Boston- ALS Finding a Cure® (ALSFAC) has awarded a $400,000 grant to a team of researchers from Massachusetts General Hospital (MGH), a Boston based private entity Nemdx Inc. (Nemdx), and the International Centre for Genetic Engineering and Biotechnology (ICGEB), Trieste, Italy, for their project “Developing a Comprehensive Blood Test for Sporadic and Familial ALS.” Dr. Ghazaleh Sadri-Vakili, Director of NeuroEpigenetics Laboratory at the Mass General Institute for Neurodegenerative Diseases and Associate Investigator at the Neurological Clinical Research Institute serves as Co- Principal Investigator.
The project will aim to establish a panel of blood biomarkers as tools to uniquely differentiate familiar and sporadic ALS patients from healthy individuals or patients with other neurodegenerative diseases. To do so, the team will conduct a series of tests to collect and compare blood samples against control groups to assess if any of the proteins in the panel may provide an early indication of disease development or progression.
“I am delighted to have received this funding from ALSFAC to begin this exciting and critical work with our colleagues to establish an ALS-specific panel of biomarkers, an urgent and unmet need in the field” said Dr. Sadri-Vakili. “Nemdx’s platform is novel and may drastically change the biomarker landscape.”
The team, consisting of Dr. Sadri-Vakili, Professor Emanuele Buratti of the International Centre for Genetic Engineering & Biotechnology (ICGEB) in Trieste, Italy, and Dr Ian Thrippleton and Vito Levi D’Ancona of Nemdx Inc., outlined a need for specific molecular biomarkers that can detect and indicate the specific ALS disease progression. This specific panel of biomarkers would allow for the classification of disease subtypes in people living with ALS, and the ability to assess early target engagement of therapeutics in clinical trials.
“We are looking forward to working closely with MGH and NemDx to help build a robust product pipeline of diagnostics specifically aimed at ALS patients” said Prof. Emanuele Buratti. “We expect that the results of this project will improve patient stratification and individually tailored treatment pathways. Most importantly, this is also an important step in the ICGEB mission to drive global access to technologies in our member countries.”
The project will span across three sites; the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, the International Centre for Genetic Engineering & Biotechnology, and Nemdx. The success of this project would allow for early diagnosis and early therapeutic intervention. The availability of panels will also assist as a prognostic tool for clinical trials.
“The Nemdx collaboration with researchers from Massachusetts General Hospital and ICGEB represents a key recognition milestone in our attempt to drastically change the biomarker landscape” said Vito Levi D’Ancona, of Nemdx Inc. “Our joint pursuit to establish an ALS-specific panel of biomarkers, supported by ALSFAC’s generous grant, marks a significant stride towards addressing the urgent need of the ALS community. The innovative approach of Nemdx’s tools platform holds the potential to revolutionize the landscape of biomarker discovery. Together, we aim to unlock early diagnostic insights, drive therapeutic advancements, and ultimately bring hope to those affected by ALS.”
For more information regarding the Healey & AMG Center for ALS, please visit our website.
Background on ALS
Amyotrophic lateral sclerosis, ALS, is the most prevalent adult-onset progressive motor neuron disease, affecting approximately 30,000 people in the U.S. and an estimated 500,000 people worldwide. ALS causes the progressive degeneration of motor neurons, resulting in progressive muscle weakness and atrophy. There are currently few FDA therapies approved for treating ALS—riluzole, edaravone (IV and oral formulation), Relyvrio, and Qalsody. Nuedexta is also used for the symptomatic treatment of pseudobulbar affect (PBA) in people with ALS.
About the Sean M. Healey & AMG Center for ALS at Mass General
At the Sean M. Healey & AMG Center for ALS at Mass General, we are on a quest to discover life-saving therapies for all individuals affected by ALS. Launched in November 2018, the Healey Center leverages a global network of scientists, physicians, nurses, caregivers, people with ALS and families working together to accelerate the pace of ALS therapy discovery and development.
Under the leadership of Merit Cudkowicz, MD and a Science Advisory Council of international experts, we are reimagining how to develop and test the most effective therapies to treat the disease, identify cures and, ultimately, prevent it.
The key to our success is our tightly integrated research and clinical efforts, encouraging opportunities to bring the challenges our patients face every day into our laboratories, focusing investigations on finding solutions that will make a meaningful difference to our patients without delay. Our collaborative efforts are designing more efficient and effective clinical trials while broadening access to these trials for people with ALS.
Nemdx Inc., based in Boston, MA with a state-of-the-art wet lab in Sandwich, UK, pioneers innovative solutions for probing the dynamic interaction between proteins through the study of their activity and modifications in disease progression.
Nemdx’s proprietary, patented tools offer swift, precise, and ultra-sensitive detection, driving early disease classification and targeted interventions.
Our recent progress includes diverse tools ushering in a new era of potential discoveries in the study neurodegenerative disease such as ALS.
The ICGEB is a unique intergovernmental organisation initially established as a special project of UNIDO. Autonomous since 1994, it runs over 45 state-of-the-art laboratories, in Trieste, Italy, New Delhi, India and Cape Town, South Africa and forms an interactive network with almost 70 Member States, with operations aligned to those of the United Nations System. It plays a key role in Biotechnology promoting Research excellence, Training, and Technology Transfer to industry, to contribute in concrete terms to sustainable global development.
The sole purpose of the ALS Finding a Cure® initiative is funding research to find a cure. Our goal is to bring together some of the top minds in ALS to develop novel biomarkers and diagnostics, provide resources to help advance new therapies to the clinic, accelerate innovation in clinical trial design, and expand access to shared research resources. ALS Finding a Cure® is inspired by and is a tribute to Christie Rizzuto, who was diagnosed with ALS in 2009 at the age of 41. It is Christie, and the many others like her, who drive our work.