Optimizing Collaboration

In the past, ALS research has been fractured and duplicative with researchers working in silos—that environment creates a breeding ground for redundancy and inefficiency

The ALS ONE partnership unites leading ALS experts from a wide array of leading institutions as one team working toward a common goal.  These stakeholders regularly communicate, share data, discuss findings, and establish best practices. This partnership improves coordination among research, care, and treatment teams, ultimately reducing the duplication of efforts.

The unprecedented linking of minds and resources from Massachusetts General Hospital (MGH), University of Massachusetts Medical Center, ALS Therapy Development Institute (ALS TDI), and Compassionate Care ALS (CCALS) is unique for its leadership in efficiency, dedication to innovative research, and commitment to increasing access to care and treatment.

From The Healey ALS Platform Trial at MGH, the very first ever of it’s kind, ALS TDI’s AT-1501 (Anti-CD40-Ligand Antibody) trial, to the anti-sense oligonucleotide (ASO) trial to target the C9orf72 gene under the direction of Dr. Brown at UMass Memorial; among many others, you can feel confident that your donations are impacting cutting edge and promising collaborative research. Meet our team

Platform Trial Updates & Webinars

Weekly Webinars! Join our partners at The Healey Center for ALS at MGH weekly to continue the great communication between the patient and science community for this groundbreaking trial! 

Upcoming Webinar Dates – Fall/Winter 2020/2021:

Get up to date by watching past webinar updates:

December 1: Monthly Update
Merit Cudkowicz, MD, MSc, principal investigator, and Sabrina Paganoni, MD, co-principal investigator of the HEALEY ALS Platform Trial, provided an update on the progress of the HEALEY ALS Platform Trial and were joined by Jen DiMartino, Executive Director of ALS ONE.
Watch recording

November 19: Weekly Q&A
Sabrina Paganoni, MD, PhD presented the week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
Watch recording | Download slides (PDF)

November 12: Weekly Q&A
Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented the week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
Watch recording | Download slides (PDF)

November 5: Weekly Q&A
Sabrina Paganoni, MD, PhD presented the week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.
Watch recording | Download slides (PDF)

October 29: Monthly Update
Merit Cudkowicz, MD, MSc, principal investigator, and Sabrina Paganoni, MD, co-principal investigator of the HEALEY ALS Platform Trial, provided an update on the progress of the HEALEY ALS Platform Trial. They were joined by Troy Green from Tackle ALS and Peter Foss from ALS Finding a Cure.
Watch recording | Download slides (PDF)

October 22: Weekly Q&A
Enrollment updates, trial sites, and patient questions.
Watch recording | Download slides (PDF)

October 21: ALS/MND Alliance Clinical Trial Design Webinar
Merit Cudkowicz, MD, MSc joined researchers from Europe and Scotland to discuss new research and clinical trial models, how they will change the landscape going forward and their practical implications for patients and researchers. Watch recording.

October 15: Weekly Q&A
Sabrina Paganoni, MD, PhD covers newly active trial sites, patient navigator service, and patient questions about the HEALEY ALS Platform Trial.
Watch recording | Download slides (PDF)

October 13: CTTI Master Protocol Development Webinar for Researchers
Marianne Chase, the Senior Director Clinical Trial Operations at Mass General’s Neurological Clinical Research Institute, joined representatives from the FDA, Janssen R&D and Edgewise Therapeutics to discuss the design of master protocol clinical trials, which allow researchers to answer multiple scientific questions within a single trial. Watch recording.

October 8: Weekly Q&A
Mass General researchers Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD are joined by Sandy Morris of I AM ALS.
Watch recording | Download slides (PDF)

September 29, 2020: Monthly Update
Merit Cudkowicz, MD, MSc, principal investigator, and Sabrina Paganoni, MD, co-principal investigator of the HEALEY ALS Platform Trial, provided an update on the progress of the HEALEY ALS Platform Trial. They were joined by Kuldip Dave, PhD, of the ALS Association.
Watch recording | Download slides (PDF)

August 27, 2020: Monthly Update
Sabrina Paganoni, MD, co-principal investigator of the HEALEY ALS Platform Trial, joined Taner Dagdelen, co-founder and CEO of Unite Genomics, for a webinar on August 27 to discuss the latest updates on the trial.
Watch recording

September 29, 2020 Update: Merit Cudkowicz, MD, MSc, principal investigator, and Sabrina Paganoni, MD, co-principal investigator of the HEALEY ALS Platform Trial, provided an update on the progress of the HEALEY ALS Platform Trial. They were joined by Kuldip Dave, PhD, of the ALS Association. Watch the full webinar HERE.

