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If you have MDS, a type of blood cancer called myelodysplastic syndrome, you’re part of a rare group of people with the diagnosis. But research is ongoing. Many clinical trials are seeking new treatments and drugs to better manage this bone marrow disorder. 

What Are Clinical Trials?

Clinical trials are a type of research that has people as the subjects. They're the main way researchers can tell if a new treatment – be it a medical device, a drug, a diet, a behavior, or any other remedy – works and is safe for the general public to use. Often a clinical trial compares how a new treatment might have better results or fewer side effects than ones currently used.

Though the drugs used in a trial often aren’t FDA-approved yet – which is one purpose of the trial – the FDA does have to give the green light for a clinical trial to proceed.

Clinical trials also can reveal how health care professionals might spot the disease sooner, without needing it to worsen first. They can also find new ways to improve your quality of life.

Who’s Right for a Clinical Trial?

The answer is, all kinds of people, and very specific people. Clinical trials are highly targeted to distinct groups – such as people who don’t respond to current remedies – to better home in on how effective a new drug or treatment is. At the same time, they want to cast a net wide enough to make sure it serves diverse groups of people.

Whether you might be a good pick for a trial will depend on many things, including:

  • Your age
  • How serious your condition is: Some trials testing various new drugs are targeted to more severe cases, while others focus on milder ones.
  • Whether you’re eligible for treatments such as chemotherapy
  • If you’ve tried the treatment being tested before
  • Whether you have co-conditions, such as anemia

Even though you might meet the basic criteria, researchers will further whittle down candidates based on more in-depth reasons. For example, they may ask if your cancer has certain molecular markers that might affect the outcome. So by design, you’d likely qualify for just a handful of current clinical trials.

Why Is Diversity So Important for Clinical Trials?

Your health isn’t just something that happens in your body. It’s affected by where and how you live, so researchers weigh other things, too. They want to tightly focus the trial to better control outside factors that might skew the results. But they also want to make sure they don’t leave out certain groups when they want to study a new drug or approach. 

To make sure the trial is both diverse and narrow enough, a clinical trial might zero in on:

  • Your race or ethnic background
  • Your lifestyle
  • Where you live, as well as the presence of pollution, systemic racism, or violence there
  • Your access to nutritious food, health care, and education
  • Your sexual orientation
  • Your socioeconomic status

What Should I Ask My Care Team About Clinical Trials?

It’ll help to have some key questions ready to ask your doctor about clinical trials. These might range from information about the trial itself, to benefits and risks, to your privacy rights.

You might want to know, for example:

  • Who’ll be in charge of my care during the trial?
  • How long will the trial last?
  • Will there be side effects to this treatment?
  • How might the treatment help my condition?
  • Will my health info be protected?
  • Who pays for the trial?
  • What if I react badly to the treatment?
  • How many office or hospital visits will there be?

How Can I Find Clinical Trials on My Own?

Other than talking with your doctor or other provider, there are several sources you can check:

  • The MDS Foundation’s website has a long list of clinical trials enrolling people. It narrows the field so you can get more of an “at-a-glance” look at current trials. 
  • has a full overview of current research.
  • Sites for organizations such as the National Cancer Institute often keep a list of ongoing clinical trials. 
  • Websites or support groups for MDS may feature lists of clinical studies. 

Once you sign up to get more information about a particular trial, a researcher likely will contact you for some basic data. If you’re a potentially good fit and agree to be part of it, you’ll sign an informed consent form so they can complete a screening.

What Clinical Trials Are Going on Now?

There are always studies in progress. Some of the ones happening now are looking at: 

  • How a drug called luspatercept works for people with a certain type of myelofibrosis (MF), which some people with MDS have
  • Whether an experimental drug called RVT-200 is safe and can cut the number of transfusions needed by people with milder MDS 
  • How certain FDA-approved drugs might work together
  • New ways to cut or ease symptoms and side effects of existing MDS treatment for better quality of life for people with the disease

Show Sources

Photo Credit: Yuguang Zhang / Getty Images


MDS Foundation: "What is MDS?" “Clinical Trials.”

National Institute on Aging: “What Are Clinical Trials and Studies?”

American Cancer Society: “An Overview of Cancer Clinical Trial Matching Services.”

National Institute on Minority Health and Health Disparities: “Diversity & Inclusion in Clinical Trials.”

National Cancer Institute: “Questions to Ask Your Doctor about Treatment Clinical Trials,” “Treatment Clinical Trials for Myelodysplastic Syndrome.” “An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK2 Inhibitor Therapy and Who Require Red Blood Cell Transfusions.”

The Aplastic Anemia and MDS International Foundation: “Myelofibrosis (MF).”

American Society of Clinical Oncology (ASCO): “Myelodysplastic Syndromes – MDS: Latest Research.”