Gene Therapy Cures Boy with Sickle Cell Disease
Gene Therapy has been around for three decades and has been lauded as a future treatment for almost every disease from cancer to cystic fibrosis. So far, it’s use has been limited to rare diseases that affect tens of thousands, not hundreds or millions.
But recently, a young boy’s gene therapy treatment appears to have stopped the symptoms of the disease There are hundreds of thousands of people who suffer from very painful symptoms worldwide – 100,000 alone in the United States – so this is very welcome news.
Currently, people with the disorder are given blood transfusions to clear these painful blockages and prevent new ones. Bone marrow transplants can treat the disease, but matching donors can only be found for around 10 per cent of people with the condition. With gene therapy, strands of DNA are used to compensate for a person’s malfunctioning genes. Now a team in France seems to have developed a treatment that would work for everyone with the disorder.
“The patient is now 15 years old and free of all previous medication,” says Marina Cavazzana at the Necker Children’s Hospital in Paris, who led the team. “He has been free of pain from blood vessel blockages, and has given up taking opioid painkillers.”
Cavazzana is confident these benefits will last. “All the tests we performed on his blood show that he’s been cured, but more certainty can only come from long-term follow-up.” She says her team has treated seven other patients, who are showing “promising” progress. If the treatment proves successful in larger trials, it could bring gene therapy into widespread use.
The successful treatment is also good news that gene therapy can now help cure genetic diseases that affect large parts of the world’s population and not just rare diseases. The downside is that its expense may limit its use to richer nations. “We should be realistic in remembering that there are hundreds of thousands of sickle cell patients in less developed countries, and that the therapy is not easily exportable or adaptable to countries with less well-developed health systems,” says Stuart Orkin at Harvard Medical School.