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publicado em 11/09/2013 às 11h48:00
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Drug developed in Brazil is hoping against sickle cell anemia

Created by researchers at UNESP, the new drug showed no toxic effects of the drugs currently used

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A drug developed by researchers at UNESP to relieve the symptoms of sickle cell anemia unites the benefits of thalidomide and chemotherapy hydroxyurea already used to treat chronic disease without showing any toxic effects of the original drugs.

The molecule, patented with the name Lapdesf1 showed good results in tests with mice made at Unicamp. Scientists from both institutions are now seeking partnership with the pharmaceutical industry to conduct the first human trials.

The research represents a breakthrough for the treatment of sickle cell anemia, a disease considered extremely neglected, and help reduce various symptoms present in patients, such as pain and inflammation, said researcher Jean UNESP Araraquara Leandro dos Santos, one of the creators of the new product.

Hydroxyurea is currently one of the most widely used drugs in the treatment of sickle cell disease to be able to increase the production of another type of hemoglobin, known as fetal hemoglobin (present in most life period uterus). High levels of fetal hemoglobin decrease the polymerization defective red blood cells and reduce the risk of vascular occlusion.

Like any chemotherapy, however, hydroxyurea has adverse effects. Besides causing nausea, abdominal pain and headache, dizziness, drowsiness and seizures, may also decrease the production of bone marrow cells. It can also affect reproductive cells and lead to infertility.

Already thalidomide, originally used as a sedative and antiemetic (anti-nausea), was withdrawn from the market worldwide in the 1960s after causing an epidemic of babies born with malformations. Was subsequently reintroduced in the 1990s for the treatment of cancer, leprosy, lupus and AIDS. In Brazil, the use of thalidomide is controlled and the drug is produced by public laboratories and provided by the Unified Health System (SUS).

According to Santos, in the case of sickle cell anemia, thalidomide is interesting for their anti-inflammatory effects. "We took thalidomide subunit responsible for the beneficial anti-inflammatory effects and added to the molecule mechanism of action of hydroxyurea, related to the ability to donate nitric oxide mediator responsible for increased fetal hemoglobin. Toxicity trials done to date, the Lapdesf1 had none of the negative effects of the original drugs, "he said.

<b> preclinical tests </ b>

After preliminary experiments in vitro have demonstrated the therapeutic potential of Lapdesf1, the group of UNESP partnered with researchers from the Center of Hematology (Blood Bank) at UNICAMP for testing in mice genetically modified to develop a framework very similar to sickle cell anemia.

The tests, conducted by researchers Carolina Lanaro Hairstyle and Carla, are being carried out under a Thematic Project at Unicamp coordinated by Professor Fernando Ferreira Costa.

"The tests were performed in the laboratory of Georgia Regents University in the United States because researchers there were the most appropriate animal model for this type of test," said Hairstyle.

About 20 rats, complete 3 months of age were divided into two groups. Half were treated with Lapdesf1 for two months. The other half received only placebo.

"In the treated group, the amount of fetal hemoglobin doubled after this period similar to the results obtained with hydroxyurea. Since the amount of inflammatory cytokines had decreased more than 70% similar to the effect obtained with dexamethasone (steroid also used against inflammation). None animals showed adverse effects, "said Hairstyle.

While waiting for the possibility to start the first human trials, the group tries to unravel the Unicamp in vitro and through new animal testing the exact mechanism of action of the new drug.

With information from UNESP

   Palavras-chave:   Sickle cell anemia    Thalidomide    Hydroxyurea    Unicamp    Unesp    Jean Leandro dos Santos    Health   
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