First drug for the Parkes Weber Syndrome

INTRODUCTION:

The Parkes Weber syndrome (PWS) is a rare, congenital vascular malformation, characterized by arteriovenous fistulas, varicose veins and bone and soft tissue hypertrophy leading to limb enlargement. The treatment of PWS patients is mainly symtomatic. There is an ever-existing need to develop a safe and effective pharmacological PWS therapy.

TECHNOLOGY (INVENTION) DESCRIPTION:

New protoescigenin derivative, 28-O-{[1-(4-carboxyphenyl)-1H-[1,2,3]triazol-4-ylprotoescigenin, exhibits advantageous pharmacological properties, especially with respect to PWS. Its efficacy was demonstrated in a rat model of multi-symptomatic PWS by showing strong beneficial effects on thigh circumference, muscle weight and femoral bone length measurements as well as blood vessels and muscles as assessed by histopathological analyses. The molecule exhibits no toxicity and therefore it is a promising candidate for a pharmaceutical. The cellular and molecular basis for the therapeutic effectiveness of the molecule pointes is probably determined by its strong anti-inflammatory endothelial effects.

ADVANTAGES OVER EXISTING SOLUTIONS:

Currently, there is no effective pharmacological therapy for PWS patients. The available treatment is mainly symptomatic. Compression therapy is used to reduce symptoms of chronic venous insufficiency and lymphatic edema. In selected cases invasive procedures are performed. Surgical treatment is difficult and may require several intravascular procedures, such as embolization, sclerotherapy or classic open operations involving arteriovenous fistula ligation. In severe cases of ischemic extremities amputation is the only therapy.

DEVELOPMENT STATUS (STAGE):

Chemistry: laboratory scale synthesis Preclinical: in vitro (human endothelial cell) and in vivo (rat PWS model)

PUBLICATIONS:

Rat Model of Parkes Weber Syndrome. Krzysztof Bojakowski, Gabriela Janusz, Iwona Grabowska, Oliwia Zegrocka-Stendel, Agnieszka Surowiecka-Pastewka, Magdalena Kowalewska, Dorota Maciejko, Katarzyna Koziak. 2015, PLoS ONE 10(7)

IP PROTECTION STATUS:

granted patents: EP3061765B1 and EP2939534B1

TECHNOLOGY / IP OWNERS :

Medical University of Warsaw - the sole owner