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Academic Publications by IntraBio’s Scientific Founders




Effects of acetyl-DL-leucine in patients with cerebellar ataxia: a case series.

Strupp, M., Teufel, J., Habs, M., Feuerecker, R., Muth, C., van de Warrenberg, B., Klopstock, T., Feil, K.

A paper exhibiting the significant benefit of Acetyl-DL-Leucine in the treatment of patients with cerebellar ataxia.

Journal of Neurology 260, 2556-2561 (2013)

Acetyl-DL-Leucine in Niemann-Pick type C: A case series

Bremova, T., Malinova, V., Amraoui, Y., Mengel, E., Reinke, J., Kolnikova, M., and Strupp, M.


Demonstrates that Acetyl-DL-Leucine treatment is associated with improvements in ataxia and quality of life in patients with Niemann-Pick Disease Type C.

Neurology 85, 1368-1375 (2015).

Niemann-Pick disease type C1 is a sphingosine storage disease that causes deregulation of lysosomal calcium

Lloyd-Evans, E., Morgan, A. J., He, X., Smith, D. A., Elliot-Smith, E., Sillence, D. J., Churchill, G. C., Schuchman, E. H., Galione, A., and Platt, F. M.

Defines the rare lysosomal storage disease Niemann-Pick type C as the first human disease resulting from endolysosomal calcium dysfunction, and demonstrates that correction of cellular calcium homeostasis can improve disease symptoms.

Nature Medicine 14, 1247-1255 (2008).

An anecdotal report by an Oxford basic neuroscientist: effects of acetyl-DL-leucine on cognitive function and mobility in the elderly.

Platt, F., Strupp, M.

A case study demonstrating that, in addition to being of benefit in patients with cerebellar ataxia and Niemann-Pick disease type C, acetyl-DL-leucine can increase mobility and quality of life in a healthy elderly subject.

Journal of Neurology 263, 1239-40 (2016)

A primer of NAADP-mediated Ca2+-signalling: from sea urchin eggs to mammalian cells.

Galione, A.

A review highlighting the pathophysiology of the NAADP-TPC-lysosomal calcium signalling mechanism in various cells and organs.

Cell Calcium 58, 27-47 (2014).

Molecular mechanisms of endolysosomal Ca2+ signalling in health and disease.

Morgan, A. J., Platt, F. M., Lloyd-Evans, E. & Galione, A.

A review outlining the concept of the endolysosomal system as a regulated calcium store.

Biochem. J. 439, 349-374 (2011).

Identification of a chemical probe for NAADP by virtual screening.

Naylor, E., Arredouani, A., Vasudevan, S. R., Lewis, A. M., Parkesh, R., Mizote, A., Rosen, D., Thomas, J. M., Izumi, M., Ganesan, A., Galione, A., and Churchill, G. C.

A paper outlining the use of in silico drug discovery software to generate a selective inhibitor of NAADP/Two-Pore Channel-mediated calcium signalling.

Nat. Chem. Biol. 5, 220-226 (2009).

Sphingolipid lysosomal storage disorders.

Platt, F. M.

A recent review of the mechanisms underlying lysosomal storage diseases.

Nature 510, 68-75 (2014).

Expression of Ca2+-permeable Two-Pore Channels (TPC) rescues NAADP-signalling in TPC-deficient cells.

Ruas, M., Davis, L. C., Chen, C. C., Morgan, A. J., Chuang, K. T., Walseth, T. F., Grimm, C., Garnham, C., Powell, T., Platt, F. M., Biel, M., Wahl-Schott, C., Parrington, J., and Galione, A.

Recent paper showing that Two-Pore Channels are integral components of the NAADP-sensitive Ca2+ release mechanism in the endolysosomal system.

Embo J. 34, 1743-58 (2015)

Shape-based reprofiling of FDA-approved drugs for the H1 histamine receptor.

Vasudevan, S. R., Moore, J. B., Schymura, Y., and Churchill, G. C.

Discovery of new anti-histamines by computational methods.

Journal of Medicinal Chemistry 55, 7054-7060 (2012).

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