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Annual Report - Central Manchester University Hospitals - NHS ...

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Research Team Helps Children With MPS<br />

A stem cell research laboratory for mucopolysaccharide (MPS) diseases<br />

is investigating treatments which may help around 400 children in<br />

<strong>Manchester</strong> - and many more nationally. The lab was set up at the Royal<br />

<strong>Manchester</strong> Children’s Hospital by Drs Rob Wynn, Ed Wraith and Brian<br />

Bigger.<br />

The nine-strong research team is using a multidisciplinary approach<br />

to investigate stem cell and gene therapies to improve bone marrow<br />

transplantation in MPS and related diseases.<br />

MPS diseases are genetic disorders which affect 1 in 26,000 people,<br />

mainly children. In each type of the disease, the patient lacks a specific<br />

enzyme needed to break down glycosaminoglycans (GAGs), types of<br />

carbohydrate that are essential in connective tissues and are used by the<br />

body to help cells to communicate with each other.<br />

These GAGs are essential for the body to work normally, but when too<br />

many build up, as happens in cells from patients with MPS diseases,<br />

they cause severe progressive mental decline, heart and other problems<br />

as well as bone and joint disease in some cases. Severe forms of MPS<br />

usually result in death in early childhood.<br />

Milder forms of MPS can be treated with weekly injections of the<br />

missing enzyme. However, most severe forms of MPS are currently<br />

untreatable as the enzyme supplied in the blood is not able to cross into<br />

the brain or to correct bone defects. The exception is Hurler syndrome<br />

(MPS IH) which can be treated with a bone marrow transplant. Our<br />

team is looking at ways to reduce the risks and side effects involved in<br />

bone marrow transplants and to increase success rates. We’re also trying<br />

to develop gene therapies for untreatable forms of MPS.<br />

Funded by the UK MPS Society and other national MPS and children’s<br />

charities, the research group is based in dedicated laboratory facilities at<br />

the Royal <strong>Manchester</strong> Children’s Hospital. (July 2008)<br />

<strong>Manchester</strong> Royal<br />

Infirmary Performs First<br />

Chimney Procedure In<br />

The UK<br />

T<br />

The <strong>Manchester</strong> Royal Infirmary became<br />

the first hospital in the UK to perform<br />

a Chimney (or Periscope) Endovascular<br />

aneurysm repair (EVAR).<br />

A patient, aged 69, suffered an aortic<br />

aneurysm which is when the aorta, the<br />

largest artery in the body, balloons or<br />

widens. The aneurysm weakens the wall of<br />

the artery and can lead to it rupturing which<br />

can have fatal consequences.<br />

The four hour operation involved a team<br />

of three consultants, in addition to many<br />

support staff, fitting a ‘chimney’ or<br />

‘periscope’ graft to repair the weakening,<br />

instead of the usual stent graft. This had to<br />

be used as the area around the aneurysm<br />

wasn’t big enough to support the standard<br />

stent graft. If they had placed it further up<br />

the artery, the blood flow to the patient’s<br />

kidneys may have been affected resulting<br />

in kidney failure. Without this type of stent,<br />

The patient would have had to wait for a<br />

minimum of two months for a specially<br />

made ‘Branch’ graft from Australia as she<br />

was not fit for full conventional surgery.<br />

The patient would have been an<br />

exceptionally high risk for conventional<br />

surgery and because of this new technology<br />

they have not only had their aneurysm<br />

repaired but their kidney function preserved.<br />

Only a few of these chimney grafts have<br />

been done world-wide and this was the first<br />

in the UK.<br />

This new and advanced technique enabled<br />

this patient to have the life-saving procedure<br />

without a need for a large abdominal<br />

incision, therefore reducing the risk of<br />

infection and also meaning that a postoperative<br />

stay on Intensive care wasn’t<br />

needed. Instead, two, much smaller incisions<br />

were made in the groin and one in the<br />

patient’s armpit. (July 2008)<br />

page 6<br />

<strong>Central</strong> <strong>Manchester</strong> and <strong>Manchester</strong> Children’s <strong>University</strong> <strong>Hospitals</strong> <strong>NHS</strong> Trust

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