Throughout 2019, ALS News Today brought you daily coverage of key findings, treatment developments, clinical trials, and other events related to amyotrophic lateral sclerosis (ALS).
As a reminder of what mattered most to you in 2019, 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 news to patients, family members, and caregivers dealing with ALS during 2020.
No. 10 Experimental Gene Therapy Successfully Silences Key ALS Gene C9orf72, Preclinical Studies Show
A tale of two preclinical studies showed that a gene therapy candidate targeting a key ALS mutation in the C9orf72 gene was able to lessen the buildup of toxic RNA clumps and reduce the activity of the mutated gene in cells collected from a patient with frontotemporal dementia (FTD) and a mouse model of ALS.
Developed by uniQure,the therapy is designed to silence thedisease-causing gene. It works by delivering microRNAs (miRNAs) RNA molecules that regulate gene expression that target the mutated C9orf72s RNA for degradation. The results supported the continuation of uniQures gene therapy program in ALS and FTD, the company said.
No. 9 Altered Gut Microbiota in ALS Patients Could Drive Digestive Problems, Study Suggests
In the summer, a small study discovered an altered composition of gut microbes in people with ALS, which could drive digestive problems in those with the disease.
Using genetics, a research team in China found that fecal samples of people with ALS have an increase in harmful microbes of the phylum Firmicutes and a decrease in beneficial microorganisms called Bacteroidetes. The resulting poor gastrointestinal health may lead to a decline in the guts digestion and metabolism functions.
No. 8 Ibudilast-Rilutek Therapy Combo Closer to U.S. Patent for ALS and Other Neurodegenerative Diseases
At the beginning of the year, U.S. biopharmaceutical MediciNova received a notice of allowance stating that its request for a patent covering a combination of its investigational therapy ibudilast (MN-166) plus Rilutek (riluzole) was being consideredby the U.S. Patent and Trademark Office.
Ibudilast is a small molecule that reduces the activity of immune cells in the brain while supporting the growth of motor neurons, those lost in people with ALS. In a Phase 2 trial (NCT02238626), the treatment was found to work well in combination with Sanofis approved therapy Rilutek, improving patients functional activity, quality of life, and muscle strength.
The notice of allowance was the final step toward patent registration. Such registration will provide patent protection until November 2035 to the combination, for use in treating ALS and other neurodegenerative diseases.
No. 7 MediciNova Launches Phase 2b/3 Trial of Ibudilast for ALS
In June, MediciNova launched a Phase 2b/3 clinical trial to continue studying ibudilast as an add-on therapy to Rilutek. The multicenter, double-blind study (NCT04057898) will recruit approximately 230 participants, who will be randomly selected to receive either ibudilast plus Rilutek or a placebo plus Rilutek for 12 months.
The trials main goal is to study ibudilasts impact on ALS progression and functional disability by determining changes in the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score between the beginning and end of the treatment period. Secondary objectives include changes in patients muscle strength, quality of life, and respiratory function. The study also will evaluate the safety and tolerability profile of ibudilast.
No. 6 FDA Approves Pivotal Phase 2b/3 Trial for Ibudilast in ALS Patients
Just a few months earlier, MediciNova had received approval from the U.S. Food and Drug Administration (FDA) to initiate this trial. Eligible patients must have had the disease for no more than 18 months and present just mild disability.
During the trial, patients will receive Rilutekfor at least 30 days before starting a regimen of either 100 mg per day of ibudilast or a placebo for a period of 12 months. Participants may then enter an extension phase in which they will be offered the ibudilast combination for an additional six months. Top-line data is expected by December 2021.
No. 5 Phase 3 Trial of NurOwn Cell Therapy Fully Enrolls 200 Patients, BrainStorm Announces
The Phase 3 trial evaluatingBrainStorm Cell Therapeuticss cell therapy candidateNurOwn completed patient enrollment in October. The 200 participants will receive three administrations of NurOwn, or a placebo, into the spinal canal every two months. The primary goals are to demonstrate the therapys safety and its ability to slow disease progression.
