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Brain drugs can now cross the once impenetrable blood–brain barrier

New technologies to shuttle drugs into the brain are showing promise in Alzheimer’s disease, cancer and more.
By
  1. Alison Abbott

    1. Alison Abbott is a writer based in Munich, Germany.

Conceptual collage illustration showing a representation of the blood-brain barrier .

Illustration: Jasiek Krzysztofiak; photographs: Zephyr/SPL; Steve Gschmeissner/SPL; Living Art Enterprises/SPL

Daiza Gordon watched her two younger brothers die when they were adolescents. They had Hunter syndrome, a rare, incurable disease — predominantly affecting boys — in which a gene for an important enzyme is missing. Guilt compounded her grief when her attempts to resuscitate her youngest brother failed. She was just 19 years old.

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Nature 641, 1086-1088 (2025)

doi: https://doi.org/10.1038/d41586-025-01569-z

Updates & Corrections

  • Correction 04 June 2025: An earlier version of this story mistakenly stated that cells direct therapeutic IDS to the lysosome as waste. Rather, they usually send the transferrin receptor to the lysosome for processing, which brings therapeutic IDS to where it is needed. The story also referred to an adeno-associated virus as a type of adenovirus. These are distinct classes of virus.

References

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  4. Liang, X. et al. Nature Commun. 16, 4028 (2025).

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