MEDISYNERGICS, LLC — Department of Health and Human Services SBIR Phase I: R

Description

DESCRIPTION provided by applicant Human African Trypanosomiasis HAT is a disease caused by a parasitic organism trypanosoma brucei It is often referred to as andquot sleeping sicknessandquot because of the symptoms that emerge in Stage patients The disease exists in two forms depending on the causative parasite Trypanosoma brucei gambiense or Trypanosoma brucei rhodesiense T b gambiense is found in central and western Africa and causes a chronic condition that can remain in a passive phase for months or more before symptoms emerge T b rhodesiense is found in southern and eastern Africa where symptoms of the infection are more virulent and faster developing than T b gambiense Approximately one half million inhabitants of sub Saharan Africa are infected each year by the hemolymphatic Stage form of HAT Symptoms include fever headaches joint pains and itching and severe swelling of lymph nodes According to WHO an estimated deaths occurred in from the neurological Stage form of HAT Symptoms are generally more severe including confusion reduced coordination and disruption of the sleep cycle progressive mental deterioration coma and death Damage as a result of Stage HAT is irreversible Only a fraction of all infected patients are adequately treated using available therapeutic agents that are not well tolerated difficult to administer and costly for patients and health care providers Newer more effective treatments particularly for Stage of the disease have been lacking It is the goal of MediSynergics LLC to discover new agents that will broadly target Stage and Stage HAT infections with an improved safety and efficacy profile We have recently identified MS as a novel hit from our compound library that is based on a marketed drug which crosses the blood brain barrier It can eliminate Trypanosoma brucei parasites within hours in an in vitro assay It is expected that any drug which arises from our research will be safe for patients of any age be conveniently administered orally or intramuscularly on a once daily basis and provide sustained protection at the lowest cost per patient PUBLIC HEALTH RELEVANCE Human African Trypanosomiasis HAT has caused periodic major epidemics in developing countries and has defied elimination Diagnosis of Stage HAT is not unambiguous and treatment options are severely limited therefore availability of a medication that could be safely and efficaciously administered soon after exposure might increase patient compliance and reduce or eliminate further transmission of the disease A healthier population in the affected regions of Africa could well lead to economic and life style improvements for these people and reduce the risk of spreading the disease to other regions of the world including the U S