In the past few years, substantial preclinical and experimental advances have been made in the treatment of the severe monogenic skin blistering disease epidermolysis bullosa (EB). Promising approaches have been developed in the fields of protein and cell therapies, including allogeneic stem cell transplantation; in addition, the application of gene therapy approaches has become reality. The first ex vivo gene therapy for a junctional EB (JEB) patient was performed in Italy more than 8 years ago and was shown to be effective. We have now continued this approach for an Austrian JEB patient. Further, clinical trials for a gene therapy treatment of recessive dystrophic EB are currently under way in the United States and in Europe. In this review, we aim to point out that sustainable correction of autologous keratinocytes by stable genomic integration of a therapeutic gene represents a realistic option for patients with EB.
Neoadjuvant chemoradiation therapy (NACRT) is increasingly used in patients with a potentially or borderline resectable pancreatic ductal adenocarcinoma (PDA) and it has been shown to improve survival and reduce locoregional metastatic disease. It is rare for patients with PDA to have a pathological complete response (pCR) to NACRT, but such patients reportedly have a good prognosis. We report the clinicopathological findings of two cases of pCR to NACRT in PDA. Both patients underwent pancreatectomy after NACRT (5-fluorouracil, mitomycin C, cisplatin, and radiation). Neither had residual invasive carcinoma and both showed extensive fibrotic regions with several ducts regarded as having pancreatic intraepithelial neoplasia 3/carcinoma in situ in their post-therapy specimens. It is noteworthy that both patients had a history of a second primary cancer. They both had comparatively good outcomes: one lived for 9 years after the initial pancreatectomy and the other is still alive without recurrence after 2 years.
Tuberculosis is sometimes perceived as a feared killer of the past but is still a dreadful disease of mankind. Mycobacterium tuberculosis multiplies inside white blood cells known as macrophages. In infected people who don’t develop the Tuberculosis, the immune system either the bacteria or impairs bacterial multiplication. The exact mechanisms behind this are not known in detail, hampering the development of effective vaccines and treatments of the disease. Why the disease is manifested in some individual, but not in others, is not completely understood.The recent study shows that a molecule called SOCS3 is required for control of the infection. The discovery was done using an experimental infection of mice genetically modified so that they do not express SOCS3 in different immune cells. These mice were dramatically susceptible to the infection with Mycobacterium tuberculosis. SOCS3 could be a new target for vaccines to improve the protection against Tuberculosis.(Presented at the 1901st Meeting, April 14, 2015)