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
- Stem Cell Therapy Global Market Report 2021: COVID-19 Growth And Change To 2030 - Yahoo Finance - May 3rd, 2021
- Stem cell therapy for COPD: Cost, effectiveness, and more - Medical News Today - May 3rd, 2021
- Thomas Smeenk on Hemostemix's autologous stem cell therapy technology and why some call it 'the fountain of youth' - InvestorIntel - May 3rd, 2021
- Mesoblast says its stem cell treatment saved lives of severely ill COVID patients - Stockhead - May 3rd, 2021
- Antiviral T cells protected, viable as off-the-shelf treatment for painful complication stem cells - Microbioz India - May 3rd, 2021
- Investigating CAR T-Cell Therapy for Use in Different Disease Types - Targeted Oncology - May 3rd, 2021
- Should scientists be allowed to grow human embryos in a dish beyond 14 days? Is it scientifically important or morally wrong? - USA TODAY - May 3rd, 2021
- Hope, hype and exploitation the wild history of stem cell science - ABC News - May 3rd, 2021
- Brave New World is being reinvented with synthetic embryosand the right reasons - SYFY WIRE - May 3rd, 2021
- Stem Cell Therapy Market Trends Evaluation 2021 By Leading Players Updates, Consumer-Demand, Key Strategies, Consumption, Industry Development, Market... - May 3rd, 2021
- STEM NOLA Teams with Obatala Science & CellSpring to Expose Students to Careers and Medical Research at Biotech Startups - PRNewswire - May 3rd, 2021
- Breakthrough gene therapies in sight | UNC-Chapel Hill - UNC Chapell Hill - May 3rd, 2021
- FDA's Abecma Approval a 'Great Blessing' for Patients with Multiple Myeloma - Curetoday.com - May 3rd, 2021
- Enhanced inhibition of tumor growth using TRAIL-overexpressing adipose-derived stem cells in combination with the chemotherapeutic agent CPT-11 in... - May 3rd, 2021
- Global Stem Cell Therapy Market 2020-2027 Trends, industry vertical, along with the geography, delivering valuable insights | Reportspedia Clark... - May 3rd, 2021
- Rheumatoid Arthritis Stem Cell Therapy Market Report 2021 by Global Key Players, Types, Applications, Countries, Size, Forecast to 2027 Clark County... - May 3rd, 2021
- CRISPRoff Reversible Epigenome Editing Method Could Enable Safer, More Precise Therapeutics - GenomeWeb - May 3rd, 2021
- Cell Therapy Market To Expand At An expeditious Growth Rate From 2021-2027 | Thermo Fisher Scientific, Nanofiber Solutions, Advanced Biomatrix,... - May 3rd, 2021
- FTC continues to crack down on companies peddling fake COVID treatments and cures - HamletHub - May 3rd, 2021
- ThermoGenesis : The History of Cell and Gene Therapy - marketscreener.com - April 18th, 2021
- Russell Health Honored in Global Business Leaders Magazine's '20 Leading Companies of the Year 2021' - PRNewswire - April 18th, 2021
- Durable B-ALL Control With Allogeneic Transplant After CAR T-Cell Therapy - Cancer Therapy Advisor - April 18th, 2021
- CRISPR lauds easy scale-out of cell therapy - BioProcess Insider - BioProcess Insider - April 18th, 2021
- NeoProgen, Inc. Receives First Granted Patent for Method to Treat Cardiac Conditions using Neonatal Heart-derived Medicinal Signaling Cells (nMSCs)... - April 18th, 2021
- CRISPR gene therapy for sickle cell disease approved by the FDA - BioNews - April 18th, 2021
- Man's heart healed by stem cell therapy and love of an old flame - Leeds Live - April 18th, 2021
- Autologous Stem Cell Based Therapies Market Professional Report 2021 Witness Robust Expansion by 2026 SoccerNurds - SoccerNurds - April 18th, 2021
- Stem Cell Therapy Market Analysis 2021: Size, Share, Sales, Growth, Revenue, Type, Application & Forecast To 2027 SoccerNurds - SoccerNurds - April 18th, 2021
- Mastitis therapy explored | AG | kmaland.com - KMAland - April 18th, 2021
- CAR T-Cell Therapy Enters the Arena in Multiple Myeloma With Ongoing Research Ahead - OncLive - April 18th, 2021
- Leukemia Cutis: Symptoms and Treatment - Healthline - April 18th, 2021
- Stem Cell Therapy Market is Set to Witness Impressive Growth | Top Players Osiris Therapeutics, Inc. (US), MEDIPOST Co., Ltd. KSU | The Sentinel... - April 18th, 2021
- Global Stem Cell Therapy Market 2020 SWOT Analysis, Growth Rate, and Market Share By Type, Application and Global Forecast 2027 - Los Hijos de la... - April 18th, 2021
- Stem Cell Therapy Market Potential Growth, Size, Share, Demand and Analysis of Key Players Research Forecasts to 2025 - The Courier - March 8th, 2021
- Animal Stem Cell Therapy Market Size, Industry Analysis, Growth Factors, Trends, and Regional Forecast to 2027 - The Courier - March 8th, 2021
- Pericardial Injection Effective, Less Invasive Way to Get Regenerative Therapies to Heart - NC State News - March 8th, 2021
- Biomarkers Help Predict the Role of Chemotherapy in Biologic Aging - OncLive - March 8th, 2021
- Glial Cell Discovery Could Point the Way to Motor Neuron Disease Therapies - Technology Networks - March 8th, 2021
- Mouth Sores from Chemo: Symptoms, Causes, and Treatments - Healthline - February 19th, 2021
- ThermoGenesis Holdings Begins a Year of Celebration of its 35th Anniversary in the Cell Banking and Cell Therapy Industry - PRNewswire - February 19th, 2021
