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Trial Underway Testing Genetic Disease Stem Cell Therapy

Red blood cell (science360.gov)

Red blood cell (science360.gov)

A clinical trial is underway testing a therapy based on a patient’s own stem cells to treat childhood cerebral adrenoleukodystrophy (CCALD), a rare genetic disease affecting mainly boys. The trial began treating the first of an expected 15 patients testing the therapy made by bluebird bio, a biotechnology company in Cambridge, Massachusetts and Paris, France.

CCALD is a genetic disorder preventing the metabolism of some complex fatty acids, resulting in the build-up of fatty acids in brain cells, which leads to progressive loss of myelin, an insulating material around nerve cells. The condition primarily affects boys, beginning at ages 4 to 8, and results in severe loss of neurological functions and is often fatal. All types of adrenoleukodystrophy affect about 1 in 20,000 people, with CCALD comprising 30 to 40 percent of the total.

The only current treatment for the disease — which was the subject in the 1992 feature film Lorenzo’s Oil — is a transplant of healthy blood-forming stem cells from siblings or non-sibling donors, but non-siblings with acceptable stem cells are available for less than 30 percent of patients.

The company bluebird bio — the name is spelled in all lower-case characters — developed its technology based on the research of the company’s co-founder Philippe Leboulch, a researcher and lecturer at Paris University School of Medicine, Harvard Medical School, MIT, and Brigham and Women’s Hospital in Boston. The technology takes a patient’s own blood-forming stem cells from bone marrow and cultures them outside the body to create healthy replacement genes for the mutated stem cells causing the disease. The stem cells with the healthy genes are then transplanted back into the patient.

bluebird’s one-time treatment for CCALD, called Lenti-D, was tested in an early-stage/proof-of-concept study with two patients, who according to the company  have enjoyed its clinical benefits for five years, with another two patients also receiving Lenti-D treatments. The new clinical trial, at Massachusetts General and Boston Children’s Hospital will administer Lenti-D generated from the stem cells of the 15 boys, who will be followed for 24 months for signs of major functional disabilities, as well as signs of potential safety issues.

In June 2013, bluebird bio filed for its initial public offering, raising some $101 million. The company is also developing therapies for the inherited blood disorders beta thalassemia and sickle cell disease. In addition, bluebird bio is in a partnership with Celgene Corporation to develop treatments for cancer based on a patient’s own genetically modified immune cells.

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