A cell-based therapy, NurOwn works by removing specific stem cells from patients and converting them to cells that produce molecules that promote nerve tissue growth and survival. The modified cells are then returned to the patient to stimulate nerve tissue growth. NurOwn aims to safely improve abilities like swallowing, speech, handwriting, and walking in people with ALS.
No. 4 High-dose Vitamin B12 May Improve ALS Prognosis if Started Early, Study Suggests
A long-term Phase 2/3 study examining vitamin B12 as a treatment for ALS found that ultra-high doses of methylcobalamin, the physiologically active form of this vitamin, may extend survival and slow the decline in functional capacity, compared with a placebo.
The study included 373 patients, diagnosed fewer than three years earlier, across 51 sites in Japan. However, the benefits were only seen in patients diagnosed less than one year before taking the supplements, and only a trend was observed.
No. 3 CuATSM Therapy May Slow ALS Progression, Improving Cognition and Respiration in Patients, Phase 1 Trial Shows
Results from a Phase 1 clinical trial (NCT02870634) showed that CuATSM a small molecule able to selectively deliver copper to cells with damaged mitochondria slowed disease progression and improved the respiratory and cognitive function of people with ALS.
Damaged mitochondria are considered a hallmark of several neurodegenerative diseases, including ALS, and delivering copper is thought to restore the health of these organelles. Developer Collaborative Medicinal Development (CMD) began testing CuATSM in patients with sporadic and familial ALS in 2016.CMD is now planning to launch a randomized, placebo-controlled clinical trial for CuATSM to confirm these results.
No. 2 Bacteria and Fungi Found in Central Nervous System of ALS Patients, Study Reports
In March, a study discovered several species of bacteria and fungi living in the central nervous system (CNS) of people with ALS, suggesting that patients have coexisting bacterial and fungal infections.
The study built on prior research suggesting that ALS might be caused by a fungal infection. Researchers then examined frozen CNS tissue from 11 ALS patients to assess whether bacterial infections accompany fungal infections. Bacterial DNA was found in different regions of the CNS and the presence of bacteria was confirmed in neural tissue samples.
No. 1 Nerve Injury May Trigger Onset, Progression of ALS Symptoms, Rat Study Suggests
Our most-read article of 2019reported that small nerve damage may serve as an ALS trigger. Such damage may accelerate motor symptoms in rats carrying a mutation in the SOD1 gene,one of the 40 genes associated with ALS development in humans.
While rats without the mutation completely recovered leg function four weeks after induced damage to the sciatic nerve, located in the leg, SOD1-mutated animals were unable to fully recover. These rats also lost function in the uninjured leg, likely as a consequence of sustained immune activation and more severe neurodegeneration.
The induced nerve damage mimics head injury and trauma in human patients, which could explain the higher prevalence of the disease among war veterans and professional athletes.
***
At ALS News Today, we hope these stories and our regular reporting throughout 2020 contribute to informing and improving the lives of everyone affected by ALS.
We wish all our readers a happy 2020.
