ALS News Today brought you daily coverage of key findings, treatment developments, clinical trials, and other important events related to amyotrophic lateral sclerosis (ALS) throughout 2020, a year marked by the COVID-19 pandemic.
As a reminder of what mattered most to you in 2020, here are the top 10 most-read articles of last year with a brief description of what made them interesting and relevant to the ALS community.
We look forward to reporting more relevant news to patients, family members, and caregivers dealing with ALS throughout 2021.
A team of researchers in Germany found that caffeine and nicotinamide adenine dinucleotide in its oxidized form (NAD+) two powerful antioxidants improved the health of lab-grown motor neurons derived from a mouse model of sporadic ALS.
These benefits, seen in cells derived from mice either in a progressive or a stable disease state, were likely associated with a reduction in oxidative stress, a known contributor to sporadic ALS.
Of note, motor neurons, the specialized nerve cells that control voluntary movement, are progressively lost in people with ALS. Oxidative stress is an imbalance between the natural production of potentially harmful reactive oxygen species and the ability of cells to detoxify them with antioxidant agents.
In an April story, we reported AB Sciences plans to launch a Phase 3 clinical trial (NCT03127267) testing its experimental oral therapy masitinib as an add-on treatment for people with ALS, after the U.S. Food and Drug Administration (FDA) cleared its request for this study.
Masitinib is designed to block the activity of multiple cell types involved in the inflammatory and neurodegenerative processes marking ALS.
The study aims to assess whether add-on treatment with masitinib is superior to placebo at slowing functional decline in up to 495 ALS patients diagnosed in the past two years. Participants functional abilities will be assessed through the ALS functional rating scale-revised (ALSFRS-R). Both masitinib and placebo will be given in combination with Sanofis Rilutek (riluzole), an approved ALS medication.
The trial is currently recruiting patients at a single U.S. clinical site(Johns Hopkins in Maryland), but another site in Ulm, Germany, is expected to open shortly. Should study findings be positive, they are expected to support future requests for regulatory approval of masitinib as an ALS treatment.
Using different mouse models of ALS, a team of researchers in the U.S. discovered a self-destructive mechanism in mitochondria the cells powerhouses that may be one of the first triggers of motor neuron degeneration in ALS.
This mitochondrial suicide was found only in the upper motor neurons those that send messages from the brain to the spinal cord, and whose degeneration is thought to be an early disease event of ALS mice, and before any signs or symptoms of the disease were evident.
These findings suggest that currently available therapies targeting mitochondrial degeneration may help to stop neurodegeneration in ALS, supporting further research in this area.
In July, BrainStorm Cell Therapeutics announced that all ALS patients enrolled in a pivotal Phase 3 clinical trial (NCT03280056) testing NurOwn, its investigational cell-based therapy, had completed dosing.
NurOwn involves expanding and maturing mesenchymal stem cells (MSCs) collected from a patients own bone marrow into cells that produce high levels of molecules promoting nerve cell growth and survival. MSCs are stem cells that can generate a variety of other cell types.
The mature cells called MSC-NTF cells are then injected into the patients spinal canal to promote and support nerve cell repair.
In the U.S.-based trial, 189 patients with rapidly progressing ALS were randomly assigned to either a total of three injections of either NurOwn, or a placebo, given directly into the spinal canal every other month.
The studys main goal was to assess the therapys safety, and whether treatment was superior to placebo at slowing disease progression as measured by the ALSFRS-R at seven months following the first dose.
A couple of months earlier, we reported the results of a preclinical study suggesting that NurOwn may not only boost nerve cell protection and repair, but also suppress the damaging immune responses that contribute to ALS progression by promoting a shift toward an anti-inflammatory state.
BrainStorm researchers found that growing healthy B-cells and T-cells immune cells known to be involved in ALS in the lab with NurOwn suppressed the growth of pro-inflammatory cell subsets, and lowered the levels of pro-inflammatory molecules. At the same time, the therapy increased the numbers of immunosuppressive cell subsets and the levels of a major anti-inflammatory molecule.
