oligonucleotide Therapeutics Society (OTS), is a co-recipient of the 2019 Breakthrough Prize in Life Sciences for his contributions to the discovery and development of SPINRAZA® (nusinersen) and a co-recipient of the inaugural Healy Center International Prize for Innovation in amyotrophic lateral sclerosis (ALS). He also received the 2018 Hereditary Disease Foundation's (HDF) Leslie Gehry Brenner Prize for Innovation in Science for his leadership and continued commitment to developing antisense therapies for Huntington's disease (HD).

Dr. Bennett has published more than 230 papers in the field of antisense research and development, and he is an inventor on more than 175 issued patents.

Prior to joining Ionis, Dr. Bennett was associate senior investigator in the Department of Molecular Pharmacology at SmithKline and French Laboratories (currently, GlaxoSmithKline).

He received his Ph.D. in Pharmacology from Baylor College of Medicine, Houston, Texas and his B.S. degree in Pharmacy from the University of New Mexico, Albuquerque, New Mexico. He performed his postdoctoral research in the Department of Molecular Pharmacology at SmithKline and French Laboratories.

Dr. Bennett serves on the Advisory Board for the Hereditary Disease Foundation.


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Lauren Black
Distinguished Scientist, Charles River

 

An ex-FDA reviewer, Lauren Black is now a Distinguished Scientist at Charles River with more than 25 years of experience in drug development, focusing on accelerated translation to Phase I/II clinical trials.

She works mainly at the first in human (FIH) stage - in high risk diseases using novel products. Before Charles River, Dr. Black was a reviewing pharmacologist in the FDA’s CDER and CBER, during which time she assessed preclinical data, worked on many FDA guidances, and represented FDA externally on oligonucleotides, safety pharmacology, choice of relevant species, and utilization of PAD as the response index for setting human safety margins. She was a founding co-author of FDA’s Human Start Dose guidance. As a specialist in biologic therapies and targeted agents, she was elected to serve in BIO’s BioSafe committee in leadership roles, resulting in a white paper on selective uses of large animals for human risk assessment. She sits on several government, academic, and industry advisory panels discussing in vitro toxicology, portfolio prioritization, technology transfer/capitalization, and regulatory strategy. Dr. Black received her BS from Carnegie Mellon University and PhD in pharmacology from the Virginia Commonwealth University School of Medicine, where she researched GPCRs.


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Kristina Bowyer
Vice President, Patient Advocacy, Ionis

 

Kristina Bowyer joined Ionis in 1992 and currently serves as the Vice President of Patient Centric Drug Development. She founded Ionis’ Patient Advocacy program 2012 to ensure that the patient perspective is incorporated into every aspect of drug development from research through market authorization. Kristina’s expertise is in rare and neurological diseases where she has developed new strategies to capture burden of disease from both the patient and the caregiver perspectives through innovative partnerships across multiple programs and organizations, such as spinal muscular atrophy, amyloidosis, myotonic dystrophy and ALS. Ionis currently has an extensive rare disease pipeline of RNA targeted therapeutics and has established strategic alliances with global pharmaceutical companies with the expertise to successfully launch rare disease treatments and ensure broad patient access.


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Robert Brown
Professor of Neurology and Director of the Program in Neurotherapeutics, UMMS

 

Robert H. Brown Jr., MD, DPhil, the Leo P. and Theresa M. LaChance Chair in Medical Research, professor of neurology and director of the Program in Neurotherapeutics at the University of Massachusetts Medical School (UMMS) and UMass Memorial Medical Center, is an internationally known researcher and physician leading the quest to cure neurodegenerative and neuromuscular diseases.

Prior to joining the Medical School in May 2008, Dr. Brown was professor of neurology at Harvard Medical School and a neurologist at Massachusetts General Hospital. He also served as Director of the Day Neuromuscular Laboratory and of the Muscular Dystrophy Association Clinic at Mass General Hospital.

