As a parent, I understand how heavy your heart feels when a doctor tells you that your little one has spinal muscular atrophy, or what we all know as 'Spinal Muscular Atrophy' (SMA). But with the advancement of medical science, new, very promising treatments have now emerged to deal with such conditions. Today we are talking about one such very special, modern drug. That is the drug called Onasemnogene Abeparvovec.
Simply put, what is this Onasemnogene Abeparvovec?
This is a bit of a complicated name, but let's keep it simple. You may have heard of this drug by the brand name `Zolgensma`. This is not a regular pill or capsule. This is gene therapy .
Imagine, every function in our body is controlled by our genes. Just like a computer program. In SMA, there is a defect in a gene that makes a protein that is essential for muscle function. So, what this Onasemnogene Abeparvovec treatment does is to insert a copy of a healthy gene into the child's body instead of that defective gene. This healthy gene is carried to the body's nerve cells with the help of a harmlessly modified virus (adeno-associated virus).
The important thing is that this is not a treatment that will completely cure SMA. However, it can help stop the disease from progressing and help the child's muscles to function and grow stronger.
How is this treatment given?
This medicine is given in a hospital, under the full supervision of specialist doctors and nursing staff. This is an infusion treatment. Like a saline solution, this medicine is slowly given into the body over a period of about an hour. This treatment is usually given only once in a lifetime.
This treatment can be given to newborns and young children. The specialist medical team who examines the child will decide whether this treatment is suitable for your child or not. So don't worry about it.
Things you need to tell your doctor before treatment
Because this is a very advanced treatment, it is extremely important to inform the medical team of all the details about your child's health condition before starting treatment. Be sure to tell them about these things in particular.
- If the child has a heart condition.
- If you have previously reported any liver disease or changes in liver enzymes.
- If you have a blood condition such as a decrease in the number of platelets in the blood.
- If you have previously had an allergic reaction to this medicine or any other medicine or food.
- If the mother is pregnant, trying to get pregnant, or breastfeeding.
Providing this information accurately is very important for the child's safety.
What are the possible side effects of the treatment?
Like any medicine, this treatment can cause some side effects. Some are normal, but there are some things we need to be very careful about. Let's break them down clearly.
| Side effect | Description and what to do |
|---|---|
| Serious side effects that should be reported to the doctor immediately | |
| Symptoms of Liver Injury | This is the most important and most common serious side effect. Watch out for symptoms such as yellowing of the eyes or skin (similar to jaundice), dark yellow/brown urine, dark stools, pain in the upper right side of the stomach, nausea, loss of appetite, and extreme tiredness. If you notice any of these , tell your doctor immediately . |
| Allergic Reactions | If you experience symptoms such as skin rash, itching, swelling of the face, lips, or tongue, or difficulty breathing, it is an emergency. Tell your doctor immediately. |
| Unusual bleeding or bruising | Due to a decrease in platelets, bleeding may not stop even from a small wound, and bruising may appear under the skin. Inform your doctor about this as well. |
| Signs of infection | If you develop a fever for no reason, chills, a sore throat, or a cough, tell your doctor about it. |
| Side effects that are usually not serious, but should be reported to the doctor | |
| Vomiting | Your child may vomit after treatment. This usually subsides within a few days. However, if it persists or if your child is having difficulty, tell your doctor. |
What if I get an overdose?
Since this medicine is administered under medical supervision in the hospital, there is little chance of an overdose. However, if you suspect that this has happened, you should immediately inform the hospital's Emergency Treatment Unit (ETU) . You can also call the National Poisons Information Centre at the Colombo National Hospital for advice if necessary.
Things to consider during and after treatment
Blood tests
Before and after treatment, your child will need to have regular blood tests to monitor their liver function and platelet levels for a period of time. This is for your child's safety, so it is very important to have these tests done exactly on the scheduled dates as instructed by your doctor.
Handling of body fluids and waste
This is a very important point that many people do not pay attention to. After treatment, parts of this medicine (modified virus) can be excreted in the child's stool and urine for a month or more. Therefore, family members should be especially careful when changing and cleaning the child's diapers.
- Wear gloves every time you change a diaper.
- Place the used diaper in a well-sealed plastic bag and dispose of it in a separate trash can.
- After changing the diaper, wash your hands thoroughly with soap.
- If your child's clothes or bedding get dirty with feces or urine, wash them separately in hot water.
Don't worry about this. Before you leave the hospital and go home, the medical team will give you clear instructions on how to do these things.
Take-Home Message
- Onasemnogene Abeparvovec (Zolgensma) is a specific gene therapy for Spinal Muscular Atrophy (SMA), given intravenously once a lifetime.
- Although this does not completely cure the disease, it is a great help in controlling the progression of the disease and improving the child's quality of life.
- It is imperative to tell the doctor about the child's complete health history (especially liver, heart, and blood-related problems) before treatment.
- Be very careful about signs of liver damage (yellowing of the skin/eyes, dark urine) after treatment. If you notice any such signs, inform your doctor immediately.
- It is very important to follow medical advice when handling the baby's diapers and waste for several months after treatment.
- If you have any concerns about this treatment, never guess, but ask the doctor or medical team treating your child.
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