A family in Queensland, Australia, is faced with selling their dream home to raise funds for their daughter’s life-saving therapy, which will cost $3 million.
Tallulah Moon, 5, has been diagnosed with SPG56, a degenerative brain disease caused by a rare gene mutation.
Tallulah was a healthy and happy child until shortly after her first birthday, when she suddenly began to lose her motor skills.
“She was hitting all her milestones, and then all of a sudden she experienced a really steep regression — her skills just fell off a cliff and it was horrible,” Golden Whitrod, Tallulah Moon’s mother, told Fox News Digital during an interview on camera.
“She went from a little girl who was walking and talking to suddenly not being able to sit up on her own, not being able to lift her arms over her shoulders or hold her neck up,” Whitrod said.
Swallowing and choking also became a concern.
“We had gone from watching this beautiful baby blossom at 14 months, to returning to the abilities of a 4-month-old,” Whitrod recalls.
Tallulah Moon was also horrified, unable to comprehend the loss of her abilities.
“I remember her looking at us as if to say, ‘Why can’t you help me?’ said her mother. “And I could feel that as a parent. I just didn’t know what to do.”
A devastating diagnosis
At first, Whitrod hoped there would be an easy fix for whatever was causing Tallulah’s downfall.
After six months of testing and scanning, doctors performed a genetic study known as whole genome sequencing (WGS), leading to Tallulah Moon’s diagnosis of SPG56 in August 2020.
SPG56 is a type of hereditary spastic paraplegia (HSP) that usually begins around age 1 or 2 and worsens over time, causing muscle weakness and gradually robbing children of the ability to walk, talk, stand and sit, as seen in past cases.
In later stages, the disease can cause cognitive decline, seizures and even the inability to swallow.
SPG56 is one of the rarest types of HSP, affecting less than one in every million children, statistics show.
There is currently no cure for the disease.
“The doctors told us, ‘just love your baby,'” Whitrod said. “They said there was nothing they could do – that there were no treatments.”
A mother’s determination
After “coming out of the fog” after her diagnosis, Whitrod immersed herself in research, making connections with other families whose children were also living with rare genetic diseases.
One of them was Terry Pirovolakis, a Canadian father whose son was diagnosed with SGP50, a disease that is very similar to Tallulah Moon’s SPG56.
Pirovolakis immediately began researching to find a gene therapy that could help his son.
After meeting with experts from around the world, he liquidated his life savings and paid a team of researchers to begin developing the therapy. In 2022, after massive fundraising efforts, his son received the one-time treatment, which halted the progression of the disease.
Following Pirovolakis’ direction, Whitrod met with scientists at genetic institutes and carefully built her research team.
Over a three-year period, the team created an experimental gene therapy for SPG56, which Whitrod calls a “massive win.”
The next step is for the therapy to go through clinical trials to make sure it’s safe and effective for the children who need it — but the cost is too high for the vast majority of families to cover.
According to Whitrod, it will take $3 million to manufacture the drug before it can be given to Tallulah Moon in a clinical trial.
“Unfortunately, big pharmaceutical companies are simply not interested in funding research into these rare diseases, even though treatments are possible,” she said.
“We realized we had to fund it ourselves if we were going to do this.”
Walter Gaman, MD, founder of Texas Executive Medicine, emphasized the financial burden that often comes with rare diseases.
“Rare diseases, by their nature, make up a small portion of the market, which means there are few customers to absorb the cost of bringing effective drugs to market,” said Gaman, who is not related to the Whitrod family. , for Fox News Digital.
In 2003, Deloitte reported that the average cost of bringing a drug to market exceeded $2 billion, according to Gaman.
“One of the most important victories for orphan drugs came in 2017, when President Trump signed the Food and Drug Administration Reauthorization Act (FDARA) into law,” he noted.
“This was a major win for orphan therapies because it expanded the FDA’s Rare Disease Program and also expedited the review process. As a result, 2018 saw a record 59 orphan drugs given the green light. We have to build that momentum once again.”
There is still a lot of work to be done, he noted.
“We need to bring orphan drugs to market, but we also need to look at ways to make these drugs more affordable for the end user,” Gaman said.
Some possible ideas are to offer tax breaks to companies that support such drugs, or to have a tax on pharmaceutical companies that goes directly to the FDA’s Rare Disease Program, he suggested.
A ‘much bigger dream’
“We are standing on the precipice of a treatment for Tallulah and for children in her position,” Whitrod said.
“We feel like we’re almost there. But, of course, $3 million for a small Aussie family is a lot.”
The Whitrods have set up a charitable foundation called Genetic Cures for Kids, with a fundraising initiative called Our Moon Mission.
The family has raised some funds through GoFundMe donations, but it’s only a fraction of what’s needed to treat Tallulah Moon — hence their decision to sell their home.
The house, which is located on the outskirts of Stuart Park, is scheduled to go up for auction this week.
“We had hoped that some miracle would happen and we wouldn’t need to sell it and that help would come before we needed to come to this difficult point,” Whitrod said.
“But in the end, we realized that this is our last asset that we have, and this can help us get to the finish line.”
While they are sacrificing their family’s “dream home,” Whitrod says they are now aspiring to a “much bigger dream.”
“That means giving Tallulah the life she deserves — and we’re all for it.”
Meanwhile, Whitrod said, Tallulah Moon is working to fight the neurodegenerative disease through physical therapy, occupational therapy and speech therapy.
As she sets out to save her daughter, Whitrod has also set a broader goal — to help other families whose children are facing similar challenges.
“What we’re creating is not just a treatment for Tallulah that ends up in children with SPG56 — we’re creating a replicable framework, so researchers can go on to create treatments for other similar diseases,” she said.
“If we get there in time, then we can help Tallulah live the life she deserves – and not just her, but many children like her.”
For more information on Our Moon’s mission or to donate, people can visit https://ourmoonsmission.org/donate/.
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