Biogen/Ionis ALS drug extends survival in rodents
A drug being developed to treat amyotrophic lateral sclerosis (ALS) by targeting mutations in the SOD1 gene has turned in promising results in mice and rat models of the degenerative brain disease. The mutations cause the gene to be overly active, and the drug, called BIIB067, reduces levels of the SOD1 protein. A team led by researchers at Washington University School of Medicine in St. Louis reported that BIIB067 extended survival and reversed signs of neuromuscular damage in the animals as compared to a placebo. The increase in lifespan was about 22%, they said. The drug, which is being developed by Biogen and Ionis, is currently in phase 1/2 clinical trials. The preclinical results were reported in the Journal of Clinical Investigation. (Release)
A novel target for preventing malaria
Researchers led by Yale University have identified a protein produced by malaria parasites in order to evade the immune system. And they’ve developed a vaccine that targets the protein, called Plasmodium macrophage migration inhibitory factor (PMIF). In two mouse models of malaria infection, the vaccine, which is RNA-based, prevented reinfection, they reported in the journal Nature Communications. The research demonstrated that PMIF helps complete the malaria parasite’s life cycle by ensuring transmission to new hosts. The Yale team is collaborating with Novartis to further develop the vaccine for clinical trials. (Release)
Scientists edit out inherited blood disorder in mouse embryos
Gene-editing techniques such as CRISPR-Cas9 have generated excitement because of their potential to cure inherited diseases, but the risk of off-target effects has limited the potential applications of the technology. Scientists at Carnegie Mellon and Yale University are reporting that they used a different gene editing technique to correct the genetic mutation that causes the blood disorder beta thalassemia. Their technique involved using a nanoparticle to deliver a peptide nucleic acid (PNA): a synthetic protein combined with elements of DNA and RNA. The PNA “unzips” the two strands of DNA and sets into motion a repair mechanism that fixes the faulty gene, the researchers reported in the journal Nature Communications. One injection in utero corrected 6% of the mutations—enough for a cure, they said. (Release)