Clinical Trial Support

Funding for rare disease clinical trial was granted in 2008, and the project “Treatment for Aromatic L-amino acid decarboxylase (AADC) deficiency”, was launched at National Taiwan University, department of medical genetics in 2010. The team would like to make a special thanks to Dr. Y.F. Chang, the President of Evergreen Group, whose sponsorship has played an important role in shedding the light to the drug development of AADC. TFRD entrusted Powell Gene Therapy Center in Florida, USA, to generate the viral vectors for the study of AADC. These viral vectors are then donated to National Taiwan University Hospital for clinical trials. The research team has completed phase III trial to ensure the safety and stability of the vectors. There are 10 AADC patients participated in the clinical trials currently; the work that has been done not only brings hope to the patients but also establishes the milestone of genetic treatment in Taiwan.

The foundation has accepted and sponsored a project at Taichung Veterans General Hospital, “Efficacy assessment of using Eculizumab in treating Kohlmeier-Degos Disease,” in 2015. The Dego’s disease is presumably caused by viral infection, dysfunction of immune system, or others. A few literatures have indicated the use of Eculizumab for treatment, and doctors have found improvement in some patients. However, since Dego’s disease is neither on the list of rare diseases nor the Eculizumab’s indication, the treatment expenses are not covered by National Health Insurance. Patient families experience tremendous financial pressures. To support Taichung Veterans General Hospital’s clinical trials on the treatment of Eculizumab on Dego’s disease, and to protect patients’ rights, the foundation has granted one year funding for the clinical trials.