Sickle cell disease should be treated with a half-matched donor approach, Elizabeth Tracey reports

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Sickle cell disease was thought to be cured using gene therapy, but longer term follow up reveals that while the condition improves many are not cured. Now a new approach developed at Johns Hopkins and around the world uses ‘half-matched’ donors, near relatives of a person with sickle cell, to cure the disease in most. Richard Brodsky, a sickle cell expert at Hopkins and one of the developers, explains.

Brodsky: Gene therapy becomes only relevant to people who you absolutely can't find a donor for. Why would you offer gene therapy to a 25 year old woman when you can do an approach that is 1/10th the cost, cures the disease and has an excellent chance of preserving her fertility? There's no good reason to be offering gene therapy to patients. The whole point of the gene therapy gene editing was because of the 1980s only 10% of patients were eligible for a transplant.        :32

Brodsky notes that most people with sickle cell disease are eligible. At Johns Hopkins, I’m Elizabeth Tracey.