ALS Research Briefs

by Margaret Wahl on Thu, 2011-03-03 10:53

ALS research news, including insights about TDP43, experimental therapies based on cyclosporin, heat shock proteins and stem cells, and more evidence for a smoking-ALS link

Loss of TDP43's normal function could be ALS contributor

New mouse studies suggest that loss of the normal function of the TDP43 protein, and not just an abnormal "toxic gain of function" of this protein, may be contributing to loss of nerve cells in at least some cases of amyotrophic lateral sclerosis (ALS). Most studies to date have implicated misfolded TDP43, mislocalized TDP43 and/or overly long-lasting TDP43 in ALS pathology. To read the entire paper without charge, see Dysregulation of the ALS-associated gene TDP-43 leads to neuronal death and degeneration in mice. See also ALS-Causing TDP43 Overstays Its Welcome and Why Does ALS Spread?

Heat shock proteins may become therapies

ALS Biopharma is designing possible therapeutic agents for ALS based on the "heat shock protein 70" family of proteins. Levels of naturally occurring heat shock proteins (HSPs) increase when cells are exposed to heat or other types of stress. The biotech company is hoping HSP70-based therapies will relieve the stress on ALS-affected motor neurons.

CNS-penetrating cyclosporin compound in development

Maas Biolab now has patent protection and Orphan Drug designation (which provides financial incentives for development of drugs for rare diseases) for its cyclosporin-based compound Mitogard. Cyclosporin (also spelled "cyclosporine"), an immunosuppressant, is believed to have neuroprotective properties. Mitogard is designed to treat ALS and other neurodegenerative diseases by being infused directly into the cerebrospinal fluid, thus bypassing natural barriers around the central nervous system. Maas Biolab is conducting animal studies to assess the safety of Mitogard. See the Feb. 9, 2011, press release Maas Biolab Wins World Cyclosporin Brain Formula Patents.

Stem cell safety trial moving forward

Biotechnology company Neuralstem, which is conducting a phase 1 clinical trial of its patented neural stem cells in people with ALS at Emory University in Atlanta, announced in February that analysis of safety data from the first nine trial participants is complete. The company will now move to the last group of ALS patients in this part of the trial. See Neuralstem's Feb. 10, 2011, press release, Neuralstem Updates ALS Clinical Trial Progress. Also in February, the company received U.S. Food and Drug Administration (FDA) orphan drug designation for its human spinal-cord-derived neural stem cells. See Neuralstem's Feb. 9, 2011, press release, Neuralstem Receives FDA Orphan Designation for Spinal Cord Stem Cells for Treatment of ALS. For details about the trial, see Emory ALS Center: Stem Cell Trial.

Smoking again tied to ALS risk

A study that included more than a million men and women, 832 of whom developed ALS, supports the previously proposed hypothesis that cigarette smoking is a risk factor for the disease. See Smoking and risk of amyotrophic lateral sclerosis: a pooled analysis of 5 prospective cohorts. See also Smoking Raises Risk of ALS.

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