$100,000

Sean M. Healey and AMG Center for ALS

In December 2022, we proudly awarded a $100,000 grant to The Sean M. Healey and AMG Center for ALS.

This grant of $100,000 to the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital will support their tireless work to discover new ways to diagnose and treat this disease.

More information about the Healey Center

‍

$160,000

ALS Therapy Development Institute

In August 2023,  we awarded $160,000 to support the ALS Therapy Development Institute (ALS TDI) and its mission to end ALS. To help achieve this mission, ALS -TDI is collectively building the world’s largest ALS data bank, allowing them to connect the dots for a cure.

Our grant was directed to expand the ALS Research Collaborative (ARC). This global initiative partners with ALS patients worldwide to gather data about their disease and then shares this data with researchers across the globe through a cloud-based data-sharing platform.

‍

More information about ALS Therapy Development Institute

‍

$225,000

Vaccine and Immunotherapy Center (VIC) at Massachusetts General Hospital

In September 2023, we awarded $225,000 to the Vaccine and Immunotherapy Center to fund the work of Mark C Poznansky, MD, PhD, and Ruxandra F. Sirbulescu, Ph.D. Specifically, we supported the Phase 1 Study of Repeated Infusion of Donor B Cells into Patients Diagnosed with ALS.

The B-cell is a key regulatory cell in the immune system; it acts by producing antibodies, antigen-presenting cells, supporting other mononuclear cells, and contributing to inflammatory pathways directly. This study involves repeated infusion of donor B-cells into patients diagnosed with ALS. Two patients with advanced ALS have to date received such infusions, which, even when repeated, appear to be safe and well-tolerated. The purpose of this study is to establish a more substantial basis for the safety of this procedure in humans and to begin to understand the effects of B-cell dosing on a patient's disease progression and immunologic profile.

More information about the Vaccine & Immunotherapy Center

‍

$100,00

Duke ALS Clinic

In March 2023, we granted $100,000 to support the ambitious researchers at Duke University. We were impressed with their approach and granted them significant funding to support their Replication Of ALS Reversals, or “ROAR” trials which will use the Deep Integrated Genomic Analysis Platform to test treatments against 4 different subclassifications of ALS.

‍More information about Duke ALS Clinic

MitoSense, Inc.

‍

In December 2023, we invested in MitoSense, Inc., a research and development company discovering treatments for neurodegenerative diseases. 

Research efforts for the last 10 years have been devoted to mitochondrial dysfunction in cancer, which led to evaluation of it in neurodegenerative diseases.  This resulted in the discovery and development of Mitochondria Organelle Transplantation (MOT™) technology. They have successfully demonstrated proof of concept for this cellular biotherapy which holds great promise for millions suffering from neurodegenerative diseases such as ALS, Parkinson's, Huntington’s, Alzheimer's, and other neurodegenerative diseases. 

More information about MitoSense, Inc.

$300,000

Houston Methodist Neurological Institute

In December 2023, we funded Dr. Stanley Appel’s study of Extracellular Vesicles Derived From Ex Vivo Expanded Regulatory T Cells Modulate In Vitro and In Vivo Inflammation.  Neuroinflammation is known to exacerbate motor neuron dysfunction, which can ultimately lead to motor neuron death. This study supports the therapeutic potential of expanded Treg Extracellular Vesicles to suppress pro-inflammatory response, support motor neuron life, and slow the progression of human disease.

‍More information about Houston Methodist Neurological Institute

$75,000

ALS Cure Project

In February 2024, we funded ALS Cure Project’s The 3ML: The Multi-Modal Machine Learning Project. The goal of The 3ML Project is to create an objective measure of function and decline in people living with ALS. By using machine learning and AI, analyze differences in bio measures such as MRI’s or proteomics from blood plasma taken at multiple points in time correlated with the patient’s functional rate of decline measured from speech over the same time period. The ultimate goal is to create an immense longitudinal data repository to support the understanding of disease as well as a functional objective measure that can replace the current subjective ALS-Functional Rating Scale which is the standard current measure of decline in ALS patients.

