The essence of the idea is an usage of new carriers for delivery of active substance – daidzein(DZ). It is an isoflavonoid compound of plant origin(found in legumes) which inhibits glycosaminoglycanes(GAGs) synthesis. Obtained carriers increase drug bioavailability and reduce side effects. Our initial studies revealed reduced GAG concentration in Human Skin Fibroblasts(HSF) without negative influence on cell populations. Small stimulation of HSF proliferation was noticed. Free daidzein administration results on focal distribution in fibroblasts cells, because of its hydrophobic character.
After administration of daidzein with developed carriers, regular cell dispersion of active substance was observed. This proves free carriers penetration and their even distribution into HSF cytoplasm. Stability of carriers systems under different physiological conditions was investigated with good results. Obtained carriers’ components are capable of reacting with GAGs. Introduction of DZ to their structure increases the expected result.
The delivery of daidzein with obtained carriers may help to achieve better outcomes of mucopolysaccharidosis type III(MPS III, Sanfilippo disease) therapy. MPS type III is a rare genetic lisosomal storage disease. The essence of this disorder is the impaired activity of GAG degrading enzymes. Due to the extremely low incidence of this disease, the therapy uses substances belonging to orphan drugs group. Because of this fact, MPS therapy is very expensive. Daidzein as a substance, which reduces the concentration of glycosaminoglycans could lower costs of MPS therapy and increase the quality of patients’ life.
Our goal at this stage of the project development is to find a partner who will be involved in the clinical implementation process. Good results predestine our materials for confirmation in a GLP laboratory. Further studies in vitro and in vivo are required. In the long term clinical trials are also necessary. An important part of daidzein pharmacokinetic studies is to investigate blood-brain barrier penetration.
We are looking for cooperation with:
- reagents providing partners
- research centers, working with animal models
- medical centers conducting therapy for patients with lisosomal storage diseases, especially MPS
Meet the Presenter: Iwona Gawlik MD (Faculty of Medicine Jagiellonian University)
Iwona Gawlik, graduate of Faculty of Medicine Jagiellonian University, Bachelor of Chemistry, Doctoral student at the Faculty of Chemistry Jagiellonian University. Since 2013 she has been involved in the synthesis and application of novel drug carriers for daidzein. This research has been carried out in Nanotechnology of Polymers and Biomaterials Group at the Faculty of Chemistry, Jagiellonian University in Kraków. She is a laureate of „Generacja Przyszłości” grant from the Ministry of Science and Higher Education of Poland.
Contact details: tel: +48 501-701-493, e-mail: iwona.gawlik [at ] uj.edu.pl