Total Posts: 6
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 2019 - ALS News Today
- Stem Cell Therapy for Kidney Failure [Last Updated On: April 1st, 2011] [Originally Added On: April 1st, 2011]
- Dogged by pain -- stem cell therapy for dogs [Last Updated On: June 24th, 2013] [Originally Added On: April 3rd, 2011]
- Dogs now getting stem cell therapy [Last Updated On: April 8th, 2011] [Originally Added On: April 8th, 2011]
- Stem Cells for Spinal Cord Injury: Community Outreach San Diego 2011 - Trish Stressman [Last Updated On: April 11th, 2011] [Originally Added On: April 11th, 2011]
- Stem Cell Treatment Doing Wonders For Autistic Boy [Last Updated On: April 14th, 2011] [Originally Added On: April 14th, 2011]
- Is Stem Cell Therapy right for your pet [Last Updated On: April 14th, 2011] [Originally Added On: April 14th, 2011]
- Regenerative Medicines [Last Updated On: May 19th, 2011] [Originally Added On: May 19th, 2011]
- Stem Cell Therapy for Multiple Sclerosis [Last Updated On: June 24th, 2013] [Originally Added On: May 20th, 2011]
- Adult Stem Cell Therapy for Multiple Sclerosis [Last Updated On: May 23rd, 2011] [Originally Added On: May 23rd, 2011]
- Heart Failure Patient After Adult Stem Cell Therapy [Last Updated On: June 24th, 2013] [Originally Added On: May 30th, 2011]
- Stem Cell Therapy For Alzheimer's/Dementia [Last Updated On: June 24th, 2013] [Originally Added On: June 2nd, 2011]
- Stem Cell Therapy For Alzheimer's/Dementia [Last Updated On: June 7th, 2011] [Originally Added On: June 7th, 2011]
- Stem Cell Therapy Injections [Last Updated On: June 24th, 2013] [Originally Added On: June 8th, 2011]
- Clinical Advances in Adult Stem Cell Therapy: Dr. Jorge Paz [Last Updated On: June 24th, 2013] [Originally Added On: June 11th, 2011]
- Stem Cell Therapy MS: Sam Harrell and The Superbowl [Last Updated On: June 12th, 2011] [Originally Added On: June 12th, 2011]
- Stem Cell Therapy MS: Sam Harrell and The Superbowl [Last Updated On: June 24th, 2013] [Originally Added On: June 14th, 2011]
- Lou Gehrig's Disease (ALS): UCSD Team's Stem Cell Therapy Rationale [Last Updated On: June 14th, 2011] [Originally Added On: June 14th, 2011]
- Stem Cell Injection Treatment - Stem Cell Therapy [Last Updated On: June 18th, 2011] [Originally Added On: June 18th, 2011]
- edivet America Dog Arthritis Stem Cell Therapy Stem Cell Therapy for Equine 1 [Last Updated On: June 24th, 2013] [Originally Added On: June 23rd, 2011]
- Lou Gehrig's Disease (ALS): UCSD Team's Stem Cell Therapy Approach [Last Updated On: June 25th, 2011] [Originally Added On: June 25th, 2011]
- Adult Stem Cell Therapy Clinical Advances - Dr. Jorge Paz in San Diego, CA March 2011 [Last Updated On: July 3rd, 2011] [Originally Added On: July 3rd, 2011]
- Adult Stem Cell Therapy Clinical Advances - Dr. Jorge Paz in San Diego, CA March 2011 [Last Updated On: June 24th, 2013] [Originally Added On: July 15th, 2011]
- Duffy Gets Stem Cell Therapy [Last Updated On: June 24th, 2013] [Originally Added On: July 15th, 2011]
- WAVE3 News Adipose Stem Cell Therapy [Last Updated On: June 24th, 2013] [Originally Added On: July 16th, 2011]
- Lou Gehrig's Disease (ALS): UCSD Team's Stem Cell Therapy Approach [Last Updated On: June 24th, 2013] [Originally Added On: July 16th, 2011]
- Clinical Advances in Adult Stem Cell Therapy - Dr. Jorge Paz Rodriguez (Miami) [Last Updated On: June 24th, 2013] [Originally Added On: July 21st, 2011]
- Stem Cell Therapy for Cerebral Palsy - Holly Catalano [Last Updated On: June 24th, 2013] [Originally Added On: August 2nd, 2011]
- Bone Marrow Aspiration - Stem Cell Therapy [Last Updated On: August 5th, 2011] [Originally Added On: August 5th, 2011]
