Advanced therapy medicinal products (ATMPs), which include gene therapy medicinal products, somatic cell therapy medicinal products and tissue-engineered products, are at the cutting edge of innovation and offer a major hope for various diseases for which there are limited or no therapeutic options. They have therefore been subject to considerable interest and debate. Following the European regulation on ATMPs, a consolidated regulatory framework for these innovative medicines has recently been established. Central to this framework is the Committee for Advanced Therapies (CAT) at the European Medicines Agency (EMA), comprising a multidisciplinary scientific expert committee, representing all EU member states and European Free Trade Association countries, as well as patient and medical associations. In this article, the CAT discusses some of the typical issues raised by developers of ATMPs, and highlights the opportunities for such companies and research groups to approach the EMA and the CAT as a regulatory advisor during development.

(Gut 2000;46:679-687)

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A previously reported effect of mechanically agitated dilutions of antiserum raised against IgE was investigated using the loss of metachromatic staining properties of human basophil leukocytes as a model. A series of 24 blind experiments was performed in which we determined the number of toluidine blue-stainable basophils after incubating with vortexed or non-vortexed dilutions of anti-IgE. Tenfold serial dilutions were used, in the range 10(21) to 10(30) (6.6 x 10(-26) to 6.6 x 10(-35) M anti-IgE). We found no evidence for a different effect of strongly agitated dilutions, compared to dilutions made with minimal physical agitation. In fact, in our hands no effect of extreme dilutions was shown at all. We conclude that the effect of extreme dilutions of anti-IgE, reported by Davenas et al., needs further clarification and that in this process the reproducibility of results between experimenters should be carefully determined.

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