On October 14, 2022, the ALS Therapy Development Institute scientists and guest presenters gathered at the ALS TDI Summit to provide the community with updates on research from the past year.
Dr. Fernando Vieira, CEO/CSO of TDI, emphasized how far we have come in ALS research – with more papers published about ALS in the past 9 years than in all of previous history and the recent approval of a new treatment for the disease.He also highlighted how little we still understand about ALS – and the need for more research and more effective treatments. “We’re proud to be the top funder of the ALS Therapy Development Institute’s critical work,” said Lynne Nieto, who attended the Summit with Augie’s Quest President, Shannon Shryne.
5/31/22: Eledon Pharmaceuticals announced positive topline results today from their Phase 2 trial of tegoprubart (formerly AT-1501) demonstrating safety, target engagement, and biomarker response in patients with ALS. Eledon’s release stated:
Tegoprubart was well-tolerated, with no drug-related serious adverse events
Dose dependent target engagement was demonstrated, and ALS associated pro-inflammatory biomarkers were both observed and significantly reduced in a dose dependent manner
Target engagement and level of pro-inflammatory biomarker reduction were associated with a trend in the slowing of disease progression as measured by ALSFRS slope when compared to a cohort from the ALS PRO-ACT database
Biomarkers significantly reduced included biomarkers also associated with IgA nephropathy and kidney allograft transplant rejection
“I’m proud to be the Chairman at Augie’s Quest to Cure ALS and ALS TDI, and excited to see the next phase of development for tegoprubart – as our work is far from finished. This milestone showcases ALS TDI’s ability to create quality therapeutics in their lab, and Augie’s Quest is proud to fund this critical science. Together, we will find a cure for ALS.” said Augie Nieto. Augie’s Quest has been the lead funder of this critical science granting over $72 million to ALS TDI under Augie’s leadership.
Tegoprubart is an antibody therapeutic targeting CD40L, a well-validated biologic target with a broad therapeutic potential. The Phase 2a trial was a 12-week, open label, dose escalating, safety, and biomarker study. The endpoints of the study were safety and tolerability, and changes in pro-inflammatory biomarkers.
“Neuroinflammation is a driving force in the pathogenesis and progression of ALS. The ability to suppress inflammatory responses may translate into clinical benefit,” said Stanley H. Appel, MD, Co-Director of the Houston Methodist Neurological Institute and Chair of the Stanley H. Appel Department of Neurology at Houston Methodist. “These results reinforce the exciting potential of tegoprubart as a promising therapy for patients with ALS.”
After being invented at ALS TDI with funding from Augie’s Quest to Cure ALS, tegoprubart (previously called AT-1501) was licensed to Anelixis Therapeutics, a for-profit biotech company that oversaw the completion of a Phase 1 safety trial in 2019 (Augie Nieto was chairman of Anelixis). In 2020, Anelixis was acquired by Eledon Pharmaceuticals, a for-profit clinical-stage drug development company. Eledon is developing precision therapies that target the CD40 Ligand pathway for use in organ and cellular transplantation, and for the treatment of autoimmune and neurodegenerative disease (including ALS).
“We are excited by the results from the phase 2 trial and by tegoprubart’s emerging clinical profile. The positive data represent a significant step in the development of tegoprubart as a potential treatment for people living with ALS,” said Steve Perrin, President and Chief Scientific Officer at Eledon Pharmaceuticals. “We thank all of the patients and families, as well as the investigators, who have supported us over the years for their commitment to finding a novel therapeutic option for ALS.”
Ending ALS will require many treatments to meet the needs of every individual living with this disease, and Augie’s Quest will continue to raise the funds and awareness urgently needed to advance cutting-edge research, fast-track effective treatments and ultimately, find a cure for ALS.
Watertown, MA – The ALS Therapy Development Institute (ALS TDI) and Augie’s Quest to Cure ALS are proud to honor the commitment of Liz Bodine to facilitate more effective ALS drug discovery at ALS TDI, the world’s most comprehensive drug discovery lab dedicated solely to ALS.
In recognition of a recent $1.5 million grant from Augie’s Quest, coupled with a $650,000 grant from 2019, ALS TDI has installed a plaque in the Discovery Biology Suite that honors Liz Bodine, the many generous and committed friends who donated in her honor, and Augie’s Quest to Cure ALS.
