Hope For A Cure


Jenna & Patrick’s Foundation of Hope

Cystinosis Research Update

Research Overview

September 8, 2020


It is worth noting, that Jenna & Patrick’s Foundation of Hope passes 100% of funds raised to the Cystinosis Research Foundation, where the dollars are put to work judiciously and quickly.*

Stem Cell Trial at UCSD:
The FDA stem cell trial is currently in phase one. There will be six patients total in phase one. Jordan, patient #1 is doing well.  At nearly a year post‐transplant, he remains off of his previously life‐saving medications and is doing remarkably well, (he might say he “is cured”). Patient #2 is also doing well! The most recent patient to the trial, Jacob, is 22 years old. Jacob’s stem cells were recovered last week. The plan is for Jacob to return to UCSD the first week of November for the transplant. Patient #4 should begin the process in January. In spite of the pandemic, the study is moving along (very carefully and safely). By this time next year, all six patients should be transplanted. Jenna and Patrick will not be candidates for phase one of the trial, due to their age and declining kidney function, but they look forward to learning the parameters for phase 2‐ and beyond‐ next year!

Research Looking forward:
The Cystinosis Research Foundation Scientific Review Board recently approved eleven new grants, four research study extensions and one grant for equipment totaling over $2.6 million in new research projects. For a complete list of research grants awarded last month please click on this link https://www.cystinosisresearch.org/grants‐awarded/  It is important to point out that these studies benefit the world of medical research at large. Much of what is learned in these studies of Cystinosis is translated to use for other diseases.

We just announced the Autumn 2020 call for research applications. There will be $2.5 million available for new grants and we hope to receive some interesting applications. While we are in the midst of a pandemic, medical research keeps marching on!

We are so grateful for your support.


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