BrainStorm announced in June that patient dosing in its Phase 3 trial evaluating NurOwn in people with ALS remained on track, despite occasional treatment scheduling changes due to the COVID-19 pandemic.
The company attributed the trials successful advancement during the pandemic to coordination among its six U.S. clinical sites, support and guidance from the FDA, and the fact that its main goal based on the ALSFRS-R could be assessed by phone.
Top-line data were shared before the years end, as anticipated by BrainStorm, and are under review by the FDA.
In April, ALS News Today reported onSeneca Biopharmas plans to launch a Phase 3 clinical trial to assess the safety and effectiveness of NSI-566, its leading stem cell treatment candidate, in adults with ALS.
The decision was supported by previous positive data from a Phase 1 (NCT01348451) and Phase 2 (NCT01730716) clinical trial and a meeting with the FDA that provided guidance on how to best design and conduct the upcoming late-stage trial.
NSI-566 treatment involves the injection of fetal spinal cord stem cells into a patients spinal cord, where they mature into nerve cells that surround and support motor neurons. These mature cells also produce certain molecules that promote motor neuron growth and survival.
Results from the previous studies confirmed NSI-566s safety, and suggested that the therapy may help to prevent further functional decline in ALS patients, when compared with data from other ALS trials.
A small study in Italy suggested that creatinine kinase a marker of muscle damage could be used as a biomarker to predict the rate of disease progression in people with ALS.
By analyzing this enzyme in 126 ALS patients, the researchers found that creatinine kinase levels were significantly higher in people with slow progressing disease compared with those with fast progressing disease, and that these differences were sustained over time.
Further analyses in mouse models of ALS confirmed these findings, and suggested that the slow progression was associated with greater muscle mass and a better ability to counter disease mechanisms for longer periods.
Elevated creatinine kinase blood levels also seemed to be specific to ALS among neurodegenerative diseases, suggesting that the muscle may be a therapeutic target in ALS.
In January, we reported that a Phase 1/2a clinical trial (NCT03482050) testing AstroRx, Kadimastems investigational cell therapy, had completed dosing a second group ofALS patients.
AstroRx delivers healthy, mature astrocytes derived from human embryonic stem cells to a patients spinal cord to compensate for diseased astrocytes and to prevent motor neuron loss. Astrocytes are star-shaped cells that normally support and protect nerve cells, but are abnormal in ALS.
Data from the first group of patients given the lowest therapy dose showed that the treatment was safe and slowed the rate of disease progression over the first three to four months following dosing. Results from the second group (given a higher dose) went on toconfirm these promising three-month findings of a single treatment.
Our most-read article of 2020 concerned the discovery that an abnormal uptake of metals from chromium to zinc during childhood is associated with ALS in adults.
By analyzing teeth samples from 36 ALS patients and 31 unaffected people with a powerful technology, the researchers were able to establish and assess differences in temporal profiles of metal exposure. They found that ALS patients had greater exposure to several metals at various developmental stages, starting as early as birth.
These findings were confirmed in mouse models of ALS, both in their teeth and in their brains, suggesting that abnormal metal metabolism may contribute to several molecular changes that could increase the susceptibility of motor neurons to premature damage.
While deficiencies and excess of essential elements and toxic metals are known to contribute to ALS, researchers were now able to provide an idea of when these metabolic abnormalities start. The results also suggested that metal metabolism could be a viable therapeutic target to prevent or halt ALS.
At ALS News Today, we hope these stories and our reporting throughout 2021 help to better inform and improve the lives of everyone affected by ALS.
We wish all our readers a happy 2021.
Marta Figueiredo holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from the University of Lisbon, Portugal. She is currently finishing her PhD in Biomedical Sciences at the University of Lisbon, where she focused her research on the role of several signalling pathways in thymus and parathyroid glands embryonic development.