“We are indebted to Liz for her dedication in supporting our effort to find effective treatments for ALS that will meaningfully slow the progression of the disease. The way her friends and family rallied around our cause in her honor is a testament to the light and love she shared with everyone she touched,” said Augie Nieto, Chairman of Augie’s Quest to Cure ALS.”
ALS, also known as Motor Neuron Disease (MND), Lou Gehrig’s Disease, and Charcot’s disease, is a progressive neurodegenerative disease which attacks motor neurons in the brain and spinal cord resulting in the wasting away of muscle and loss of movement. Every 90 minutes, someone is diagnosed with the disease, and the average lifespan is 3-5 years.
“On behalf of my beloved wife, Liz, I would like to express our deep gratitude to Augie’s Quest and ALS TDI for this honor,” said Murray Bodine. “Liz was deeply inspired by the work being funded by Augie’s Quest at the Institute and it gave her purpose and comforted her to know that she was able to contribute to the efforts to find a cure for ALS. She, of course, shares this honor with her many friends and her family who lovingly stood by her and supported her during these past few years.”
ALS TDI’s CEO and Chief Scientific Officer, Fernando Vieira M.D., says that the discovery of new drug targets is vital to the development of effective treatments for ALS. “I am thankful I had the honor of meeting Liz’s husband, Murray, and so many of the friends that made this donation possible last October,” he added.
The Discovery Biology Team will work to strengthen existing and future research efforts by studying mechanisms of clinical ALS and using the knowledge to facilitate more effective drug development in ALS at the Discovery Biology Suite
Dedicated scientists at ALS TDI are working every day to invent and discover the many treatments that will be needed to cure ALS. ALS TDI’s work has enabled multiple therapies for ALS to move forward into human clinical development, and they will continue to discover drugs until there are effective treatments for everyone with ALS. Augie’s Quest to Cure ALS has been the lead funder of their work since 2005.
About ALS Therapy Development Institute
As the Drug Discovery Engine for ALS, the ALS Therapy Development Institute (ALS TDI) discovers and invents ALS treatments and partners to advance them into clinical trials. It is the first and largest nonprofit biotech focused 100% on ALS research. ALS TDI incorporates all aspects of drug discovery under one roof to find treatments as quickly as possible.
Based in Watertown, MA, ALS TDI employs a team of industry trained, drug development experts with more than 300 years of combined experience. ALS TDI is internationally recognized as a leader in preclinical and translational ALS research, and partners with pharmaceutical companies and biotech organizations all around the world. Rated a four-star nonprofit on Charity Navigator, ALS TDI spends 87 cents of every dollar raised on finding effective treatments and cures for ALS. Visit www.als.net for more information.
About Augie’s Quest to Cure ALS
Augie’s Quest to Cure ALS is a nonprofit committed to changing the experience of people living with ALS by fast-tracking cutting-edge research to advance effective treatments and an ultimate cure. The organization is galvanizing thousands to join this fight, confronting ALS in an entirely new way, and driving innovative research forward, and at an accelerated pace. Augie’s Quest was founded in 2005 byAugie Nieto, the successful fitness industry mogul behind Lifecycle and Life Fitness who has been living with ALS since 2005 and whose life story was chronicled in the award-winning film, Augie. Please join our Quest at www.augiesquest.org.
Phil Green became a champion for the ALS community after his ALS diagnosis in 2018. We are proud to have his support on the Augie’s Quest Leadership Council.
Clinical trials are a critical part of research. When a scientist is developing a new therapy or treatment for a disease, clinical trials are how they make sure it’s safe and effective before it can become available to anyone who needs it. But for someone with amyotrophic lateral sclerosis, or ALS, a disease that has no known cure, clinical trials are about so much more than moving science forward — they’re their only shot at curing, and sometimes even slowing down, their disease.
Navigating clinical trials with ALS can seem daunting, especially when you’re not familiar with scientific research. So we sat down with Phil Green, an ALS Champion and advocate since his diagnosis in 2018 and a member of Augie’s Quest Leadership Council, to clear up some of the mystery surrounding clinical trials from a patient perspective. Drawing from his own clinical trial experience and ALS advocacy work, he gave these pointers for anyone wondering if a clinical trial is right for them.
There’s a limited window where you might qualify.
