Stem cells enable personalised treatment for bleeding disorder

April 5, 2013
Cells from patients' blood could be developed as treatments for heart and circulatory diseases.

(Medical Xpress)—Scientists have shed light on a common bleeding disorder by growing and analysing stem cells from patients' blood to discover the cause of the disease in individual patients.

The technique may enable doctors to prescribe more effective treatments according to the defects identified in patients' .

In future, this approach could go much further: these same cells could be grown, manipulated, and applied as treatments for diseases of the heart, blood and circulation, including heart attacks and haemophilia.

The study focused on (vWD), which is estimated to affect 1 in 100 people and can cause excessive, sometimes life-threatening bleeding. vWD is caused by a deficiency of von Willebrand factor (vWF), a blood component involved in making blood clot. vWF is produced by endothelial cells, which line the inside of every blood vessel in our body. Unfortunately, they are difficult to study because taking biopsies from patients is invasive and unpleasant.

A group led by Dr Anna Randi at the National Heart and Lung Institute, Imperial College London used a new approach to investigate the disease. Dr Richard Starke, a British Heart Foundation Intermediate Fellow and lead author of the study, took routine blood samples from eight patients with vWD, extracted stem cells called , and grew them in the lab to yield large numbers of endothelial cells.

By testing these cells, they were able to analyse each patient's disease in unprecedented detail. In some patients, the scientists found new types of defect, which may enable them to recommend improved treatments. Professor Mike Laffan, a collaborator in the study and in charge of patients with VWD at Hammersmith Hospital in West London, is looking to apply these findings to reduce severe bleeding in these patients.

Dr Randi believes that endothelial progenitor cells could become an invaluable resource for testing for vWD and other diseases. "We will be able to test the effects of a range of compounds in the patients' own cells, before giving the drugs to the patients themselves," she said.

This approach could have impact far beyond vWD. Endothelial cells derived from blood could also be isolated and reinjected into someone recovering from a heart attack, to help them grow new blood vessels and repair the injured heart tissue. Dr Starke says this approach avoids the main problem with transplant therapies, in which the immune system tries to destroy the foreign material. "The patients would receive their own cells, so they wouldn't face the problems of rejection," he said.

Work is well underway towards achieving this goal, but blood-derived endothelial cells are only now being explored. "There are already many studies where patients have been injected with to see whether damage to the heart could be repaired, and there are some promising results," says Dr Randi. "The door is open to such treatments, and our studies are a step towards identifying the right cells to use."

The group's previous research has already thrown up pointers for potential new treatments. Aside from producing vWF to form clots, endothelial cells are responsible for forming new blood vessels. In their last paper, the group showed that vWF is actually needed to build healthy blood vessels. Some patients with vWD suffer severe bleeding from the gut because defects in vWF cause their to develop abnormally. "There are drugs already being used in other diseases which target abnormal blood vessel, that could be useful to stop bleeding in some vWD ," says Randi. "Nobody would have thought of using them to treat vWD, but by testing them on the patient's own endothelial cells , in the laboratory, we can find out if these drugs work before giving them to the patient."

Scientists are now interested in the possibility of using as a treatment in themselves. For instance, haemophilia, the hereditary which affected Queen Victoria's family, might one day be treated by taking these cells from a patient and replacing the gene that causes the disease, then putting them back into the patient.

Explore further: Research findings breathe new life into lung disease

More information: RD Starke et al. 'Cellular and molecular basis of Von Willebrand Disease: studies on blood outgrowth endothelial cells.' Blood April 4, 2013 vol. 121 no. 14 2773-2784 doi: 10.1182/blood-2012-06-435727

Related Stories

Research findings breathe new life into lung disease

October 24, 2012
It turns out the muscle cells on the outside of blood vessels have been wrongly accused for instigating lung disease. New research shows that while these muscle cells are responsible for constricting or dilating the blood ...

New patient-friendly way to make stem cells for fight against heart disease

November 29, 2012
funded by the British Heart Foundation (BHF), Medical Research Council (MRC) and Wellcome Trust – have today published a patient-friendly and efficient way to make stem cells out of blood, increasing the hope that scientists ...

Researchers discover new blood vessel-generating cell with therapeutic potential

October 16, 2012
Researchers at the University of Helsinki believe they have discovered stem cells that play a decisive role in the growth of new blood vessels. If researchers learn to isolate and efficiently produce these stem cells found ...

New evidence of the benefits of home dialysis for kidney patients

June 21, 2011
Researchers at St. Michael's Hospital have found more evidence of the benefits of home dialysis for patients with kidney failure.

Recommended for you

Molecular hitchhiker on human protein signals tumors to self-destruct

July 24, 2017
Powerful molecules can hitch rides on a plentiful human protein and signal tumors to self-destruct, a team of Vanderbilt University engineers found.

Researchers develop new method to generate human antibodies

July 24, 2017
An international team of scientists has developed a method to rapidly produce specific human antibodies in the laboratory. The technique, which will be described in a paper to be published July 24 in The Journal of Experimental ...

New vaccine production could improve flu shot accuracy

July 24, 2017
A new way of producing the seasonal flu vaccine could speed up the process and provide better protection against infection.

A sodium surprise: Engineers find unexpected result during cardiac research

July 20, 2017
Irregular heartbeat—or arrhythmia—can have sudden and often fatal consequences. A biomedical engineering team at Washington University in St. Louis examining molecular behavior in cardiac tissue recently made a surprising ...

Want to win at sports? Take a cue from these mighty mice

July 20, 2017
As student athletes hit training fields this summer to gain the competitive edge, a new study shows how the experiences of a tiny mouse can put them on the path to winning.

'Smart' robot technology could give stroke rehab a boost

July 19, 2017
Scientists say they have developed a "smart" robotic harness that might make it easier for people to learn to walk again after a stroke or spinal cord injury.

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.