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Jen VanHoutan gives her daughter Laine a kiss June 20, 2017. Laine died March 21, 2018, of a rare, inherited genetic disorder of the nervous system. (Alyssa Pointer/Chicago Tribune/TNS)

Alyssa Pointer

DOWNERS GROVE, Ill. — Less than a year after the U.S. Food and Drug Administration approved the first drug to treat her rare disease, a Downers Grove girl died at home with her parents, who spent nearly a decade crusading for the treatment, at her side.

Laine Manning VanHoutan, 12, died of CLN2 Batten Disease, a rare, inherited genetic disorder of the nervous system that typically begins in childhood.

“The disease is just ruthless,” her mother, Jen VanHoutan said, adding that she and her family took comfort in knowing their “adventure princess” was finally free of the illness that robbed her of her ability to walk, swallow and communicate. “She’s whole again.”

Jen and Tracy VanHoutan set out on a tireless quest to find a CLN2 cure in 2009 after their son, Noah, was diagnosed. At the time, the disease was always terminal. The search for treatment became even more dire when genetic tests revealed that Laine, then 3, also had CLN2 Batten Disease, even though she had not yet shown any symptoms.

The VanHoutans traveled overseas to identify scientists working on potential therapies to replace the missing enzyme that led to the disease. They pleaded with the U.S. Food and Drug Administration for less stringent clinical trial requirements and lobbied Congress to allocate more money to rare disease research. They became leaders in the rare disease community, and organized fundraisers to raise more than $1.5 million to fuel the search for a Batten disease cure.

During one trip to urge scientists along in their research, Tracy VanHoutan met with doctors as Laine, a blond-haired, fairy-loving, tea-party-throwing little girl played nearby.

On April 27, the family’s efforts paid off when the FDA approved the first known drug found to dramatically slow the effects of CLN2 Batten disease. The treatment is expensive and does not eliminate the disease. Results thus far have found that children treated early in the disease’s progression are able to halt further deterioration almost completely.

But the treatment, called Brineura, came too late for the VanHoutan children. Noah died in March 2016, just shy of his 12th birthday. Laine had been in hospice care for several months, and was struggling to breathe so much in recent weeks her parents hesitated to leave on trips to promote rare disease research.

“This is going to be our kids’ legacy,” Jen VanHoutan said.

Despite the disease that stole Laine’s childhood, her parents did their best to keep allowing her exciting experiences. She skied white-powdered mountains in Keystone, breathed the fresh evergreen air in Snowmass and felt sand between her toes on Florida beaches. She saw Cinderella’s castle at Disneyworld and frolicked in the grass at the Easter Egg Roll at the White House.

“She deserved to be able to live,” Jen VanHoutan said.

Laine’s death touched off an outpouring of compassion from both the rare disease community across the U.S. and neighbors in Downers Grove. Hundreds of people — many with children struggling or deceased after a rare disease — sent sympathies in comments on Facebook, while friends lined their suburban street with luminarias.

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The VanHoutans plan to keep advocating for rare disease research and improved treatments for CLN2 Batten Disease, even though Noah and Laine are gone, they said.

In a story about their family’s crusade published in the Tribune in July 2017, Jen VanHoutan compared Noah and Laine to lotus flowers — beautiful, strong and determined even though they bloomed amid mud, murky water and ugliness.

Jen VanHoutan said she smiles picturing Laine and Noah together now in heaven.

“I know he was waiting for her since he got there,” she said.

Information about the family’s efforts to fight the disease and raise money for research is available at NoahsHope.com.

Copyright 2018 Tribune Content Agency.

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