Nav: Home

Largest ever US study launches to research causes and genetics of blood diseases

May 25, 2016

The National Myelodysplastic Syndromes Natural History Study (The National MDS Study) is underway, the ECOG-ACRIN Cancer Research Group and its collaborators announced today. This new study, funded by the National Institutes of Health's National Heart, Lung, and Blood Institute (NHLBI), and performed in collaboration with the National Cancer Institute (NCI), will collect detailed information and biological samples from 2000 adults with myelodysplastic syndromes (MDS) and 500 more patients receiving care for a persistent low red blood cell count (anemia) that cannot be explained. Its purpose is to build a national resource to be used by scientists in future research.

A third group will be formed as a comparison cohort by selecting 1000 patients who will be screened in the study because of symptoms of MDS, but who will be found to not actually have one of the blood disorders. In total, the study will enroll up to 3500 patients, making it the largest-ever prospective study of MDS in the U.S.

It is hoped that this national resource will help researchers to identify the causes and genetic makeup of these serious and sometimes fatal diseases. Other research could lead to new and better ways to diagnosis and treat these conditions.

MDS occur when the blood-forming cells in the bone marrow are damaged and have problems making new blood cells. Considered a type of cancer, these abnormal blood cells fail to grow properly and die sooner than normal cells, leaving affected individuals with low blood counts and a shorter lifespan. Treatment options depend on the disease severity at diagnosis and are limited in their effectiveness.

About 30,000 people every year develop MDS, which occur mostly in adults over 60 years of age and more often in men than women. Common symptoms of MDS include fatigue, unusual bleeding, bruises, and tiny red marks under the skin, paleness, and shortness of breath.

Many people with MDS develop a serious or life-threatening anemia. About one-third of people with MDS develop acute myeloid leukemia, an aggressive blood cancer.

There are many questions about the causes of MDS and what patients can expect during the course of the disease. Unfortunately, MDS lacks a large collection of patient-related disease information and human tissue samples, such as diseased blood and bone marrow samples, which provide the opportunity for scientific research and breakthroughs. Such resources are already in place for other more common diseases, but not yet for MDS.

People 18 years of age or older who agree to participate in The National MDS Study will donate blood, bone marrow, and other body tissues (eyebrow hair, cells from the insides of the cheeks, and skin) to give researchers information to study how these blood disorders change over time. Patients will also answer quality of life questionnaires on how the disease affects their physical and emotional well-being over time.

This study requires the participation of a large network of physicians who support medical research and who examine people experiencing the symptoms of MDS. A number of organizations are collaborating on this effort. For patient recruitment, which is expected to take five to seven years, the NCI is contributing access to its two large cancer research networks, the NCI National Clinical Trials Network (NCTN) and the NCI Community Oncology Research Program (NCORP).

Physicians may enroll their patients in this study if they and their hospital, practice, or cancer center are a member of any cancer research group that belongs to either NCI network. These groups are the Alliance for Clinical Trials in Oncology, ECOG-ACRIN Cancer Research Group, NRG Oncology, and SWOG.

ECOG-ACRIN Cancer Research Group, which is leading the study, has added The National MDS Study to its portfolio of active clinical trials in leukemia, thus streamlining its implementation at clinical centers nationwide.

Patients' blood, bone marrow, and other body tissues will be processed and stored at a central laboratory and biorepository at the Moffitt Cancer Center and its M2Gen subsidiary in Tampa, Fla. Patient information and data from patient samples will be linked and stored centrally at a data coordinating center, under the supervision of The Emmes Corporation, which is coordinating the trial.

At the end of this study, the collected data and specimens will be transferred to the NHLBI and will be available to scientists throughout the country for their own research studies. In this way, The National MDS Study will enable scientists to answer questions about MDS that up to this point have been impractical to study at a single institution or even among small groups of researchers.
Visit the official website,, to obtain more information about the study.

The scientific leadership for The National MDS Study is as follows:

Study Chair

Mikkael A. Sekeres, MD, MS
Cleveland Clinic Taussig Cancer Institute

Study Deputy Chair

Steven D. Gore, MD
Yale Cancer Center

Study Statistician

Donald M. Stablein, PhD
The Emmes Corporation

Principal Investigator, Central Laboratory/Biorepository

Pearlie K. Burnette, PharmD, PhD
Moffice Cancer Center

Quality of Life Chair

Gregory A. Abel, MD, MPH
Dana-Farber Cancer Institute


The ECOG-ACRIN Cancer Research Group is a membership-based scientific organization that designs and conducts cancer research involving adults who have or are at risk of developing cancer. ECOG-ACRIN comprises nearly 1100 member institutions in the United States and around the world. Approximately 12,000 physicians, translational scientists, and associated research professionals from the member institutions are involved in Group research, which is organized into three scientific programs: Cancer Control and Outcomes, Therapeutic Studies, and Biomarker Sciences. ECOG-ACRIN is supported primarily through National Cancer Institute research grant funding, but also receives funding from private sector organizations through philanthropy and collaborations. It is headquartered in Philadelphia, Pa. For more information, visit, call 215.789.3631, and follow the organization on Twitter: @eaonc.

