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Hemophilia B Gene Therapy Shows Promise in Clinical Trial

Gene therapy to restore clotting factor IX levels in blood might provide long-term treatment pf patients with hemophilia B. A phase I/II multi-center, open-label, clinical trial has tested a liver-directed, AAV based gene therapy called FLT180a that uses a synthetic viral capsid and a gain-of-function protein to boost factor IX levels long-term. The trial has found, a one-time injection sustained synthesis of normal levels of factor IX protein in the liver in nine of ten patients, across four doses, but needed immunosuppression.

Mapping Magnetic Stimulation Induced Neuroplasticity in Major Depression

A new study provides evidence that changes in brain connectivity induced by repetitive transcranial magnetic stimulation (rTMS) in patients with treatment-resistant depression may indicate macro-level neuroplasticity. Acute changes in neuroplasticity induced by rTMS may also indicate interindividual variability in rTMS treatment response and therefore could be used as a biomarker. The study found strong predictive associations in connections between prefrontal regions and regions involved in emotion, memory and movement.

Parkinson Patients Show Metabolic and Inflammatory Changes in Trial

A randomized, double-blinded, placebo-controlled, Phase I clinical study on newly diagnosed patients with Parkinson’s disease, naive to standard dopaminergic therapy, showed mild, clinically significant alterations in the brain metabolome upon oral treatment with nicotinamide riboside for a month at a dose of 1000 mg/day. The treatment was safe with increased levels of NAD (nicotinamide adenine dinucleotide) in the brain, in addition to related metabolites in the cerebrospinal fluid (CSF).

Anxiety Linked to Gut Microbial Metabolite in Mouse and Human

A study comparing mice engineered to synthesize the metabolite 4EPS, to mice that cannot, shows the metabolite passes into the brain and dampens maturation of oligodendrocytes, thinning the myelin insulation around axons. This results in anxiety-like behavior in mice that can be reversed by drugs that promote myelination. In an accompanying human study, the authors show a drug that adsorbs 4EPS from the human gut alleviates anxiety in adolescents with autism.

Trial Finds Experimental Immunotherapy Reduces Most Children’s Sensitivity to Peanuts

A clinical trial funded by the National Institutes of Health has found peanut oral immunotherapy in children aged 1 to 3 years with severe peanut allergy safely desensitized most children to peanuts and induced remission of peanut allergy in one-fifth. The immunotherapy consisted of a daily oral dose of peanut flour for 2.5 years. Remission was defined as being able to eat 5 g of peanut protein, without having an allergic reaction six months after completing immunotherapy. The youngest children and those who started the trial with lower levels of peanut-specific antibodies were most likely to achieve remission.

Hemophilia Gene Therapy Trial Achieves Sustained Clotting Factor Levels

Results from a multicenter international phase 1/2 clinical trial are the first to show improved production of coagulation factor VIII over prolonged period, leading to reduction or complete elimination of bleeding events. The trial infuses SPK-8011, a new recombinant AAV vector engineered to express factor VIII in human liver cells, in 18 men with hemophilia A and demonstrates a 91.5% decrease in bleeding episodes.

Parkinson’s Disease Trial Reveals Defects in Blood-Brain Barrier

A phase II clinical trial led by scientists at the Georgetown University Medical Center that tests the efficacy of the drug nilotinib approved for leukemia, in the treatment of Parkinson’s Disease suggests that the drug targets vascular and autophagy defects in the progression of Parkinson’s Disease. Nilotinib, 300 mg, reverses these effects via altering miRNA expression, suggesting epigenomic changes that may underlie long-term disease-modifying effects.

Biomarker Identified for Early Antidepressant Effects of Deep Brain Stimulation

Acute behavioral changes and long-term antidepressant response can be reliably elicited by surgically targeting and stimulating the subcallosal cingulate (SCC) gyrus area 25, a brain area implicated in depression. While the clinical effectiveness of DBS over the course of six months of treatment has been repeatedly demonstrated, there are differences in the timeline of recovery across different patients. A new study identifies an electrophysiological biomarker that can help optimize this experimental procedure in treating treatment-resistant depression and gain insights into the mechanisms underlying the efficacy of deep brain stimulation.

Asthma Worsens with Repurposed Drug Licensed for Psoriasis

A medicine used to treat psoriasis worsens asthma symptoms in patients with severe asthma, according to a randomized, placebo-controlled, double-blind, clinical trial result published in the New England Journal of Medicine. The drug risankizumab, a humanized, monoclonal antibody, targets the proinflammatory cytokine IL-23. IL-23 inhibition has been an effective treatment strategy for psoriasis and Crohn’s disease. Molecular profiling of sputum samples showed the drug reduced factors that prevent infections in airways.

Infrared Light Improves Memory in Pilot Clinical Trial on Healthy Individuals

A pilot clinical trial conducted by scientists at Durham University in collaboration with Maculume Ltd., provided evidence that self-administration of near-infrared light at 1068 nm wavelength twice daily may improve memory function in healthy, middle-aged people with normal intellectual capacities. The study included 14 healthy people, aged 45 and above who self-deliver six minutes of PBM-T (photobiomodulation therapy) twice daily over four weeks and a placebo group of 13 healthy individuals who wore a dummy PBM-T helmet. The authors said the therapy might have the potential to help people living with dementia and other disorders such as Parkinson’s, traumatic brain injury, and motor neuron disease.