The project is headquartered at the Muscular Dystrophy Research Center (CPDM) in Ribeirão Preto, São Paulo State, Brazil. This center was created as a result of a partnership between the Brazilian Association of Friends of Muscular Dystrophy (AADM) and the University of Ribeirão Preto (UNAERP).

The head of AADM and CPDM is Edna Maria Pupin. Edna is the mother of Murilo, a 26 year old man with DMD. She is also the "mother and heart" of this Fight in Brazil and to this research center.

The main and only goal of CPDM is to study muscular dystrophy. The University of Ribeirão Preto is the principal supervising university but other universities such as the University of Sao Paulo will be involved as the project becomes larger.

CPDM was initially built using funds contributed by parents who become associated with AADM. The equipment was purchased with financial support from the State of São Paulo Research Foundation (FAPESP) and Petrobras, a Brazilian oil company. At present, parents from Brazil make contributions to keep the project going.

NEW: April 2005, the Norwegian legacy "Erik Allums legat for Duchenne Muskeldystrofi" supported the Brazilian Project with NKR 150,000 (ca USD 24,000).  

The investigators team is supervised by Prof. João Bizario, PhD in Cellular and Molecular Biology, in Ribeirão Preto.

The research is exclusively destined to study new drugs and treatment to Duchenne/Becker Muscular Dystrophies.

Currently, the projects that are being developed at CPDM can be fitted in two principal areas:

I) Drug test:

Responsibel for this area is Prof. Lucélio Bernardes Couto, PhD in Pharmacy. 

l-couto@uol.com.br

All drugs that will be investigated have been approved by the FDA (Food and Drug Administration) but for other applications. The basic reason is that the pharmaceutical companies absolutely refuse to petition the U.S. FDA for diseases other than those already approved by FDA for that particular drug. Because these drugs are on the market there is a wealth of information for each of them. Each drug that will be tested has certain properties that we feel will help alleviate the symptoms of this terrible disease. These drugs will first be tested in mdx mice and GRMD (Golden Retriever Muscular Dystrophy) dogs. The pertinent pharmaceutical companies have already completed safety studies on such animals but refuse to divulge their data to us. If the animal testing shows positive results, clinical trial studies under the laws and guidelines of Brazil entities will be initiated in clinics around the world that will agree and sanction our protocol.

II) Cellular and molecular biology studies:

Responsibel for this area is Prof. Maria Cristina Ramos Costa, PhD in Biochemistry and Molecular Biology

mcrcosta@usp.br

We are currently establishing primary culture of myoblasts from mdx mice with the objective of transfecting these cells with constructions of dystrophin and/or utrophin, reinfuse them and observe the expression of the protein. We are studying changes in gene expression profile, using semiquantitative RT-PCR and western blotting, during the natural progression of mdx dystrophinopathy (or accelerated by compulsory physical exercise). Furthermore, we have standardized immunocytochemistry, immunohistochemistry, molecular biology and biochemistry protocols as additional tools for drug test projects.

See photos:

Since the early 1990s, steroids have been used to delay the progression of the disease but there are many adverse side effects. It is our contention that if steroids show efficacy then there must be other drugs that would provide successful treatments without the adverse side effects of steroids. The following are fruitful areas of research:

On behalf of Fight for a Future

Berit