Mitsubishi Tanabe Pharma America (MTPA) will present initial data on efforts to create an oral version of edaravone, a key drug in the treatment of ALS that is currently only available intravenously, during the International Symposium on ALS/MND in Glasgow, Scotland. The symposium will be held Dec. 7-9. MTPA is expected to present results that demonstrate that oral edaravone is processed in the body in the same manner as the infused formulation.
An intravenous formulation of edaravone, Radicava, was approved by the U.S. Food and Drug Administration to treat ALS in May 2017.
“We are happy to learn that MTPA is working on an oral formulation of Radicava. This new formulation could make it easier for people living with ALS to take the drug. As we learn more, we will be sure to update our community,” stated Dr. Neil Thakur, executive vice president of mission strategy at The ALS Association.
Continue reading Mitsubishi Tanabe Pharma America to Present Data Showing Oral Version of Edaravone Processed Similarly to IV Version in Patients
In a promising new study by Drs. Robert Brown and Christian Mueller at the University of Massachusetts Medical School report that a type of viral gene therapy using synthetic microRNAs (miRNAs) targeting the ALS SOD1 gene is safe and effective in nonhuman primate macaques (monkeys). The ALS Association provided $1.7 million in funding for this study, which demonstrated an efficient reduction of the SOD1 protein without side effects. This paves the way forward for further development of this potential therapy.
Familial (inherited) ALS accounts for 10 percent of ALS cases. Of that percentage, 20 percent of cases are caused by mutations in the SOD1 gene, the first gene discovered to cause ALS in 1993, by co-author Dr. Robert Brown. Over the years, numerous studies have shown that mutant SOD1 is toxic to motor neurons, the cells that die in ALS, via multiple identified mechanisms. In addition, misfolding of toxic of SOD1 proteins have been identified in sporadic (non-inherited) cases of ALS.
With this in mind, researchers have targeted SOD1 for potential ALS therapies. For example, studies in a SOD1 mouse model demonstrated that silencing (decreasing) SOD1 delayed disease onset, increased survival time, and reduced muscle loss and motor and respiratory issues.
In this study published in high impact journal Science Translational Medicine, Mueller, Brown, and team administered miRNA to the central nervous system (CNS) in macaques, via spinal cord delivery. MicroRNAs are a part of the inherent gene silencing pathways found in both plants and animals. They are designed to specifically bind to target sequences found on messenger RNAs (the instruction book to make protein) to prevent toxic proteins from being made in the first place. This technique can effectively “turn off” toxic genes and proteins, which in this case is SOD1.
Continue reading Potential New Therapy Silences SOD1 Gene in Non-Human Primates
By Russell Mikunda
Before being diagnosed with ALS, I had no idea that veterans are twice as likely to be diagnosed with the disease. Even knowing what I know now, I would still serve my country.
I was in the Navy for eight years, reporting for duty on three different ships during my time in the service. During my second deployment, we were off the coast of Beirut when things started to heat up during the early 1980s.
I’m very proud to be a veteran. The VA (Department of Veterans Affairs) takes good care of me. But sometimes I worry that everybody with ALS doesn’t get that same high level of treatment I do.
Continue reading Guest Post: ‘I Would Still Serve My Country’
By Stan Williams
Last year, I sat in the Washington, D.C., offices of my four elected Indiana representatives and saw in their eyes how my words, and the words from my wife, pierced their hearts.
Attending the National ALS Advocacy Conference left me with the unmistakable conclusion that those with the most to lose should be speaking the loudest. We must explain to our elected officials how their inaction affects people living with ALS.
Our real-life experiences of my own battle with ALS, including my wife’s challenges as my caregiver, had an impact on the Congressmen. They listened intently. Some even shed a tear or two.
Continue reading Guest Post: ‘Those With the Most to Lose Should Be Speaking the Loudest’
By Stacy Crowder
There’s no sugarcoating it. ALS is terrible.
Unlike many fathers this Halloween, I couldn’t carve a pumpkin with my kids. I also couldn’t help them put on their costumes or take them trick-or-treating.
I didn’t know much about ALS before my diagnosis, but I became an expert pretty quick. I also got to work fighting – and not just for me but to bring hope to everyone living with ALS.
Continue reading Guest Post: ‘I’m Really Fighting for My Family’
Why motor neurons die in ALS largely remains a mystery. In a study funded by The ALS Association, Dr. Anne Hart, professor of neuroscience at the Carney Institute for Brain Science at Brown University, and her colleagues gained insight into why some types of motor neurons die, while other don’t. A paper about the study was recently published in PLOS Genetics.
Interestingly, Dr. Hart and her team used microscopic worms, called C. elegans, which maintain conserved ALS genes seen in humans to create the first precise worm models for SOD1 ALS, the second most common genetic cause of inherited ALS, using gene editing.
Motor neurons are the cells that die in ALS. Basically, a person has two types of motor neurons. Upper motor neurons (UMNs) connect the primary motor cortex in the brain (area of the brain that controls voluntary movement) to the spinal cord. UMNs use glutamate, a type of neurotransmitter, to send signals and communicate with each other.
Lower motor neurons (LMNs) connect the spinal cord to muscle and use acetylcholine as a neurotransmitter. (See diagram below.) It is not clear why, in some people with ALS, one or both types of motor neurons die.
Continue reading Precise ALS Worm Model Gives Insight into How Motor Neurons Die
By Kaisha Johnson and Cliff Whitlock, The ALS Association Golden West Chapter
More than 1,200 participants, volunteers, and members of the community came together for a family-friendly day of celebration, exercise, education, awareness, music, food, and family fun at The ALS Association Golden West Chapter’s 14th annual Napa Valley Ride to Defeat ALS and Walk on Saturday, September 22.
Together, these dedicated individuals raised more than $920,000 on event day toward a $1 million goal to fund the mission priorities of the Golden West Chapter in care services, advocacy, and research.
Held at the California Veterans Home in Yountville, the annual Fall event brings incredible energy and an outpouring of support for those affected by ALS in California and around the world. Many participants said it was the best ride they’ve attended.
There was an incredible outpouring of community support, including more than 140 volunteers who set up, checked in participants, served food and drinks, supported rest stops and the six bike routes, cheered on all of our incredible riders and walkers as they crossed the finish line, and so much more.
Continue reading Guest Post: Putting the Brakes on ALS – The Power of Community