Update on Saving Chloe Saxby, August, 2017:
Thank you for supporting our family in our bid to save Chloe and close to 200 children around the world suffering with this monster of a disease.
The last 6 months have been a stark reminder that we really are racing against the clock to find a cure for Vanishing White Matter Disease. Little Chloe has experienced over 60 seizures this year which is taking its toll on her little body. Her hands are much more shakey, her speech has deteriorated and she slumps forward in her wheelchair. But our brave little fighter is hanging in there, with her beautiful smile and cheeky personality, we are more deterined than ever to put an end to this disease, not only for Chloe but for all children.
There are only two Professors in the World that are working on a treatment for Vanishing White Matter Disease. We had the opportunity to meet with both of them at the United Leukodystrophy Conference last month, where they updated us on their latest findings. Over the past 12 months, both Professors have made some very promising progress towards a treatment for VWM Disease which gives us renewed HOPE.
So far, your donations have enabled Professor Orna Elroy-Stein to:
- Expand her small team of scientists working on VWM Disease by hiring additional Research Assistants
- Help purchase vital testing equipment and the Confocal Microscope which has enabled the team to accelerate their research.
However urgent funding is now required to accelerate the next phase of testing in our bid to save Chloe and the current generation of children suffering with VWM Disease.
After talking to the professors at the conference, both have categorized what their current funding needs are:-
Professor Orna Elroy-Stein:
$500,000 for a key piece of labratory equipment called a MALDI TOF machine which is a powerful tool for drug screening and will enhance Orna's research
$500,000 for outsourcing of testing which can be done many times faster in a much larger commercial lab
Professor Marjo Van der Knaap
$250,000 to secure two PHD students for the next couple of years
$500,000 for the next phase of testing of the promising compounds that have been identified as potential treatments for VWM Patients.
Professor Van der Knaap to date has recieved grants for her research and has made signifiant progress toward a treatment, however, her research has been put on hold due to the grant funding being depleted.
Chloe does not have time to lose. This disease has already robbed her of her ability to walk, next it will take away her ability to see, hear, eat, hold her head up and she will die at an early age if the cure does not come in time. With funding, this treatment can come in time.
Thank you from the bottom of our hearts for helping us in our race against time.
Determined, hopeful and eternally grateful,
Nyree, Grant, Maddy & Chloe xx
Update on Saving Chloe Saxby, Feb, 2017:
After an illness free winter, Chloe has deteriorated over the last couple of months due to the onset of seizures and once again makes us realise how much we are in a race against time.
We have had some promising communication from the professor who is heading up the small research team that is working on a cure for VWM disease. She has isolated four compounds that showed promising results in her lab, however, the next phase of testing will need to be conducted. Outsourcing this testing is the best way to save time as the big commercial laboratories can handle much larger amounts of testing and have the equipment to do so. This does take a considerable amount of funding, which is why we started the Saving Chloe Saxby Campaign. Unfortunatley, because this disease is so rare, the fundraising is left up to the individual families. Although Chloe is the face of the VWM campaign in Australia, all of the funds raised will go to speeding up the cure - to help approximately 170 children around the world suffering with this disease.
This cure is going to come, but without funding it will not come in time to save Chloe and the current generation of children suffering with VWM Disease.
Once again, from the bottom of our hearts, thank you to all who have donated and also to those who have so given generously of their time to help us.
If you could continue to share SavingChloeSaxby.com we would be very grateful.
Determined, Hopeful and Eternally Grateful.
The Saxby Family
We have reached the quarter of a million dollars milestone!! We are so grateful to everyone who has donated so generously, not only financially, but of their time, their compassion, spiritually and emotionally.
We have HOPE, - and if you have hope, you have everything.
The monies raised will be put towards the purchase of a confocal microscope. We believe this will exponentially decrease the time it is going to take to find this cure. We will keep on fundraising untill we reach our goal of $2.6 Million. With this funding we will continue to buy key pieces of research equipment and expand the research team.
Once again, we thank everyone for your support.
Determined, Hopeful, and Eternally Grateful
Nyree, Grant, Maddy and Chloe.
Together we can save Chloe Saxby
Watching our precious little girl deteriorate every day is every parent's worst nightmare, but it is our agonizing reality. Until the age of three, our beautiful little girl was running around, healthy and happy. She had just started dance lessons and loved them.
Then overnight, she lost the use of her legs.
After eight months of testing Chloe was diagnosed with Vanishing White Matter Disease, an extremely rare, degenerative and terminal brain disease. VWM is a condition that destroys Myelin, the brain's white matter. In doing so it permanently affects transmission of brain signals to the rest of the body.
This is a disease that doesn't allow children to be children. A bump to the head, high temperature, cold/flu, stress or fright could kill Chloe.
Chloe is now seven, this monster of a disease has already robbed Chloe of her ability to walk, it will then paralyse her little body so she won't be able to hold her head up, talk, see hear or eat. She will experience a lot of pain, suffering and die at an early age....If the cure is not found in time.
There is hope!
Scientists researching VWM have had some encouraging breakthroughs recently. Professor Orna Elroy-Stein from one of only two research facilities in the world working on a cure, is testing new treatments with positive results.
However progress to reach the clinical trials stage is hampered by a lack of funding. With only 172 reported cases worldwide, pharmaceutical companies don't see it as commercially viable. So it is left up to the families affected to raise the funds to save our children.
And we are in a race against time. The life expectancy is typically only between 5-10 years from disease onset. Speeding up the progress to reach clinical trials stage will come at a cost of approximately $2.6 million.
The cumulative breakdown of our fundraising goals is:
1st Milestone: $840k - to fund a specialised Confocal Microscope to speed up & further the already promising research
2nd Milestone: $1.4m – reaching this will also fund a Fluorescence
Activated Cell Sorter and hiring of additional research assistants
3rd Milestone: $1.7 mil – will additionally fund live cell imaging and MRI analysis equipment
4th Milestone: $2.6m – to enable an outsourced program to facilitate characterisation of the drug-like molecule with the potential to lead to pre-clinical stage
Chloe is a patient of Dr Hugo Sampaio, Neurologist at Sydney Children's Hospital - "In the world of rare diseases research, the last decade has seen tremendous efforts to establish international collaborations. There are currently two major research collaborations underway aiming to find a cure for Vanishing White Matter Disease, both of these are overseas. The research taking place under Professor Orna Elroy-Stein at the University of Tel Aviv is showing some promise and may be lifesaving for Chloe and other children like her".
We can't stand by whilst this disease ravages Chloe's body and watch it take everything from her. Our gorgeous girl is a ray of sunshine and deserves the right to make her mark on this world.
This cure is going to come, please help us find it in time to save Chloe
Let's stop this disease so the next time parents are told this soul destroying news there is a treatment available! This can only happen with your help. We really hope you will assist us in raising these lifesaving funds, every dollar counts. Even if you can only share this with as many people as you know, we will be forever grateful.
All funds raised will go towards finding the cure.
Thank you from the bottom of our hearts.
Grant, Nyree, Maddy and Chloe Saxby
Saving Chloe Saxby is part of our own non-profit Australian Registered Charity with Deductible Gift Recipients Status.
Chloe Saxby and Vanishing White Matter Disease Incorporated
Charitable fundraising number: CFN/23384 ABN: 59 276 626 144