To qualify for any clinical trial, you must meet certain requirements. For example, to enroll in many ALS trials, you cannot be more than two years beyond your first symptoms. And since it can take a long time from when symptoms first start to finally get a diagnosis — sometimes a year or more — you’ll want to act fast.
The researchers also need to be sure that your ALS is progressing at a certain pace before you start the trial. Unlike other diseases, there are no known biomarkers in the blood that researchers can look for to see if the therapy is working, and there’s no growth or tumor they can measure, as in cancer. With today’s knowledge of ALS, the only way to determine if a therapy is working is by measuring your disease progression, so if you aren’t progressing enough to begin with, researchers won’t be able to tell if the therapy is helping.
You’ll have to take some initiative.
You should always talk to your doctor about clinical trial opportunities and any questions you might have. But your doctor might not be aware of every ALS trial that’s happening around the country, so it’s a good idea to do your own research on what trials are open and where. You can search for ALS on clinicaltrials.gov, the NIH’s clinical trials database, or you can use the ALS Signal Dashboard created by our friends at I Am ALS to search for clinical trials by state, trial phase, genetic target and more. Then, it’s up to you to consult with your doctor and reach out to the trial site to find out if you qualify.
It’s a big commitment.
Clinical trials ask a lot of their participants. Though some trials may enable remote participation, the majority require you to travel to the clinic for every visit, and if you’re unable to drive, you’ll need to arrange your own transportation. It can be a big time and financial commitment, especially if the clinic is out of town.
“From a personal commitment level, the investment is pretty high,” Phil said. You’re also committing your body to a therapy that may or may not work, and if you’re participating in a double-blind controlled study, like Phil did, there’s a fair chance — in his case, a 50/50 shot — that you’re in the placebo group and not getting any real treatment at all.
But if it does work, the payoff can be huge. Since the study hasn’t been published yet, Phil doesn’t know for sure if he got the investigative treatment or a placebo, but he’s almost certain he got the treatment because his disease stopped progressing for a year. “If I was in the placebo group, how can I explain how my disease progression stopped for the majority of 2019?” he said.
No matter the outcome, you’re helping people with ALS everywhere.
Phil Green and Lynne Nieto addressing the crowd at the 2021 Cure ALS Golf Classic to support Augie’s Quest
Even if the treatment doesn’t work, or if you get a placebo, you’re still making an invaluable contribution to research for ALS cures — one that will help everyone with ALS, now and in the future.
“Without the ALS community’s participation in these trials, a therapy that might help us will never reach the market,” Phil said. “So we need to be the research partners for these companies so they can bring their potentially impactful therapy to everyone else who currently doesn’t have access to these investigational therapies.”
You won’t be left in the dark.
So what happens once you enroll? Every trial is different, but each trial site has a clinical trial coordinator who will map out the study for you and make sure you understand what’s happening every step of the way.
In Phil’s case, he received a schedule at the start and was deliberately walked through what would happen and when. “I felt very informed up front, which I thought was very helpful,” he said. He was also encouraged to reach out to the trial coordinator by text or email at any time, so he always had a direct communication line for any questions or concerns he might have.
You need to find the right fit for you.
Phil’s top advice for someone looking to enroll in a clinical trial?
“Find a trial that will meet your individual needs.”
Consider how long the trial will run, how many trips you’ll have to make to the trial site or if you’ll be able to check in at your home clinic, what interventions it will entail, what side effects you might experience, and whether you might be compensated for some of your out-of-pocket expenses (like flights and hotel stays if you’re traveling out of state). These are all questions you’ll want to ask when you first reach out to the trial coordinator.
You still have other options to participate in research.
Even if you don’t qualify for a clinical trial, or if you decide clinical trials aren’t for you, you might be able to enroll in an observational study, another important part of clinical research.
“There are so many observational trials you can participate in that not only help advance the research and understanding of the disease, but also can give you insights into your own progression and help you stay on top of your own personal journey,” Phil said. While you won’t gain access to a potentially helpful new therapy, you’ll still be contributing to ALS research by providing important data to scientists studying the disease.
Augie’s Quest is proud to support our research partners at the ALS Therapy Development Institute (ALS TDI) to fund critical science and drug development through work happening at the Augie’s Quest Translational Research Center. In 2021, through a partnership with the Eddie and Jo Allison Smith Family Foundation, Augie’s Quest also funded all new enrollees of the Precision Medicine Program (PMP).
