No one should outlive their child. Losing two children to the same rare disease is a pain that most parents can never understand and hopefully never will. According to a news report on a local Chicago TV station, one families tragedy may be averted if a newly developed treatment continues to show results after initial successes.
The VanHoutan family lost daughter Laine and her brother, Noah, right around their 12th birthday. To give the disease some context, Batten disease is described as giving a child both ALS and Alzheimers, causing a decline in both physical and mental ability.
But there is good news on the horizon for the first time for patients and families that suffer from this inherited disease. A new treatment recently approved by the FDA is not only slowing the progression of the disease, but in some cases patients are showing improvement. The findings were recently published in an article in the New England Journal of Medicine.
Brineura was approved to treat a specific form of Batten disease. It’s the first treatment approved for the CLN2 type in patients three years old and up who have symptoms. In this case, Brineura was able to extend the ability of patients to continue walking. Prolonging a patient’s ability to walk is a major improvement for quality of life and a big win for patients with Batten Disease.
“It really is the holy grail of drug development to see an effect like that.”
— Tracy VanHoutan,
father of two victims of
Batten disease is a genetic nervous disease that usually strikes children between the ages of 5 & 10. Most patients with Batten disease eventually die from it by their late teens or early twenties. Batten disease is rare and only occurs in about 2 to 4 of every 100,000 live births in the United States.
Unfortunately for the VanHoutan family the developments did not appear in time to prolong the lives of their children. Laine died at the end of April. To honor the lives of the children, the family has created a charity and will be hosting a 5K to raise funds for Batten disease research.
Support for Batten disease can be found on a special group on Facebook devoted to the patients, families and care givers who struggle with this truly devastating childhood disease.