A Dying Girl's Family Takes On Big Pharma Over Drug Access
A U.S. pharmaceutical company testing a drug meant to slow down child dementia refuses to offer the medication to a terminally ill young girl in Germany. Her family is fighting the decision.
MUNICH — Hannah suffers from child dementia, and the 9-year-old will die within the next few years. Special medication could help, but the pharmaceutical company that produces this medication doesn't want to give it to Hannah.
The Vogel family was in Spiekeroog for their summer holiday when Hannah's problems became obvious. She was suddenly unable to cycle anymore, and she needed help when she descended the stairs, or would instead go down by sliding one step at a time on her bottom. "That's when we realized something was seriously wrong," says Stefanie Vogel, Hannah's mother.
The family has known about what's ailing Hannah since February. She was diagnosed with the very rare hereditary disease NCL2, also known as CLN2, or child dementia. It is one of the worst diseases a child can develop, caused by a single genome in Hannah's DNA being defective, which in turn means her cells are unable to "digest" her body's waste products. The body is essentially poisoning itself over a long period of time. Walking, talking and writing are already difficult tasks for Hannah, but her steady decline will worsen over time. In the end, she'll be unable to do anything and will die in a few years' time. "After we received the diagnosis, we just cried for weeks on end," Hannah's mother says.
But there could be hope for Hannah. An American pharmaceutical company called Biomarin has developed a drug for NCL2 that is currently being clinically tested in four different countries. There are even 12 children in Hamburg who are receiving this drug, and some parents of these children say that the medicine, BMN 190, has stopped progression of the disease. Biomarin itself has noted the drug's "encouraging results."
But though it would be legally possible to offer the drug to Hannah, the company refuses to give her the medication. Biomarin cites its "ethical responsibilities" towards all patients, saying its goal is the quickest possible approval of the drug. Company officials argue that approval could be endangered if the drug were to be given to a patient outside of the clinical trial parameters.
The company's position is incomprehensible to Hannah's parents, who have decided to fight the decision. They have created both an online petition and a Facebook page to put pressure on Biomarin. "You simply have to help a terminally ill child if you have the means to do so," Stefanie Vogel says.
They have even found a physician who would be able to treat Hannah with the drug should it become available to her. "Some children from the study have already received the drug for over a year without any side effects worth mentioning," says metabolism expert Thorsten Marquardt of the University Hospital of Münster. This is also verified by Angela Schulz, who is conducting the study with BMN 190 at the University Hospital Hamburg-Eppendorf: ‘We are glad that, so far, we have not noticed any serious side effects,’ she says.
She doesn't want to comment on some of the success stories behind BMN 190 because a scientific evaluation would have to take place beforehand. But it is nonetheless clear that this situation presents a dilemma for her. "It is heartrending to exclude children from this trial who could benefit from it."