One of the first contacts we made after Grace was diagnosed with INAD was to the NBIA organization. NBIA stands for Neurodegeneration with Brain Iron Accumulation, and their headquarters is located in Portland, OR, under the direction of Dr. Susan Hayflick.
Actually, it was our geneticist at Children's Hospital that made the initial contact for us. When I contacted the NBIA, they already knew who we were, which was a nice transition for us in a very scary time. I spoke on the phone to Allison Gregory, who works with Dr. Hayflick. She took some of Grace's information, and talked to me more about INAD and what it meant for our family.
We have kept in touch with Allison and the NBIA over the last few years, and this summer, she asked about some of our fundraising efforts. I told her we had been blessed to live in a very generous community, and that we had been able to raise around $130,000 in the two years since Grace has been diagnosed.
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| Grace was SO excited to help out with the OHSU natural history family study. |
Most of that funding (around $100,000) has been donated to Washington University in St. Louis, MO, to Dr. Paul Kotzbauer. He and his research team are studying potential treatment options for children with INAD.
In the course of our conversations, Allison told me the NBIA was applying for a grant to start a natural history family study for PLAN families (and INAD is a type of PLAN). PLAN stands for PLA2G6 neurodegeneration, which refers to the gene that is affected. When it presents itself in infants and toddlers, it's known as INAD.
Allison and Dr. Hayflick were applying for a grant with the FDA, but were looking for the support of INAD families to get started, and they knew our INAD family community was strong. In the past three years, INAD families all over the world have raised more than $500,000 and have funded two research studies. She said they were asking for the INAD families to pledge $30,000 for the next three years to get the study started, and they hoped to supplement the research with the FDA grant.
What this study will do is gather data and information, and lots of it about the INAD kids and their families. With two active research studies, the group wants to be ready for clinical trials when the time is right. After talking to two of the other moms, we decided this was something we definitely wanted to get behind. Our iCan for INAD crowdrise online fundraising account had close to $13,000 in it that we thought would be a good start. I said that Kyle and I had about $28,000 from A Grace Filled-Journey, and that we would fill in the remainder.
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| Dr. Hayflick and her team at OHSU, and they are grateful for the donation too! |
So, this week, Grace and I got a cashier's check for $17,500, and mailed it to Dr. Hayflick at the Oregon Health and Science University in Portland, OR. It felt really good to be able to jump in and send funding when it was truly needed. Thanks to all of our generous followers, the NBIA will be able to get started on this study immediately. We still hope and pray that the FDA grant funding comes through, and they should hear in December.
Kyle and I are still planning to financially back Dr. Kotzbauer and his work at Washington University. He will finish up the second year of his study in the spring, and is excited that he and his research team continue to make progress. They should be able to start screening some drugs in the coming months, and he plans to keep us updated on his funding needs.
From the bottom of our hearts, we could not be more grateful to each and every one of you that have donated to INAD research over the last two years. We want you to know what a difference your generous gift has made, and continues to make. Together we will #BeatINAD.
mlh
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