Total Posts: 45
Ins holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Cincias e Tecnologias and Instituto Gulbenkian de Cincia. Ins currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.
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Top 10 ALS Stories of 2020 - ALS News Today
- [PDF] Stem Cell Therapy Market Size Strong Revenue and Generated Opportunities UNLV The Rebel Yell - UNLV The Rebel Yell - August 31st, 2021
- Mike Tysons weird stem cell treatment has helped transform incredible body at 55 - Daily Star - August 31st, 2021
- Acute Monocytic Leukemia: Causes, Symptoms, and Treatment - Healthline - August 31st, 2021
- UC Davis and the School of Medicine set new records in research funding - UC Davis Health - August 31st, 2021
- NCCN Issues Updated Guidelines Regarding Third COVID-19 Vaccine Booster for Those With Cancer - Cancer Network - August 31st, 2021
- DHEC Supports Awareness and Education during National Sickle Cell Awareness Month - SCDHEC - August 31st, 2021
- The emerging roles of the gut microbiome in allogeneic hematopoietic stem cell transplantation - DocWire News - August 31st, 2021
- Global Cryopreservation Equipment Market Report 2021-2028 - Growing Acceptance for Regenerative Medicine & Increasing Needs of Biobanking... - August 31st, 2021
- Operational Highlights and Financial Results for the Year Ended June 30, 2021 - GlobeNewswire - August 31st, 2021
- Event to benefit 2 families with kids fighting cancer - Youngstown Vindicator - August 31st, 2021
- Cryo-Cell International Announces Uplisting to The Nasdaq Capital Market - GlobeNewswire - August 31st, 2021
- Incyte and MorphoSys Announce the European Commission Approval of Minjuvi (tafasitamab) in Combination With Lenalidomide for the Treatment of Adults... - August 31st, 2021
- Thomas Smeenk with an update on the FDA Phase II clinical trial program of the Hemostemix blood-derived, stem cell therapeutics product (ACP-01) -... - August 31st, 2021
- 2021 Pipeline Insight Report on Myocardial Infarction - Featuring AstraZeneca, Mesoblast and Boehringer Ingelheim Among Others -... - August 31st, 2021
- Mini brains grown in the lab sprout primitive eyes - Freethink - August 31st, 2021
- BeiGene and EUSA Pharma Announce China NMPA Approval of QARZIBA (Dinutuximab Beta) for Patients With High-Risk Neuroblastoma - Business Wire - August 18th, 2021
- Study Calls for COVID-19 Vaccination in Patients With Cancer to Enable Optimal Treatment Delivery During Pandemic - OncLive - August 18th, 2021
- Treatment of AML in Older Patients Almost At 'The Holy Grail' - Curetoday.com - August 18th, 2021
- Animal Stem Cell Therapy Market Identify Key Drivers, Trends, Latest Innovations, Business Senario, Demand With Outlook by 2026 - The Market Writeuo -... - August 18th, 2021
- 8 Tips for Coping With a Diagnosis of PPMS - Everyday Health - August 18th, 2021
- More positive results from CRISPR trial for sickle cell and thalassaemia - BioNews - August 18th, 2021
- Belumosudil Granted Full Approval for Treatment of Chronic GVHD by FDA - Cancer Network - July 22nd, 2021
- Dr. Martin on the Role of Maintenance Therapy With Rituximab in MCL July 15th 2021 Peter - OncLive - July 22nd, 2021
- FDA tells Magenta to pump the brakes on blood cancer trial before it starts to develop new dosing test - FierceBiotech - July 22nd, 2021
- Stemson Therapeutics Secures $15M Series A Funding to Cure Hair Loss - Business Wire - July 22nd, 2021
- Events planned to support 5-year-old's cancer treatments - The Augusta Chronicle - July 22nd, 2021
