Natalie’s Wish Fundraiser and Celebration – A Wish Comes True

We are astounded by your extraordinary generosity and commitment to the CRF research program. Your donations will allow us to continue to fund life-saving research that is changing the lives of those living with cystinosis. The Natalie’s Wish Fundraiser and Celebration raised a remarkable $1,350,332 for cystinosis research. Thanks to your steadfast commitment to research, we are able to accelerate the research process and move closer to better treatments and a cure every day.

As a direct result of your support, CRF was able to fund millions in research that has led to new discoveries about cystinosis and to the approval of two of the most significant advancements in the treatment of cystinosis – an FDA-approved delayed-cysteamine treatment and an FDA-approved stem cell clinical trial – allowing those affected by cystinosis to dream of a life free of the disease.

We honor and thank the five patients who volunteered to participate in the UC San Diego stem cell and gene therapy clinical trial. Their participation has yielded information that has broadened our understanding of the disease and its complications. It is because of their courage that we have renewed hope that we can stop the progression of cystinosis and find a cure.

Thank you for your dedication to research and to our beloved children and adults with cystinosis. We have more work to do but with you by our side, we will soar to new heights!

View “A Wish Granted” our heartwarming 2022 video featuring the Partington family and their journey with cystinosis. 

A Wish Granted
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A Welcome Message from Nancy, Jeff and Natalie Stack
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Stem Cell Trial: Jordan Janz
Patient 1 Update
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Stem Cell Trial: A Parent’s Perspective
Barb and Jeff Kulyk discuss Jordan’s Journey
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Stem Cell Trial: Jacob Seachord
Patient 3 and his mother, Rocky, discuss their experience
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Stem Cell Trial Update
From CRF-funded researcher Stéphanie Cherqui, PhD
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A Newly Diagnosed Family
Brandon & Mel Parsel share Henley’s Story
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Research Update: Neuromuscular Disease
CRF-funded researcher Reza Seyedsadjadi, MD
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Research Update: Corneal Cystinosis
CRF-funded researcher Morgan Fedorchak, PhD
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Thank You to our Donors
From the CRF families
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Dear Family and Friends, 

This month we celebrate another extraordinary milestone. The fifth patient, our daughter, Natalie Stack, received the stem cell transplant on March 29th. To date, five of the six patients approved for the trial, have been successfully transplanted. We thank Stéphanie Cherqui, PhD, for discovering the treatment and dedicating her career to those with cystinosis. We honor and thank the five adults who volunteered for the stem cell trial; it is because of their courage that we have hope that this treatment might stop the progression of cystinosis or be the cure.

Over time, we will learn how successful the treatment is, but what we know today is that because of you and your steadfast support, our children and adults have a chance to dream of a life free of cystinosis. 

Cystinosis is a relentless disease that eventually destroys every organ in the body. We must continue to fund research to improve current treatments and prolong the lives of those affected by cystinosis. Since 2003, CRF has awarded 212 research grants, 8 extension grants and 9 equipment grants making us the largest fund provider of cystinosis research in the world. 

We have funded research that has led to an FDA-approved treatment and an FDA-approved clinical trial greatly improving the lives of those with cystinosis. We have brought hope to the cystinosis community, but the need to fund vital and life-saving cystinosis research continues. 

Over the past 19 years, you have shared in the highs and lows that we have experienced along our journey toward a cure and now it is time to celebrate all that we have accomplished together.  Join us in the month of April as we present our fundraising program – an inspiring new CRF video and updates from dedicated CRF researchers. Together we honor our CRF community and the brilliant researchers fighting to create better treatments and a cure for cystinosis! 

Since CRF was first founded, Natalie’s wish – to have my disease go away forever –  has united us in our fight against cystinosis, and though we cannot be together in person this year, we will always be united in our hope for a cure. 

With love and gratitude,   

Nancy and Jeff Stack

With your constant support, we have achieved what many thought was impossible – an FDA approved treatment and an FDA approved clinical trial allowing those affected by cystinosis to dream of a life free of the disease. We have brought hope to the cystinosis community, but the need to fund vital and lifesaving cystinosis research continues. 

Over the past 19 years, you have shared in the highs and lows that we have experienced along our journey toward a cure and now it is time to celebrate all that we have accomplished together!

Your dedication to Natalie’s wish — to have my disease go away forever — has united us in our fight against cystinosis and brings us closer than ever in our quest for a cure!

Although circumstances once again have prevented us from safely gathering, please join us for our Natalie’s Wish Fundraiser to celebrate our CRF community and the brilliant researchers working to discover better treatments and a cure for cystinosis.

This year, our annual Natalie’s Wish fundraiser is a video program that features a new video update on CRF, project updates from key CRF researchers, a heartwarming story from a newly diagnosed family, and an update from two of the volunteers in the stem cell and gene therapy clinical trial. You don’t want to miss the special message from our family and patient community who want to express their gratitude to all those who support our research program and our families.

We are pleased to share our fundraising program with you throughout the month of April. The program will be available to view and explore any time that is convenient for you. While we have faced continued challenges because of the pandemic, our research teams have been steadfast in their work every day to find better treatments and a cure. We have reached milestones and have made new discoveries about cystinosis, but we have more work to do and more research to fund.

We are grateful for your past commitment to our research program and thank you for helping us raise funds this year so that we can continue to support ground-breaking research that will undoubtedly lead to a cure for cystinosis.

Thank you!

A Welcome Message from Nancy, Jeff and Natalie Stack
Watch Video
Stem Cell Trial: Jordan Janz
Patient 1 Update
Watch Video
Stem Cell Trial: A Parent’s Perspective
Barb and Jeff Kulyk discuss Jordan’s Journey
Watch Video
Stem Cell Trial: Jacob Seachord
Patient 3 and his mother, Rocky, discuss their experience
Watch Video
Stem Cell Trial Update
From CRF-funded researcher Stéphanie Cherqui, PhD
Watch Video
A Newly Diagnosed Family
Brandon & Mel Parsel share Henley’s Story
Watch Video
Research Update: Neuromuscular Disease
CRF-funded researcher Reza Seyedsadjadi, MD
Watch Video
Research Update: Corneal Cystinosis
CRF-funded researcher Morgan Fedorchak, PhD
Watch Video
Thank You to our Donors
From the CRF families
Watch Video

We have missed all of you, and although we hoped we would be able to meet in-person in 2021, we could not safely do so. This year, our annual Natalie’s Wish fundraiser is an interactive video program that features an update on CRF, project updates from key CRF researchers, a heartwarming story from a newly diagnosed family, and an update from two of the volunteers in the stem cell and gene therapy clinical trial. You don’t want to miss the special message from our family and patient community who want to express their gratitude to all those who support our research program and our families.

We are pleased to share our fundraising program with you today. The program will be available to view and explore any time that is convenient for you. While we have faced challenges because of the pandemic, our research teams have continued to work every day to find better treatments and a cure for our adults and children with cystinosis. Thank you for your past commitment to our research program and thank you for helping us raise funds this year so that we can continue to fund ground-breaking research that will undoubtedly lead to a cure for cystinosis.

Thank you for joining us for our Natalie’s Wish Fundraiser to celebrate our CRF community and honor the brilliant researchers working to create better treatments and a cure for cystinosis. Your generosity and commitment to funding cystinosis research raised $1.9 million!

Your dedication to Natalie’s wish – to have my disease go away forever – has united us in our fight against cystinosis and brings us closer than ever in our quest for a cure!