Because von Hippel-Lindau disease (VHL) can cause several different problems, MD Anderson brings together a team of experts from several disciplines to treat the disease. They work together to personalize your care, ensuring the most advanced and most effective treatments possible.
Surgery, including minimally invasive and laser surgery in some cases, is a commonly used treatment for many VHL-associated problems. Like all surgeries, surgery for VHL disorders is most successful when performed by a specialist with a great deal of experience in the particular procedure.
MD Anderson’s surgeons are among the most skilled and renowned in the world. They perform a large number of surgeries for VHL disease each year, using the least invasive and most advanced techniques.
A new targeted therapy is also available patients with VHL-related hemangioblastomas and of the brain and spine, kidney cancer and pancreatic neuroendocrine tumors. This promising therapy may allow some patients to avoid surgery for specific tumors.
Our von Hippel-Lindau disease treatments
If you are diagnosed with VHL, your treatment will depend on locations of the tumors and cysts that develop and the problems they cause.
Since surgery is one of the primary treatments for the disease, doctors will often try to coordinate surgeries for different growths. This limits the number of procedures a patient undergoes, reducing recovery time and the trauma of surgery on the body.
Endolymphatic sac tumor treatment
Endolymphatic sac tumors are removed through minimally invasive surgery. If surgery is not possible, these tumors are treated with radiation therapy.
If hemangioblastomas are small and/or slow-growing, they may not need treatment. Tumors that are large, fast-growing or symptom-causing are surgically removed or may in some cases be treated with focused-beam or stereotactic radiation therapy.
In addition to surgery, a newly developed targeted therapy is available to VHL patients with hemangioblastoma of the brain and spine. This therapy may help patients avoid surgery. Doctors are studying how to best integrate this targeted therapy into hemangioblastoma treatment plans.
Retinal hemangioblastomas found on the outer regions of the retina can be treated with laser photocoagulation (treatment with laser beams that heat and destroy tumors), or cryotherapy (treatment with extremely cold probes that destroy the tumor). Vitrectomy, or surgical treatment, should be considered for patients with detachment and/or scarring of the retina.
Tumors in very delicate areas, like the optic disc, usually are not treated because of the damage treatment might cause.
Kidney cancer treatment
Kidney tumors usually are surgically removed when they grow larger than three centimeters in diameter or if they grow quickly. In addition, MD Anderson offers advanced treatments that can help some people, including:
- Partial nephrectomy, surgical removal of part of the kidney
- Radiofrequency ablation, which uses radiofrequency waves to destroy the tumor
- Cryoablation, which uses extreme cold to remove tissue
In addition to surgery, a newly developed targeted therapy is available to VHL patients with kidney cancer. This therapy may help patients avoid surgery. Doctors are studying how to best integrate this targeted therapy into kidney cancer treatment plans.
If kidney cancer spreads beyond the kidney, it becomes challenging to treat.
Immunotherapy and chemotherapy help some people. New therapies include anti-angiogenesis (blood vessel starving) medications, which stop blood vessels from growing, and targeted therapies that help your body fight the disease on a molecular level. Learn more about kidney cancer treatment.
Pancreatic tumors and cysts treatment
Cysts and cystadenomas in the pancreas generally do not require treatment. Rarely, if a cyst becomes very large, your doctor may shrink it by using a needle to draw fluid from the cyst.
Pancreatic neuroendocrine tumors require close follow up because they may spread, which can be very dangerous. Generally, if a known or suspicious pancreatic neuroendocrine tumor (NET) becomes larger than 3 centimeters, it is surgically removed.
In addition to surgery, a newly developed targeted therapy is available to VHL patients with pancreatic NETs. This therapy may help patients avoid surgery. Doctors are studying how to best integrate this targeted therapy into pancreatic NET treatment plans.
Surgery is the main treatment for pheochromocytomas. To prevent complications from severe high blood pressure caused by high catecholamine levels during surgery, you must take medicine for two to three weeks before the procedure.
Removal of a pheochromocytoma may require taking out the whole adrenal gland. This is called an adrenalectomy. Sometimes the surgeon can “spare” the cortex (outer layer) by removing only the tumor and the adrenal medulla. Sometimes, minimally invasive laparoscopic surgery, which uses a smaller incision and may help you recover quicker, is possible.
If both adrenal glands are removed, you will need to take medicine to give your body the hormones that usually are made by the adrenal gland.
von Hippel-Lindau disease clinical trials
As one of the nation’s leading research centers, MD Anderson offers clinical trials of new treatments for von Hippel-Lindau disease. Learn more about clinical trials.
Treatment at MD Anderson
von Hippel-Lindau disease is treated in a special VHL Clinic in our Genitourinary Center.
MD Anderson patients have access to clinical trials offering promising new treatments that cannot be found anywhere else.
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People with a rare, inherited disorder called von Hippel-Lindau disease develop cancerous and noncancerous tumors in up to 10 different parts of their bodies.
“Having von Hippel-Lindau disease can be overwhelming,” says Eric Jonasch, M.D., who heads MD Anderson’s von Hippel-Lindau Clinic. “Tumors recur frequently throughout your lifetime, and they could only be removed surgically – until now.”
