Novel Trypanocidal Compounds for the Treatment of Stage 1 and 2 HAT.

Period of Performance: 04/25/2016 - 03/31/2017

$300K

Phase 1 SBIR

Recipient Firm

Medisynergics, LLC
FARMINGTON, CT 06032
Principal Investigator

Abstract

? DESCRIPTION (provided by applicant): Human African Trypanosomiasis (HAT) is a disease caused by a parasitic organism, trypanosoma brucei. It is often referred to as sleeping sickness because of the symptoms that emerge in Stage 2 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 1, form of HAT. Symptoms include fever, headaches, joint pains and itching and severe swelling of lymph nodes. According to WHO, an estimated 10,000 deaths occurred in 2010 from the neurological, Stage 2, 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 2 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 2 of the disease, have been lacking. It is the goal of MediSynergics, LLC to discover new agents that will broadly target Stage 1 and Stage 2 HAT infections, with an improved safety and efficacy profile. We have recently identified MS-08 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 72 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.