Medicines used for ovarian and breast cancer, called PARP inhibitors, might be repurposed to treat people with amyotrophic lateral sclerosis (ALS), a study in cells suggests. This suggestion came from researchers’ work with veliparib (ABT-888), an investigational PARP inhibitor, that found it effectively reduced TDP-43 aggregates — a hallmark of…
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The U.S. Food and Drug Administration has given positive feedback to MediciNova’s Phase 3 developmental plan for its investigational therapy ibudilast (MN-166) for amyotrophic lateral sclerosis (ALS). Following the FDA’s guidance and suggestions, the company will design a trial that determines the maximum benefit of the treatment,…
Treatment with EHP-102, an investigational cannabinoid-derived medicine, reduced weight loss, preserved motor neurons of the spinal cord, and lowered the abnormal reactivity of astroglial cells in a mouse model of amyotrophic lateral sclerosis (ALS), new research shows. Findings were recently published in the study, “Neuroprotective effects of…
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Noxopharm and Nyrada Discover Potent Inhibitor of Key Protein Involved in ALS, Other Conditions
The pharmaceutical development company Noxopharm and its majority-owned subsidiary, U.S. biotechnology firm Nyrada, have discovered a potent inhibitor of the protein IRAK4, a “master switch” critical to the development of inflammatory autoimmune diseases, such as amytrophic lateral sclerosis (ALS). IRAK4 has emerged as a key switch in the…
Specific patterns of a misfolded protein — superoxide dismutase 1 (SOD1) — are found in the brain and spinal cord of patients with sporadic amyotrophic lateral sclerosis (ALS), according to a new study, supporting research suggesting that misfolded SOD1 plays a role in this ALS subtype. The research, “…
More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…
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Parkinson’s Therapy Azilect Added to Rilutek May Delay Fast-progressing ALS, Phase 2 Trial Shows
Adding Azilect (rasagiline) — a medication already approved to treat Parkinson’s disease — to Rilutek (riluzole) may be able to slow the advancement of fast-progressing amyotrophic lateral sclerosis (ALS), a Phase 2 trial suggests. However, its positive safety results are being challenged by other physicians, who raise concerns that…
Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are caused by different patterns of inflammation in the brain, which may help explain why these two illnesses are so distinct. The German study, “Different neuroinflammatory profile in amyotrophic lateral sclerosis and frontotemporal dementia is linked to the…
A newly developed method can help characterize the types of misfolded proteins present in patients with amyotrophic lateral sclerosis (ALS), according to findings in a thesis. The recently published doctoral thesis, titled “Structural investigation of SOD1 aggregates in ALS: identification of prion strains using anti-peptide antibodies,” was written…
Retrotope is providing its investigational fatty acid being developed for Friedreich’s ataxia, RT001, to people with amyotrophic lateral sclerosis (ALS) under an expanded access program, also called “compassionate use.” Individual patients with life-threatening or severely debilitating diseases can petition the U.S. Food and Drug Administration, through their physicians,…
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