Huntington’s Disease Successfully Treated for the First Time

Huntington’s Disease Successfully Treated for the First Time

Introduction

For many years, Huntington’s disease has been one of the most feared brain disorders. It slowly takes away a person’s ability to move, think, and live independently. Families who carry the gene for Huntington’s disease often describe it as a life sentence, because there has never been a cure or effective treatment.

But now, for the very first time, scientists and doctors are reporting a breakthrough. A new treatment has shown real success in stopping, slowing, or even reversing some of the damage caused by Huntington’s disease. This news is giving hope not only to patients and families but also to the wider medical world.

In this article, we will explain what Huntington’s disease is, why it has been so difficult to treat, what the new treatment involves, and what this breakthrough means for the future. We will use simple, easy-to-understand language so that everyone can follow along.

What Is Huntington’s Disease?

Huntington’s disease (often called HD) is a rare, inherited condition. This means that a person gets it because of a problem in their DNA, passed down from their parents. If one parent carries the faulty gene, there is a 50% chance that their child will also have the disease.

The disease affects the brain, especially areas that control movement, thinking, and emotions. Over time, the brain cells in these areas die, causing a wide range of problems.

Main Symptoms of Huntington’s Disease:

1. Movement problems – People may start to have uncontrolled movements, twitches, and jerks. Later, they may find it hard to walk, talk, or even swallow food.

2. Thinking problems – Memory, decision-making, and planning become more difficult.

3. Emotional problems – Depression, anxiety, mood swings, or even aggressive behavior can happen.

When does it start?

Most people start showing symptoms between the ages of 30 and 50. Once it begins, the disease usually gets worse over 10–25 years.

Why is it so feared?

Because it is caused by a gene mutation, people who know Huntington’s runs in their family often live with constant fear. They may not want to take a genetic test, because finding out they have the gene can be emotionally painful. And until now, even if someone tested positive, there was nothing doctors could do to stop it.

Why Has Huntington’s Been So Hard to Treat?

There are a few reasons:

1. It is genetic – The disease comes from a problem in the DNA itself. Unlike infections (which can be killed with antibiotics) or cancers (which can sometimes be cut out), fixing DNA errors has always been a big challenge.

2. The brain is complex – The brain has billions of neurons. Treating just the right cells, without harming others, is very difficult.

3. It slowly destroys brain cells – Once brain cells are dead, they usually cannot grow back. So, even if treatment is found, doctors need to stop the disease very early or find ways to repair damaged brain tissue.

Because of these reasons, for decades scientists could only focus on medicines that treated symptoms like depression or movement problems. These did not stop the disease itself. Patients and families were desperate for something more.

The First Successful Treatment

Now, for the first time, researchers are reporting a treatment that directly targets the cause of Huntington’s disease, not just the symptoms.

What is the new treatment?

The treatment is based on gene silencing technology. In simple words, scientists found a way to “switch off” the faulty Huntington gene so that it stops making the harmful protein that kills brain cells.

• Normally, the faulty gene makes a protein called huntingtin.

• This protein builds up in the brain and becomes toxic.

• The new treatment tells the body: “Stop making so much of this bad protein.”

How was it tested?

• Patients were given the treatment through an injection into the fluid around the spinal cord.

• This allowed the drug to travel to the brain.

• Scientists then measured the amount of toxic protein in the spinal fluid.

• The results showed that protein levels dropped dramatically.

This is the first time

The disease runs through families, relentlessly kills brain cells and resembles a combination of dementia, Parkinson's and motor neurone disease.

An emotional research team became tearful as they described how data shows the disease was slowed by 75% in patients.

It means the decline you would normally expect in one year would take four years after treatment, giving patients decades of "good quality life", Prof Sarah Tabrizi told BBC News.

The new treatment is a type of gene therapy given during 12 to 18 hours of delicate brain surgery.