Today we are going to talk about a somewhat complex, yet very modern medical treatment that many people have not heard of. Its name is Elivaldogene Autotemcel. It is marketed under the name SKYSONA. This is not a regular pill or capsule. This is a specific treatment for a very rare and serious neurological condition called cerebral adrenoleukodystrophy (CALD). Let us explain this treatment in a simple way that you can understand.
What really happens with this medicine?
To understand this, let's take a small example. Imagine that the nerve cells in our brain and spinal cord are like electrical wires. These electrical wires have a plastic sheath (insulation) around them, which helps the electricity to travel smoothly without leaking. Similarly, our nerve cells also have a protective sheath called 'myelin' around them. This sheath is what allows nerve messages to travel quickly and accurately.
In a disease called cerebral adrenoleukodystrophy (CALD), a genetic defect prevents the body from producing a protein needed to maintain this 'myelin' sheath. This protective sheath is then gradually destroyed, impairing the functioning of the nervous system.
Simply put, this treatment, called Elivaldogene Autotemcel, involves taking cells from the patient's own body, genetically modifying them in a lab, and giving them the ability to produce the missing protein. The modified cells are then reintroduced into the patient's body. This is also called gene therapy . This helps control the worsening of symptoms.
Things your doctor should know before starting this treatment
Because this is a very complex treatment, it is essential that your medical team is fully aware of your health condition. Before starting treatment, you should definitely tell your doctor if you have any of these conditions:
- If you currently have any type of infection (such as fever, cold, cough).
- If you have allergies to this medicine (elivaldogene autotemcel), other medicines, foods, dyes, or preservatives.
- If you are pregnant or trying to get pregnant .
- If you are breastfeeding your baby .
How is the treatment given?
This is not a home remedy. This treatment is given in a hospital or specialized clinic by a trained medical team.
This medicine is injected into the body like saline through a vein. The entire process is carried out under the close supervision of doctors and nurses.
Before your treatment, you will be given a special information leaflet called a MedGuide . It contains a lot of important information about this medicine, the procedure and the risks. It is very important to read and understand it carefully.
This treatment is also given to children over 4 years of age, but special precautions and criteria are considered.
What if you take too much medicine?
Since this is administered under medical supervision in a hospital, the chances of this happening are very low. However, if there is any suspicion that this has happened, the National Poisons Information Centre at the Colombo National Hospital or the hospital's Emergency Treatment Unit (ETU) should be informed immediately.
Things to consider during and after treatment
After receiving this treatment, doctors will constantly monitor your health. For this, you will need to take special care of certain things.
- Bloodwork: After treatment, you will need to have your blood tested on a regular schedule. This will check whether the treatment is working, whether the new cells are working properly, and whether there are any side effects.
- Risk of infection: This treatment may temporarily weaken your immune system, which increases your risk of infection. If you develop symptoms such as fever, chills, cough, or sore throat , do not self-medicate. Call your healthcare team immediately . Also, try to stay away from people who are sick as much as possible.
- Donating blood: You cannot donate blood after receiving this treatment.
- Cancer risk: With modern gene therapies like this, there is a very small risk of developing some types of blood cancer. Don't be alarmed by this, but be open about this risk and how it will be monitored with your doctor.
- Special advice for men: If you are a man undergoing this treatment, you must use a condom when having sex with a woman who is of childbearing potential. This method of protection should be followed for 6 months after treatment. If you suspect that your partner is pregnant during this time, inform your doctor immediately.
What are the possible side effects?
Like any medication, this treatment can cause side effects. Some of them require immediate medical attention, while others may be normal.
| Side effect | Description / Symptoms |
|---|---|
| Serious side effects that should be reported to the doctor immediately | |
| Allergic reactions | Skin rash, itching, swelling of the face, lips, tongue or throat. |
| Bleeding | Blood in the stool or black stools, vomit that looks like coffee grounds, urine that is red or dark brown, small red/purple spots on the skin, and light bruising. |
| Infection | Fever, chills, cough, sore throat. |
| Symptoms of epilepsy (Seizures) | Having a fit ( seizure )-like condition. |
| Side effects that are usually not dangerous, but should be reported to the doctor if they persist | |
| Digestive system problems | Constipation, stomach pain, diarrhea, vomiting, nausea. |
| Other minor ailments | Loss of appetite, headache, mouth or throat sores and pain. |
This list does not include all possible side effects. In addition, if you experience any unusual symptoms, talk to your doctor about them.
Take-Home Message
- Elivaldogene Autotemcel (SKYSONA) is a highly specific gene therapy for the rare disease Cerebral Adrenoleukodystrophy (CALD).
- This treatment is provided in a hospital under the supervision of a team of specialist doctors.
- Before treatment, be sure to tell your doctor all your health information (especially infections, allergies).
- Be very careful about signs of infection and bleeding after treatment. If you have any concerns, inform your doctor immediately.
- Men receiving treatment are required to use a condom during sexual intercourse for the entire duration of the treatment.
- Don't be afraid to talk to your doctor about any questions or concerns you may have.
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