- HER2-Specific CAR T Cells Induce Early Efficacy Without Dose-Limiting Toxicities in Pediatric CNS Tumors - OncLive - July 22nd, 2021
- A mother's love: Two years after devastating crash, Abby Roby has hope - London Free Press (Blogs) - July 22nd, 2021
- Roswell Park Team Shows Dendritic-Cell Vaccines Can Be Paired With Standard Therapy for Breast Cancer - Newswise - July 22nd, 2021
- Introducing the 3D bioprinted neural tissues with the potential to 'cure' human paralysis - 3D Printing Industry - July 22nd, 2021
- Investing in stem cells, the building blocks of the body - MoneyWeek - June 25th, 2021
- Discrepancies Between Real-World Patterns of Care and Recommendations Based on Clinical Trials Exist in MCL - OncLive - June 25th, 2021
- Catalent to Acquire RheinCell Therapeutics, Strengthening a Path Towards Industrialization of Induced Pluripotent Stem Cell-based Therapies - PR Web - June 25th, 2021
- Disparities in Utilization of Autologous Stem Cell Transplantation as Consolidative Therapy for Multiple Myeloma: A Single Institution Retrospective... - June 25th, 2021
- Vita Therapeutics Raises $32 Million in Oversubscribed Series A Financing Led by Cambrian Biopharma to Advance the Development of Therapies to Treat... - June 25th, 2021
- Updates in the treatment of peripheral T-cell lymphomas | JEP - Dove Medical Press - June 25th, 2021
- Novel Tafasitamab Combination Finds a Role in Second-line DLBCL Treatment - Targeted Oncology - June 25th, 2021
- Global Nerve Repair and Regeneration Devices Market to Reach $11. 8 Billion by 2026 - GlobeNewswire - June 25th, 2021
- 5 potential benefits of exosome treatment - AZ Big Media - June 8th, 2021
- Orion and the Blood Service launch collaboration to develop new CAR T-cell cancer therapy - Yahoo Finance Australia - June 8th, 2021
- Improvements in the Treatment of Patients With MCL Over the Past Decade - Oncology Learning Network - June 8th, 2021
- Novo Nordisk partners with Heartseed on heart failure cell therapy - PMLiVE - June 8th, 2021
- Citius Pharmaceuticals to be Added to Russell 2000 Index - BioSpace - June 8th, 2021
- IN8bio Completes Treatment of First Cohort in Phase 1 Clinical Trial with Gamma Delta T-Cell Therapy in Patients with Newly Diagnosed Glioblastoma... - June 8th, 2021
- Regenerative medicine: moving next-gen treatments from lab to clinic - Pharmaceutical Technology - June 8th, 2021
- Global $30+ Billion Cell Therapy Bioprocessing Market to 2028: Market Opportunities in Automated Procedures to Produce Cell Therapies - PRNewswire - June 8th, 2021
- Global Stem Cell & Regenerative Medicine Market Report Forecast to 2027 3M Group Novartis AG Integra Gene Therapies The Manomet Current - The... - June 8th, 2021
- Novel CAR-T Cell Therapy Produces Early and Deep Responses in Certain Patients with Multiple Myeloma - Curetoday.com - June 8th, 2021
- BioRestorative Therapies to Present at the Emerging Growth Conference on June 9, 2021 - StreetInsider.com - June 8th, 2021
- Innovent Biologics and IASO Biotherapeutics to Present Updated Data from its Anti-BCMA CAR-T Therapy in Relapsed/Refractory Multiple Myeloma at... - June 8th, 2021
- High Rate of Response Noted in MCL and CLL With Cirmtuzumab Plus Ibrutinib - Cancer Network - June 8th, 2021
- Dr. Ghosh on the Role of Off-the-Shelf CAR T-Cell Therapy in Myeloma June - OncLive - June 8th, 2021
- Triplet Combo of Polatuzumab Vedotin, Rituximab and Lenalidomide Safe, Effective to Treat Relapsed/Refractory DLBCL - Cancer Network - June 8th, 2021
