Zoe's INAD Battle Fund
Zoe has been diagnosed with Infantile Neuro Axonal Dystrophy (INAD), a rare genetic, degenerative, neurological condition. Children with the condition live until age 5-10. We (parents, Christine Hamshere and Steve Lightfoot) are raising money to take Zoe to America to participate in a clinical trial of a drug which we hope will halt and possibly reverse some of the symptoms. Please help us to try and save Zoe from INAD.
Zoe was born in 2015 and grew up as a normally developing child until around 18 months, when it was questioned why she wasn't yet walking. She could stand at furniture but couldn't take steps without support. At first it was considered to be caused by hypermobile joints, but shortly after Zoe turned 2 it was apparent that she was developmentally behind and had lost some skills.
In January 2018 Zoe had an MRI brain scan which revealed significant brain damage, cerebellar atrophy. The part of the brain that affects balance and co-ordination, speech and swallowing had formed normally as a baby and then shrivelled.
Then began a series of tests to try and find out the cause of the atrophy. In April 2018 we received the devastating news that Zoe was diagnosed with INAD. The Great Ormond Street Hospital description is that it "is a rare inherited disorder affecting the nerve axons (which are responsible for conducting messages) in the brain and other parts of the body, causing a progressive loss of vision and of physical and mental skills....Eventually the combination of the diseased brain and physical weakness becomes too great to sustain life, and death usually occurs between the ages of five to ten years. Parents and carers will be aware of the child’s increasing frailty, and death is usually relatively peaceful and expected when the time comes."
We have been told there is no miracle cure. The NHS have been fantastic, but all they can do is support Zoe as her symptoms present. Since the diagnosis, Zoe has lost mobility and her speech, swallowing has also deteriorated, and she has some leg stiffness. She is still a bright bubbly fun loving beautiful girl. She has a great sense of humour, an infectious laugh, and still tries to wave and blow kisses. She loves bouncing along to songs and interaction with others, cuddling the neighbours dog and her toy rabbits She has a good understanding of what is going on around her, and loves it when you copy her actions and sounds, especially drumming. She has a smile that lights up the room. We are desperate to keep her this way for as long as we can.
We approached a drug company in America that are developing a study drug to help children with INAD. INAD is a gene variation that causes cell damage when fatty acids are broken down. The study drug is a stabilised fatty acid that protects cells from being broken down. In initial trials the study drug stopped the decline in patients and returned some lost skills. More info is available from the link on this page https://www.retrotope.com/publications/ under October 2018 publication.
Zoe has now been invited to America to participate in a drug trial. Only 15 children worldwide will be on the trial. The first trip is at the start of December. The second will be in January 2019.
Please help us to fund taking Zoe to America. We will need to make 4 trips in a year as part of the trial, and then potentially ongoing after that.
Since the diagnosis we have moved house to a property suitable for disabled house adaptions. The Council are helping with the adaptions but we still need to fund some of the works. Zoe started at a special needs school in September and we are having to transport her there. We need to purchase a wheelchair accessible vehicle so we can take Zoe out comfortably. We also have another baby on the way. We have been told that the baby will be INAD free.
Please help us to fund the trips to America. Any funds that are not spent on this will go towards adapting the house for Zoe, transporting her to school, and supporting all the charities that are helping us out. For example Martin House children's hospice have offered to look after Zoe when the baby arrives, which we are extremely grateful for. SNAPS Yorkshire have been providing invaluable support for us, and rebound therapy for Zoe. Little Hiccups have helped us to take Zoe out on day trips as well as regular play sessions. Leeds Mencap have been providing nursery care and amongst other things helped us apply for the school place, and got us into the queue for house adaptions.
Please help us to try and save Zoe from INAD. Thank you for your support,
Christine & Steve
It's been a busy couple of months! Zoe seems to be responding well to the drug trial. She is sleeping better, has improved head control and there are signs that her digestion may be improving too. We won the school transport appeal so that Zoe is now transported to school. We have purchased a wheelchair accessible car so we can take Zoe out in her buggy. The first car was written off within a week by a stolen van, thankfully it was swiftly replaced and no permanent injuries sustained. The house adaptions are underway. Zoe's baby brother arrived safely into the world last week. Zoe had a short respite stay at Martin House during the birth. See photo of Zoe meeting her brother in hospital. We have booked our flights back to America in July, we all intend to make the trip.
We made our second trip to America, and are back home now. Pleased to report that Zoe has started on the drug trial.
Thank you all so much for your help to raise the funds to take Zoe to America to participate in the drug trial. We have been blown away by your generosity and can't believe we raised the funds within 3 weeks. Thank you to every individual and business/organisation that donated, organised fundraising events, helped us out, and shared our campaign. Thank you also to the media for raising the profile of our campaign. Thank you also for all the messages of support. We have made our first trip to America, we went at the end of November, and returned on Saturday morning. Zoe has been though a lot of baseline tests, including EEG to test for seizure activity, sight, hearing, blood tests, and skills testing. We return to America early January for further tests and to return with the drug for Zoe. We then make a trip in the summer and another in January 2020. This should complete the trial, but beyond this we may make further trips if the trial is successful in halting / reversing the symptoms. Fingers crossed it will be! Your support is keeping us all going. Thank you xxx