A Leap Forward to Cure a Neglected Disease

$6,500
Raised
100%
Funded on 2/12/14
Successfully Funded
  • $6,500
    pledged
  • 100%
    funded
  • Funded
    on 2/12/14

About This Project

The World Health Organization estimates that as many as 11 million people worldwide are infected with the parasite that causes the deadly Chagas disease. Chagas is a leading cause of heart failure in Latin America. In the U.S., Chagas appears to be increasing in prevalence due to migration of people from endemic areas. Our findings show that a drug already in use for other purposes stop the parasite and the progressive heart disease that causes death in the patients.

Ask the Scientists

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What is the context of this research?

Our laboratory is exploring new uses for abandoned and approved therapeutic drugs to be used in chronic fibrotic diseases, such as the heart fibrosis or scaring produced in chronic chagas disease.

Chagas disease is caused by the parasite T. Cruzi. Recent studies have demonstrated that the protein TGFβ1 plays a major role in the infection by the parasite and development of the chronic disease in humans.

We have discovered that an FDA -approved drug can inhibit TGFβ1-mediated fibrosis and at the same time inhibit the life cycle of this parasite in the host. Moreover, the patent on this drug is expiring, which may make it more affordable in developing countries.

What is the significance of this project?

Chagas disease is recognized by the World Health Organization as one of the world’s most neglected tropical diseases. It is caused by the intracellular parasite Trypanosoma cruzi, and is a widely distributed debilitating human illness, affecting millions of people in Central and South America. Chagas is a leading cause of heart failure in Latin America. Currently more than 400,000 people are chronically infected in the USA.

Although more than a century has passed since the discovery of Chagas disease, the development of an effective treatment still eludes us. No therapeutic approach to target Chagas-induced heart scarring, which leads to death, is presently available.

The process of developing a new therapeutic drug is long and difficult. The average length of time from target discovery to approval of a new drug is more than 13 years, and the failure rate exceeds 95 percent. However, the drug re-purposing that we are proposing here is a more cost-effective and expedient approach to attain worldwide access to effective treatment.

We have discovered that daily administration of an established and well-tolerated drug that is on the market for other purposes inhibits the infection and proliferation of the parasite in a mouse model of Chagas disease, and prevents the consequent heart scarring and death. The drug is able to inhibit a common pathway that is involved in the progression of the parasite infection and also heart scarring. Importantly, this drug is going off patent protection, which will make it much less expensive.

The downside of the patent expiration is that it is not economically favorable for companies to invest in research on drugs that have gone off patent protection. At this juncture, modest support will allow us to complete our work with the animal model, as the final obstacle to overcome before the drug can be used in clinical trials for people afflicted with Chagas disease.

What are the goals of the project?

We have data showing the efficiency of the of an FDA-approved drug in infected cells and animal models of Chagas. The drug is available on the market in tablets for oral administration, including pediatric formulation. This crowdfunding proposal will allow us to determine the most efficient dose of the oral-administrated drug in animal models of Chagas disease.
After meeting this goal, we will be able to propose a clinical trial for patients afflicted with chronic chagas disease.

This proposal will involve a collaborative effort between Dr. Sanchez at Tulane University, in New Orleans, and the adjunct Tulane Professor, Dr. Eric Dumonteil. Dr. Dumonteil is full professor at the Universidad Autonoma de Yucatan, Mexico.
Dr. Sanchez’s laboratory has a focus on scarring and Dr. Dumonteil is an expert on Chagas disease.

Budget

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The funding will support collaboration between Dr. Sanchez's lab. and Dr. Dumonteil. No salary support is requested for the investigators. Rather, the budget presented here will be used to cover supply costs, animal care expenses and reagents for this proposal.

Endorsed by

This is a fantastic project that should shorten the path toward a new treatment for Chagas disease.
This research will help to alleviate the severity of cardiac symptoms in chronic Chagas disease cases. This could be a huge step toward alleviating the disease burden in individuals currently infected with the parasite.

Meet the Team

Cecilia G. Sanchez
Cecilia G. Sanchez

Team Bio

Dr. Sanchez is an assistant professor in the Department of Medicine, Division of Pulmonary Diseases. Her laboratory focuses on understanding the mechanisms of tissue fibrosis. Her goal is to develop new therapeutic paradigms for chronic fibrotic disorders, including idiopathic pulmonary fibrosis, scleroderma and chagasic cardiomyopathy. Dr. Sanchez's laboratory is interested in the re-purposing of drugs already available on the market to treat other diseases. Dr. Sanchez was born in the U.S., but spent her childhood in Colombia, where Chagas is endemic. Having undergone multiple surgeries during the course of her childhood, she has always been interested in the ways in which science and medicine can be used to better people's lives.

Cecilia G. Sanchez

Dr. Sanchez is an assistant professor in the Department of Medicine, Division of Pulmonary Diseases. Her laboratory focuses on understanding the mechanisms of tissue fibrosis. Her goal is to develop new therapeutic paradigms for chronic fibrotic disorders, including idiopathic pulmonary fibrosis, scleroderma and chagasic cardiomyopathy. Dr. Sanchez's laboratory is interested in the re-purposing of drugs already available on the market to treat other diseases. Dr. Sanchez was born in the U.S., but spent her childhood in Colombia, where Chagas is endemic. Having undergone multiple surgeries during the course of her childhood, she has always been interested in the ways in which science can be used to better people's lives.


Project Backers

  • 8Backers
  • 100%Funded
  • $6,500Total Donations
  • $53.14Average Donation
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