Jonasch led a clinical trial that recently resulted in the Food and Drug Administration’s (FDA’s) approval of the first-ever drug to treat von Hippel-Lindau disease (VHL).
“The drug, belzutifan, is a complete game-changer for patients who have waited decades for an effective treatment,” he says. “Their quality of life is about to dramatically improve.”
Genetic mutation leads to tumor growth
Von Hippel-Lindau disease is caused by a mutation in the VHL gene. Normally, this gene keeps cell growth in check. But when the gene mutates, the gene malfunctions and cells multiply out of control. Masses of cells develop into cancerous and noncancerous tumors in the brain, spine, eyes, ears, lungs, kidneys, pancreas, liver, adrenal glands, and reproductive organs.
As fast as surgeons removed them, new ones appear.
“It’s an endless cat-and-mouse game,” Jonasch says. “We can never predict when or where tumors will show up. Our goal is to stay ahead of them.”
A constant cycle of surveillance, surgery and recovery
To do this, doctors monitor patients from head to toe to identify new tumors and track the growth of existing ones.
“We scan and examine them at least once every two years,” Jonasch says.
When tumors arise, they’re left untouched for a while – the opposite of how most cancers are treated.
“Knowing our patients will develop multiple tumors throughout their lives, we deliberately wait to operate until their tumors grow large enough to damage an organ or spread to other areas,” he explains. “The goal is to control patients’ cancer, and at the same time, spare their organs from the scarring caused by multiple, repeated surgeries. When we do operate, we take out all the possible tumors we can, and leave as much of the organ as possible.”
This constant cycle of surveillance, surgery and recovery places a tremendous psychological and physical burden on patients, Jonasch says.
“They walk around with the Sword of Damocles hanging over their heads. It’s not an ideal way to live,” he says, “but it’s all they had – until now.”
Clinical trial for von Hippel-Lindau disease
With the FDA’s recent approval of belzutifan, Jonasch expects to see the number of von Hippel-Lindau patients who will need surgery significantly drop.
The FDA approved the drug after a clinical trial involving 61 patients at MD Anderson and 10 other cancer centers produced encouraging results.
The clinical trial was designed to test the drug’s effectiveness in VHL patients who developed a type of kidney cancer called renal cell carcinoma. This is one of the most common cancers in people with von Hippel-Lindau disease. At some point in their lives, about 70% of VHL patients will develop renal cell carcinoma.
After taking belzutifan daily for 18 months, almost half of participants saw their kidney tumors shrink at least 30%.
The drug also shrank tumors in the brain, spine and pancreas.
“Because von Hippel-Lindau disease produces tumors throughout the body, our clinical trial participants had other types of cancer in addition to kidney cancer,” Jonasch says.
91% of pancreatic neuroendocrine tumors (a rare type of pancreatic cancer), and 30% of hemangioblastomas (slow-growing tumors in the brain or spine), shrunk by at least 30%. In a handful of participants, tumors disappeared altogether.
Most participants whose tumors didn’t shrink still got promising news – their tumors stopped growing and remained stable while on belzutifan.
“The drug kept their tumors at bay,” Jonasch says. “When the stable disease numbers are added to the shrinkage numbers, 98% of participants benefited from the medication.”
FDA officials were so impressed by the data that they approved the drug not only for renal cell carcinoma – the original intention of the study – but also for brain and spine hemangioblastomas and pancreatic neuroendocrine tumors.
“Belzutifan fills a huge and previously unmet medical need,” Jonasch says. “This is the first time we have a highly effective medication to treat three types of tumors associated with von Hippel-Lindau disease.”
“The drug was relatively easy to tolerate,” Jonasch says, “compared to the surgery patients would have needed without it.”
The science behind belzutifan’s success
Belzutifan works by blocking a protein called HIF-2a, which fuels the growth of cancers.
For years, the protein was considered undruggable. It was slippery, and experimental drugs that potentially could have turned off its cancer-promoting action wouldn’t stick to it. But when scientists discovered a tiny pocket on HIF-2a’s surface, they inserted various drug candidates into it and began testing their effectiveness. Eventually, they found a drug that worked. That drug would later become known as belzutifan.
“Patients and their families finally have a reason to hope,” says Jonasch of the discovery of belzutifan. “Most will see their tumors shrink with this new drug, and the cycle of endless surgeries will be broken.”
He points to the 61 clinical trial participants.
“Altogether, the group had an average of 20 surgeries every year before joining the trial. After starting belzutifan, only two needed an operation. The drug nearly eliminates the need for surgery.”
Future research may benefit additional kidney cancer patients
The next step, Jonasch says, is to launch future clinical trials to determine whether belzutifan can help prevent tumors from forming in the first place.
He also wants to know if the drug will be useful in treating other cancers, and he’s already looking at its effect on kidney cancer patients who don’t have VHL disease.
“More than 70,000 people are diagnosed with kidney cancer each year, and most of them don’t have von Hippel-Lindau disease,” Jonasch says. “Their cancers have a very similar genetic makeup to VHL-related kidney cancers. Wouldn’t it be great if the drug works for them, too?”
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