Subscribe to the BioNews newsletter for free

Advanced Search

Search for

Like the Progress Educational Trust on Facebook



Gentler bone marrow transplants treat adults with sickle cell

14 December 2009

By Ailsa Stevens

Appeared in BioNews 538

Adults with the inherited blood disorder 'sickle cell anaemia' may be treated using blood stem cells transplanted from a healthy tissue-matched donor, scientists based at the US National Institute of Diabetes and Digestive and Kidney Diseases in Bethesda, Maryland, have shown. The treatment successfully boosted levels of normal red blood cells in nine out of 10 of the adults who have so far been given the experimental therapy, according to a study published in the New England Journal of Medicine last week.

Normally red blood cells are doughnut-shaped, but in people with sickle cell anaemia they become banana or 'sickle' shaped when they shed their oxygen load, making them prone to getting stuck in the small blood vessels. This triggers bouts of extreme pain and can potentially cause strokes and permanent damage to many different organs.

Red blood cells originate from blood stem cells found in the bone marrow, which is why transplanting bone marrow from a matched donor can potentially provide a cure for patients with sickle cell anaemia. Standard bone marrow transplants involve first destroying all of the patient's own bone marrow using a dose of chemotherapy, radiation, or a combination of the two. This aspect of the treatment is dangerous for adults, but the procedure has been successfully used to cure some 200 children with sickle cell disease.

In an attempt to develop a treatment which would be safe for adults, the researchers used a less aggressive form of radiation to treat ten patients aged 16-45 with sickle cell anaemia, before giving them a bone marrow from a tissue-matched donor. Because the treatment only partially killed off their own blood stem cells, the patients produced a mixture of both normal and defective red blood cells, but the normal red blood cells were able to compensate for the defective ones because they live longer, said the researchers. 'Because sickle red blood cells only live about six or seven days and normal red blood cells live 120 days, if you can get a little bit of the donor cells in there, the donor cells take over,' John Tisdale, one of the lead authors of the study, told Reuters.

'The simplicity, low toxicity, and high efficacy of this approach make it feasible for use at most transplantation centers,' Matthew Hsieh, the lead author, and colleagues concluded. However, the need for a period of intensive care in a highly sterile environment subsequent to the treatment will make it unsuitable for developing countries, where the condition tends to be most prevalent, noted New Scientist. It is thought that the increased prevalence seen in warmer climates may be because carrying the condition confers resistance to malaria.

Following the treatment, patients face a lifetime of taking powerful drugs in order to prevent their own immune system from attacking the donor blood stem cells. It is not yet clear whether it may be possible to wean the patients off of these drugs, which weaken the immune system making them prone to illness and infection.

The New England Journal of Medicine | 10 December 2009
LA Times | 10 December 2009
Med Page Today | 09 December 2009
Reuters | 09 December 2009
WebMD | 09 December 2009
New Scientist | 10 December 2009


17 October 2011 - by Mehmet Fidanboylu 
The blood condition sickle cell disease may be reversed by turning off a single gene, according to scientists in the USA. By inactivating a single gene in red blood cells the researchers were able to alleviate symptoms of the disease in mice, offering the hope of a potential new treatment for humans...
03 October 2011 - by Dr Kimberley Bryon-Dodd 
A study analysing three different, but closely related, African populations has identified that a cancer gene is present at a surprisingly high frequency; akin to those usually associated with evolutionary advantages...

29 June 2009 - by Dr Will Fletcher 
The discarded placentas of newborn babies have been identified as a new and more plentiful source for harvesting stem cells by researchers from the Children's Hospital and Research Center, Oaklands, US. The more traditional stem cell source, blood from the umbilical cord, only contains enough stem cells for a transplantation to be useful on infants or children. Placentas contain far more stem cells and, when combined with blood from the umbilical cord, would mean that enough stem cell materia...
02 June 2008 - by Dr Charlotte Maden 
Researchers at Johns Hopkins University School of Medicine in the US have found a faster and more efficient way to reprogramme cells into embryonic-like stem cells so that they can be used to study genetic disorders such as sickle cell anaemia. The study was published in the...
10 December 2007 - by Katy Sinclair 
By Katy Sinclair: Researchers, reporting in Science, have announced the successful treatment of a mouse with sickle-cell anaemia using stem cell lines created from adult mouse cells. Rudolf Jaenisch, of the Whitehead Institute for Biomedical Research in Cambridge Massachusetts, and Tim Townes, of the University of Alabama at Birmingham, worked...

Be the first to have your say.

You need to or  to add comments.

By posting a comment you agree to abide by the BioNews terms and conditions

- click here to enquire about using this story.

Published by the Progress Educational Trust


Public Conference
8 December 2017

Speakers include

Professor Azim Surani

Professor Magdalena Zernicka-Goetz

Professor Robin Lovell-Badge

Sally Cheshire

Professor Guido Pennings

Katherine Littler

Professor Allan Pacey

Dr Sue Avery

Professor Richard Anderson

Dr Elizabeth Garner

Dr Andy Greenfield

Dr Anna Smajdor

Dr Henry Malter

Vivienne Parry

Dr Helen O'Neill

Dr César Palacios-González

Philippa Taylor

Fiona Fox

Sarah Norcross

Sandy Starr


Good Fundraising Code

Become a Friend of PET HERE and give the Progress Educational Trust a regular donation