The PMP is the most comprehensive and longest running translational research study in ALS. People from around the world share their data on movement, lifestyle, medical history, genetics, biomarkers, voice recordings and patient cell biology. By collecting the unbiased data, researchers are able to better understand ALS. This incredible program comes at no cost to its participants and is funded thanks to generous Augie’s Quest donor support.
The decision is yours.
Clinical trials are a crucial part of scientific research, and especially for diseases like ALS, they’re a path to discovering truly helpful therapies — and hopefully, one day, cures.
“We’ll never have FDA-approved therapies for ALS that are somewhat helpful if people don’t participate in clinical trials,” Phil says. Your clinical trial participation benefits the greater ALS community, but it also stands to benefit you, he explains.
“Clinical trials are an opportunity to try an investigational therapy that might actually help slow down your disease progression,” Phil says. “We already know what the alternative is, so right now our only chance at hope is through these trials.”
Ultimately, the decision to participate in clinical research is one that only you can make for yourself. But if you’re having trouble weighing the pros and cons, Phil suggests talking it through with other members of the ALS community who’ve been where you are today. “I think leveraging people who walked in your shoes or sat in your wheelchair is critical,” he says. “I like to think that we are the experts on living with this disease, and I’m not a doctor so I’m not going to tell you what to do, but I can tell you what I’ve done and hopefully that can help you make a decision.”
Augie’s Quest is committed to funding cutting-edge research to find effective treatments, and ultimately cures, for ALS. Your support means hope for people living with ALS. Donate today.
Every 90 minutes, someone is diagnosed with ALS and will be challenged to choose how they spend their remaining years. Augie’s Quest is determined to change the future of ALS — because we believe that ALS is curable; it’s just underfunded.
Meet Ellen Corindia — a United States veteran, wife, dear friend of Augie’s Quest and ALS Champion. When Ellen was 26 years old, she was diagnosed with ALS.
“Being diagnosed with ALS is like being shoved out of a skydiving plane not knowing if your parachute will open. Hitting the ground and death is inevitable, yet we all hope for a lengthy descent with time to enjoy the view. I chose to become a monthly donor to Augie’s Quest in the hopes that more people can have a lengthy parachute ride until the day when life stops shoving people out of the plane in the first place! Please consider joining me. I have never seen a more promising time in ALS therapeutic development. We are getting there. It will continue to take year-round financial commitments. A little bit from lots of people will get us to effective treatments.” – Ellen Corindia
This year, Augie’s Quest to Cure ALS has continued to fast-track the most promising ALS treatments yet. In May 2021, we presented our research partners at the ALS Therapy Development Institute (ALS TDI) with $500,000 in grant funding to help continue making critical advancements in our understanding of ALS and develop new treatments for those living with ALS today.
Eledon Pharmaceuticals announced in August 2021 that its Phase 2 study of AT-1501 in people with ALS should be fully enrolled by year’s end, with results expected between April and September 2022. Due to our initial funding of ALS research, the first AT-1501 subject was enrolled by Eledon Pharmaceuticals in their Phase 2 clinical evaluation trial in November 2020.
The AT-1501 research initiative marks the very first time in history a non-profit organization research project has ever reached this stage of drug development — incredible news that was made possible thanks to generous Augie’s Quest supporters.
In addition to the critical work happening at the Augie’s Quest Translational Research Center at ALS TDI, Augie’s Quest is very excited to support an emerging research project at Duke University led by Richard S. Bedlack Jr., MD, PhD, studying the potential role of the gut microbiome in ALS progression and reversals.
People with ALS have markedly different disease progression rates, and a rare few even have ‘reversals’, recovering most or all of their lost motor function. While demographics, clinical characteristics and genetics can predict some of this variability, none of these completely explains the different progressions rates. Understanding the factors that influence the variability in disease progression may lead to new treatments that slow the disease down in all patients.
We are determined more now than ever, to continue funding the best science and most promising research to fast-track effective treatments and find a cure for this devastating disease. Today, there is real hope, like never before. But we need your support in this critical fight. Join our Giving Circle and support ALS research 365 days a year.
If curing a debilitating and deadly disease were as simple as driving on a highway, would you take the fast lane? Of course, you would! That’s exactly what we’re doing — we are determined to aggressively pursue our mission to find a cure for ALS by funding the most promising research and science. Because we believe ALS is curable; it’s just underfunded.
