Innovation in Myelodysplastic Syndromes: Current Treatment and The Future of MDS 

Myelodysplastic Syndromes (MDS) is a complicated disease to diagnose and treat. Yesterday, we looked at the origin of  MDS, what early diagnosis looked like and where MDS treatment started. Today, we’ll look at where we are now and what the future of MDS treatment looks like. 

Understanding Current Treatments For Myelodysplastic Syndromes 

In yesterday's article, we talked about the history of an MDS diagnosis and early treatment options. While we still have a long way to go in regard to treating MDS, patients now have options to treat their disease. These options fall into the following categories:

• Supportive Care
• Growth Factors
• Chemotherapy
• Stem Cell Transplant

We’ll get into the specifics of these treatment options.

Supportive Care 

Supportive care doesn’t treat the disease itself. Rather, supportive care targets symptoms of the disease. These treatments can be used alone or in combination with other MDS treatments. Majority of supportive care in MDS targets anemia. 

Treating Low Red Blood cells (Anemia) - There are two main ways doctors will go about treating anemia in MDS patients. The first is usually injections of a manmade version of the growth factor erythropoietin which can aid the bone marrow in growing and producing more red blood cells. 

If a patient doesn’t find success with a growth factor, they may need to receive red blood cell transfusions. Patients may be given a cell maturation agent along with the transfusion which can help them go longer between transfusions.

Treating Low White Blood Cell Count (Neutropenia) - Patients with low white cell counts are at an increased risk of infection, and of those infections becoming serious. Patients are encouraged to take measures to try to prevent catching infections. If a patient does notice signs of an infection, they should alert their doctor. Patients with a bacterial infection will be treated with an antibiotic. 

If the infection does turn serious, patients may be given a white blood cell growth factor to help increase their white blood cell count to help them better fight off the infection.

Treating Low Platelet Count (Thrombocytopenia) - Patients with low platelet counts may be given platelet transfusions or growth factors. If these treatments do not help patients with their symptoms (easy bruising and excess bleeding) they may be given an antifibrinolytic agent, a type of drug that helps to reduce bleeding by blocking the breaking down of blood clots. 

Chemotherapy

Unlike supportive care, the purpose of chemotherapy is to directly target and treat the cancer in the bone marrow. There are several different kinds of chemotherapy now available to MDS patients. The decision on what type of chemotherapy to use will depend on the patients goals and general health. 

Standard Chemotherapy Drugs

Standard chemotherapy drugs are used specifically to attack and destroy MDS cells. Standard chemotherapy drugs are most often used in younger, otherwise healthy, High Risk-MDS patients as they are needed at high doses to be effective, and can be especially hard on the body. Patients who receive high dose standard chemotherapy are the most likely to go into remission, but the side effects can be serious and potentially life threatening. 

Hypomethylating Agents 

Hypomethylating agents help to slow the growth of MDS cells by blocking DNA that helps cancer cells to grow. They also help promote healthy cell maturation and formation. 

Immunosuppression Agents

Immunosuppressive agents help to suppress a patient's immune system. Suppressing the immune system allows stem cells the chance to grow and make new blood cells, but does come with risks associated with a lowered immune system.  

Immunomodulating Drugs

Immunomodulating drugs help doctors alter a patient's immune system. This allows doctors to target and depress only specific areas of the immune system, keeping the patient at lower risk of complications as their full immune system is not affected. 

Stem Cell Transplant

A stem cell transplant (SCT) is currently the only way to “cure” MDS. A younger, relatively healthy patient may qualify to receive a SCT. Patients who are older or in poor general health, which unfortunately makes up a majority of MDS patients, may not be good candidates for an SCT because of the toll the procedure can take on the body.

As a part of this treatment, patients are given high-dose chemotherapy to kill the cells in their bone marrow. They are then given new stem cells. Typically, an allogeneic SCT is used for MDS patients. This type of SCT requires that the stem cells come from a donor. Patients can be matched to a donor, not related to them, who’s HLA type is similar to the patients cell type, but results are best when the donor is closely related to the patient (like a brother or sister) and the donor's HLA type is a close match to the patient's cell type. 

Future of Myelodysplastic Syndromes Treatment 

Clinical trials let us see into the future of treatment. In August of 2023, Rebloyzl received FDA approval for use in LR-MDS patients who are ESA naive. This was a monumental approval for the MDS community. This approval marked the first new treatment option for patients in years. 

In July of 2023, another promising trial for MDS patients was discontinued. This trial was in a new class of treatments called monoclonal antibodies. Monoclonal antibodies are antibodies that are created in a lab by researchers who have a specific antigen (protein on the surface of a cell) they want to target. Once a target antigen has been identified, researchers can produce large quantities of the antibody specifically created to stick to the antigen. The monoclonal antibodies can then be given to a patient who has the target antigen to help their immune system recognize and attack those cells more effectively. 

While the Gilead monoclonal antibodies trial was discontinued, the science behind it is still very exciting and shows a lot of potential to revolutionize the way MDS and other cancers are treated.

Be A Part of The Future of MDS Treatment

Innovation in MDS treatment is exciting. Every step forward and every approval gets us closer to an MDS cure. As we can see, treatment development in MDS is still young. While this is frustrating for many patients, it creates the important opportunity for MDS patients to be actively involved in the development of new treatment options.

Clinical trials are what make new treatment options possible and they require dedicated patients willing to try something new. If you are interested in learning more about clinical trials, stay tuned next week when we’ll debunk common clinical trial myths. If you want to see if there are any clinical trials that you qualify for, use our Clinical Trial Finder to find one you qualify for or to get help signing up for a trial.