Neuralstem to Offer Experimental Stem Cell Therapy in Colorado
Neuralstem plans to offer its experimental stem cell therapy to ALS patients in Colorado under the new "right-to-try" law recently passed in Colorado. According to this law, demonstrating safety is sufficient for administering the drug to terminal patients. As Neuralstem's human spinal cord-derived stem cell therapy called NSI-566 has been shown in a Phase 1 trial to be safe in humans, it qualifies under the new law. The Company has already begun training surgeons on delivery of the therapy. Whether patients will pay for the treatment is yet to be determined. Also, whether this therapy will truly benefit patients is an open question. Without further clinical trial testing to determine efficacy, it's unclear whether NSI-566 has any positive effect on ALS. For more info: Fierce Biotech via The ALS Forum Newsletter
New Method for Reprogramming Cells to Model Neurodegenerative Diseases
Induced pluripotent stem cells (iPSCs), adult cells genetically reprogrammed into an embryonic stem cell-like state, are becoming a powerful tool for research and drug development in ALS. iPSCs derived from sporadic and familial ALS patients are being used to understand mechanisms of the disease and to screen for candidate ALS drugs. For more on iPSCs, see The ALS Forum
VA Expedites Access to Housing Grants for Veterans with ALS
The Department of Veterans Affairs announced that veterans with ALS and those on active duty automatically are eligible for Specially Adaptive Housing Grants. The grants provide veterans with up to $68,000 to adapt their homes. For more info, see the VA's release on the subject
VA Biorepository Brain Bank Seeks Veterans with ALS
The U. S. Department of Veterans Affairs (VA) is seeking veterans who have ALS and who live in the United States to enroll in its VA Biorepository Brain Bank, a human tissue bank that collects, processes, and stores tissue samples for use in future research. Tissue samples from veterans who have ALS may help researchers determine what causes the disease and why veterans have a higher risk of developing it. To participate or to ask questions, call the VA toll free at 866-460-1158 or at 857-364-6748 or 857-364-3877. For more info on this, see VA Biorepository Brain Bank
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 email@example.com.
FDA Approval Given for Phase II clinical trial of Brainstorm's Nurown Adult Stem Cell Therapy Technology
Brainstorm Cell Therapeutics has been given FDA approval to move forward with a Phase II clinical trial with NurOwn in patients with ALS (recruitment info to be announced). The Phase I study held in Israel showed "encouraging signals of efficacy in individuals receiving NurOwn treatment, in which a dramatic improvement was observed during more than two consecutive visits ... . NurOwn differentiates bone marrow-derived mesenchymal stem cells (MSC) into "MSC_NTF" cells. These neuron-supporting cells secrete elevated levels of neurotrophic, or nerve-growth, factors for PROTECTION of existing motor neurons, PROMOTION of motor neuron growth, and RE-ESTABLISHMENT of nerve-muscle interaction. For more info: Brainstorm
Notes from the 2014 Annual Meeting of the American Academy of Neurology
Tirasemtiv: May help improve breathing and muscle strength, but tolerability remains a considerable obstacle. Further analysis of the results of the Tirasemtiv Phase IIB clinical trial is ongoing.
Ozanezumab: May do more than help recommect motor neurons to muscle fibers ... it may boost populations of oligodendrocytes (key motor neuron support cells that appear to be lost in people with ALS). A Phase II clinical trial of ozanezumab is ongoing, with nearly 300 people participating. The first results could be released as early as the end of 2014.
Neupogen: Contains the neuroprotective substance granulocyte colony stimulating factor (G-CSF), which aims to protect motor neurons from further damage by reducing inflammation. It may also help create new nerve cells by mobilizing stem cells to the central nervous system from the bone marrow. A clinical trial in Germany is planned for as early as later this year.
Stem Cell Therapies: For any potential stem cell therapies to be truly useful in the clinic, doctors need to be able to track them to make sure the stem cells are settling in (engrafting) in people with ALS where they are needed -- throughout the spinal cord. Research is being done to meet this challenge.
For more info on any of these stories: ALS-TDI
High-Calorie Diet May Help in ALS
A study shows that a high-carb, high-calorie tube feeding formula was safe and well-tolerated in a small number of ALS patients at a late stage of the disease. Those in the study in the high-carb, high-calorie group gained more weight and experienced fewer adverse events, including deaths, than those on tube feedings designed to replace expended calories and maintain weight or those on a high-fat, high calorie formula, which may not have been well absorbed. For more info: MDA-ALS Magazine