ABSTRACT
The creation of red blood cells for the blood transfusion markets represents a highly innovative application of regenerative medicine with a medium term (5-10 year) prospect for first clinical studies. This article describes a case study analysis of a project to derive red blood cells from human embryonic stem cells, including the systemic challenges arising from (i) the selection of appropriate and viable regulatory protocols and (ii) technological constraints related to stem cell manufacture and scale up to clinical Good Manufacturing Practice (GMP) standard. The method used for case study analysis (Analysis of Life Science Innovation Systems (ALSIS)) is also innovative, demonstrating a new approach to social and natural science collaboration to foresight product development pathways. Issues arising along the development pathway include cell manufacture and scale-up challenges, affected by regulatory demands emerging from the innovation ecosystem (preclinical testing and clinical trials). Our discussion reflects on the efforts being made by regulators to adapt the current pharmaceuticals-based regulatory model to an allogeneic regenerative medicine product and the broader lessons from this case study for successful innovation and translation of regenerative medicine therapies, including the role of methodological and regulatory innovation in future development in the field.
Subject(s)
Erythrocytes/cytology , Inventions , Regenerative Medicine/legislation & jurisprudence , Animals , Biomedical Research , Cells, Cultured , Clinical Trials as Topic , HumansABSTRACT
AIM: The aim of the study was to evaluate the results of the use of flutamide at low doses for the therapy of the iperandrogenism in adolescents. METHODS: The study enrolled 35 young women with acne and irsutism; 31 had polycystic ovary syndrome (PCOS) and 4 periferic iperandrogenism. In other 8 young women, sexually active, the flutamide has been associated with the hormonal contraceptive. On the three young women with iperinsulinism it has been decided to associate the flutamide with the metformina. All the young women were checked each month for the liver functional. Before the beginning of the therapy the menstrual situation, the Body Mass Index (BMI), the Ferriman' s and Cremoncini's score, the ovary's ultrasound aspect, and the hormonal order were evaluated. Follow-up was made after three months and after six months after the beginning of the therapy with flutamide 62.5 mg/die. RESULTS: Only in 4 cases the therapy has been suspended due to collateral effects, soon regressed after one week of the treatment interruption. The results have demonstrated a overwhelming improvement of the peripheral symptoms of iperandrogenism in all patients. CONCLUSION: The authors hope that flutamide could enter in the list of medicines normally used to treat the beauty flaws of policistic acne and to restore a hormonal order associated to an effective contraception.
Subject(s)
Acne Vulgaris/drug therapy , Androgen Antagonists/therapeutic use , Flutamide/therapeutic use , Hirsutism/drug therapy , Hyperandrogenism/drug therapy , Polycystic Ovary Syndrome/drug therapy , Adolescent , Androgen Antagonists/administration & dosage , Androgen Antagonists/adverse effects , Body Mass Index , Child , Female , Flutamide/administration & dosage , Flutamide/adverse effects , Follow-Up Studies , Humans , Time Factors , Treatment Outcome , Young AdultABSTRACT
Ossifying lipomas independent of bone tissue are very rare. A literature review revealed that only few cases of ossifying lipoma independent of bone tissue have been reported. None of the cases have been reported in the international radiology literature. In addition, there are no reports concerning the MRI features of this entity. We describe CT and MRI findings in a case of ossifying lipoma of the parapharyngeal space.
