Moonshots for Unicorns seeks to treat ultra-rare conditions | News

After diagnosing their young daughter with an extremely rare genetic disorder, two Danville doctors have combined their professional and personal lives to fill a gap in modern medicine that they have become particularly sensitive to over the past year.

Geri and Zachary Landman’s youngest daughter Lucy – now 14 months old – has been diagnosed with a disorder in her PGAP3 gene for which there is no treatment or cure on the market, making her one of about 50 patients worldwide.

While living with their daughter’s disorder and founding the nonprofit Moonshots for Unicorns have since become flashpoints in the Landmans’ lives, with numerous hurdles to overcome in order to develop a treatment and cure, they said it the first thing was to get a diagnosis place was a challenge.

“One of the tenets of medicine is if something doesn’t change what you’re doing, then why waste the cost or time on testing,” said Zachary Landman. “It’s a chicken-and-egg situation because we don’t test for it because there is no treatment for it, and there is no treatment because there is no test for it.”

It was Geri Landman’s expertise as a pediatrician and the couple’s professional connections in the medical world that saw her persistence in finding a diagnosis for her daughter pay off.

“Geri was really concerned,” Zachary Landman said. “She said, ‘I’m not just a mom, I’m not crazy, I’m a pediatrician,’ and sent several videos of her doing increasingly weird things like falling over and not being able to sit up on her own.”

He added that in many cases, the disorder leads to the diagnosis of autism and epilepsy without further investigation into the actual cause of the symptoms.

“If Geri hadn’t been a pediatrician and if I hadn’t worked at Stanford and if our neurologist hadn’t happened to have had a personal connection with Geri while we were in the hospital and also happened to have been a neurologist as a genetics specialist, Lucy would never have had that diagnosis.” received,” said Zachary Landman.

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But despite their luck at getting a diagnosis, the Landman family was devastated by the result, and at first there was no hope in sight.

“Initially we didn’t believe it, but of course it’s true and the follow-up investigations have shown it to be true. So we took a week off and we cried, and her older sisters cried a lot too,” he said.

The Landmans would later learn that the sense of helplessness they felt was all too common for families of extremely rare disease patients. But unlike most, both doctors were able to use their expertise to push for solutions.

“I reached out to Geri and said we can lie in bed and feel sorry for ourselves for the rest of our lives, feel sorry for Lucy, or we can do something about it,” Zachary Landman said.

The couple, who met while visiting UCSF, quickly jumped back into action.

“We just read every single piece of research paper on PGAP3, and we just emailed every author, every scientist in the world who worked on it within two to three weeks,” Zachary Landman said. “We got the science, we got the pathophysiology of the disease as well as anyone did at the time.”

Their efforts resulted in a whirlwind of meetings with physicians, scientists and pharmacy CEOs around the world, and an upcoming meeting with White House officials.

The good news, the Landmans discovered, was that gene therapy showed promise for treating PGAP3 and other extremely rare genetic diseases. The bad news, however, was that the rarity of the condition meant a lack of market demand.

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“Ultimately, our health care system is designed for for-profit, market-sized developments, so it’s not the fault of the people or scientists working on drug development, but we’ve been told numerous times that… PGAP3 would be perfect if you had 1,000 kids on the world – better yet in the United States because they can afford it, then we can absolutely recover the cost of developing the drug,” said Zachary Landman.

With the goal of both developing a treatment for their daughter and addressing the lack of access to gene therapy for extremely rare diseases, the Landmans started the non-profit organization Moonshots for Unicorns.

“Unfortunately, no drug company, government or investor is interested in funding this research because there just aren’t enough children with Lucy’s genetic disorder — we have to be,” Geri Landman said on a GoFundMe page for the endeavor.

Geri Landman also emphasized that she and her husband’s doctors put their daughter in a unique position for development and treatment.

“No child in the world with this condition has had an opportunity to receive effective treatment so early in life, which means there’s nothing we can do about what we can do for them,” said Geri Landman.

Zachary Landman noted that while the couple are fortunate to be able to remedy the lack of treatment, the organization’s mission is to provide an equal opportunity for all.

“We look at Lucy and say we want to get her treated as soon as possible,” Zachary Landman said. “[It’s]about $2.5 million to develop a treatment. That’s why we created Moonshots – to hopefully have it, so no parent has to go to sleep at night being told what we’ve been told, what’s been told, that there is no treatment and no cure, and nothing you can do for your child can do.”

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With Moonshots for Unicorns, the Landmans also want to help other families of loved ones with extremely rare genetic disorders feel less alone and find strength in numbers. Zachary Landman, who comes from a family of doctors, said he’s also trying to spread a message his father left him.

“My dad was actually a pediatrician his entire career, and he’s taken care of…probably kids like Lucy his entire career,” Zachary Landman said. “The advice he gave me was he said, ‘Look, it really sucks and you can’t see it now, but over time I’ve found through years of working with families that your family is often closer grows together and becomes much stronger because of this.'”

Zachary Landman said that was the case with his own family and that the renewed connections between them served as further fuel for Moonshots for Unicorns.

“For anyone who’s recently been diagnosed or who’s just going through it for the first time, we’re only five months into this, but we’re really grateful for how it’s brought our family together and kind of aligned us,” he said. “It gives you a kind of completely different perspective on life, and that certainly applies to Geri and me.”

For more information on Moonshots for Unicorns and updates on the latest developments in research at the organization’s pop-up lab, visit

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