Does anyone in your family, perhaps your child, have a condition called Neurofibromatosis Type 1 (NF1)? You may be worried about the painful, difficult-to-remove, plexiform neurofibromas (PN) that come with this condition. But today we're talking about a new, promising drug that's now approved for use in adults and children to treat the condition. It's called Gomekli (mirdametinib).
What is Gomekli? How does it work?
Think about it, our body has different signaling pathways for cells to grow. In NF1, one of these signaling pathways becomes overactive, leading to the formation of tumors around the nerves. These tumors sometimes grow around important areas like nerves, blood vessels, and internal organs, making them very difficult to remove surgically, and sometimes impossible.
This is what Gomekli does. It blocks two enzymes in our body called MEK1 and MEK2. These enzymes are the ones that send the signals that the tumors need to grow. So when Gomekli stops that signal pathway, the tumors grow less, and sometimes the tumors shrink and start to shrink . Simply put, this is a good alternative treatment for people who can't have surgery.
How do you use this medicine?
Your doctor will determine the right dose of Gomekli for you or your child, based on your height and weight .
This treatment is usually done in a 28-day cycle.
- You should take this medicine twice a day, every 12 hours .
- You need to take the medicine in this way for 21 days in a row .
- Then you have to take the medicine for 7 days .
- When the 28 days are complete, a new cycle begins again.
Gomekli comes in capsules. However, for those who have difficulty swallowing capsules, there is also a grape-flavored tablet that can be dissolved in water and taken. Here's how to make it:
1. Put the number of pills prescribed by the doctor into a medicine measuring cup.
2. Add 5 or 10 milliliters of water to it.
3. Swirl the cup for about two to four minutes until the tablets are completely dissolved. Once dissolved, the liquid will turn white and look a bit milky.
4. Drink it right away from the cup. Or you can suck it up with an oral syringe .
5. After you finish drinking, add another 5-10 ml of water to the same cup, stir, and drink that as well. This will help the remaining medicine enter the body.
6. If you don't drink it within 30 minutes of making it, throw away the remaining liquid.
What do studies say about the success of the Gomekli drug?
The efficacy and safety of this drug has been confirmed in a clinical study called the ReNeu study. It involved 111 children and adults aged 2 years and older with inoperable PN tumors.
The results are really encouraging.
| Result | Adults | Children |
|---|---|---|
| Shrinkage of the tumor size by at least 20%. | 41% | 52% |
| Results lasted 12 months or longer for more than 88% of respondents. | 88% | 90% |
The most important thing is that these results have started to appear in as little as 4 months after starting treatment. It usually takes about 7-8 months to see the first results.
But remember, the results of these studies are not the same for everyone . The results may vary depending on your situation.
How do you know if the medicine is working?
The main way to know this is through MRI scans that your doctor will order. These can check to see if the tumors are shrinking in size.
Plus, you'll start to notice the difference yourself.
- Pain reduction: The pain caused by the tumor is significantly reduced.
- Being able to move around better: If you had difficulty moving due to tumors, these will be reduced and it will be easier to perform daily tasks.
- Improved quality of life: Overall, you start to feel better than before.
In the study, good results, such as pain reduction, were seen even after about 3 months of treatment.
What are the most common side effects?
Like any medicine, Gomekli can cause some side effects. However, most of these can be managed.
| Common side effects | Simple things to manage |
|---|---|
| Skin rash, nausea/vomiting, diarrhea, muscle and joint pain, fatigue. |
|
| In children, stomach pain, redness/swelling of the skin around the nails, and heart problems may also occur. | Report any of these symptoms to your doctor immediately , especially if you have heart problems. |
Most importantly: If you experience any side effects, even if they are minor, be sure to tell your doctor . He or she may lower the dose, stop taking the medication for a while, or give you a different medication to control the side effects.
Other special things to know about this medicine
There are several things to consider before starting Gomekli and during treatment.
1. Eyesight: This medicine can sometimes affect the eyes. Therefore, you should have a complete eye examination before starting treatment. You should also have your eyes checked as directed by your doctor during treatment. If you experience any symptoms such as blurred vision, tell your doctor immediately.
2. Heart function: An echocardiogram is performed before starting the medication, which measures heart function. This is repeated every 3 months for the first year of treatment.
3. Pregnancy: This medicine may be harmful to the fetus .
- If you are a woman of childbearing potential, you will be given a pregnancy test before starting treatment. You must use a highly effective form of birth control during treatment and for 6 weeks after stopping the medication.
- If you are a man, you should use effective birth control during treatment and for 3 months after stopping the medication. Talk to your doctor about this openly.
Take-Home Message
- Gomekli (mirdametinib) is a new treatment approved for use in adults and children with inoperable tumors (PN) associated with the condition Neurofibromatosis Type 1 (NF1).
- This blocks the signals needed for tumor growth, helping the tumor shrink and reduce pain.
- It is very important to have regular eye, heart, and MRI scans under the supervision of your doctor during treatment.
- Side effects may occur, but most of them are manageable. Tell your doctor immediately about any discomfort.
- You should never start, stop, or change the dosage of medication without medical advice.
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