Does anyone in your family, or someone you know, have a congenital vision impairment? Sometimes these are caused by genetic diseases that are passed down from generation to generation. Day by day, vision can decrease, and eventually become completely invisible. So, today we are going to talk about the latest, most advanced treatment in medical science that treats such a rare, hereditary eye disease. Its name is Voretigene Neparvovec.
Simply put, what is Voretigene Neparvovec?
This is not a medicine like the pills, capsules, or ointments we usually use. This is called Gene Therapy . The brand name of this is LUXTURNA .
This treatment is used to treat a specific hereditary vision impairment that affects the retina of our eyes. Specifically, it treats a vision problem caused by a defect in the RPE65 gene in our body.
So, what is this gene therapy?
Imagine that there is a big book with instructions on how everything in our body should work. This book is our genes . Sometimes, on one page of this book, that is, in one gene, there can be a mistake, an error. The error in the gene called RPE65 is one such thing.
This treatment, called Voretigene Neparvovec, uses a harmless virus as a carrier to insert a correct copy of the gene into cells that have the faulty gene. It's like replacing a page with the wrong instructions with a new page with the correct instructions. After this, the eye cells are able to function properly again, and through this, the lost vision can be restored to some extent or even stopped further vision loss.
This is truly a major leap forward in medical science. It is the only great hope that there is now a successful treatment for a previously incurable condition.
Things you need to tell your doctor before undergoing this treatment
Because this is a very specific treatment, it is essential that your doctor is aware of your complete health history. Be sure to tell him or her about the following:
- Other eye diseases: Any other eye disease or vision problem you already have (e.g. cataracts, glaucoma).
- Allergies: Please tell us if you have any allergies to this medicine (voretigene neparvovec), any other medicine, food, dyes, or preservatives.
- Pregnancy and breastfeeding: Be sure to tell your doctor if you are pregnant, trying to become pregnant, or breastfeeding.
How do you use this medicine?
This is not a home remedy. This is an injection into the eye . This is done by a specially trained eye surgeon in a very controlled, clean environment, like a hospital operating room. So there is nothing to worry about, your safety is ensured to the fullest extent possible.
Can this be given to small children?
Yes. This treatment can be given to children as young as one year old, under appropriate circumstances. However, the final decision about it is made by the pediatrician and eye surgeon who examines your child. If you are giving this treatment to a child, it is very important to talk to the medical team about it thoroughly and explain everything.
What happens if you take too much medicine?
Since this is given in a hospital, under the full supervision of doctors, the chances of an 'overdose' occurring are very low. However, if there is any suspicion that such a thing has happened, the hospital staff will take immediate action. Normally, in such a case , the National Poisons Information Centre at the Colombo National Hospital or the nearest Emergency Treatment Unit (ETU) is informed.
Things to consider during and after treatment
During and after treatment, doctors will monitor you closely. You also have a great responsibility.
If you notice any changes in your vision - such as decreased vision, blurred vision, black spots floating in front of your eyes, increased sensitivity to light, or eye pain or redness - tell your doctor right away . This is very important.
What are the possible side effects?
As with any medical treatment, there are some possible side effects. Be especially aware of the following.
| Side effects that should be reported to the doctor immediately | |
|---|---|
| Side effect | Description |
| Allergic reactions | Skin rash, itching, swelling of the body, especially of the face, lips or tongue. |
| Bleeding from the eye | Bleeding inside or outside the eye. |
| Vision changes | Blurred vision, seeing black dots or nets floating before the eyes, seeing flashes of light. |
| Eye pain | Persistent or severe eye pain. |
| Increased eye pressure | Feeling of pressure inside the eye. This will be checked by the doctor. |
| Cataract | Blurred vision after treatment. |
This list may not include all side effects. If you experience any unusual symptoms, talk to your doctor about them.
Take-Home Message
- Voretigene neparvovec (LUXTURNA) is a specific gene therapy for a hereditary eye disease caused by a defect in the RPE65 gene.
- This treatment is performed in a hospital by a specialist eye surgeon, who injects an injection into the eye .
- Before treatment, give the doctor a complete description of all your medical conditions, allergies, and whether or not you are pregnant.
- After treatment, if you experience any changes in vision, eye pain, or redness, notify your doctor immediately .
- This is not a treatment for everyone. Whether this treatment is right for you or not will be determined after a thorough medical examination.
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