‍More information about ALS Cure Project

$70,000

Duke ALS Clinic

In February 2024, we funded Dr. Richard Belack’s Pilot Trial to Target Biological Mechanisms Connected to the Body's Microbiome and investigate the Safety, Tolerability and Preliminary Efficacy of BL-001 Probiotic in People with ALS. 
The microbial ecosystem of the gut, known as the gut microbiome, is increasingly recognized as a potential mediator of both GI function and key bioactive disease-modulating metabolites in Central Nervous System diseases. More specifically, alteration of microbiome composition and/or defective host-microbiome interface has been suggested to contribute to the pathogenesis of neurological disorders, including ALS.  Conversely, the microbiome has also been identified as a source of key factors contributing to neuroprotection. There is growing evidence that microbiome-mediated metabolites modify neuronal transmission, synaptic plasticity, myelination, neuroinflammation, and complex host behaviors that might drive the pathogenesis of ALS.

‍More information about the Duke ALS Clinic

$250,000

‍Sean M. Healey & AMG Center for ALS

In February 2024, we funded Dr. Matthew Nolan’s Exploring the Effect of Statins to Restore Stathmin-2 Function and Promote Motor Neuron Regeneration. Dr. Matthew Nolan’s research will be conducted under the direction of Clotilde Lagier-Tourenne, MD, PhD, with administrative oversight by Merit Cudkowicz, MD, MSc. Dr. Nolan conducted large-scale drug screens to identify compounds that might target and re-normalize STMN2 levels which contribute to abnormal TDP-43 levels in ALS patients. Surprisingly, one of the drug classes that appeared to show benefit was a class of medications called statins – compounds widely used to lower cholesterol in individuals with heart disease. This remarkable finding suggests there could be a role for statin treatment in people with ALS.

‍More information about Dr. Matthew Nolan

$100,000

The Robert Packard Center, ALS Research at Johns Hopkins

In February 2024, we funded The Packard Center, which supports more than 180 scientists, technicians, and lab personnel who collaborate on projects and openly exchange key resources meant to advance the field. The spark for these fruitful exchanges comes, in part, from regular monthly investigator meetings that are required of Center scientists, as well as an annual research symposium. An active Scientific Director and Scientific Advisory Board featuring many of the world’s top ALS researchers help to set the overall strategic direction of the Packard Center, ensuring that each team works on a crucial piece of the overall puzzle.

More information about The Robert Packard Center

‍

$250,000  

Massachusetts General Hospital Department of Neurology’s Sean M. Healey & AMG Center for ALS

In April 2024, we funded the Massachusetts General Hospital Department of Neurology’s Sean M. Healey & AMG Center for ALS. The gift will support the research project Exploring the Effect of Statins to Restore Stathmin-2 Function and Promote Motor Neuron Regeneration, which is conducted by Matthew Nolan PhD, an early career investigator mentored by Clotilde Lagier-Tourenne, MD, PhD, the Araminta Broch-Healey Endowed Chair in ALS. Dr. Lagier-Tourenne and her collaborators have discovered that normal stathmin-2 (STMN2) function is critical to motor neuron health and outgrowth. Thus, loss of STMN2 appears to contribute to the muscle weakness characterizing ALS. The team has found that in cell models, normalizing STMN2 using gene-targeted therapies helps to rescue motor neurons, which potentially could restore function in people with ALS.

“We at Hop On A Cure are thrilled to support this innovative and accessible potential new therapy,” said Hop On A Cure CEO Nic Shefrin. “We are also excited to champion promising young talent, like Dr. Nolan, who have committed themselves to ALS research.”

“The work that we do at the Healey & AMG Center would not be possible without the support of foundations like Hop On A Cure” said Dr. Lagier-Tourenne. “My team and I are grateful, and excited to be able to move forward with this research that will benefit the lives of people with ALS and their families.”

For more information about the Healey & AMG Center for ALS, click here.