- Income, Education Affect Treatment and Survival in MDS - DocWire News - June 8th, 2021
- Polatuzumab With Rituximab and Lenalidomide Shown Safe and Effective for Relapsed/Refractory DLBCL - Targeted Oncology - June 8th, 2021
- Early Promise Observed With Lisaftoclax in Relapsed/Refractory CLL and Other Hematologic Malignancies - Targeted Oncology - June 8th, 2021
- Mustang Bio to Host Key Opinion Leader Webinar on MB-106 CD20-Targeted CAR T for - GlobeNewswire - June 8th, 2021
- FDA Lifts Hold on bluebird bio's Sickle Cell and Beta-Thalassemia Gene Therapies - BioSpace - June 8th, 2021
- Abecma Continues to Improve Survival in Heavily Pretreated Patients With Multiple Myeloma - Curetoday.com - June 8th, 2021
- HSCT-Sparing, Chemotherapy-Free Treatment of Ph+ ALL With Ponatinib/Blinatumomab Leads to 100% CR Rate - Cancer Network - June 8th, 2021
- Fate Therapeutics Highlights Positive Interim Data from its Phase 1 Study of FT516 in Combination with - GlobeNewswire - June 8th, 2021
- Action Taken by Governor Phil Scott on Legislation - June 7, 2021 - Office of Governor Phil Scott - June 8th, 2021
- Cancer research: New advances and innovations - Medical News Today - June 8th, 2021
- Naturally Treat & Heal The Cause of Pain with Regenerative Therapies - The Mountaineer - May 15th, 2021
- CAR T-Cell Therapy UCARTCS1A Shows Early Activity in Relapsed/Refractory Myeloma - OncLive - May 15th, 2021
- Rinri Therapeutics Secures Innovate UK Funding Grant for 3.2m Project to Advance its Novel Cell-Based Therapy to Restore Hearing Loss - PRNewswire - May 15th, 2021
- With $52 Million Series A, Appia Bio Anticipates a Bright Future in Cell Therapy - BioSpace - May 15th, 2021
- Genenta Phase I/II Glioblastoma Data at ASGCT Show Temferon Delivered Tumor-Focused Interferon ExpressionData presented at the 2021 American Society... - May 15th, 2021
- Safety and Efficacy of Consolidation Therapy with Ipilimumab Plus Nivolumab after Autologous Stem Cell Transplantation - DocWire News - May 15th, 2021
- Fascination with stem cell sensation leads to Fulbright scholarship - Monash University - May 15th, 2021
- Omeros' Narsoplimab Pivotal Trial Data to Be Shared as an Oral Presentation at the European Hematology Association Congress - Business Wire - May 15th, 2021
- Munshi Explains Staging, Prognosis, and Treatment for a Patient With Acute Graft-vs-Host-Disease - Targeted Oncology - May 15th, 2021
- KD-PACE Salvage Therapy for Aggressive Relapsed Refractory Multiple Myeloma, Plasma Cell Leukemia and Extramedullary Myeloma - DocWire News - May 15th, 2021
- Icy Microneedle Patch Delivers Cell Therapy, Then Melts - Freethink - May 15th, 2021
- FDA Responds to Creative Medical Technology Holdings Regarding Its ImmCelz IND for Stroke Treatment - BioSpace - May 15th, 2021
- Global Stem Cell and Platelet Rich Plasma (PRP) Alopecia Therapies Market 2021 Type and End-use Industry 2026 Orange County Hair Restoration Center,... - May 15th, 2021
- Allogeneic Mesenchymal Stem Cell Segment Is Expected To Lead In the Global Rheumatoid Arthritis Stem Cell Therapy Market over the Forecast Period,... - May 15th, 2021
- Advanced Therapy Medicinal Products Market Size Worth $21.2 Billion By 2028: Grand View Research, Inc. - PRNewswire - May 15th, 2021
- New connection between metabolism and red blood cell development - Harvard Gazette - May 15th, 2021
- Heart attack recovery aided by injecting heart muscle cells that overexpress cyclin D2 - The Mix - May 15th, 2021