Patient & Caregiver Q&A Article

Melissa’s Question:

Melissa is a patient new to our community.

What is IDH1? To expand on this What are IDH1 and IDH2? and what happens when IDH1 gets broken?

This Answer refers to:

Broken IDH1 in cholangiocarcinoma

This has been written by Professor Guzzba

Professor Guzzba is a fictional Molecular Biologist and Scientist Character, who’s job is to simplify the complex into a manner that you can better understand and effectively apply.

“Ah, welcome aboard the Bio Sub once again, my brave explorers! Today, we’re diving deep into the wonders—and sometimes dangers—of Cell City. As we sail through the vast Cytoplasm Ocean, we see all the important components floating around, working together to keep the cell alive and thriving.

First, let’s take a trip to the Nucleus, where the instructions for everything are stored. Inside, we find the IDH1 gene, which holds the instructions for making the IDH1 enzyme. The IDH1 gene sends out a message called mRNA, carrying these instructions. This messenger swims out of the nucleus and into the Cytoplasm Ocean, where it reaches a special floating factory called the ribosome.

The ribosome reads the message and builds the IDH1 enzyme. And now, the real work begins! The IDH1 enzyme floats through the Cytoplasm Ocean, looking for a special resource called isocitrate, which comes from sugar. Once it finds isocitrate, it transforms it into something called alpha-ketoglutarate (a-KG).

This alpha-ketoglutarate is then sent to the Mitochondria Islands—the power plants of Cell City. Here, another worker, IDH2, is waiting inside the mitochondria. IDH2 takes the alpha-ketoglutarate and turns it into ATP, the magical energy units that power everything in Cell City. The mitochondria send this ATP all over the cell, like electricity powering a city, making sure every part of the cell has the energy it needs to grow, repair, and function properly.

But explorers, not everything is smooth sailing in Cell City. Sometimes, the IDH1 gene can get damaged. And when that happens, instead of creating the helpful alpha-ketoglutarate, it starts producing a dangerous toxic waste called 2-hydroxyglutarate (2-HG). This waste pollutes the Cytoplasm Ocean, choking the cell’s environment and stopping everything from working the way it should.

With the ocean polluted, Cell City can’t repair itself properly. This pollution can even encourage cancer to grow, turning a healthy city into a place where disease takes over. So, while IDH1 and IDH2 usually keep things running smoothly, when IDH1 is damaged, it’s like spilling toxic waste into the ocean, disrupting everything and allowing cancer to sneak in.

And that, my friends, is why it’s so important that our little workers, IDH1 and IDH2, stay healthy to keep Cell City thriving and full of energy!”

But here’s the good news—science is working hard to solve the problem of broken IDH1 enzymes! When the IDH1 gene gets damaged and starts making that harmful toxic waste, scientists have come up with a smart plan to stop it. They’ve developed a special drug that sends in a blocking agent to stop the broken IDH1 enzyme from producing the toxic 2-HG.

Think of it like a protective shield that stops the damaged enzyme from making the harmful waste that pollutes the Cytoplasm Ocean. While the IDH1 enzyme remains broken, the blocking agent helps keep the toxic waste under control, preventing it from damaging Cell City further.

By stopping the toxic waste production, the cancer cells can’t keep growing and spreading, helping to keep things stable. Scientists are helping to slow down the disease and protect other healthy Cell Cities from harm.

It’s a great breakthrough—a stepping stone that helps us keep fighting to stop the pollution and give Cell City a better chance to stay healthy!

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