We are proud to partner with the ALS Therapy Development Institute (ALS TDI) to fund critical science and drug development at the Augie’s Quest Translational Research Center and are excited for new and emerging scientific developments.
ALS is a disease we can tackle; a problem we can solve. But still, ALS research funding lags significantly behind other neurological diseases. However, we see hope and a cure just around the corner. That’s because every dollar we raise drives innovative science and research forward, at an accelerated pace. And with every new discovery, we’re making real progress in managing ALS — including one of the most promising drug treatments yet in AT-1501.
Due to our initial funding, the first AT-1501 subject was enrolled by Eledon Pharmaceuticals in their Phase 2a clinical evaluation trial in November 2020. The AT-1501 research initiative marks the very first time a non-profit organization has ever reached this stage of drug development, in any disease indication. Augie’s Quest to Cure ALS is proud to have provided the significant funding for this project.
As CEO of Eledon Pharmaceuticals, David-Alexandre Gros tweeted, “There would be no AT-1501 without Augie and Lynne Nieto and Augie’s Quest”. Under Augie’s personal oversight and continued leadership, more than $150 million has been raised for ALS research.
With help from supporters like you, we can continue funding the most robust pipeline of potential ALS treatments in the world. And more than any other time, we now have the best shot at success. We are working day in and out to provide funding for the next promising ALS treatments, and as quickly as possible. There is hope for those living with ALS. We just need more support to stay the path and help create a world where we can say Augie’s Cure instead of Lou Gehrig’s disease.
With your help, we see a world with new hope for countless people living with ALS, their families, caregivers and friends. With every gift, you help fast-track effective treatments and an ultimate cure for this devastating disease. Because ALS is curable; it’s just underfunded. Support our Quest to find a cure by making a donation today.
Augie’s Quest to Cure ALS is proud to provide $500,000 to our research partners at the ALS Therapy Development Institute (ALS TDI). The grant funding will help ALS TDI continue making critical advancements in our understanding of ALS and develop new treatments for those living with ALS today.
We are determined more now than ever, to continue funding the best science and most promising research to fast-track effective treatments and find a cure for this devastating disease. Grant funding will be put to immediate work within the Augie’s Quest Translational Research Center at ALS TDI and will support:
The discovery of new drug targets by using pluripotent stem cells: a type of stem cell that has emerged as a significant source of stem cells for ALS research.
Expanded drug discovery model development to move possible ALS treatments from the lab to the clinic as quickly as possible.
The process to investigate and validate potential biomarkers in human blood: one potential way to improve earlier diagnosis of ALS would be to identify specific biomarkers that can be identified early in the disease process.
The Augie’s Quest Translational Research Center is part of the ALS Therapy Development Institute, the world’s most comprehensive lab dedicated solely to ALS research. Located outside of Boston, ALS TDI is a non-profit biotech that spans every aspect of drug discovery research and therapeutic development. Through the Augie’s Quest Translational Research Center, ALS TDI is able to partner with ALS patients to spearhead discovery of effective therapies for this devastating disease.
Translational research has emerged as an increasingly important component of drug discovery in recent years. The main focus of translational research is to obtain an in-depth understanding of mechanisms that drive human disease and to integrate this information into all stages of drug development from early discovery to clinical trials.
“I’m so proud of the work being done at the Augie’s Quest Translational Research Center at ALS TDI. We speak to newly diagnosed families every week who are hoping for a cure, and we’re focused on funding the best science to make that a reality in the future.” – Augie Nieto
Today, there is real hope like never before. We remain committed to finding a cure for ALS and your generous donation helps fast-track effective treatments and an ultimate cure for this devastating disease.
When we envision a titan of industry, we often think of a serious, iron-fisted business mogul residing behind a long, polished table in a high-rise boardroom overlooking the world, disconnected from the everyday struggles and realities of the average person.
Augie Nieto shatters this mythological ideation of yesteryear’s out-of-touch tycoon with just a crack of his mischievous smile. He’s at the top of a very small class of unique, powerful, and innovative entrepreneurs who are successfully running their endeavors with a hands-on approach, creating and collaborating with people face-to-face in the most authentic of new business and philanthropic models.
With an ever-present twinkle in his eyes, Augie is the leader of this new school of tough-as-nails, hardworking, yet compassionate and empathetic CEOs and leaders with a certain sense of ethos not evident in previous generations.
