Sometimes, small changes in our genes can cause major health problems. hATTR amyloidosis is one such rare, but very serious genetic condition. In this, a protein produced by our liver accumulates incorrectly and damages the nerves. This can cause many problems such as difficulty walking, pain, and numbness. But now there is a new treatment for this. Today, let's talk about this new drug called Wainua.
How does the medicine Wainua work?
To understand this, let's first take a simple look at hATTR disease. Our body has a protein called transthyretin (TTR). This is made by our liver. Due to a small change in the TTR gene in a person with hATTR disease, this protein folds incorrectly, clumps together, and deposits in the body as clumps called 'amyloid'. It's like curdling milk. These amyloid clumps are mostly deposited in the nervous system. Then the nerves are damaged and their function is impaired.
Now let's look at what the Wainua vaccine does. The drug Wainua directly targets the gene that makes the faulty TTR protein. Simply put, it 'silences' the gene that gives the wrong instructions.
This reduces the amount of abnormal TTR protein produced by the liver. This in turn reduces the deposition of amyloid in the nerves, helping to control the progression of nerve damage.
This is a monthly injection that is injected under the skin. What's special is that it comes in an 'auto-injector' designed for the patient or their caregiver to use at home.
How do I know if this treatment is right for me?
Your doctor will decide if Wainua is right for you. This treatment is usually suitable for the following people:
- h If you are an adult who has been confirmed to have ATTR Amyloidosis .
- If the disease has caused nerve damage .
Your doctor will consider several factors before making this decision.
- Family history : Check to see if anyone in your family has had this disease.
- Symptom Check: Your symptoms will be thoroughly examined.
- Genetic testing: A blood test is done to confirm the TTR gene mutation.
- Imaging tests: Tests such as MRI scans can also look for amyloid deposits in nerves and other organs.
How has the success of the Wainua medicine been tested?
Before any new drug is released into the market, extensive research is conducted to determine its quality, safety, and effectiveness. The Wainua drug has also undergone a clinical trial called NEURO-TTTransform.
This research mainly looked at:
1. How does the Wainua drug change the TTR protein level in the blood?
2. How will this affect the symptoms related to nerve damage in hATTR patients?
They used special methods to measure these.
- Neuropathy Impairment Score (NIS): This is a special scoring system. It measures muscle strength, reflexes, and sensation. The lower the score, the better the condition of the nerves.
- Quality of Life (QOL-DN): This measures the patient's fatigue, ability to perform daily activities, and mental state. Here too, lower scores indicate better quality of life.
The success of Wainua was confirmed by comparing the results of this study with the results of patients in another study who were given a placebo containing no medication.
| How the research participants were selected | |
|---|---|
| Inclusion Criteria | Exclusion Criteria |
| - Ages 18-82 - People with stage 1 (walking without assistance) or stage 2 (walking with assistance) of nerve damage due to hATTR - Those with a confirmed mutation in the TTR gene | - People with other types of Amyloidosis - People with nerve damage due to other causes such as diabetes - Those who have had or are about to have a liver transplant - People receiving certain other treatments for hATTR |
What were the main results of the research?
The results of the study were very encouraging. Those who received Wainua treatment for 66 weeks had significantly better results than those who received a placebo.
- The levels of the harmful TTR protein in their blood had decreased significantly.
- Neurological symptoms (NIS scores) were significantly controlled.
- Also, their quality of life had clearly improved.
What are the side effects of Wainua and how to manage them?
Like any medication, Wainua can cause some minor side effects. The most common are:
| Side effect | How to manage |
|---|---|
| Decreased vitamin A levels | Take the vitamin A supplement exactly as your doctor prescribes. Never take more than your doctor tells you to. If you notice any changes in your vision, such as decreased night vision or dry eyes, tell your doctor right away. |
| Vomiting | Drink plenty of water and fluids. Avoid oily and heavy foods. Eat slowly. If the vomiting does not stop after a few days, definitely talk to your doctor. |
These are the main side effects. In addition, talk to your doctor about any symptoms that are bothering you.
How do you know if the treatment is successful?
Your doctor will monitor your health regularly. This will include blood tests to check your TTR levels. They will also check to see how well your neurological symptoms are improving. Over time, you may start to notice improvements in your muscle strength, ability to perform daily tasks, and mood.
The most important thing is, do not stop the vaccination for any reason or change the vaccination schedule without the doctor's advice.
Are there any problems with other medications or foods?
According to current research, no adverse interactions have been found between Wainua and other medications or foods. However, new information may come to light in the future.
Therefore, before you start this treatment and during it, give your doctor a complete description of all other medications, vitamins, supplements, and Ayurvedic products you are using. This is very important for your safety.
Take-Home Message
- Wainua is a novel, monthly vaccine treatment approved for the rare genetic condition hATTR Amyloidosis.
- This reduces the body's production of an abnormal protein that damages nerves.
- This helps control the nerve damage caused by the disease and improve the quality of life of patients.
- Since a decrease in vitamin A levels is a major side effect, it is essential to take the vitamin A supplement prescribed by your doctor as prescribed.
- Always talk to your doctor before starting or stopping any treatment, and if any side effects occur.
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