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Congenital heart disease infants treated with adult stem cells.

BALTIMORE, Md., December 20, 2016 -- In a randomized clinical study conducted among babies with congenital heart defects, researchers here have begun testing the use of adult stem cells from bone marrow to treat children with hypoplastic left heart syndrome (HLHS).

University of Maryland School of Medicine (UM SOM) surgeons are injecting the cells into the babies' hearts during open-heart operations.

The Interdisciplinary Stem Cell Institute (ISCI) at the University of Miami Miller School of Medicine is supplying the stem cells.

Even with extensive surgical treatments, HLHS babies still do not have optimal outcomes. The researchers hope the cells will increase the babies' chances of survival.

HLHS limits the heart's ability to pump blood from the heart to the body.

The premise of this clinical trial is to boost or regenerate the right ventricle, the only ventricle in these babies, to make it pump as strongly as a normal left ventricle. The researchers hope the therapy will be "a game-changer" for these patients.

The first two patients, who were both four months old when the stem cells were injected, are doing well after their surgery, physicians said.

Allogeneic stem cells can be used in other human beings without creating an immune response, which could cause the body to reject the cells.

The cells are a type of adult stem cell (found in both children and adults) that can develop into tissue- or organ-specific cells. Mesenchymal stems cells (MSCs) can be harvested in advance, expanded in culture, and stored for use later.

The allogeneic nature of the MSCs makes it possible for stem cells from one bone marrow donor to provide all the stem cells for this study. Researchers elsewhere are taking a different approach to strengthen the HLHS heart, with autologous cells, stem cells taken from the HLHS patient's own umbilical cord, for use in that specific patient.

In adult patients, MSCs in the heart have been shown to reduce scar tissue, reduce inflammation, cause new small vessels to grow, and stimulate the heart to regenerate itself, causing heart muscle cells and cardiac stem cells to grow.

Current HLHS treatment options are either a heart transplant or a series of three open-heart reconstructive surgical procedures to connect the left and right sides of the heart. However, even with a transplant or the reconstructive surgical series, children with HLHS have an average five-year survival of only 50-60 percent.

In this Phase 1 safety and efficacy study, allogeneic MSCs are injected into the heart muscle during the second of the three reconstructive surgeries, typically performed at approximately four months of age.

A total of 30 patients with HLHS will be enrolled in the study.

Fifteen patients will receive six-to-eight stem cell injections each, based on the size of the heart, while 15 control patients will not receive the cells.

This is an open-label trial, in which researchers and participant families will know whether or not the cells are administered.


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Title Annotation:In The Clinic...
Publication:Stem Cell Research News
Article Type:Clinical report
Date:Jan 16, 2017
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