Spanish researchers have cured a disease using pseudo-embryonic stem cells for the first time.

The team, led by scientists from Barcelona's Centre for Regenerative Medicine, has corrected a genetic defect in cells belonging to three sufferers of Fanconi's anaemia, a serious blood disease.

But the process was carried out in the laboratory and there is still a long way to go before clinical trials on humans can be envisaged.

"We are not curing Fanconi's anaemia, we are curing it in vitro," said Anna Veiga, Director of the stem cell bank at the centre and co-author of an article published in Nature recently. "So we are demonstrating that is possible and that when we have overcome the other obstacles, we can start talking of a cure."

The researchers used gene therapy to eliminate the disease from skin cells taken from patients then converted them into induced pluripotent stem cells or pseudo-embryonic cells. These act like embryonic stem cells and have a similar capacity for changing into different kinds of cells, a process known as cell differentiation.

The process makes use of known techniques but combines them for the first time. It holds the promise of producing stem cells specific to each patient and, as these are generated from the patient's own cells, the risk of rejection would be eliminated.

Tests have now begun on rats but Veiga emphasises that much still has to be done before tests can begin on humans. Four genes are used to transform the adult cells into pseudo-embryonic ones and one of these can cause cancer. Until the technique is improved and cells can be produced in proper conditions, clinical trials will have to wait.

Currently the only example of a clinical trial being carried out by a reputable research centre is in the US where scientists are looking for safe ways of reversing spinal injury.
But unorthodox treatments are being offered in many countries by organisations with dubious scientific credentials and Veiga believes the general public should be wary of their claims.

"If you go online, you will find all kinds of organisations promising miracle cures for serious diseases like Alzheimers or Parkinsons - they even claim they will get rid of your wrinkles - but most have no scientific basis and are done without any quality control," she said.

She cites the case of a young Israeli man who twice underwent treatment with foetal stem cells in Russia for neuronal disease. Two years later Israeli doctors found him to have several brain tumours.

"You can't just give someone stem cells and say if they work great, if not then tough," said Veiga. "If you inject them into the heart of someone who has had a heart attack, you need to know what those cells will do - will they stay put and turn into cardiac cells? Or will they do something else?"

rebecca.warden@uw-news.com

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