- Dr. Chein discusses Autologous Stem Cell Therapy - Palm Springs [Last Updated On: June 24th, 2013] [Originally Added On: August 12th, 2011]
- Stroke Victim Improved With Stem Cell Therapy. More at http://www.stemcellfusion.com [Last Updated On: June 24th, 2013] [Originally Added On: August 14th, 2011]
- Dr. Craig Saunders Adult Stem Cell Therapy [Last Updated On: August 15th, 2011] [Originally Added On: August 15th, 2011]
- Embryonic stem cell therapy in China (www.esctherapy.com) [Last Updated On: June 24th, 2013] [Originally Added On: August 16th, 2011]
- Ying Liu discusses IPS cell therapy for ALS [Last Updated On: June 24th, 2013] [Originally Added On: August 16th, 2011]
- Stem Cell Therapy: Healing Force of the future [Last Updated On: September 19th, 2011] [Originally Added On: September 19th, 2011]
- Benefits of STEM CELL THERAPY - Adipose Tissue [Last Updated On: September 22nd, 2011] [Originally Added On: September 22nd, 2011]
- Stem Cell Therapy Skin Repair and Anti-Wrinkle Cream [Last Updated On: June 24th, 2013] [Originally Added On: September 24th, 2011]
- Cardiac Stem Cell Therapy [Last Updated On: June 24th, 2013] [Originally Added On: September 24th, 2011]
- Holly Huber on Recovery: Stem Cell Therapy for Multiple Sclerosis Community Outreach Dallas, TX 2011 [Last Updated On: June 24th, 2013] [Originally Added On: September 24th, 2011]
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- A Touch of Love® - Stem Cell Therapy for Pets-Dr. Danny DeRose-Part 3 [Last Updated On: September 27th, 2011] [Originally Added On: September 27th, 2011]
- Adult Stem Cell Therapy for Multiple Sclerosis [Last Updated On: June 24th, 2013] [Originally Added On: September 28th, 2011]
- Multiple Sclerosis Stem Cell Therapy News Coverage [Last Updated On: June 24th, 2013] [Originally Added On: October 1st, 2011]
- The Spinal Cord Journey - Stem cell therapy stories from three spinal cord injury patients [Last Updated On: October 6th, 2011] [Originally Added On: October 6th, 2011]
- Peyton Manning Stem Cell Therapy: Some Doctors Cry Foul Over European Treatment - Video [Last Updated On: October 13th, 2011] [Originally Added On: October 13th, 2011]
- Stem-Cell-Therapy-in-China.wmv - Video [Last Updated On: October 13th, 2011] [Originally Added On: October 13th, 2011]
- Randolph veterinarian uses new stem cell therapy on ailing pets - Video [Last Updated On: June 24th, 2013] [Originally Added On: October 13th, 2011]
- Stem Cell Therapy for Rheumatoid Arthritis - Video [Last Updated On: June 24th, 2013] [Originally Added On: October 14th, 2011]
- Holly Huber on Recovery: Stem Cell Therapy for Multiple Sclerosis Community Outreach Dallas, TX 2011 - Video [Last Updated On: October 14th, 2011] [Originally Added On: October 14th, 2011]
- Affordable Stem Cell Therapy in Guatemala (2hrs from Miami) - Video [Last Updated On: June 24th, 2013] [Originally Added On: October 15th, 2011]
- Stem Cell Research: Macular Degeneration - Video [Last Updated On: October 18th, 2011] [Originally Added On: October 18th, 2011]
- Duffy Gets Stem Cell Therapy - Video [Last Updated On: October 18th, 2011] [Originally Added On: October 18th, 2011]
- VetCell's StemRegen - the original and best stem cell therapy for equine tendon injuries - Video [Last Updated On: October 20th, 2011] [Originally Added On: October 20th, 2011]
- Macular degeneration - Stem Cell therapy (English subtitles) - Video [Last Updated On: October 20th, 2011] [Originally Added On: October 20th, 2011]
- Stem Cell Therapy for Rheumatoid Arthritis - Community Outreach Dallas - Darnell Morris - Video [Last Updated On: June 24th, 2013] [Originally Added On: October 21st, 2011]
