New Understanding. New Possibilities.

For nearly 2 decades, researchers have been exploring new ways to treat myelodysplastic syndromes (MDS). Now, a deeper understanding of how the immune system impacts disease progression is bringing us closer to a different treatment approach: immunotherapy.


Understanding MDS

Before learning about the new scientific developments in myelodysplastic syndromes (MDS), it may be helpful to refresh your general knowledge about the disease.


MDS is a group of rare blood cancers in which the bone marrow stops making enough healthy blood cells.

Bone marrow is the soft, spongy tissue inside the bones. Stem cells in the bone marrow create immature blood cells called blasts. These blasts mature and develop into 3 types of blood cells: red blood cells, white blood cells, and platelets.

In MDS, the stem cells in the bone marrow are cancerous. This means that they do not function properly and do not mature the way they should.

This leads to:

  • Too many abnormal blast cells
  • Too few healthy, mature blood cells (cytopenia)
  • Overall weakened immune system
Blood cell development

Symptoms of MDS vary depending on which kind of blood cell is affected or the type of cytopenia.

Even though MDS can be very serious, some people do not experience symptoms or their symptoms are so mild that they can be confused for other conditions. Since most people with MDS are older than 65 years old, symptoms of MDS are sometimes brushed off as signs of aging.

This can lead to a delay in diagnosis.

For many people, a diagnosis of MDS comes as a shock. With the help of patient advocates from the MDS Alliance, we’ve compiled a list of associations and advocacy groups that provide useful information and ways to find support.

Find support and resources here >

Low levels of red blood cells (anemia) can cause fatgue or shortness of breath. Low levels of white blood cells (leukopenia) can cause frequent infection or fever. Low levels of platelets (thrombocytopenia) can cause frequent bleeding, such as nosebleeds, or easy bruising.

There are 6 subtypes of MDS and 5 risk scores.

To determine the subtype of MDS, doctors use the 2016 World Health Organization (WHO) Classification of MDS. Subtype is based on blood results, the appearance of the bone marrow, the number of blast cells, and any genetic changes.

Doctors use a tool called the Revised International Prognostic Scoring System (IPSS-R) to assign 1 of 5 risk scores: very low, low, intermediate, high, very high.

Risk scoring helps to predict someone’s prognosis and the likelihood that their MDS will progress to a more aggressive type of blood cancer called acute myeloid leukemia (AML). Different risk scores require different treatment approaches.

Learn more about the IPSS-R and risk scoring here >

What is IPSS-R scoring based on? Chromosome test results (cytogenics) from bone marrow biopsy at diagnosis. Blood count results (hemoglobin, platelets, absolute neutrophils) at diagnosis. % of blast cells in the bone marrow at diagnosis.

MDS is considered higher-risk if it is classified as intermediate, high, or very high. Higher-risk MDS is more severe and more likely to progress to AML than lower-risk types. It can have a serious physical and emotional impact on the people who live with it, and their caregivers.

Treatments for higher-risk MDS can be frequent and time-consuming and may cause side effects such as fatigue and a weakened immune system.

Living with higher-risk MDS can make it difficult to see family and friends and do everyday activities.

But after nearly 15 years without advances in treatment, there’s finally hope on the horizon.

19% Very Low, 38% Low, 20% Intermediate, 13% High, 10% Very High, ~40% of patients are diagnosed with higer-risk MDS (Intermediate, High and Very High)


Understanding Immune Dysfunction

Advances in MDS treatment start with a closer look at immune dysfunction and its impact on MDS progression.


The immune system is the body’s natural defense system. Bone marrow and white blood cells are important parts of the immune system. These cells circulate throughout the body and protect it against intruders like bacteria, viruses, and cancer cells.

Multiple immune cell types: T cells, natural killer cells, macrophages, and DCs

MDS disrupts the production of healthy immune cells. This leads to a weakened, or dysfunctional, immune system that doesn’t protect the body the way it should.

When a healthy immune system encounters abnormal or cancerous cells, it recognizes, attacks, and destroys them.

In MDS, the dysfunctional immune system is unable to find and kill the cancerous stem cells, abnormal blast cells, unhealthy blood cells, and weakened immune cells.

This allows MDS to progress.

Current treatments for MDS treat the cancerous cells but do not fix the immune dysfunction that may prevent the body from fighting back. Now, researchers have identified an immune system target that might help the immune system to regain function. This type of therapy is called immunotherapy.

Dysfunctional Immune System


Possibilities in Immunotherapy

An immune system target called TIM-3 could be the key to bringing immunotherapy to MDS.


Immunotherapy boosts the body’s own immune system to fight cancer and other diseases.

Different immunotherapy drugs act on different parts of the immune system. Immunotherapies that target proteins found on cancer cells and certain types of immune cells, such as T cells, have been successfully used to treat solid tumor cancers and blood cancers.

To date, immunotherapy has not been successfully used to treat cancers of the bone marrow, like MDS, also known as myeloid disease.

Now, researchers have identified a potential immune system target in MDS: TIM-3 receptors.

Heroic immune system

Receptors are found on the surfaces of cells and tell the cells what to do. TIM-3 is a receptor that is found on dysfunctional immune cells and cancer cells in MDS. This makes TIM-3 a potential target for immunotherapy.

TIM-3 receptors on dysfunctional immune cells tell them to turn off. This allows the cancer cells to escape and multiply. TIM-3 receptors on cancer cells tell them to grow and spread. Together, the effects of TIM-3 on both kinds of cells increase the speed of disease progression.

The role of TIM-3 receptors on cancer cells and T-cells
Hope Highway

Researchers are optimistic that targeting TIM-3 could slow the growth and spread of cancer cells and help the immune system regain its ability to fight back.

This novel approach, called immuno-myeloid therapy, could bring the benefits of immunotherapy to people with higher-risk MDS for the first time. Finally, hope is on the horizon.



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If you or a loved one is living with higher-risk MDS, you’re not alone. It can help to talk to others who are going through similar experiences.

Connect with others through the MDS Alliance.  


Novartis is proud to collaborate with the MDS Alliance on New Understanding. New Possibilities. Together, we are spreading the word about exciting advances in MDS.

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