Can a repurposed Parkinson’s drug slow ALS progression?
Ropinirole, a drug used for Parkinson’s disease, shows promise in slowing the progression of amyotrophic lateral sclerosis (ALS), early research suggests.
Investigators randomly assigned 20 individuals with sporadic ALS to receive either ropinirole or placebo for 24 weeks. During the double-blind period, there was no difference between the groups in terms of decline in functional status.
However, during a further open-label extension period, the ropinirole group showed significant suppression of functional decline and an average of an additional 7 months of progression-free survival.
The researchers were able to predict clinical responsiveness to ropinirole in vitro by analyzing motor neurons derived from participants’ stem cells.
The study was published online in Cell Stem Cell (2023 Jun 1. doi: 10.1016/j.stem.2023.04.017).
The trial was sponsored by K Pharma. The study drug, active drugs, and placebo were supplied free of charge by GlaxoSmithKline.
West Nile infections rising in the U.S.
Several signs are pointing to an impending surge in the number of human cases of West Nile virus in several regions of the United States. West Nile virus is spread by infected mosquitoes and currently there is no cure or virus-specific treatment. In rare cases, it can be deadly. It can infect humans, birds, horses, and other mammals.
West Nile Virus is the leading cause of mosquito-borne disease in the continental United States. As of Aug. 8, 126 human cases had been identified across 22 states, according to the Centers for Disease Control and Prevention.
“Particularly here in California, it’s peak risk right now,” said Vicki Kramer, PhD, chief of vector-borne diseases in the California Department of Public Health. She said scientists there are seeing higher mosquito and infected mosquito numbers.
Dead birds are tested for the virus and by Aug. 4, 181 of the 913 birds tested in California have been positive, three times the total testing positive by that time in 2022.
“Last year at this time, we had 60 positive dead birds out of 817 tested,” Dr. Kramer said.
Myasthenia gravis drug gets FDA nod
The Food and Drug Administration (FDA) has approved rozanolixizumab (Rystiggo, UCB) to treat adults with generalized myasthenia gravis (gMG) who are positive for anti-acetylcholine receptor (AChR) or anti–muscle-specific tyrosine kinase (MuSK) antibody, the drug’s manufacturer, UCB, has announced.
gMG is a rare autoimmune disease of the nerve muscle junction. Anti-AChR and anti-MuSK antibody-positive gMG are the two most common subtypes. Rozanolixizumab is the first FDA-approved treatment for adults with both subtypes.
Rozanolixizumab is a subcutaneous-infused humanized IgG4 monoclonal antibody that binds to the neonatal Fc
receptor (FcRn), reducing the concentration of pathogenic IgG autoantibodies.
U.S. approval is based on results of the phase 3
MycarinG study involving 200 patients with AChR or MuSK autoantibody-positive gMG. Patients were randomly assigned to one of two rozanolixizumab groups (7 mg/kg or 10 mg/kg) or placebo for 6 weeks.
As reported in The Lancet Neurology (2023 May. doi: 10.1016/S1474-4422[23]00077-7), rozanolixizumab led to statistically significant improvements in gMG-specific outcomes, including everyday activities such as breathing, talking, swallowing, and being able to rise from a chair.