HPBCD was tested for revealing the symptoms of Cockayne syndrome in mouse model: a surprise

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Cockayne syndrome is a hereditary form of premature aging caused by a
gene mutation. The disease affects more organs including the skin which
becomes hypersensitive to ultraviolet radiation. Subcutaneous fat is
lost in the course of the disease progress. To date there is no cure for
Cockayne syndrome. Majora et al. recently presented their results in
Science Translational Medicine on a potent drug candidate
suberoylanilide hydroxamic acid (SAHA) which may represent a therapeutic
option for this disease [1]. Laboratory test animals (mice) were involved in
testing of efficacy of SAHA. Mice (both wild type and gene-deficient)
were divided into various groups. One group received only drinking
water, the other received 2-hydroxypropyl beta cyclodextrin (HPBCD)
solution, while the third received SAHA solubilized by HPBCD. Each group
were sub-divided into species exposed or non-exposed to UV-light.
Surprisingly one control group of the gene-deficient species receiving
HPBCD without SAHA (but not exposed to UV-light) showed a phenotype that
was less pronouncedly damaged than those receiving standard drinking water. Dosage
and DS of HPBCD were unfortunately not disclosed in the article. The
authors explain this phenomenon by a possible analogy found for
Niemann-Pick-C disease, namely that HPBCD can improve
autophagic/lysosomal functions [2]. Nevertheless, UV-irradiated
gene-deficient mice were not responding to HPBCD “treatment” without
application of SAHA.

All in all, each publication demonstrating that
HPBCD alone may show the sign of therapeutic effect is a magnificient
step towards rising the scientific profile of these carbohydrates.

[1] Majora et al.: Sci. Transl. Med. 10, eaam7510 (2018)

[2] Davidson et al.: PLOS ONE 4, e6951 (2009)

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