STAT

Research using brains-in-a-dish forces a radical rethinking of Huntington’s disease

The new understanding, that Huntington's may start before birth, is surprising because it has long seemed like a prototypical neurodegenerative disease that doesn't strike until adulthood.

Even allowing for the fact that these were lilliputian brains, they were not behaving at all according to plan. From the first days of the tiny lab-grown organs’ development, primitive “progenitor cells” romped out of their birthplaces in the deep interior and quickly turned into neurons and glia, specialized cells that do the brain’s heavy lifting, from thinking and feeling and moving to boring old neurological housekeeping. But the cells were jumping the gun.

In healthy developing human brains, progenitor cells spend a good chunk of prenatal existence simply reproducing, vastly increasing their numbers and postponing becoming other brain cells. The impatient progenitor cells, however, were in cerebral organoids — minuscule 3-D versions of the brain — created from the cells of people with Huntington’s disease in hopes of mimicking the patients’ actual brain development decades earlier.

It was new evidence that, in their understanding of this devastating genetic illness, scientists know only half the story: In addition to being a neurodegenerative disease, it is also neurodevelopmental, starting

You’re reading a preview, subscribe to read more.

More from STAT

STAT2 min read
STAT+: Pharmalittle: We’re Reading About An Amgen Obesity Drug, A Senate Bill On Shortages, And More
Amgen will no longer develop an early-stage obesity pill, and will instead focus on a more advanced injectable candidate to compete with Wegovy and Zepbound.
STAT2 min read
STAT+: Pharmalittle: We’re Reading About AstraZeneca CEO Pay, Alternatives To WuXi And More
And so, another working week will soon draw to a close. Not a moment too soon, yes? This is, you may recall, our treasured signal to daydream about weekend plans.…
STAT2 min read
STAT+: Pharmalittle: We’re Reading About FTC Fighting ‘Junk’ Patents, Pfizer Direct-to-consumer Plans, And More
The FTC expanded its campaign against pharmaceutical companies for filing what it calls “junk” patent listings for 20 different brand-name treatments.

Related Books & Audiobooks