However, he’s extremely different from his counterparts.
Augie can’t walk.
He can’t talk.
He can’t even pound his fist on that iconic boardroom table.
That’s not entirely what makes Augie stand out from some of our most influential leaders. While Augie is no longer a mover and shaker in a literal sense, he is one of the most motivated and enigmatic people you will ever meet.
You can see it in his eyes and the way he looks at a complicated problem, like a strategy in a chess game, where he’s working out his next move in his head. Augie still does what Augie does best: he’s a thinker and creator. These immutable characteristics of Augie’s energetic mind are what fuels his tireless work and the incredible path he’s taken since his devastating ALS diagnosis in 2005 when he was at the top of his professional and personal game
Augie was in the prime of his life, at an ageless 47 years old, when this strong, adventurous, competitive specimen of a man started experiencing the early tell-tale symptoms of ALS. He and those around him were shocked to see his fit, toned, and youthful body reject the simplest of physical commands. To imagine his boundless physical energy and zest for outdoor adventure, travel, and his everyday freedom slipping away was immeasurable and would normally sink the strongest of wills.
However, Augie isn’t like most people. ALS picked a fight with the wrong guy.
From entrepreneur to warrior philanthropist
Augie’s close friends and allies can all attest that ALS has met its match in Augie. His sheer will, competitive spirit, cutting sense of humor, and sharp mind that solved countless complicated business problems took over as Augie shifted gears to take on the toughest fight for his life and the lives of thousands of people living with ALS.
Augie has always been on some kind of quest. From his early, self-starter days launching a popular line of gym and fitness equipment under the renowned Lifecycle brand to ultimately becoming the founder and President of his very successful umbrella company, Life Fitness, Augie has always been a leader in lifestyle, exercise, fitness, and the pursuit of better health. He’s the original social influencer and branding guru of the health and fitness industry.
By design, Augie was the perfect candidate to take on a disease like ALS.
After learning the stark reality that ALS is not only incurable but ranks extremely low in widespread support and research for treatment and cures, Augie set his sights on defeating his disease with the gusto and business acumen that launched so many successful past endeavors. With the same charisma and drive Augie brought to running his successful business and boardrooms, along with his deep network of close friends and influential connections, Augie began his new life’s work. He co-founded Augie’s Quest to Cure ALS and partnered with the ALS Therapy Development Institute in a multi-pronged attack on ALS.
From the very start, Augie’s mission was clear and simple: to find a cure for ALS. Augie quickly learned that to find a cure, he needed to raise immediate funds and an urgent awareness to launch a full-scale revolution in ALS research. To learn that ALS was not only incurable but underfunded just wasn’t the answer Augie was looking for.
A game-changer in the fight against ALS
Early in his fight, Augie realized that awareness wasn’t going to be enough to change the abysmal ALS research landscape. It was going to take money — a lot of money.
With a powerful cadre of long-time friends, family, and business innovators, Augie founded one of the most effective and creative teams to face fundraising for ALS full-time. From events, corporate sponsorships, and good old-fashioned tireless networking and advocating, Augie’s efforts are beginning to pay off in a big way.
Since he first founded Augie’s Quest in 2005, his team has raised millions of dollars strictly for ALS research and still is. With the launch of their funding and support for the Augie’s Quest Translational Research Center at the ALS Therapy Development Institute (ALS TDI) in 2020, Augie’s Quest donors are literally changing the game in the fight against ALS. With their support, this groundbreaking research center is dedicated to continuing the quest by identifying new drugs like AT-1501 to fight ALS.
In keeping with a reputation for results, the Augie’s Quest Translational Research Center has integrated with the ALS TDI’s Precision Medicine Program (PMP), the most comprehensive and longest-running translational research study in ALS. PMP researchers study data collected from the Augie’s Quest Translational Research Center to develop a variety of personalized treatment approaches for individuals with ALS. Partnerships like this will ultimately lead to innovative therapeutic discoveries and new approaches to treating a spectrum of ALS patients.
Augie doesn’t take “no” for an answer
As we’ve come to learn, Augie is a lifelong competitor and doesn’t take “no” for an answer. It’s that same stubborn tenacity that has allowed him to outlive his ALS diagnostic class of 2005. Miraculously, he’s the only survivor left from that year.
