FDA Grants Fast Track Designation to NurOwn
The FDA has designated BrainStorm Cell Therapeutics' NurOwn as a "Fast Track" produce for the treatment of ALS. The FDA's Fast Track program ia designed to expedite the review of drugs and biologics intended to treat serious conditions and meet unmet medical needs through increased meetings and written communications with the FDA. A Phase 2 clinical trial of NurOwn is still recruiting participants. For more info on this trial: See ALS-TDI: NurOwn
Have you been seeing information on Facebook and other social media regarding Ubiquilin-2? The study referred to in those articles was published back in 2011! To see the original story, see the ALS-TDI: Ubiquilin-2
Endoplasmic Reticulum Protein Protects Motor Neurons from ALS
When ALS attacks motor neurons, certain types of motor neurons resist, while others surrender. If scientists can figure out why some neurons hang tough, they may be able to offer that same protection to those that succumb. ALS-resistant motor neurons may draw defense from a protein called Sil1, which is in short supply in the sensitive neurons. For more info on the Endoplasmic Reticulum Protein and Sil1, see The ALS Forum
Targeting Pharmaco-resistance Pumps Improves Ruluzole Efficacy in ALS Mice
The central nervous system is adept at protection from toxins and utilizes specialized transporters in the endothelial cells of the blood-brain-barrier to pump them out. BUT...this can also rid the brain and spinal cord of potentially beneficial drugs. For info on a potential therapy to overcome this, see The ALS Forum
ALS Clinical Trials
ALS-TDI monitors ALS clinical trials worldwide. For a list of the 112 trials being monitored, see ALS-TDI Clinical Trial Database
Individuals affected by ALS are at an increased risk of serious and possibly life-threatening complications from the flu, so it's important to stay informed and take steps to protect themselves and their families.
Weather and MDA
If you are impacted by severe weather and need MDA services -- including help repairing or replacing durable medical equipment that's been damaged by a storm or information about local groups offering emergency assistance and/or shelter, contact MDA by calling (800) 572-1717 or emailing firstname.lastname@example.org.
Diaphragm Pacing System Clinical Trial
In 2012, MDA committed funds to help support a clinical trial designed to determine whether the NeuRx Diaphragm Pacing System is effective in people with ALS. This experimental device is a respiratory treatment strategy in which electrodes are implanted into the diaphragm using minimally invasive surgery. The diaphragm can then be stimulated to contract, mimicking normal breathing action. The device can be used with or without other ventilation strategies, such as positive-pressure ventilation.
The clinical trial is still open to new participants. For more information, see:
New Mouse Model with SOD1 Mutation
May Model Early ALS
A new mouse model is available to researchers that may prove useful for modeling selective neuronal susceptibility in ALS and screen for effective therapies based on measures other than survival. For more details: The ALS Forum
ALS Mechanisms and Models
At the Society for Neuroscience Meeting held in November, 2014, over 30,000 neuroscientists came together to discuss cutting-edge research on development, genetics, molecular mechanisms, tools, and therapeutics in all areas of neuroscience. For highlights from the nanosymposium on "Motor Neuron Disease Mechanisms and Models," where researchers gathered to share their latest findings about mechanisms and models of ALS and other neuromuscular diseases, see: The ALS Forum
Mitophagy Pathway Links ALS and Parkinson's Disease
Researchers have linked genes for ALS and Parkinson's Disease in the same pathway, a cellular methanism to degrade decrepit mitochondria. For more info, see The ALS Forum
ALS Registry Findings
The data is derived from four nationwide administrative databases and a self-reporting web portal that were integrated into a single registry. In addition to serving as a comprehensive and systematic resource on the current state of ALS, the registry could potentially provide a large dataset for environmental risk analysis, if the data use and privacy constraints are resolved. To read more about this valuable new resource and the potential collaborations surrounding it, see The ALS Forum