Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment Guidance for Industry | FDA
The FDA recently released a new guidance document to assist sponsors in the clinical development of drugs and biological products for the treatment of ALS. Specifically, this guidance addresses the FDA’s current thinking regarding the clinical development program and clinical trial design for drugs to support an indication for the treatment of ALS. Click here to read more.
Kadimastem Announces Promising Interim Results of Cohort A of Its Phase 1/2a Clinical Trial in ALS | BioSpace
Kadimastem Ltd., a clinical stage cell therapy company, announced promising interim results of its Phase 1/2a clinical trial for the treatment of patients with ALS. The trial aims to evaluate the safety and preliminary efficacy of injecting AstroRx®, an “off-the-shelf” clinical-grade astrocyte cell product, into the spinal cord fluid of ALS patients. Click here to read the full article.
New gene therapy targeting C9orf72-ALS begins Phase 1 clinical trial in the UK | Dr Keith Mayl & Dr Ahmad Al Khleifat | MND Research Blog
Researchers at King’s College Hospital, led by Professor Christopher Shaw, have embarked on the first gene therapy clinical trial for ALS patients with a C9orf72 gene mutation. Since the mutated gene produces toxic products, blocking the gene with gene therapy might be a useful approach to treatment. Click here to read the full article
Kennedy’s Disease vs ALS: How muscle patterns can aid diagnosis and perform as a novel biomarker | MND Research Blog | Martina Slapkova
Researchers from University College London found that muscle MRI can help distinguish ALS from Kennedy’s disease based on the way specific muscle groups deteriorate in each condition. The method can also help assess the severity of the disease. Check out the full story here
Add-on Masitinib Slows Progression of ALS, Final Phase 2/3 Trial Results Show | ALS News Today | Ana Pena
In a Phase 2/3 clinical trial, AB Science demonstrated that Mastinib in combination with Rilutek (riluzole) slowed functional decline of patients with ALS who have typical disease progression by 27% after 11 months of treatment. AB Science is now setting up a Phase 3 trial to confirm the results and gather more data in support of a new marketing application to the European Medicine Agency (EMA). Click here to view the full article.
Small-Molecule Compounds ID’d That May Lessen Protein Buildup in Cells Under Stress, an ALS Hallmark| ALS News Today | Patricia Inacio
A study at UC San Diego School of Medicine identified small-molecule compounds that reduced the accumulation of TDP-43, a molecular hallmark of ALS. While these preliminary findings require further investigation, it is hoped that this work may lead to new therapies for the disease. Click here to read the full article.
By Capturing Patients’ Feelings of Dyspnea, DALS-15 Scale May Ably Spot Those Needing Help with Breathing | ALS News Today | Ana Pena
Researchers at Otto-von-Guericke University in Germany developed a patient-reported scale to quantify dyspnea in ALS — the Dyspnea-ALS-Scale (DALS-15) — to help doctors evaluate respiratory problems and to support decision-making regarding NIV. A DALS-15 validation study showed that the scale was highly reliable, and confirmed that dyspnea cannot be fully captured using objective parameters like spirometry or blood gas analyses. Click here to view the full article.
Tailored treatment for ALS poised to move ahead | Nature Medicine | Carrie Arnold
The US Food and Drug Administration (FDA) has provisionally approved an experimental personalized treatment for a young woman with ALS. The therapy, known as an antisense oligonucleotide (ASO), is designed to disable mutated strands of genetic code to prevent them from producing a protein that causes neurological damage. Click here for the full article
Study Adds to Evidence that Smoking Increases Risk for ALS | ALS News Today | Jose Marques Lopes
Cigarette smoking has been proposed as a risk factor for developing ALS and accelerating disease progression. However, methodological concerns hamper definitive conclusions of whether smoking causes the disease. Click here for the full article.
Shocking: New Zealand death rate for motor neuron disease five times global average | Hawthorne Caller | Mike Billings
The Global Burden of Disease (GBD) study – the world‘s largest scientific effort to qualify the health loss from more than 300 major diseases, injuries and risk factors – found the mortality rate for MND in New Zealand was as high as 2.2 deaths per 100,000 people. Click here for the full article.
Swim training improves muscle strength in mouse model of ALS | ALS News Today | Joana Carvalho
Swim training reduced the loss of muscle strength associated with ALS (5% less between 11 and 15 weeks), and increased citrate synthase activity by 26% compared with ALS mice that did not undergo swim training. Click here for the full article.
ALS Researchers Discover New Biomarker and Drug Target | GEN
A research team led by scientists at Harvard University said it has found a potential new biomarker and drug target for ALS. Published in Nature Neuroscience, the study relied on stem cell models of human motor neurons to reveal the gene STMN2 as a potential therapeutic target. Click here for the full article.
Major breakthrough in MND drug trial | Mercury
Australian scientists have developed a groundbreaking drug which postpones the development of Motor Neuron Disease. After a six-month drug trial, researchers at the Florey Institute and the University of Melbourne claim the drug CuATSM has slowed progress of the disease by 70 per cent in 32 patients. Click here for the full article and video.
New Aussie drug gives hope to treat MND | SBS NEWS
Australian researchers have developed a drug called CuATSM which has been found to slow the progression of motor neurone disease. Click here for the full article.
New Zealand has highest motor neurone mortality rate in the world | Stuff | Hannah Martin
New Zealand has the highest mortality rates for motor neurone diseases in the world, new research has found. But little is known about why — sounding alarm bells for experts.
New Zealand has world’s highest death rate from motor neuron disease | News Hub | Jamie Ensor
A global study has revealed NZ has the highest mortality rate from MND, with 2 kiwis dying from the disease each week. According to the study, the risk of developing the disease is 1 in 300.
Shocking:New Zealand death rate for motor neuron disease five times global average | NZ Herald | Emma Russell
NZ’s MND death rate is the highest in the world and five times the global average, a new study has found. Experts are warning this shocking new finding should be a major wake-up call. Click here for full article
Study reveals shocking death rates for Kiwis with motor neurone disease | NZ Herald
Dr Emma Scotter, who led the study, said NZ doesn’t have higher MND mortality rates just due to living longer, or having a greater proportion of older people in our population. “It’s something other than just an age effect,” Scotter said. Click here for the full article
NZ has highest rate of motor neurone disease of any country in the world | 1 News Now
A new study has found NZ has the highest rate of MND of any country in the world. Auckland Uni scientists found the MND mortality rate is as high as 2.8 deaths per 100,000 people. Internationally, the average is just 1.67 deaths per 100,000 people. Click here for full article and video clip
Study: Athletes and footballers may be at greater risk of disease which killed Celtic legend Jimmy Johnstone | Herald Scotland | V.Weldon
Intense exercise could increase the risk of developing motor neurone disease (MND), research suggests. People who are the most physically active, such athletes and footballers, could be as much as 26 per cent more likely to go on to suffer from the devastating condition.