Acute myeloid leukemia (AML) is a type of blood cancer that grows quickly and is difficult to treat. Patients with this condition often face difficult treatment journeys, especially when there’s a relapse after therapy or if it does not respond at all. Scientists have been working on targeted medications to fight this cancer for the last several years, and one of the results of these efforts is Gilteritinib, also known as ASP2215 in research.
This medicine has been an important option for people with AML who have a gene change called the FLT3 mutation. It operates differently from standard chemotherapy, with a lot of patients already recording improved results. The real questions most people have are: How does it work, and what should we know about it? That’s what this article is going to cover.
Understanding AML and FLT3 Mutations
The bone marrow is where blood cells are made, and that’s where AML starts. Instead of growing into healthy cells, the marrow makes too many immature cells called blasts. These blasts push out normal cells, making it hard for the body to fight off infections or stop bleeding.
Around one in three AML patients have changes in a gene called FLT3. This gene usually helps blood cells grow, but when it mutates, it makes cancer grow. Patients with this mutation often see the cancer return faster and don’t respond well to common treatments.
How Gilteritinib Was Developed
Researchers looked for ways to block the activity of mutated FLT3. Early drugs worked for some patients but not all, with resistance emerging in some cases, enabling the cancer cells to survive.
This led to the development of medicines that are stronger and more selective. One such medicine is Gilteritinib (ASP2215) FLT3/AXL inhibitor, which was developed by Astellas Pharma and approved by the U.S. FDA in 2018. It got approved shortly after a large clinical trial showed a higher survival rate compared to chemotherapy. If you’re looking for the medicine to buy, it is sold under the brand name Xospata.
How Gilteritinib Works
This medicine belongs to the tyrosine kinase inhibitor class, a medication that stops some proteins from fueling cancer development. This is how it works:
- FLT3 inhibition: Gilteritinib targets the FLT3 protein from sending out signals that aren’t normal.
- AXL inhibition: It also blocks AXL, another protein that can help cancer resist treatment.
- Overcoming resistance: The treatment works even after other FLT3 medicines stop working by attacking both FLT3 and AXL.
This dual action is why the drug is often referred to as a Gilteritinib (ASP2215) FLT3/AXL inhibitor in research papers.
Who Can Use this Medicine?
Adults with AML that has come back or not responded to treatment are the main group of people who use Gilteritinib. Doctors use a diagnostic test that has been approved to first check for FLT3 mutations, and may prescribe this drug if the mutation is present.
Researchers are also studying whether it can be used with other drugs to treat newly diagnosed cases of AML. These studies are still going on, and the results will help decide how to use them in the future.
How It Is Taken
Gilteritinib is an oral medication, which comes in tablet form and is taken once a day. Patients should take it at the same time every day, with or without food.
It should be taken with water and swallowed completely. Skipping doses or stopping without talking to a doctor can affect the success of the treatment.
Benefits Seen in Trials
In clinical studies, patients who took Gilteritinib lived longer on average than those who had standard chemotherapy. Some people achieved complete remission, which means that blood tests could no longer detect their cancer. Some of the most important findings from the trials are:
- Improved overall survival rates
- Responses across different FLT3 mutation types
- A likelihood of remission even in heavily pretreated patients
Some Possible Side Effects
Gilteritinib, like many medication, has side effects. Most patients can tolerate it better than chemotherapy, but some people have serious reactions.
Some common side effects are:
- Diarrhea
- Tiredness
- Raised liver enzyme levels
- Decreased red blood cells or platelets
Some serious side effects are:
- Infections like pneumonia or sepsis
- Fever with low white blood cells (febrile neutropenia)
- Differentiation syndrome, a rare but deadly reaction in which cancer cells suddenly mature
While using this medicine, patients need to go for regular check-ups and blood tests.
Important Safety Notes
- Stay away from grapefruit products since they interfere with how the drug is processed in the liver.
- St. John’s Wort and other herbal products may make the medication less effective.
- Doctors usually check heart rhythm with an ECG since the medicine may rarely cause QT prolongation.
Why AXL Inhibition Matters
Some patients who took earlier FLT3 inhibitors saw their cancer return quickly. Scientists discovered that AXL, another protein, played a role in this. It acted as a backup pathway for cancer cells.
Gilteritinib stops this escape route by also blocking AXL. This sets the medicine apart from older treatments and helps patients achieve longer responses. You can visit https://www.nature.com/ to learn more about AXL inhibition.
What Doctors Are Studying Next
Gilteritinib has revolutionized the way we think about relapsed AML, although research is still going on. Researchers are still asking questions like:
- Can it be combined with chemotherapy in those who have just been diagnosed with AML?
- Can it be paired with other targeted medications to prevent resistance?
- Will it function better if you use it earlier in treatment instead of later?
The answers may expand the use of this medicine in the future.
Conclusion
Gilteritinib gives patients with relapsed or hard-to-treat AML hope when there aren’t many other options. It might not be suitable for everyone, but for those with FLT3 mutations, it has become an important treatment. It already plays a major role in treating AML today, but more studies will help us figure out how to use it in the future. The first step in deciding if this medicine is right for you is to talk with your healthcare team about genetic testing and the treatment options that are available.
