Older Children with Spinal Muscular Atrophy Improve on Nusinersen
Long-term treatment with the anti-sense oligonucleotide led to motor benefits in an extension trial of children 2 to 15 years old.
NEWS
Could Greasing the Wheels of Lipid Processing Treat Alzheimer’s?
Speakers at AD/PD 2019 reported that AD risk factors mess up lipid metabolism in glial cells. In cellular models, speeding the clearance of fats lessened pathology.
Could CD33 Be the Microglial Target for Stimulating Phagocytosis?
Inhibiting the receptor activates microglia to mop up debris, making CD33 an attractive therapeutic target.
Parsing How Alzheimer’s Genetic Risk Works Through Microglia
Scientists at AD/PD 2019 see a Goldilocks of microglial activation: Both too little and too much is bad in an injured brain. How could a therapy make it just right?
Life Without Microglia: Rare Cases of CSF-1R Mutations Paint a Grim Picture
In people carrying two mutated copies of the trophic receptor, important brain structures, such as the corpus callosum, never developed.
APP Upp: Mutation Nixes Six Amino Acids from Aβ, Spurs Aggregation
Presented at AD/PD, the discovery by scientists in Uppsala is the first APP deletion found to cause Alzheimer’s disease. The same group found the Swedish and Arctic APP mutations.
Electrode Detects Aβ Aggregates in Alzheimer’s Plasma
The method purportedly distinguishes patients from controls with more than 90 percent sensitivity and specificity.
Expression, Expression, Expression—Time to Get on Board with eQTLs
A tool of modern genetics, expression studies link GWAS hits to specific cell types, providing clues to pathogenesis. Microglia come up again and again.
Virtual Exhibit Hall
Alzforum encourages users to visit the Virtual Exhibit Hall, where companies showcase their newest initiatives, products, and services. We welcome Dash Genomics, Inc. and Abcam, which join our other exhibitors — Biogen, BioLegend, BrainXell, NanoString Technologies, and the Jackson Laboratory.
SPOTLIGHT
AD/PD Meeting in Lisbon Hosts a Humbled Field
Held in romantically beautiful Lisbon, the 14th AD/PD conference was the biggest thus far, drawing 3,982 attendees from 73 countries, according to Abraham Fisher, its longtime lead organizer. But as the field grows, it has little to celebrate, and the mood reflected that. Trialists were appealing for patience and pointing to deepening clinical trials skills as they learn from setbacks on investigational therapies, most recently of lanabecestat, crenezumab, and aducanumab. Trials targeting tau were not yet reading out, but the medical food Souvenaid posted modestly positive results. At the other end of the bench-to-bedside spectrum, basic science is diversifying. Next-gen genetics is spilling out gene variants which, together with RNAseq, are energizing research into glial and lipid biology. Debate about the amyloid hypothesis, a fixture at AD/PD, evolved toward a focus on genetic variability in how a person’s innate immune system responds to amyloid deposition. Ab itself? Nothing special about it. It aggregates into an irritant as the brain’s ability to degrade it wanes with age. It starts things off, but other factors later dominate the disease. Or so researchers now think. Read Madolyn Rogers and Gabrielle Strobel’s coverage.
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