AUGUST 27, 2020 Update: Sabrina Paganoni, MD, co-principal investigator of the HEALEY ALS Platform Trial, joined Taner Dagdelen, co-founder and CEO of Unite Genomics, for a webinar to discuss the latest updates on the trial. Watch the full webinar HERE.

AUGUST 20, 2020 Update: We are pleased to share this newly released informative video from our partner, Dr. Merit Cudkowicz, which highlights the progress and gives additional insight, into the The Healey ALS Platform Trial – the first and only of it’s kind to test multiple drug therapies at once. “The goal is not to stop and slow down the illness. We want to stop the illness, we want to reverse it and we want to prevent ALS from ever happening in the first place”. – Dr. Merit Cudkowicz. We call that ALS DONE and your support of ALS ONE will help further this important breakthrough trial. Watch the full video HERE.

AUGUST 3, 2020: We are excited to announce that the first ever ALS Platform Trial has officially begun! Five centers are now open for enrollment in The Healey Center for ALS at MGH’s groundbreaking platform trial, led by Dr. Merit Cudkowicz and team, and it is anticipated that additional sites will be opening over the next days and weeks. As the first ever platform trial for ALS, the HEALEY ALS Platform Trial is testing three proposed drug regimens and will add two more over the next several months. The three experimental treatments include, (in no particular order): Zilucoplan, a small macrocyclic peptide inhibitor of complement component 5 [C5], developed by UCB Ra Pharmaceuticals, Inc.; Verdiperstat, an oral myeloperoxidase inhibitor, developed by Biohaven Pharmaceutical Holding Company Ltd., and CNM-Au8 nanocrystalline gold, an intracellular nanocatalyst to support cellular bioenergetics developed by Clene Nanomedicine, Inc.

To read more about the start of this highly anticipated trial, please click HERE.

To watch the launch video, please click HERE.

Individuals living with ALS who are interested in participating in this trial are encouraged to complete the Patient Interest Form, which can be downloaded HERE.

JULY 8, 2020: The latest updates on The Healey ALS Platform Trial were provided via zoom webinar on July 7, 2020. This hour-long informative session by Dr. Merit Cudkowicz and team includes many answers to frequently asked questions by pALS and families. The webinar, in it’s entirety, may be viewed by clicking HERE.

Since discovering the first gene that causes ALS over 20 years ago, Dr. Robert Brown of the University of Massachusetts Memorial Medical School, has worked to develop gene therapies for people with ALS. Through an ALS ONE partnership with MGH, the team is now altering genes in patients using a harmless virus delivered in a single dose, potentially revolutionizing care and treatment for people with genetic forms of ALS.

Researchers at ALS TDI are developing a drug that restores immune system balance and reduces inflammation. Meanwhile, a team at MGH has developed new imaging tools that measure inflammation in patients with ALS. The ALS ONE partnership has hastened the development of this promising anti-inflammatory therapy to bring it from the lab to patients—and preclinical work is under way.  

Posted Date: October 23, 2020

MEASURING FATIGUE IN ALS: A study by lead investigator, James Berry, MD, at the Sean M. Healey and AMG Center for ALS at MGH. OVERVIEW: Looking for healthy volunteers and individuals with motor neuron disease (ALS, PLS, HSP, SMA) to complete a survey study to help us learn more about fatigue in individuals with motor neuron disease.

ELIGIBILITY CRITERIA: People with MND and Healthy Participants who are at least 18 years of age and able to complete a series of questionnaires online.

WHAT HAPPENS IF I SIGN UP?

