Innovation in Myelodysplastic Syndromes: Diagnosis and Initial Therapies

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

Understanding The History Of A Myelodysplastic Syndromes Diagnosis

MDS is a relatively new diagnosis. It wasn’t until 1982 when the first classification of MDS was published in the French-American-British (FAB) group. This is when MDS first became a formal diagnosis, though the initial understanding of the disease was at a very basic level. At that time, MDS was defined and characterized by its refractory anemia and excessive blasts in the bone marrow. Prior to the 1980’s, MDS was called vitamin and iron resistant anemia. The refractory anemia that was part of the defining characteristics of early MDS diagnosis refers to anemia that persisted after treatment to manage vitamin and iron levels.

In the 1990’s, a more complete understanding of MDS began to emerge. The cytogenetic factors, that we now understand play a large role in MDS, were just starting to be identified. These mutations and genomic factors found in MDS patients then became a part of the diagnosis. In 2001, we moved from the FAB classification of MDS to the World Health Organization (WHO) classification. The main difference in these two classifications was the blast percentage. The FAB classification required >30% blasts for an MDS diagnosis, while the WHO classification required >20%.

Innovation in Myelodysplastic Syndromes Treatment 

FDA approved disease-modifying therapies have only been available for MDS patients since 2004. This means that MDS treatment is still relatively young in the areas of research and development.

Before 2004 (and even still today), erythropoiesis-stimulating agents (ESA’s) were used off-label to treat MDS patients. ESA’s work by stimulating the bone marrow to produce more red blood cells. These therapies were not seen to directly alter patients' disease, rather they targeted anemia, one of the symptoms of MDS.

In the 2000’s and 2010’s, MDS patients' treatment options were decided mainly by whether the patient was considered to have lower or higher-risk disease. Patients with lower-risk disease were given ESA’s as a first line treatment to treat their anemia. Lenalidomide began to be used for patients with the chromosomal abnormality, del(5q). Patients with higher-risk disease were given hypomethylating agents like azacitidine and decitabine. Hypomethylating agents work by suppressing the DNA that allows cancer cells to grow. 

Limited options for patients meant that there was a lot of work that needed to be done. Dedicated researchers and clinicians have spent years studying MDS and what can be done to cure this disease. Tomorrow, we’ll talk about where we are now in regard to treatment options available to patients and what current clinical trials tell us about the future of treatment in MDS.

Be A Part of The Future of MDS Treatment

As we can see, treatment development in MDS is still young. While this is frustrating for many patients, it creates the incredibly 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 a bunch of common clinical trial myths. If you want to see if there are any clinical trials that you qualify for, you can use our Clinical Trial Finder to find one or to get help signing up for a trial.