This year has been a challenging one for our Quest with the loss of our inspiration and co-founder, Augie Nieto.
Our mission to find a cure for ALS (Amyotrophic Lateral Sclerosis) and improve the lives of those affected by this devastating disease remains our top priority.
We’re proud of the progress made this year at the Augie’s Quest Translational Research Center. Here are just some of the ways your support has made a tangible impact:
1) Using advanced cell biology techniques, the Augie’s Quest Translational Research Center (AQ TRC) initiated collaborative studies to explore two new drug targets – medicine intervention points – in motor neurons from people with ALS grown in the the lab.Developed new techniques for growing and studying axons – the long connections between neurons and muscles – in the lab. These long connections break down in ALS. Understanding how they break down and discovering ways to stop that is important for developing effective treatments.
2) Initiated important studies of the how cells from people with ALS handle or mishandled critical RNA molecules. RNA molecules carry the instructions from human genes to the rest of the cell so that the cells can function properly. This is impaired in ALS and will likely need to be repaired. Scientists in the AQ TRC are exploring treatments that might address this dysfunction.As the end of 2023 approaches, we urge you to make your tax-deductible donation and help change the future for countless people living with ALS, their families, caregivers, and friends.
Incurable is Unacceptable. Together, we can create a future without ALS.
The ARC expansion includes three key changes: Doubling the Size of the Study – allowing us to gain new insights into ALS by gathering data from a larger and more diverse population.
Inclusion of Asymptomatic ALS-Related Gene Carriers – which has the potential to provide us with new insights into processes that delay or accelerate ALS disease onset or progression.
Integration of Electronic Health Records (EHRs) – enabling us to fill in information gaps and gain profound insights into the development and progression of ALS Read the full announcement to learn more about these important updates.
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.
By joining the Augie’s Quest Giving Circle, you’ll join a special community composed of passionate and inspirational people who are determined to make a difference in the ALS landscape. When you choose to support ALS research 365 days a year, you are choosing to change the future of ALS.
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.
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.
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.
