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Discovery In "Bubble Boy" Disease Gene Therapy

om gene therapy," Shou said. "Although several XSCID patients treated elsewhere with gene therapy have developed leukemia, patients with other types of genetic blood disorders have not developed cancer following gene therapy. So our discovery suggests that patients with XSCID are especially vulnerable to this serious complication but that people with other genetic blood diseases may not be at as high a risk."

Sorrentino's team discovered a clue to what makes XSCID mouse models--and children with XSCID--particularly vulnerable to leukemia caused by gene therapy. Specifically, they analyzed the bone marrow cells of XSCID mice to determine the size of the population of primitive stem cells that had not yet produced B and T cells. This population of cells is the target for gene therapy, Sorrentino noted. The researchers found that this population of primitive stem cells was significantly expanded.

"The many primitive cells stalled in their development greatly increases the odds that gamma C genes will insert themselves into oncogenes in at least a few of them," Sorrentino said. "That is probably is a major reason that XSCID patients are particularly vulnerable to developing leukemia from gene therapy."

In addition to this large population of primitive cells in XSCID patients, the ability of gamma C to insert into an oncogene and act as an on-signal contributes to the risk of gene therapy for this immune deficiency disease, Sorrentino said. Another risk factor is the lack of the Arf gene to put the brakes on the overgrowth of cells that turn cancerous because of gene therapy.

"Without the Arf gene to force cancerous cells to commit suicide, these cells continue to divide and cause leukemia," he said. "This suggests that the patients with XSCID in France who developed leukemia following gene therapy may also have abnormalities in either Arf or another tumor suppressor gene."

The apparent lower risk of cancer
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