- Meet the women hoping to recruit more stem cells donors from Black communities - CTV News - February 19th, 2021
- CAR T-Cell Therapy Carves Out a Role in Multiple Myeloma - OncLive - February 19th, 2021
- AI project to power the future of stem cell manufacturing - TechCentral.ie - February 17th, 2021
- Promising Results for Stem Cell Treatment of Degenerative ... - February 14th, 2021
- Stem Cell Therapy for Heart Disease - Cleveland Clinic - February 14th, 2021
- Stem Cell Therapy for Stroke Recovery | University of ... - February 14th, 2021
- Stem Cell Treatment: Future of Modern Medicine | Science Times - February 14th, 2021
- Researchers may have found a miracle cure for baldness - BGR - February 14th, 2021
- Types of leukemia: Prevalence, treatment options, and prognosis - Medical News Today - February 14th, 2021
- Jasper Therapeutics Announces Launch of New Clinical Trial with National Heart, Lung, and Blood Institute to Evaluate JSP191 in Sickle Cell Disease -... - February 14th, 2021
- Global Stem Cell Therapy Market Set to Reach USD 214.5 Million by 2024 - The Courier - February 14th, 2021
- Keep it Flowing: Combating COVID-19 Blood Shortages in Cancer Treatment - Curetoday.com - February 14th, 2021
- Network-based screen in iPSC-derived cells reveals therapeutic candidate for heart valve disease - Science - February 14th, 2021
- AlloVir Research Presented at the 2021 Transplantation & Cellular Therapy Meeting Digital Experience - Business Wire - February 14th, 2021
- Orchard Therapeutics talks the benefits of HSC therapy - BioProcess Insider - February 14th, 2021
- Protein Discovery in the Development of New Hearing Hair Cells Could Lead to Treatments for Hearing Loss - SciTechDaily - February 7th, 2021
- Stem cells efficacy confirmed in treating ototoxic hearing loss - Korea Biomedical Review - February 4th, 2021
- Groundbreaking Trial Shows Umbilical-Cord Derived Mesenchymal Stem Cell Infusions Save Lives of Sickest COVID-19 Patients - HospiMedica - January 22nd, 2021
- Massive Growth in Canine Stem Cell Therapy Market Set to Witness Huge Growth by 2027| VETSTEM BIOPHARMA, Cell Therapy Sciences, Regeneus, Aratana... - January 22nd, 2021
- Autologous stem cell transplantation versus bortezomib for the first line treatment of systemic light chain amyloidosis in the UK - DocWire News - January 22nd, 2021
- Promising Steps Toward Retinal Cell Transplants to Fight Blindness - HealthDay News - January 22nd, 2021
- Hemostemix Announces the Bread Contract with the Department of Foreign Affairs, Trade & Development Canada - InvestorIntel - January 22nd, 2021
- Trending News on Targeted Oncology, Week of January 22, 2021 - Targeted Oncology - January 22nd, 2021
- Stem Cell Therapy Market Size, Growth Opportunities, Trends, Key Players and Forecast to 2027 - The Courier - January 22nd, 2021
- Time To Book Profit In Fate Therapeutics Stock After A 5x Rally? - Forbes - January 22nd, 2021
- Reid Merryman, MD, on Study Findings Investigating Prognostic Value of ctDNA for Patients with DLBCL - Cancer Network - January 22nd, 2021
- Co-founder Thomas Smeenk on the benefits of Hemostemix autologous stem cell therapy - InvestorIntel - January 12th, 2021
- Covid-19 Impact On Stem Cell Therapy Market 2020 Huge Potential in Future Growth by 2027 | Magellan, Medipost Co., Ltd, Osiris Therapeutics, Inc.,... - January 12th, 2021
- Adipose Derived Stem Cell Therapy Market Analysis and Forecast, 2020-2026 Coherent Market Insights | BioRestorative Therapies, Inc., Celltex... - January 12th, 2021
- New COVID-19 Treatment Uses Stem Cells - Freethink - January 12th, 2021
- Creative Medical Technology Holdings Files Investigational New Drug Application (IND) with FDA for Treatment of Stroke using ImmCelz Regenerative... - January 12th, 2021
- Global Cell Freezing Media for Cell Therapy Market 2020 Dynamics Analysis, Size Witness Growth Acceleration During 2025 - KSU | The Sentinel Newspaper - January 12th, 2021
- Gamida Cell to Present Full Data from Phase 3 Study of Omidubicel at TCT, the Combined Transplantation and Cellular Therapy Meetings of ASTCT and... - January 12th, 2021
- Cate Dyer of StemExpress is Named Businesswoman of the Year! - PRNewswire - January 12th, 2021
- Adocia Files Patent on a Hydrogel Scaffold for Cell Therapy in the Treatment of Type 1 Diabetes - Business Wire - January 12th, 2021
- Gene Therapy Market Estimated to Record Highest CAGR by 2024 - KSU | The Sentinel Newspaper - January 12th, 2021
- Stem cell treatment corrects skull shape and restores brain function in mouse model of childhood disorder - National Institutes of Health - January 9th, 2021
- Doctors Make Medical Breakthrough In Treating Severe Cases Of COVID - CBS New York - January 9th, 2021
- Global Stem Cell Therapy Market Report 2020: Market to Recover and Reach $14.76 Billion in 2023 at a CAGR of 19.62% - Forecast to 2030 - Yahoo Finance - January 9th, 2021
- Engineered stem cells that evade immune detection could boost cell therapy and I-O - FierceBiotech - January 9th, 2021