fatal disease at center of multimillion

fatal disease at center of multimillion

A Reno couple who have spent the past 10 years trying to save their daughters lives are now battling biotech and pharmaceutical companies that they claim stole a lifesaving treatment they pioneered that could be worth hundreds of millions of dollars. District Court in Reno, Chris and Hugh Hempel and their twins Addison and Cassidy, 13, are suing for “no less than” $100 million for what the suit says is the misappropriation of confidential information and a plan to market a treatment for Neimann Pick Disease Type C.

In the lawsuit filed Jan. 5, the Hempels claim they were swindled out of a plan they had to develop a treatment that has kept their girls alive. The lawsuits says big pharmaceutical companies have used their plans and their daughter’s medical records to broker multimillion dollar deals.

Addi and Cassi Hempel were diagnosed with Niemann Pick Type C when they were 3. The rare and fatal genetic disease is often referred to as childhood Alzheimer’s disease. It causes cholesterol to build up and be trapped in cells, overloading organs and attacking the brain. Most children don’t live past their teens.

Twins Addi, left, and Cassi Hempel wait in their Renown Regional Medical Center hospital beds in 2009 for infusion pumps to be attached to ports

(Photo: RGJ file)

But after receiving the devastating news this would kill their girls, the Hempels went to work, achieving something unheard of in the medical community, according to Dr. Caroline Hastings of the UCSF Benioff Children’s Hospital in Oakland. She has treated the girls.

The Hempels found researchers who were about to publish a study on the effects cyclodextrin, a simple sugar compound, has on mice with Niemann Pick Type C.

They formed a team, with Hastings, and successfully lobbied the Food and Drug Administration for approval to treat their daughters using that compound.

Many called them crazy to try something untested, untried and unstudied on humans.

had other doctors say they wouldn’t have agreed to do what we did, Hastings said.

remember early on someone asked me, is the worst that can happen from trying this? Hastings said.

Today, the girls continue to suffer from seizures and periods of dementia. Just a week shy of their 14th birthdays, they have lost their ability to walk and talk but still recognize and communicate with their parents through verbal sounds, touching and kisses.

They require constant, 24 hours a day, seven days a week care at home.

Chris and Hugh Hempel and their attorneys would not comment on the lawsuit, but the 25 page court document reads like a David vs. Goliath story.

In August, Chris Hempel wrote on Facebook, massive fraud has been perpetrated not only against the twins and our family but our researchers, doctors, hospitals, donors and community. Cyclodextrin has been stolen and now sold for $200 million dollars!
fatal disease at center of multimillion
! It’s going to a billion++ dollars when approved. This was a drug funded and created by all of you who helped us.

Cydan, funded by big name pharmaceutical companies including Pfizer, one of the largest pharmaceutical companies in the world. Its website says it was the first orphan drug accelerator, named for the Orphan Drug Act of 1983. The act is meant to encourage the development of medicines for rare diseases. It grants seven years of exclusivity for drugs that treat a specific condition that affects fewer than 200,000 people, making a drug that previously pharmaceutical companies have not allocated time and resources for because of the possible limited financial gain. According to the suit, Cydan first approached the Hempels about a partnership to develop cyclodextrin in 2013. Soon after reaching out to the Hempels and agreeing to a joint venture, Cydan formed Vtesse, a company dedicated to developing drugs for Niemann Pick Disease Type C and other rare diseases. In April 2017, Vtesse was purchased by Sucampo Pharmaceuticals for $200 million, in large part because of the same treatment, the suit says, the Hempels were in contract to develop. In December 2017, Sucampo was purchased by Mallinckrodt, a global pharmaceutical company, for $1.2 billion.

(Photo: Provided to the RGJ)

“Cydan, Vitesse, and Sucampo swooped in to misappropriate the Hempels’ confidential information and trade secrets, developed a directly competitive treatment, and sold it to an international pharmaceutical company for hundreds of millions of dollars,” the lawsuit said.

“Cydan’s, Vtesse’s, and Sucampo’s misconduct robbed the Neimann Pick Type C community of its power to fund and control future developments in NPC treatments, and their ill gotten gains must be disgorged.

In a statement, Sucampo said, we cannot comment on active litigation, we will defend our position vigorously.

When Addi and Cassi were born on Jan. 23, 2004,
fatal disease at center of multimillion
Chris and Hugh Hempel were overjoyed.

She dreamed of being a soccer mom, and as the babies grew so did their energy. The couple took hundreds of photos documenting their seemingly perfect life.