Benserazide

The aim of the study is to assess impact of Fecal Microbiota Transfer (FMT) on clinical symptoms of Parkinson's disease. Assesment of tremor, slowness of movements and balance problems before and after FMT will be performed. The effect of FMT on frequency of constipations, which are common among Parkinson disease patients and have negative impact on quality of life and drug absorption will also be assessed. Detailed assessment of absorption of levodopa, which is the golden standard of treatment of Parkinson disease, is planned. It is planned to recruit 40 patients with diagnosis of Parkinson disease and indications for colonoscopy (constipations, age >50 years). Patients will be randomly assigned to the group receiving treatment with FMT or identically looking placebo. It will be administered to intestine during colonoscopy. Patients will be assessed by neurologist few times after the procedure. Psychological assessment and examination of gait and balance by physiotherapist is also planned. The last assessment will be performed after 12 months to see if the clinical effect can be observed for such a long time. The composition of the intestinal microbiota will be carefully assessed before and after the procedure in order to identify pathogens that may affect the course of the disease.

Beta-thalassemias and hemoglobinopathies are serious inherited blood diseases caused by abnormal or deficiency of beta A chains of hemoglobin, the protein in red blood cells which delivers oxygen throughout the body.The diseases are characterized by hemolytic anemia, organ damage, and early mortality without treatment. Increases in another type of (normal) hemoglobin, fetal globin (HbF), which is normally silenced in infancy, reduces anemia and morbidity. Even incremental augmentation of fetal globin is established to reduce red blood cell pathology, anemia, certain complications, and to improve survival.
This trial will evaluate an oral drug discovered in a high throughput screen, which increases fetal globin protein (HbF and red blood cells expressing HbF)and messenger ribonucleic acid (mRNA) to high levels in anemic nonhuman primates and in transgenic mice. The study drug acts by suppressing 4 repressors of the fetal globin gene promoter in progenitor cells from patients. The drug has been used for 50 years in a combination product for different actions - to enhance half-life and reduce side effects of a different active drug- and is considered safe for long-term use.
This trial will first evaluate 3 dose levels in small cohorts of nontransfused patients with beta thalassemia intermedia. The most active dose will then be evaluated in larger subject groups with beta thalassemia and other hemoglobinopathies, such as sickle cell disease.