These Twins Have Been Slowly Deteriorating Since Age 3, Due to Rare Childhood Alzheimer’s
When her twins, Addison and Cassidy, were born, Chris Hempel dreamed of being a soccer mom. It wasn’t until the active and curious girls were three years old that they began getting noticeably clumsier and less vocal and finally received the diagnosis that would shut down so many of their parents’ dreams for them: childhood Alzheimer’s.
Childhood Alzheimer’s is a name given to a variety of rare diseases that cause symptoms similar to Alzheimer’s disease in young children. One of the most common of these diseases is Niemann-Pick Type C, a storage disorder in which cholesterol builds up in the cells, causing vital organs to be overloaded and the brain to stop functioning normally over time.
Most children diagnosed with this rare disease don’t live to see the end of their teen years. When Addi and Cassi were given their diagnosis, the family was told they wouldn’t live beyond the age of ten.
But Chris and her husband, Hugh, weren’t ready to give up yet. The pair had cashed out as some of the first employees at Netscape and had some extra money they could put into finding the best possible care for their daughters. So they went to work, becoming avid activists and fundraisers along the way.
Before long, the Hempels stumbled across cyclodextrin, a sugar-like compound being studied at the University of Texas. The treatment was providing positive results to patients, so the Hempels ordered some from a distributor and mixed it in water to give to their girls.
Later, after talking to a family in India whose son was the first to have the compound administered via an IV, the Hempels lobbied the FDA for permission to administer it intravenously as well. They wrote letters to the FDA acknowledging that the treatment had the potential to kill their daughters but that they believed it could also possibly save their lives and offer hope to other families in the future.
They were approved to use cyclodextrin intravenously, and the girls got their first dose in 2009. But the obstacles kept coming. Tests showed that the compound was not able to cross the blood-brain barrier and that the only way to make it effective for the brain would be to administer it through a port directly to the brain or through spinal injections. After another appeal to the FDA, the Hempel twins were the first ever to receive cyclodextrin treatment this way.
It has always been the Hempels’ hope to continue to research and develop cyclodextrin treatment and make it affordable for other families. So when they were approached by a drug company called Cydan, they were eager to work together toward a solution. They created a legal agreement with the company and shared their daughters’ full medical histories and research data in an attempt to get the drug fully approved and available to other families.
In 2014, however, they stopped receiving communication from Cydan, just as it was opening Vtesse, a daughter company built to develop cyclodextrin products. Now cyclodextrin is on a fast-track towards FDA approval, a status the Hempels believe it could not have achieved without the data they provided. But when it gets approved, there’s no longer any promise that the pharmaceutical companies will stick to the affordable prices that were part of their agreement with the Hempels.
The family is now in the middle of a nasty lawsuit and is hoping to win so that the pharmaceutical company will not be able to set sky-high prices on the drug that has done so much for their daughters and others with Niemann-Pick Type C.
Addi and Cassi are now 14 years old and showing some measure of improvement. Their hearing has improved and the worsening of their mobility and vocal abilities has slowed down significantly. They are no longer able to walk or talk, but they communicate with their parents through noises and touch. However, if a big pharmaceutical company is allowed to take exclusive rights to cyclodextrin, the treatment may become more expensive than they and others like them are able to afford.
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