ECOG-ACRIN Cancer Research Group

Related Cancer Articles:

Cancer mortality continues steady decline, driven by progress against lung cancer
The cancer death rate declined by 29% from 1991 to 2017, including a 2.2% drop from 2016 to 2017, the largest single-year drop in cancer mortality ever reported.
Stress in cervical cancer patients associated with higher risk of cancer-specific mortality
Psychological stress was associated with a higher risk of cancer-specific mortality in women diagnosed with cervical cancer.
Cancer-sniffing dogs 97% accurate in identifying lung cancer, according to study in JAOA
The next step will be to further fractionate the samples based on chemical and physical properties, presenting them back to the dogs until the specific biomarkers for each cancer are identified.
Moffitt Cancer Center researchers identify one way T cell function may fail in cancer
Moffitt Cancer Center researchers have discovered a mechanism by which one type of immune cell, CD8+ T cells, can become dysfunctional, impeding its ability to seek and kill cancer cells.
More cancer survivors, fewer cancer specialists point to challenge in meeting care needs
An aging population, a growing number of cancer survivors, and a projected shortage of cancer care providers will result in a challenge in delivering the care for cancer survivors in the United States if systemic changes are not made.
New cancer vaccine platform a potential tool for efficacious targeted cancer therapy
Researchers at the University of Helsinki have discovered a solution in the form of a cancer vaccine platform for improving the efficacy of oncolytic viruses used in cancer treatment.
American Cancer Society outlines blueprint for cancer control in the 21st century
The American Cancer Society is outlining its vision for cancer control in the decades ahead in a series of articles that forms the basis of a national cancer control plan.
Oncotarget: Cancer pioneer employs physics to approach cancer in last research article
In the cover article of Tuesday's issue of Oncotarget, James Frost, MD, PhD, Kenneth Pienta, MD, and the late Donald Coffey, Ph.D., use a theory of physical and biophysical symmetry to derive a new conceptualization of cancer.
Health indicators for newborns of breast cancer survivors may vary by cancer type
In a study published in the International Journal of Cancer, researchers from the UNC Lineberger Comprehensive Cancer Center analyzed health indicators for children born to young breast cancer survivors in North Carolina.
Few women with history of breast cancer and ovarian cancer take a recommended genetic test
More than 80 percent of women living with a history of breast or ovarian cancer at high-risk of having a gene mutation have never taken the test that can detect it.
More Cancer News and Cancer Current Events

Trending Science News

Current Coronavirus (COVID-19) News

Top Science Podcasts

We have hand picked the top science podcasts of 2020.
Now Playing: TED Radio Hour

Climate Mindset
In the past few months, human beings have come together to fight a global threat. This hour, TED speakers explore how our response can be the catalyst to fight another global crisis: climate change. Guests include political strategist Tom Rivett-Carnac, diplomat Christiana Figueres, climate justice activist Xiye Bastida, and writer, illustrator, and artist Oliver Jeffers.
Now Playing: Science for the People

#562 Superbug to Bedside
By now we're all good and scared about antibiotic resistance, one of the many things coming to get us all. But there's good news, sort of. News antibiotics are coming out! How do they get tested? What does that kind of a trial look like and how does it happen? Host Bethany Brookeshire talks with Matt McCarthy, author of "Superbugs: The Race to Stop an Epidemic", about the ins and outs of testing a new antibiotic in the hospital.
Now Playing: Radiolab

Speedy Beet
There are few musical moments more well-worn than the first four notes of Beethoven's Fifth Symphony. But in this short, we find out that Beethoven might have made a last-ditch effort to keep his music from ever feeling familiar, to keep pushing his listeners to a kind of psychological limit. Big thanks to our Brooklyn Philharmonic musicians: Deborah Buck and Suzy Perelman on violin, Arash Amini on cello, and Ah Ling Neu on viola. And check out The First Four Notes, Matthew Guerrieri's book on Beethoven's Fifth. Support Radiolab today at