Sixteen years after his initial diagnosis, and outliving everyone’s expectations, Augie is still personally involved in the day-to-day work toward the next promising ALS treatment. He knows we are very close to a breakthrough. That’s what keeps him going, along with his infectious sense of humor and affinity for telling a good joke or two.
Yes, Augie may not be able to talk. However, he’s still the most magnetic voice in the room.
To learn how Augie’s sheer will and steadfast belief in what Augie’s Quest is doing, and how that drives him beyond the limits of human strength and endurance, see Augie’s incredible story in the feature film-length documentary “Augie”.
Would you be surprised to learn that vital research and clinical trials required to approve a new drug therapy are extremely expensive? You might also be shocked to discover that ALS research is abysmally underfunded. Those are two disheartening facts about ALS that our founder Augie Nieto discovered early on in his fight against this devastating disease.
As a result, when Augie founded Augie’s Quest to Cure ALS, it was his steadfast mission that all funds raised were invested directly towards the cure for ALS. Our team has kept true to his mission as it was clear early on to Augie that Augie’s Quest needed to be laser-focused to find a cure and fund the best ALS research.
Augie’s Quest Translational Research Center & AT-1501
One of our biggest accomplishments came in 2020, when we launched our funding and support for the Augie’s Quest Translational Research Center at the ALS Therapy Development Institute (ALS TDI). The center is dedicated to continuing our quest in identifying new drugs like AT-1501 to fight ALS.
The AT-1501 research initiative marks the very first time in history a non-profit organization research project has ever reached this stage of drug development — incredible news that was made possible thanks to our generous supporters.
Due to our initial funding of ALS research, the first AT-1501 subject was enrolled by Eledon Pharmaceuticals in their Phase 2 clinical evaluation trial in November 2020.
In August 2021, Eledon Pharmaceuticals announced that the Phase 2 clinical trial of AT-1501 — which shows promise to improve muscle function, slow disease progression and improve survival for people living with ALS — is likely on track to be fully enrolled by the end of 2021.
It’s estimated that 54 adult patients with ALS in the United States will be enrolled in the study at 12 separate ALS treatment sites. Once patients are established in the study, they will be administered four ascending doses of AT-1501 via IV infusion in each of the sequentially enrolling cohorts. Each participant will receive six bi-weekly infusions of AT-1501 over an 11-week period.
We are proud to partner with ALS TDI to fund critical science and drug development at the Augie’s Quest Translational Research Center and are excited for new and emerging scientific developments.
The need to discover and fund more research and clinical trials like AT-1501
Partnering with the Eddie and Jo Allison Smith Foundation, Augie’s Quest is funding the first 100 patients to be enrolled in ALD TDI’s Precision Medicine Program this year.The data collected from volunteer ALS patients in this program is expected to be an important discovery, and further supports the belief that multiple therapies will be needed to treat and cure ALS. We are determined to continue to fund the best research to make these drug therapies across the ALS spectrum a reality.
While Augie’s Quest is hyper-focused and dedicated to specifically curing ALS, there is notable evidence that the AT-1501 therapy might also be an effective ally in fighting against other diseases like Alzheimer’s disease and Lupus. The ripple effect of giving to Augie’s Quest to fund drug therapies like AT-1501 is very heartening.
Augie’s personal focus in the fight is stronger than ever
Augie’s extensive network of friends and family can attest to how sharp Augie’s mind continues to be. His closest friends remark that Augie seems to be even more focused and attentive than in his pre-ALS life, due to having to transfer his boundless physical energy and enormous creativity to successfully power his new life’s work to find a cure for ALS. Augie’s relentless drive, his sense of humor, and his refusal to accept the word “no” have been the key components to our success in fundraising for the cure for ALS.
As CEO of Eledon Pharmaceuticals, David-Alexandre Gros tweeted, “There would be no AT-1501 without Augie and Lynne Nieto and Augie’s Quest”. Under Augie’s personal oversight and continued leadership, more than $150 million has been raised for ALS research.
How you can help fund the fight
Donor support has been critical in making AT-1501 a possible therapy reality for those living with ALS, and we will continue to support our research partners at ALS TDI and CEO Fernando Vieira lab’s incredible research efforts. Our goal for 2021 is to return to our pre-pandemic funding levels to drive new and exciting therapy research opportunities at ALS TDI. Today, there is real hope like never before.