JACKSONVILLE, Fla. — "I just want there to be some hope... life with VHL is different from what most other people deal with... I've had to deal with it two ways one as a patient and one as a parent and I think it's harder to deal with it as a parent."
The words echo in Robert Kramer's office as he talks about about his experience with living with Von-Hippel-Lindau disease, as well as taking care of his son who also has it.
Luckily for both of them, there is a new drug coming to market that Kramer is hoping to get his son on as soon as possible that will help combat Von-Hippel-Lindau as well as Renal Cell Carcinoma.
Welireg, also known by its drug name of belzutifan, is a drug recently approved by the FDA that has shown to shrink brain tumors by a significant amount.
Dr. Othon Iliopoulos led a Zoom call with the VHL Alliance and showed graphs that showed Welireg working to shrink tumors in one of his patients brains.
Kramer has been on the clinical trial for Welireg for the past three and a half years, and he says The worst side effect he had was fatigue; that's coming from a man who says he normally can go "one-hundred twenty miles an hour" and isn't used to being tired.
The biggest side effect in question however, is the possibility of infertility due to Welireg. According to Iliopoulos, it's still too early to know exactly whether this is the case.
When looking at the tests done on animals, some test subjects were shown to gain infertility. Both the VHL alliance and experts are hoping that this drug can eventually be used to not only treat VHL and RCC, but also other forms of cancer.
To quote Kramer, “this is a game changer for a lot of people.”
He went on to tell me that as he was talking to the geneticist when his son found out he had VHL.
“I had my hands in my face crying and then the geneticist said, there may not be a cure in your time, but there possibly will be in your son’s time.”
Fast-forward to now, and the FDA approves a drug that fights VHL two months ahead of its scheduled time to be approved.
What seem to be the side effects that most people have experienced is anemia and nausea.
For Kramer, if he was tired enough, he would experience blurry vision and brain fog. Since he is someone who lives in Arizona, he is used to climbing mountains. Due to using Welireg, it made him feel more exhausted than he usually would be. “Once I got to the top of the mountain I felt like I was at ten-thousand feet so I was sucking air,” says Kramer.
Luckily, in the past three and a half years that he has been on the clinical trial for Welireg, he feels that his body has acclimated to the drug.
When it comes to insurance, since the drug is not yet out on the market, it's difficult to know if and/or how much insurance companies will cover the cost of Welireg.
However, as more time passes and this drug becomes widely available, we will be able to determine how insurance companies react to this new drug and how much they will cover.