Commission demands reform on experimental cell therapies

The reputation of stem cell research is being undermined by unscrupulous clinics offering unproven therapies, says a report published last week.

The Lancet Commission on Stem Cells and Regenerative Medicine brought together a number of leading scientists to review progress and consider challenges facing the discipline.

'Regenerative medicine offers transformative potential for the future of patient care. But that potential could be jeopardised by low-quality research and a loss of public trust in stem cell science,' commented The Lancet's editor-in-chief Dr Richard Horton.

In a sense, the field has been a victim of its own hype. 'Excitement about possible treatments for incurable diseases like multiple sclerosis and Parkinson's disease, inflated by media reports, has led desperate patients and families to a proliferating number of poorly regulated clinics peddling untested and potentially ineffective therapies,' the report says.

Stem cell therapies have been shown to be extremely effective in a number of skin and blood disorders, including patients with severe combined immunodeficiency – the former 'bubble babies' born without functioning immune systems – and look promising for other blood disorders such as beta-thalassemia (see BioNews 920). However progress has been significantly slower in other tissues.

Professor Giulio Cosso of the University of Manchester, who lead the commission, explained that in other organs, 'fixing' the cells is not enough.

'As soon as you move to diseases that affect the muscles, the brain and the heart, you can't remove the diseased tissue. You add your healthy or cured cell to 95 that have not been cured,' he said.

The gap between public expectations and the number of proven treatments has allowed a market to spring up offering unproven treatments, often at high prices.

A well-known example is Davide Vanonni, who despite holding no medical or scientific qualifications offered stem cell treatments for neurological conditions in Italy before being convicted of conspiracy and fraud in 2015 (See BioNews 878).

Last month the FDA announced that it would be taking a tougher stance against US clinics offering unproven stem cell treatments (see BioNews 916). Treatments using only the patient's own cells are not subject to the same level of regulation as drugs under US law if the cells are only 'minimally manipulated' – and similar loopholes exist in many jurisdictions.

But the report argues that 'new therapies expose patients to risks, some of which are difficult to predict even with inbuilt safeguards' and that use in humans needs proper governance.

The commission's recommendations broadly fall into calls for improvement in four areas: science, funding models, governance and public engagement.