Spoonbill Foundation

*Last Updated November 2019. Please scroll down for information on how to donate directly to the Spoonbill Foundation and to see how much money has been raised so far.

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Pantothenate kinase-associated neurodegeneration (PKAN) is a rare neurologic disease that affects both children and adults. People with PKAN lack a chemical necessary to metabolize a vitamin in the brain. Without normal levels of this metabolite, part of the brain degenerates, causing severe problems with walking, coordination, vision, speech and swallowing. The illness is particularly cruel in childhood, when uncontrollable twisting movements can be extreme, causing pain and even bone fractures. Awareness is not affected, making the suffering imposed by the disease especially poignant. Many children with PKAN die before the age of 10.

We use genetically engineered animal models to study PKAN and test potential drugs to treat the disease. The two most important animal models in PKAN are a fruit fly (studied in the Sibon / UMCG lab the Netherlands) and a mouse (studied in the Hayflick-Hogarth / OHSU lab in the US).

Working collaboratively, we have identified a compound that we believe may change the course of this terrible disease

Here’s why:
• When we feed the compound to a PKAN mouse, it fixes all signs (biomarkers) of disease in the brain.
• When we feed the compound to PKAN cells from humans, the abnormal biomarker levels change to match the cells from healthy people.
• We need only tiny doses to completely fix the signs of disease in the mouse brain, yet even very high doses of the compound cause no bad side effects in the mouse.
• The same abnormal biomarkers in the PKAN mouse brain are also abnormal in the blood of people with PKAN. This is important, because it gives us an objective, biochemical way of measuring the disease and its response to drug treatments.

We are calling this compound ‘CoA-Z’. We have met with the United States Food & Drug Administration and the agency is supportive of our plan to move the compound quickly into humans. We anticipate that regulatory agencies in Europe and around the world will follow the FDA’s lead.

WE CAN’T DO THIS WORK WITHOUT FUNDING

We have completed much of the work to get to this point with grant funding from our universities, but we have much left to do. Here is what we need to keep moving forward:

step1

Make CoA-Z and get it ready for testing in people with PKAN

• $350,000 to make the first large batch of CoA-Z (includes testing the method for making the compound large-scale, manufacturing it to the high standards required for use in humans, and putting it into a form patients can take)

• $70,000 to perfect the biomarker blood test (includes developing efficient methods for testing and interpreting the results in a large number of patients)

step2

Test CoA-Z in people with PKAN• Even a small clinical trial typically costs $6-22 million! Our novel approach, in which people do all assessments close to their homes, will reduce the cost substantially. However, we estimate it will still cost approximately $35,000 per patient to run a clinical trial of CoA-Z. In order to have a robust trial with as many participants as possible, we anticipate we will need to raise approximately $2 million.

We are determined to eventually make CoA-Z available to everyone at the lowest cost possible

To this end, we have formed sister non-profit foundations in the Netherlands and US to further develop CoA-Z. We need your help to raise further funds to reach our goal. Donations are tax-deductible and are being accepted via PayPalDonate or check.

Donations
To donate through PayPal: Donate Here

Please make checks payable to:
Spoonbill Foundation
c/o Immix Law Group
600 NW Naito Parkway, Suite G
Portland, OR 97209

The Spoonbill Foundation is recognized as a nonprofit organization by the IRS.
IRS Code: Section 501(c)(3)
Tax Identification Number: 81-5080532

Why create a Foundation?
The reason we chose to create nonprofit foundations to develop this compound is because we want to retain control over CoA-Z and its price. If we had partnered with a pharmaceutical company, then they would have control over the final product and what price they chose to charge families. Unlike a for-profit corporation, our goal is not to make money. We want to get the compound to everyone who needs it at the lowest possible cost. You can learn more about the mission of the Spoonbill Foundation here: https://spoonbillfoundation.org

Track the donations!

We’ve added a way for you to keep track of the donations that are coming in and how much closer we’ve gotten to our goal. The total includes the amount needed for all the steps combined. A budget breakdown of how we reached this estimate can be found above.

We will be updating this tracker/thermometer weekly.
Last Updated: 11/2/2019 (6,027 added through Spoonbill)

*We will now be adding the donations collected through our sister organization Lepelaar Stichting to the overall total.
Clinical Trial Timeline
For many of you (and for us too!) it might feel as if you have waited forever for the clinical trial to begin. We hope this timeline will give you an idea of all that has been accomplished, with your help, since the discovery of the PANK2 gene, as well as our next steps: http://nbiacure.org/spoonbill-foundation/timeline