❤️ Help Us Give Zeccai More Time

Help Our 4-Year-Old Grandson Fight Sanfilippo Syndrome

There are some things no grandparent should ever have to write.

When our grandson Zeccai was just 2 years old, our family received devastating news: Zeccai had been diagnosed with Sanfilippo syndrome Type A (MPS IIIA), a rare and terminal genetic disease that progressively damages a child's brain and nervous system.

Sanfilippo has sometimes been described as “childhood Alzheimer's.” Children may begin life developing normally, but the disease gradually causes them to lose abilities they have worked so hard to learn — including speech, cognitive abilities, motor skills and other developmental functions.

Zeccai is now 4 years old. And he is fighting every day.

Zeccai Was Given a Chance

When Zeccai was only 2, he was accepted into a clinical trial for GC1130A at UCSF Benioff Children's Hospital in Oakland, California.

GC1130A is an investigational treatment being studied for children with Sanfilippo Type A. It delivers a replacement version of the enzyme that Zeccai's body is missing directly into the cerebrospinal fluid through an intracerebroventricular access device. The goal is to get the enzyme directly to the brain and central nervous system.

The UCSF study is specifically investigating GC1130A delivered through an intracerebroventricular access device.

Zeccai was the first child in the world to receive this treatment.

At only 2 years old, while most children his age were learning new words, playing with toys and discovering the world around them, Zeccai was facing experimental treatment for a disease that had no FDA-approved therapy to alter its underlying course.

His parents had to make an incredibly difficult decision: take the chance on an experimental treatment because doing nothing meant watching this devastating disease continue to take pieces of their little boy away.

What Is Sanfilippo Syndrome Type A?

Sanfilippo syndrome Type A is caused by changes in the SGSH gene, which result in a deficiency of an enzyme called sulfamidase, also known as heparan N-sulfatase.

This enzyme normally helps the body break down a complex sugar called heparan sulfate.

Without enough of the enzyme, heparan sulfate builds up inside cells, particularly affecting the brain and central nervous system. Over time, this buildup causes progressive neurological damage.

Children with Sanfilippo Type A can gradually lose developmental abilities, including language, cognition and motor function. The disease is progressive and life-limiting.

The Device That Makes Zeccai's Treatment Possible

To receive GC1130A, Zeccai has an Ommaya reservoir, also called an intracerebroventricular access device.

An Ommaya reservoir is a small device surgically placed under the scalp with a catheter that reaches into a brain ventricle containing cerebrospinal fluid. It provides doctors with repeated access to the fluid surrounding the brain so medication can be delivered directly into the central nervous system.

For Zeccai, this device is what allows his investigational treatment to be delivered directly where it is needed.

But having a device that enters the brain comes with very real risks.

Potential complications of an Ommaya reservoir include infection, meningitis, bleeding or hemorrhage, catheter misplacement, blockage or malfunction, cerebrospinal-fluid leakage, and other neurological complications. Some complications can require additional surgery.

Every time Zeccai receives treatment through this device, his family carries the fear that something could go wrong.

Yet they keep going. Because Zeccai is worth fighting for.

✈️ Every Two Weeks, They Travel Across Colorado and California

Zeccai and two family members travel from Colorado Springs to Oakland, California every two weeks for his treatment. The program pays for most of Zeccai's travel expenses, which is a tremendous help to his family.

Even with that assistance, this journey has placed a significant emotional and physical toll on the family. Two family members have to take time away from work and their regular schedules to travel with Zeccai for his treatments every two weeks. They are constantly balancing work, family responsibilities, travel and the needs of a little boy who is fighting a devastating disease.

This journey has created an enormous hardship for Zeccai's family.

Most importantly, there is the emotional toll of repeatedly traveling across the country and putting their little boy through treatment, knowing the risks involved with his Ommaya reservoir.

His mom and dad are doing everything they possibly can to care for him while also being there for their daughter, little Elliana, who is just one year old. She loves her brother and is growing up alongside this journey, too.

The Cost of Fighting for Our Son and Grandson

Fighting Sanfilippo doesn't happen just inside a hospital.

It happens every day.

It means rearranging work schedules.

It means traveling across the country every two weeks.

The trial does pay for flights, hotels, transportation, food and countless other expenses, however the trial is ending in November.

This means medical bills and ongoing care.

And now there is a newly approved treatment that could potentially give Zeccai another opportunity.

Because FAYUVI is a newly approved gene therapy, the exact cost of treatment and all of the expenses associated with receiving it are not yet known to our family. We know that a treatment like this has the potential to be extremely costly, and there may also be significant expenses involved with evaluation, specialized medical care, travel and follow-up treatment.

We simply don't know yet what the total cost will be.

But we don't want the day to come when Zeccai's doctors tell us that he may have an opportunity and his family has to say, “We can't afford it.”

That is why we are asking for help now.

We want to be prepared.

We want Zeccai's family to have the ability to pursue every medically appropriate opportunity available to him without finances being the reason they have to walk away.

We Are Asking for Your Help

We are starting this fundraiser because we want to give Zeccai every opportunity available to him.

We want to help his family continue his current treatment and travel.

We want to help with the enormous expenses they have already faced.

And if Zeccai's doctors determine that FAYUVI is appropriate for him, we want his family to be financially prepared to pursue that opportunity.

We know the amount we are asking for may seem substantial.

But the cost of this journey has already been substantial — financially, physically and emotionally.

And because we don't yet know what the cost of FAYUVI treatment will ultimately be, we are trying to prepare for the possibility of significant medical expenses rather than waiting until the last minute to ask for help.

❤️ Every Donation Can Make a Difference

If you can donate, please know that your contribution is helping give Zeccai and his family options.

A $10 donation matters.

A $25 donation matters.

A $50 donation matters.

A $100 donation matters.

Whatever you are able to give, it all adds up.

If you cannot afford to donate, please don't feel bad. Sharing Zeccai's story is also an incredibly meaningful way to help.

You never know who might see this fundraiser.

You never know whose heart might be touched by Zeccai's story.

And you never know whether one share might reach someone who can make a tremendous difference for his family.

Please donate if you can. Please share if you can. And please keep Zeccai in your thoughts and prayers.

❤️ What We Are Really Trying to Raise Is Time

We aren't simply trying to raise money.

We are trying to raise time.

Time for Zeccai to laugh.

Time for him to play with his little sister.

Time for him to hug his mom and dad.

Time for more birthdays.

More Christmas mornings.

More family pictures.

More ordinary days that most of us never think twice about.

❤️ Please Help Us Fight for Zeccai

Sanfilippo has already asked this little boy and his family to endure more than any family should have to endure.

But Zeccai keeps fighting.

His parents keep fighting.

His family keeps fighting.

And now we are asking you to fight alongside them.

If you are able, please make a donation today.

If you can't donate, please share this fundraiser with your friends, family, coworkers and community.

Every dollar. Every share. Every prayer. Every person who helps spread Zeccai's story matters.

Please help us give Zeccai, his mom, his dad and his little sister Elliana a little more hope.

Please help us give this beautiful little boy a chance at more time.

❤️ Donate. Share. Pray for Zeccai.

Thank you from the bottom of our hearts for standing beside our family.

— Zeccai's grandparents and family