- Adult Stem Cell Therapy for Congestive Heart Failure [Last Updated On: June 24th, 2013] [Originally Added On: October 21st, 2011]
- Stem Cell Therapy for Critical Limb Ischemia: First Choice or Last Ditch? - Video [Last Updated On: October 22nd, 2011] [Originally Added On: October 22nd, 2011]
- WAVE3News Adipose Stem Cell Therapy - Video [Last Updated On: October 22nd, 2011] [Originally Added On: October 22nd, 2011]
- Stem Cell Therapy in Cardiac Disease - Video [Last Updated On: June 24th, 2013] [Originally Added On: October 24th, 2011]
- Stem Cell Treatment for Autism: Community Outreach Miami, Florida - May 2011 - Video [Last Updated On: June 24th, 2013] [Originally Added On: October 24th, 2011]
- Pre Post n 6 Months Post Stem Cell Therapy of Dystonic Cerebral Palsy - Video [Last Updated On: June 24th, 2013] [Originally Added On: October 24th, 2011]
- Stem Cell Treatment for Rheumatoid Arthritis - "I am basically pain free." - Video [Last Updated On: October 26th, 2011] [Originally Added On: October 26th, 2011]
- UMBILICAL CORD STEM CELL THERAPY - Video [Last Updated On: June 24th, 2013] [Originally Added On: October 26th, 2011]
- Stem Cell Therapy for Peripheral Artery Disease - Video [Last Updated On: June 24th, 2013] [Originally Added On: October 26th, 2011]
- Multiple Sclerosis Adult Stem Cell Therapy - Video [Last Updated On: June 24th, 2013] [Originally Added On: October 26th, 2011]
- Stem Cell Therapy for Multiple Sclerosis Stimulates IDO - Video [Last Updated On: June 24th, 2013] [Originally Added On: October 26th, 2011]
- HIV/AIDS: Spotlight on Stem Cell Research - John Zaia - Video [Last Updated On: October 26th, 2011] [Originally Added On: October 26th, 2011]
- Stem cell treatment by Adiva Health Care India after Spinal Cord Injury - Video [Last Updated On: October 27th, 2011] [Originally Added On: October 27th, 2011]
- Stem Cell Treatment for Spinal Cord Injury - Video [Last Updated On: October 28th, 2011] [Originally Added On: October 28th, 2011]
- Lou Gehrig's Disease: Stem Cell Therapy Pre-Clinical Studies - Video [Last Updated On: October 28th, 2011] [Originally Added On: October 28th, 2011]
- Stem Cell Treatment for Spinal Cord Injury - Video [Last Updated On: June 24th, 2013] [Originally Added On: October 28th, 2011]
- Holly Huber on Recovery: Stem Cell Therapy for Multiple Sclerosis Community Outreach San Diego CA - Video [Last Updated On: June 24th, 2013] [Originally Added On: October 28th, 2011]
- Stem Cell Treatment for Rheumatoid Arthritis - "I got my life back" - Video [Last Updated On: October 28th, 2011] [Originally Added On: October 28th, 2011]
- Adult Stem Cell Therapy Following Heart Attack and Stroke - Video [Last Updated On: October 28th, 2011] [Originally Added On: October 28th, 2011]
- Stem Cells - Treatment for Autism - Video [Last Updated On: November 2nd, 2011] [Originally Added On: November 2nd, 2011]
- Stem Cell Treatment Diabetes - Video [Last Updated On: November 7th, 2011] [Originally Added On: November 7th, 2011]
- Stem Cell Therapy: Psoriatic Arthritis Treatment - Video [Last Updated On: June 24th, 2013] [Originally Added On: November 8th, 2011]
- Stem Cell Therapy Cure Hair Loss. More at http://www.stemcellfusion.com - Video [Last Updated On: June 24th, 2013] [Originally Added On: November 10th, 2011]
- BioLogic Stem Cell Therapy - Video [Last Updated On: November 16th, 2011] [Originally Added On: November 16th, 2011]
- BioLogic Stem Cell Therapy - Video [Last Updated On: June 24th, 2013] [Originally Added On: November 16th, 2011]
- AVS Stem Cell Therapy - Video [Last Updated On: June 24th, 2013] [Originally Added On: November 17th, 2011]