Dr. Brown graduated magna cum laude from Amherst College in 1969 with a degree in biophysics. In 1973, he completed a doctorate of philosophy in neurophysiology at Oxford University and received his medical degree from Harvard Medical School in 1975. Following medical school, Dr. Brown completed his internship in internal medicine at Peter Bent Brigham Hospital and a residence in neurology at Massachusetts General Hospital. From 1980 to 1983, Dr. Brown was a research fellow in neuroscience at Children's Hospital in Boston. In 1975, Brown joined Harvard Medical School as a clinical fellow and rose to the rank of professor of neurology in 1998.


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Valerie Estess
Founder & Director of Research, Project ALS

 

Valerie Estess is a co-founder of and director of research for Project ALS, a non-profit 501(c)3 whose mission is to find and fund promising ALS research toward effective treatments and a cure.

Sisters Valerie, Jenifer, and Meredith Estess started Project ALS in 1998, when Jenifer, a thirty-five-year-old theater and film producer, was diagnosed with ALS.

Over two decades, Project ALS and its world-leading research teams have contributed to meaningful breakthroughs in ALS genetics, motor neuron and stem cell biology, disease modeling, and drug screening.

With Jenifer Estess, Valerie is co-author of Tales from the Bed: A Memoir. She lives in New York.


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Toby Ferguson
Head of Neuromuscular Development Unit, Biogen

 

Toby is a neuromuscular neurologist and neuroscientist who joined Biogen in 2013. His professional experience has been focused on developing treatments for neuromuscular disease, with a focus on ALS. He is currently the Head of the Neuromuscular Development Unit at Biogen and leads the neuromuscular clinical development group. Toby plays a key role in developing ALS clinical trials, and in driving preclinical strategy within neurodegenerative and neuromuscular disease. His group also works closely with the scientific and biomarker, and commercial teams at Biogen, Ionis, and other external collaborators to identify novel disease targets and to develop the needed tools for efficient clinical development.

At Biogen, Toby has advanced multiple programs into the clinic for ALS, DM1, and Parkinson’s disease. In ALS, one program (tofersen for SOD1 ALS) is in the final stages of clinical testing, and multiple additional programs are in early clinical testing including a program for C9ORF72 ALS and two programs for the broader ALS population. He is dedicated to the successful development of meaningful therapeutics for ALS and strongly believes that collaboration across industry, academia, and advocacy organizations is crucial to providing people with ALS the therapies they need and deserve.

Prior to Biogen, Toby had a clinical neuromuscular neurology practice and a lab focused on peripheral axon injury and regeneration at Shriners Research Center and Temple University in Philadelphia. Toby trained in neurology and neuromuscular disease at the University of Pennsylvania. He obtained an MD and PhD (Neuroscience) at the University of Florida and maintains an interest in axon regeneration and degeneration.


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David Fischer
Executive Director, Discovery Sciences, Charles River

 

David has led numerous early stage drug discovery programs in rare and orphan disease indications. He brings expertise in complex and primary cell-based assays, including iSPC and hESC stem cell models and human primary cell models. David holds a degree in Chemistry and a PhD in molecular Genetics, both from Leiden University.

Focus areas include cystic fibrosis, Huntington’s disease, ALS, Usher III Syndrome, Duchenne muscular dystrophy, and neurodegenerative disorders (Alzheimer’s and Parkinson’s disease.)

During his postdoctoral fellowships at the Netherlands Institute for Neuroscience in Amsterdam (an Institute of the Royal Netherlands Academy of Arts and Sciences) and the Free University Amsterdam, he focused on Alzheimer’s and Huntington’s Disease and mentored two graduate students, at the University of Amsterdam and at Leiden University.

Over 50 patent applications and peer-reviewed papers.


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Erin Fleming
Director of External Operations, Project ALS

 

Erin Fleming is Director of External Operations at the Project ALS Therapeutics Core at Columbia (THE CORE), where she oversees day-to-day operations and manages interactions with THE CORE's collaborators.

