Myelodysplastic syndromes (MDS) are treated based on type and risk assessment, but traditional therapies may not work for everyone. Clinical trials offer a way to explore emerging treatments and advance current medical knowledge for others living with MDS.

Myelodysplastic syndromes (MDS) affect how your bone marrow makes new blood cells. In MDS, bone marrow produces immature blood cells (red, white, or platelets) that are abnormally formed, dysfunctional, and often short-lived.

Sometimes referred to as bone marrow disorders, MDS are now widely recognized as cancers. Several different types exist, each defined by unique clinical features.

If you’ve been diagnosed with MDS, treatment depends on many different factors, and for some people, clinical trials may hold the most benefits.

Clinical trials are research studies investigating the safety and effectiveness of emerging medical interventions. They’re conducted in phases, using human volunteers to evaluate the benefits, risks, adverse reactions, and best practice applications of new medications, procedures, or other therapies.

While clinical trials are often recommended when a condition hasn’t responded to conventional treatment, other reasons a clinical trial for MDS might be right for you include:

  • having reduced-cost access to a multidisciplinary team of medical professionals
  • receiving disease-specific, personalized care
  • gaining access to the latest treatment options before they’re publicly available
  • the possibility of improved health outcomes
  • being an active part of medical research that may help future generations
  • adding racial or ethnic diversity to help inform effective treatment development

Clinical trials are often available even for early stages of a condition, though eligibility for each trial will vary. In MDS, for example, some trials might seek participants who are considered low-risk while others may want to focus on participants with treatment-resistant MDS.

According to the American Cancer Society, considering a clinical trial for MDS makes sense for many people because there’s currently no universal “best” MDS treatment option, and MDS can become harder to treat over time.

Are there any risks in participating in a clinical trial?

There’s always some risk when participating in a clinical trial. Adverse effects are possible and aren’t always predictable or expected. You may experience discomfort, pain, or even life threatening complications.

This is part of the purpose of a clinical trial — to determine who benefits under what circumstances and who experiences negative effects and why. Different clinical trial phases can carry different levels of risk, but all risks are carefully monitored throughout the process.

Clinical trials also follow very specific guidelines designed to accurately convey results. They may require significant time commitments for appointments and testing, and there may be lifestyle restrictions that affect your activity, diet, or regular habits.

MDS are not a single condition. They’re a group of bone marrow cancers with unifying characteristics. Their diversity and varying severity creates a broad range of treatment possibilities.

Stem cell transplant, supportive care (blood transfusions and blood cell growth factors), and chemotherapy make up the basics of conventional MDS treatment. Which therapies your doctor recommends depend your MDS diagnosis, current symptoms, and your overall health and personal preference.

To help improve treatment outcomes in MDS, research into new therapies in ongoing. Promising treatments in development for MDS, outlined in a 2021 review, include:

  • Hypoxia-inducible factor (HIF) prolyl hydroxylase inhibitors: Stimulate red blood cell production to relieve anemia.
  • Telomerase inhibitors: Target an enzyme associated with the growth of cancer cells.
  • Thrombopoietin-receptor agonists: Stimulate platelet production, reducing bleeding risk.
  • Oral azacitidine: Helps reactivate tumor-suppressing genes silenced in certain cancers.
  • Second-generation hypomethylating agents (HMAs): Also work to reactivate tumor-suppressing genes, similar to azacitidine.
  • Molecular targeted therapies: Target specific molecular pathways associated with MDS cell mutations.
  • Apoptosis inhibitors: Initiate cell destruction in targeted cancer cells.
  • Targeted antibodies:Trigger an immune response to destroy targeted cancer cells.

Some of these interventions are also being investigated in combination with other experimental treatments and current therapies to help boost effectiveness. Before any treatments are available for public use, the clinical trial results must show significant benefits.

An oncologist, a doctor specializing in the treatment of cancer, can direct you to MDS clinical trials that may be suitable for your individual diagnosis.

If you’re interested in researching available clinical trials on your own to discuss with your doctor later, you can search those available by visiting:

Some ongoing and recruiting clinical trials for MDS include:

Doctors understand the benefits and appeal of clinical trials, especially for conditions like MDS where treatment success can vary by individual. If you want to discuss this option with your oncologist, you can help make this conversation as informative and constructive as possible by:

  • researching available clinical trials before your appointment
  • writing down questions you have about clinical trials
  • asking your doctor why you may or may not be a good candidate for a clinical trial
  • bringing a loved one to help take notes or offer an alternative perspective after the discussion
  • discussing financial considerations of conventional therapy versus a clinical trial
  • creating a follow-up plan if you decide to apply for a clinical trial

In general, MDS outlook varies, with survival rates ranging between months to years.

There is no way to know if a clinical trial for MDS will improve your outlook in MDS. Your doctor can help you select a clinical trial that holds the most promise for your individual diagnosis.

Clinical trials for MDS aren’t just there for when traditional therapies don’t work. Clinical trials offer a variety of benefits, including reduced-cost access to a multidisciplinary team of professionals, targeted therapy, and the ability to receive MDS medications not otherwise available.

While adverse reactions are possible in clinical trials, your doctor can help you navigate the benefits and risks of participation. There’s no guarantee a clinical trial will improve your MDS outlook, but there’s a possibility you’ll see success with an emerging treatment.