  • This is a questionnaire only study.
  • All visits will be conducted remotely and online (There will not be any in-person or telephone visits).
  • There will be 5 visits over 12 months (baseline, at 4 weeks, 3 months, 6 months and 12 months).
  • For each visit, you will be provided with a link to access a series of questionnaires aimed at helping us better understand fatigue.
  • It will take you approximately 30-45minutes to complete all questionnaires.

COMPENSATION: There will be no compensation for this study. However, your participation will help develop and implement future studies to quantify the measurement of fatigue, and develop treatments for fatigue in people with motor neuron disease.

IF YOU ARE INTERESTED in participating or learning more, please click this link: https://redcap.partners.org/redcap/surveys/?s=94JPCNPJJF

Principal Investigator: James Berry, MD.

Please contact Zoe Scheier at 617-724-4663 or zscheier@mgh.harvard.edu if you are interested in hearing more details and/or if you would like to know if you are eligible to participate in this study.

Our partners at CCALS and the ALS clinics at MGH and UMass Memorial Medical Center have streamlined a common release form, allowing enhanced communication between between care teams and treatment teams, and increased referrals for high-quality care.

ALS ONE’s dedicated ALS Research Access Nurse, Judith Carey, R.N., located at the MGH Healey Center for ALS, fields over 700 calls annually from patients worldwide to assist them in navigating clinical trials for which they may be eligible. Judy is a tremendously compassionate and knowledgeable resource who connects patients with clinical trial opportunities, either at MGH or elsewhere, which helps the patients, and helps the trials run faster. Patients and families can contact Judy to learn more about clinical trial opportunities by emailing her at: jcarey8@partners.org.

Patient-Focused Solutions

Patients with ALS can’t afford to wait—the typical life expectancy upon diagnosis is 2-5 years

The partnerships forged by ALS ONE bring efficiency and improved coordination to the fields of ALS research and care. The ALS ONE patient network creates a direct pipeline from therapy discovery, to immediate implementation, and to patient services.

ALS ONE funds a team at MGH that is solely focused on designing new clinical trials to maximize efficiency. Through this dedicated team, ALS ONE partners are able to be proactive in designing effective trials and models that lead to greater success when applying for grants and greater efficiency in moving from concept to trial.   

By centralizing the Institutional Review Board (an entity that protects the rights of people in clinical trials), ALS ONE has accelerated the clinical trial process by an average of 7-12 months.

The MGH ALS imaging group is developing novel tools to measure the biological effect of experimental treatments in a way that can reduce the trial population from 400 to 30 patients, and the trial duration from 12 to 3 months. This added efficiency allows ALS ONE to test many more therapies quickly.

pALS and Families: Have you always wanted to learn more about ALS research and influence others to help improve the research process? If so, we encourage you to nominate yourself or a family member to participate in this year’s Virtual ALS Clinical Research Learning Institute (CRLI), which is being held October 2nd & 3rd. The deadline to apply is September 1st so don’t delay.  The  ALS CRLI is an annual two-day program dedicated to educating attendees on clinical research and therapy development and empowering this group to be advocates for ALS clinical research. To learn more about the ALS Clinical Research Learning Institute and what it means to become an ALS Research Ambassador, please view this WEBINAR and, if interested, download the application HERE.

Supporting Clinical Trials

Researchers are faced with myriad challenges that make it difficult to secure necessary data and benchmarks while they work to move novel therapies forward 

ALS ONE is investing in technologies that help ease the research process and better enable patients to meet with clinicians and attend clinical trials in a safe environment. 

The NCRI at MGH serves as the coordination center for ALS ONE clinical trials, as well as the coordination and data management center for the Northeast ALS Consortium, a group of more than 100 academic centers worldwide dedicated to finding new ALS therapies.

The NCRI at MGH serves as the coordination center for ALS ONE clinical trials, as well as the coordination and data management center for the Northeast ALS Consortium, a group of more than 100 academic centers worldwide dedicated to finding new ALS therapies.

ALS ONE funds a transportation program through CCALS to provide rides for patients so they can attend clinical appointments and trials at affiliated institutions.