Previously, Erin was associate director at Project ALS, project manager at the start-up biotech Applied Therapeutics, and project director at the boutique life sciences consulting firm Clearpoint Strategy Group. She holds a BA in English and Comparative Literature from Columbia University.


Headshot of Lori Hermstad
Lori Hermstad
Alex & Jaci’s Mom and Patient Advocate

 

Lori Hermstad, 54, grew up in Storm Lake, Iowa, is a graduate of Buena Vista University with a Bachelors in Education. Today Lori resides in Spencer, Iowa, with her husband Jeff.

Lori and Jeff welcomed their beautiful twin daughters, Alexandria (Alex) and Jaci on August 22, 1993. The little red-headed full of life bundles of joy made their lives complete.

Sadly, Alex passed away from ALS on February 14, 2011, after a six year battle at the age of 17. Jaci was heartbreakingly diagnosed on February 14, 2019. Tragically, Jaci passed away on May 1, 2020, at the age of 26.

Lori has been an advocate for ALS research for many years. Both Alex and Jaci have donated their tissue to leading ALS scientists, Dr. Robert Brown and Dr. Neil Shneider.

Jaci was a trailblazer by being the first person to try the newly developed ASO for FUS-ALS aptly called jacifusen. Lori continues to advocate for treatment and a cure working with Congress and governmental legislators.Lori’s life is a patchwork of hope and determination, buttressed by her faith and all that she has experienced.


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Neil Shneider
Director of the Eleanor and Lou Gehrig ALS Center

 

Neil Shneider, MD, PhD, is a physician-scientist with expertise in neuromuscular development and disease. He is a graduate of Harvard College and of the MD-PhD program of the Columbia University College of Physicians and Surgeons, and he was Chief Resident of the Harvard Longwood Neurology Training Program.

Dr. Shneider completed his graduate work in the laboratory of Dr. Richard Axel and his postdoctoral fellowship in the laboratory of Dr. Thomas Jessell. After several years in the Intramural Program of the National Institute of Neurological Disorders and Stroke, Dr. Shneider returned to Columbia University as Assistant Professor of Neurology in the Neuromuscular Division. He is a member of the Center for Motor Neuron Biology and Disease and of the H. Houston Merritt Clinical Research Center. Dr. Shneider has clinical expertise in the diagnosis and care of patients with ALS and related motor neuron diseases.

Dr. Shneider is Director of the Eleanor and Lou Gehrig ALS Center at Columbia University, Co-Director of The Project ALS Therapeutics Core at Columbia, and the Claire Tow Associate Professor of Motor Neuron Disorders at Columbia.


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Jinsy Andrews
Associate Professor of Neurology and Director of Neuromuscular Clinical Trials, Columbia University

 

Jinsy A. Andrews, M.D., M.Sc., FAAN is an Associate Professor of Neurology, in the Division of Neuromuscular Medicine and serves as the Director of Neuromuscular Clinical Trials at Columbia University. She currently oversees neuromuscular clinical trials and cares for patients with neuromuscular disease, primarily with Amyotrophic Lateral Sclerosis (ALS). Dr. Andrews has extensive experience in all phases of human clinical trials and drug development in both the academic and industry settings. Dr. Andrews is the elected co-chair of the Northeastern ALS (NEALS) Consortium, which is a network of over 100 ALS clinical research centers internationally. She is also elected to the National Board of Trustees of the ALS Association and is a Fellow of the American Academy of Neurology (FAAN). Dr. Andrews has also received the Diamond Award for ALS clinical research from Wings Over Wall Street and the Muscular Dystrophy Association.

Dr. Andrews received her B.S. from Union College, M.Sc. in Biostatistics (Patient-Oriented Research) from Columbia University's Mailman School of Public Health and M.D. from Albany Medical College. She completed her residency training in Neurology at the University of Connecticut and served as the Chief Neurology Resident in her final year. Dr. Andrews completed fellowship training in Neuromuscular Disease/ALS and Clinical Neurophysiology at Columbia University. She is board certified in Neurology, Neuromuscular Disease, and Electrodiagnostic Medicine.