Local Doctors Launch Moonshot Research to Save Daughter
photo courtesy of Kree Photography
Sutter Health physicians Geri and Zach Landman are racing against time to save their two-year-old daughter, Lucy, who suffers from an ultra-rare gene defect.
Two East Bay doctors are racing against time to save their two-year-old daughter, who suffers from an ultra-rare gene defect.
Sutter Health physicians Geri (Pediatrics) and Zach Landman (Pain Management) were on vacation in 2022 with their three daughters when their baby Lucy contracted a cold that drained her of her energy and ability to sit up and eat normally. Multiple brain, spinal, and nerve tests proved negative, but genetic testing found Lucy has two bad copies of the PGAP3 gene, one of the many genes involved in cell communication. It's one tiny amino acid difference in one tiny protein, but it's critical.
“Her brain is developing normally, but it might not communicate properly with the rest of the body,” Zach Landman told the Forum. “Without a cure, we were told that she would never walk or talk, and she would suffer from severe seizures. Geri and I have medical training, so we read all the papers on the subject and found some startling information.”
Lucy's condition is so rare that only 30 to 50 people worldwide have this defect in their genes, Zach said, adding that his other daughters do not have the same condition.
The cure for PGAP3 is being developed at Nationwide Children’s Hospital Center for Gene Therapy right now. Scientists have used Lucy’s donated cells as the scientific building blocks for the cure. Zach says the latest round of clinical tests should be wrapped up by this Fall, and the family will know if the treatment has a chance. The procedure includes adding the proper genes into Lucy’s system using the shell of a virus structure as the carrier.
However, because there are simply not enough kids known to have Lucy's genetic disorder, no drug company, government, or investor is interested in funding this research.
Last year, the Landmans co-founded Moonshots For Unicorns (moonshotsforunicorns.org), a 501(c)3 non-profit, to raise awareness and pay for the nearly $2 million in research costs. The results would be made available to other researchers as a way to treat other gene-related issues.
“The goal is to give these kids the best shot at life,” Zach said. “About 1 percent of people in the United States have some type of genetic disease. We would use the results from Lucy's treatment like researchers do with developing flu shots. Each year, we would build on developing research to be able to treat more people.”
The Landmans have been in contact with four or five other families whose loved ones may have similar gene therapy needs that could benefit from Lucy’s story.
But in the process, their youngest daughter has taught the family resilience.
“Marrying my personal and professional life has taught me, and I look for what Lucy has given us,” Zach said. “From this experience, she taught me to be a listener and to make sure I am learning and growing.”
The Moonshots for Unicorns page (moonshotsforunicorns.org) provides information on getting involved and opportunities to donate. You can follow Lucy's journey on her Instagram page (@lucythepgap3goose).