Hello! I'm cycling 100 miles as part of RideLondon on 29th May 2022. I am taking on this challenge in order to raise money for my friend Verity Grainger who is trying to raise £500,000 in order to pay for Compassionate Treatment for her rare genetic condition MNGIE.

Verity was diagnosed with MNGIE in 2016. MNGIE (mitochondrial neurogastrointestinal encephalomyopathy) is an ultra-rare life-shortening genetic condition that blocks the production of a particular enzyme, causing a build-up of metabolites in the blood that damages mitochondria. So rare in fact, that there are only around 200 people documented to have the condition worldwide. Sadly, Verity is one of these cases. MNGIE is an unmerciful condition that impacts many areas of the body and causes it to slowly degenerate.

Unfortunately, there is no proven treatment for this condition. The only unsubstantiated option Verity has been given is to attempt a bone marrow transplantation, which comes with considerable risk. A previous trial of this in patients with MNGIE was overwhelmingly unsuccessful and the results of this make for grim reading.

There is, however, a safer treatment known as enzyme replacement therapy, which has previously been used in clinical trials to treat this condition with promising results. Enzyme replacement therapy works by encapsulating the missing enzyme in red blood cells. This allows the enzyme to survive long enough for the treatment to be effective without the body’s immune response breaking it down. This would mean regular infusions of the enzyme would be needed, around every 4-6 weeks which would allow the body to perform the function it cannot do on its own, i.e., being able to remove the mitochondria-damaging metabolites from the bloodstream.

This has been approved for compassionate use, but comes at a considerable expense, estimated to cost around £500,000! (Not including the 2.9% + £0.25p per donation JustGiving fees). This would cover the cost of a neurologist to conduct the trial, the continued manufacturing of the enzyme, and any patient costs. This will not only provide the treatment for Verity, but also her sister, Zillah who also has the same condition. This would be run at St George's University Hospital in London.

Verity's family and friends are all pulling together in order to raise the target amount required for treatment. Please take the time to read Verity's inspirational story using the link below. All donations towards my fundraising page will go directly to Verity in order to help provide her with the Compassionate Treatment she needs and deserves.

https://www.justgiving.com/crowdfunding/compassionate-treatment-for-mngie?utm_term=p7qNMEeaM

Thank you!

Dan