Transplant technique tested on mice could change how we treat a host of diseases

A new technique for bone marrow transplants tested so far only in mice could transform the treatment of a host of different diseases if replicated in humans.

A study published in Science Translational Medicine showed how bone marrow transplants could be conducted without the need for gruelling chemo and radiotherapy.

The finding may have profound implications for blood cancers and autoimmune diseases such as type-1 diabetes, rheumatoid arthritis and multiple sclerosis.

Bone marrow transplants are currently used to treat patients who are critically ill from leukaemia and other cancers of the blood but up to one in five patients die as a result of the treatment.

Many complications are caused by the fact that chemo and radiotherapy are used to eliminate existing bone marrow cells so new cells can be successfully transplanted.

But researchers at Stanford University have pioneered a way to eliminate these cells using two different antibodies instead.

In mice more than 99 per cent of the cells were wiped out.

The researchers said: ‘If it works in humans like it did in mice, we would expect the risk of death would drop form 20 per cent to effectively zero.’

The bone marrow produces blood cells and most of the cells in the body’s immune system. This means a rogue immune response could potentially be cured by a one-off transplant.

Irving Weissman, co-author of the research and professor of pathology and of developmental biology at Stanford, said: ‘There is almost no category of disease or organ transplant that is not impacted by this research.

‘If and when this is accomplished, it will be a whole new era in disease treatment and regenerative medicine.’

Instant analysis
Blood-borne diseases including leukaemia and autoimmune diseases like Addison’s disease and rheumatoid arthritis are challenging to treat and are responsible for considerable morbidity and mortality. While we have made strides in our understanding of how to detect and manage them, we have yet to be able to cure many of these diseases definitively, even if we have been able to give patients the ability to live almost normal lives.

Bone marrow transplants have had considerable success in being able to cure various blood-borne disorders; as with any transplant, though, the issue of rejection of the transplanted tissue is a serious one. To prevent this the patient has to undergo gruelling therapy in the form of chemotherapy/radiotherapy in order to kill existing cells of the bone marrow so that the new cells will ‘take’. This obviously has its own considerable potential complications.

Haemolytic stem cells are cells that can develop into any needed blood cells, including cells governing immunity, and these are the cells that are transplanted in such procedures.

This novel technique, demonstrated in mice, uses what are known as monoclonal antibodies as an alternative to chemo/radiotherapy prior to transplant. It rids the existing bone marrow of blood cells, thus allowing the new transplanted cells to ‘take up residence’ and proliferate, giving the recipient literally a whole new family of blood cells.

While the result of the bone marrow transplant is the same, ie the curing of an existing disease, the side effects from aggressive pre-transplant therapy to prepare the bone marrow are zero, thus making this modification of an existing treatment revolutionary.

The potential applications of this technique encompass a wide range of human disease; it does, however, remain experimental, and it will potentially be years before any human applicability is realised.
TSA
Research score: 2/5