Frequently Asked Questions about the new treatment for hypothalamic obesity

Although tumors affecting the brain’s hormone center are often benign and can be removed safely, many patients continue to suffer from severe long-term consequences caused by damage to the hypothalamus. For some, these complications can even be life-threatening.

Damage to the hypothalamus can disrupt an important signaling pathway in the brain. The resulting loss of control over satiety, metabolism and impulse regulation can lead to uncontrolled eating and obesity. Until now, there has been no effective treatment for this condition.

The Princess Máxima Center led the Dutch arm of a clinical trial led by Seattle Children's Research Institute looking at the benefit of setmelanotide, a drug that restores signaling related to satiety and energy expenditure in the brain.

They found that setmelanotide led to an average decrease in BMI – a way to measure healthy weight – of 20%. The researchers expect BMI to decrease further in people who continue treatment for longer than one year.

Prof. Hanneke van Santen, pediatric oncologist at the Princess Máxima Center and Wilhelmina Children's Hospital, led the Dutch arm of the clinical trial. She says: ‘Our clinical trial looking at the benefit of setmelanotide gives children with hypothalamic obesity and their families new hope. The drug’s effect was remarkable. Children who previously struggled not only with obesity but also with lethargy, emotional outbursts, and many other obesity-related problems truly seemed to come back to life. And alongside that, there was the tangible and impressive weight loss.

‘The results of our clinical trial are so compelling that I hope setmelanotide will soon be reimbursed for this patient group. That is essential if we want to offer setmelanotide to all children living with obesity caused by hypothalamic damage.’

We understand that you may have questions about this research. Below we answer some frequently asked questions. If your question is not listed, please contact communicatie@prinsesmaximacentrum.nl, stating 'New treatment for hypothalamic obesity' in the subject line. You can read our full news article here.

When will this medication become available?

This is not yet clear. Setmelanotide has now been approved by both European and U.S. regulatory authorities. The next step is to get it reimbursed on health insurance. In the Netherlands, this process is overseen by the Dutch Healthcare Authority. The drug can only become widely available once reimbursement approval has been granted.

Can I / can my child receive this medication now?

At present, setmelanotide is not yet routinely available in the Netherlands for children with hypothalamic obesity. Treating physicians therefore cannot prescribe it as standard care. Once there is more clarity about availability and reimbursement, this information will be shared with children and adults who may be eligible for treatment. We recommend asking your doctor to keep you informed.

Who is this medication intended for?

The medication is intended for children and adults with hypothalamic obesity: obesity that develops as a result of damage to the hypothalamus, for example following a brain tumor or treatment for a brain tumor. It is not intended for people with other forms obesity.

Will the medication be reimbursed?

No decision has been made yet. Researchers and clinicians hope that setmelanotide will be reimbursed in the near future so that all eligible children and adults can access the treatment.

My child has hypothalamic obesity and is being treated at the Princess Máxima Center. Do we need to sign up?

No, this is not necessary. If there are new developments that are relevant for your child, your treatment team will discuss them with you. If you have questions, you can always contact your child’s lead physician.

I am a survivor, or the parent of a survivor, with hypothalamic obesity, but I am no longer receiving care at the Princess Máxima Center. What can I do?

At this time, it is not yet possible to register for treatment with setmelanotide. Once more information becomes available about access and reimbursement, it will be shared with people who may be eligible for the treatment. In the meantime, please discuss any questions about your personal situation with your current specialist or general practitioner.

If the treatment is approved, it will only be provided through specialized expert centers for people with hypothalamic obesity. This will ensure that the treatment can be carefully monitored. In the Netherlands, expert teams for children with hypothalamic obesity are located in Utrecht and Rotterdam. Expert teams for adults with hypothalamic obesity are located in Leiden, Amsterdam, Utrecht, and Rotterdam. If the treatment is approved, you can ask your doctor for a referral to one of these centers.

Does the medication work for everyone with hypothalamic obesity?

The study results are highly promising, but as with any medication, the exact effect may vary from person to person. In the clinical trial, treatment with setmelanotide led on average to a substantial reduction in BMI and a significant decrease in hunger.

Are there any side effects?

Yes. Reported side effects included skin darkening or discoloration, headaches, and gastrointestinal symptoms such as stomach or bowel complaints. Physicians always carefully weigh the benefits of treatment against any potential risks and side effects.

Can the medication cure hypothalamic obesity?

No. The medication does not repair the underlying damage to the hypothalamus. However, the study shows that it can reduce feelings of hunger and support weight loss, resulting in improvements in overall health and quality of life.

Will I need to take the medication for the rest of my life?

That is the current expectation, although the research is still very new and long-term data remains limited. In principle, treatment would likely continue lifelong, similar to hormone replacement therapies such as thyroid hormone or hydrocortisone, or insulin therapy for people with type 1 diabetes.

Setmelanotide restores the brain signal that tells the body it is full, but it does not repair the damaged hypothalamus itself. For that reason, ongoing treatment may be needed to maintain its benefits.

Where can I go with questions?

Has your child been treated at the Princess Máxima Center and do you have questions about hypothalamic obesity? Or were you treated elsewhere? Please contact your medical team. They are best placed to advise you about your individual situation.