To: nigel bates who wrote (1304 ) 7/26/2002 5:46:46 PM From: scaram(o)uche Respond to of 2243 I guess you're off to the beach. Have a great time. People want out, at any cost........table.finance.yahoo.com finance.yahoo.com finance.yahoo.com Biggest volume day in over two years, second or third in the history of the company. I like the looks of the cross-over study. If I were a patient, I'd consider brief periods of using OGT 918 on top of ceredase. Other random stuff....... Pediatrics 2002 Jun;109(6):1170-3 Gaucher's disease and fatal hepatic fibrosis despite prolonged enzyme replacement therapy. Perel Y, Bioulac-Sage P, Chateil JF, Trillaud H, Carles J, Lamireau T, Guillard JM. Department of Pediatrics, Onco-Hematology Unit, University Hospital, 33076 Bordeaux, France. yves.perel@chu-bordeaux.fr We report on the case of a girl with type 1 Gaucher's disease, treated from age 9 to 15 with high-dose enzyme replacement therapy. This treatment did not avert the development of an extensive mutilating hepatic fibrosis warranting a liver transplantation, which was followed by death. In some cases of Gaucher's disease, alternative strategies such as fractionated or further increased ERT, gene therapy, or glucosyltransferase inhibitor should be explored. Blood 1999 Mar 15;93(6):2081-8 Immunosurveillance of alglucerase enzyme therapy for Gaucher patients: induction of humoral tolerance in seroconverted patients after repeat administration. Rosenberg M, Kingma W, Fitzpatrick MA, Richards SM. Genzyme Corp, Framingham, MA 01701-9322, USA. Alglucerase, a macrophage-targeted enzyme replacement therapy for Gaucher disease, has been successfully used for several years to improve clinical symptoms and reverse disease progression. As part of an immunosurveillance program, 1,122 Gaucher patients were monitored for antibody response to glucocerebrosidase, the active component of alglucerase. Seroconversion was detected in 142 patients (12.8%) by enzyme-linked immunosorbent assay (ELISA) and confirmed by radioimmunoprecipitation. The majority (75%) of the seroconverted population had no detectable levels of circulating inhibitory antibody as assessed by in vitro inhibition of enzymatic activity of the therapeutic molecule. Of the remaining patients with putative inhibitory antibodies, the majority had only low levels of serum inhibitory activity, which was transient. A very small number of patients were identified as developing true neutralizing antibodies, as defined by the development of antibodies that impacted clinical efficacy. Many of the patient antibody responses were also diminished with time. Eighty-two of the 142 seroconverted patients have stopped producing antibody to the molecule and appear tolerized. The mean time for humoral tolerization was 28 months from initiation of therapy. Of 64 seroconverted patients followed for at least 30 months of therapy, the tolerization rate was 93%. These results show that although 12.8% of the patients on therapy developed antibodies to the molecule, 90% of these patients became tolerized over time.