A call for Fundraising Help

For many reasons I have held off making any move or mention of raising funds for our little guy but I feel the time and opportunity has come.

Aside from the very obvious one of being so time poor, up until now I’ve been very torn between raising funds for the world of Oncology and that of Rare Metabollic diseases, namely NPC. Then I have been grappling with the issue of what do you raise funds for- research or the Organisations that support families affected. And to be honest the passed 12 months we have just had to put our head down with one foot in front of the other and move forward as best we can. Incidentally the 12th of February marks 12 months since his ALL diagnosis.

Of late I’ve been thinking more towards the NPC research and rare diseases given there seems to be quite a considerable amount of funding to pediatric Oncology. I have these grand ideas of establishing a foundation and holding an annual Fundraising ball eventually but currently time and energy is hindering these plans which is why I’m sharing this with you. Hopefully small steps will turn into big ones.

Our little guy has been very fortunate to receive many life saving and life prolonging drugs that researchers, doctors and even families have researched and campaigned for both for his ALL and NPC. Here is a link to a story from the Wall Street Journal which details the plight of some families in the US campaigning to get a treatment drug for NPC to their kids. I’m in electronic contact with all of these families and some of their doctors. I’ll forewarn you now, it’s lengthy and at times confronting but also very inspiring.

Given Ed’s ALL is a priority to be treated over his NPC he’s not really currently a candidate to be pushing for this Cyclodextrin mentioned in the article. However there is currently an oncology drug called Vorinostat which is being used to treat certain strains of Leukemia and now there is some research being conducted into using this exact same Oncology drug as a treatment (not cure) for NPC. Furthermore, just last week the Children’s Hospital at Westmead has begun a new trial using this Vorinostat for relapsed ALL.
What are the chances you ask, and yes just to remind you Ed is the only known patient in the world battling ALL and NPC. It’s early days and much more ground work to be done on this front but there is the possibility that he may begin on this drug in the coming months. I’m really pushing for this as what have we got to lose- two diseases, one drug. Too good to be true!!

I’m sharing all of this with you so you have some idea of where we are at in terms of research, drugs and current treatment plans as I have very recently been contacted by the President of the Australian Niemann Pick Foundation seeking some assistance in raising funds for a specific project and despite how time poor I am it’s a difficult one to ignore. It goes like this:

Professor Mark Wolterfang is a leading Neuropsychiatrist based at the University of Melbourne and is one of Australia’s experts in NPC. For several years he has been working with Professor Ashley Bush and a medicinal chemist doing their Post Doctorate research on an unnamed compound that they believe to be an effective treatment (not cure) for NPC. It is currently being trialled in human cell lines in NPC affected mice which they purchased from the States.

This research in the animal models is being conducted at the Howard Florey Institute based at Melbourne University. Previously the research has been funded by the National Health and Medical Research Council but they have been unsuccessful in securing any more funding in the last 2 rounds. As a result Professor Wolterfang contacted the NPC Foundation as they feel they are very close to making a breakthrough but need some financial assistance. If successful it will then be trialled in humans in the not so distant future. In order to continue with the research they are looking for $120,000 for 12 months of research. This is basically broken down into $100,000 for the Post Doctorate salary and admin costs, and $20,000 to house the NPC mice. If the funding doesn’t appear, even just half ($60,000 for 6 months) then the entire project will be closed down around April of this year.

It’s hard to say where the research will lead and if the compound will eventually come to market but I really feel this is something I need to get behind. Ed is currently on about 8-12 medications at any one time and I’m sure every single one of those drugs in their initial stages has had to go through a rigorous testing process such as this to get to market. We have been fortunate enough to reap the benefits of those before who fought for compassionate use of the Miglustat he is on (a therapy drug for his NPC) and now I feel it’s time to join in on the momentum of fundraising and support research. There are approximately 10-15 known patients in Australia with NPC so hopefully between us we’ll be able to raise the funds to keep the research going but at this point in time I need some help.

28th of February is World Rare Disease day (http://www.rarediseasedayaustralia.com.au/) and I’m hoping to pull something together around this time depending on the amount of hospital hours we clock up between now and then but I’m now putting it out there to see what you could do to help.

I’m open to anything, anybody has to offer but here is a list of suggestions of where we might start thinking:

*Prizes for a Raffle
The NPC President is in the process of putting together a raffle and aiming for some pretty major prizes- a cruise, major holiday, furniture, gift vouchers, services, whatever she can get her hands on so any leads there will be greatly appreciated. If there is something in your workplace that you think might be worth donating then let me know.

*Corporate Philanthropy
If anybody works for a major or large company and you think the company might be interested in donating I’m putting together some more formal corporate introductory letters if you need

*Host a small gathering:
Movie night, afternoon tea, girls night in, golf day, cards day, Jeans for Genes day in your workplace, even a dinner party and get people to make a small donation
Remember 28th of February is World Rare Disease Day so that might be a good day to hold it.

Or just donate.

Donations can be made via the Australian NPC Foundation website (http://npcd.org.au/) click on DONATE NOW or the details for EFT or in person at any ANZ Branch are:

Account Name: Australian NPC Disease Foundation Inc

BSB: 013-525

Account: 5722-92555

All donations over $2 are Tax Deductible

Please mark your donation for RESEARCH, although I’m sure all monies raised in the next few months will go directly to this cause.

I’ll keep you posted with any new ideas I may have, including my own little fundraiser.

Until next time, and as always thank you all so much for your love, friendship and support to our family. We are often overwhelmed by the strength and generosity of the human spirit during this time. Each and every one of you play a part in this journey and we know you help in whatever way you can, for this we are ever grateful.


3 thoughts on “A call for Fundraising Help

  1. Great work yet again Ali. Also people out there don’t forget we have the Persevere wristbands available at a cost of $3 each with all proceeds going to the Foundation for researching this matter. I am Mandy, The President of the Australian NPC Disease Foundation, and also the mum of two children, Matthew 26 and Timothy 24, both whom have NPC Disease diagnosed in 2007. So I truly “Get It”!!!! like Ali, though she definitely has coped a double whammy with the cancer for that gorgeous little man Eddy, who totally owned my heart when I met him. Hope you can get behind our joint cause and continue to support Ali with anything she needs in her time short 28 hour days.
    Mandy Jacobs

  2. Hi Ali, you folk are amazing, and our heart goes out to you as it does everyday. We have to help, just wish we had the funds to do more. We hope that you and Pete – and your Mum and Dad – can keep your energy and spirit going against all the odds. Thankyou for keeping in touch through your blog. You are amazing. Love. Diane and Malcolm

  3. Although funds are what ia currently needed, spreading awareness helps bring funds from donors. So even if you don’t have funds to spare at the moment, make sure you share this post or the Australian NPC website anywhere you can. I am a mom to an NPC angel in the US and I have found that raising awareness anywhere I can results in getting funds from people all over.

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