Myelodysplastic syndromes (MDS) linked to abnormal stem cells

July 2, 2012

Researchers at Albert Einstein College of Medicine of Yeshiva University have found that abnormal bone marrow stem cells drive the development of myelodysplastic syndromes (MDS), serious blood diseases that are common among the elderly and that can progress to acute leukemia. The findings could lead to targeted therapies against MDS and prevent MDS-related cancers. The study is published today in the online edition of the journal Blood.

"Researchers have suspected that MDS is a 'stem cell disease,' and now we finally have proof," said co-senior author Amit Verma, M.B.B.S., associate professor of medicine and of developmental and molecular biology at Einstein and attending physician in oncology at Montefiore Einstein Center for Cancer Care. "Equally important, we found that even after MDS standard treatment, abnormal stem cells persist in the bone marrow. So, although the patient may be in remission, those stem cells don't die and the disease will inevitably return. Based on our findings, it's clear that we need to wipe out the abnormal stem cells in order to improve cure rates."

MDS are a diverse group of incurable diseases that affect the bone marrow and lead to low numbers of blood cells. While some forms of MDS are mild and easily managed, some 25 to 30 percent of cases develop into an called . Each year, about 10,000 to 15,000 people in the U.S. are diagnosed with MDS, according to the National .

Most cases of MDS occur in people over age 60, but the disease can affect people of any age and is more common in men than women. Symptoms vary widely, ranging from anemia to infections, fever and bleeding. Treatment usually involves chemotherapy to destroy abnormal blood cells plus supportive care such as blood transfusions.

In the current study, lead author Britta Will, Ph.D., research associate in the department of cell biology, and her colleagues analyzed bone marrow stem cells and (i.e., cells formed by ) from 16 patients with various types of MDS and 17 healthy controls. The stem and progenitor cells were isolated from bone marrow using novel cell-sorting methods developed in the laboratory of co-senior author Ulrich Steidl, M.D., Ph.D., assistant professor of cell biology and of medicine and the Diane and Arthur B. Belfer Faculty Scholar in Cancer Research at Einstein.

Genome-wide analysis revealed widespread genetic and epigenetic alterations in stem and progenitor cells taken from MDS patients, in comparison to cells taken from healthy controls. The abnormalities were more pronounced in patients with types of MDS likely to prove fatal than in patients with lower-risk types.

"Our study offers new hope that MDS can be more effectively treated, with therapies that specifically target genes that are deregulated in early stem and progenitor cells," said Dr. Steidl. "In addition, our findings could help to detect minimal residual disease in patients in remission, allowing for more individualized treatment strategies that permanently eradicate the disease."

Explore further: The Medical Minute: What is myelodysplastic syndrome?

More information: The paper is titled, "Stem and progenitor cells in myelodysplastic syndromes show aberrant stage specific expansion and harbor genetic and epigenetic alterations."

Related Stories

The Medical Minute: What is myelodysplastic syndrome?

June 15, 2012
Television journalist and host of "Good Morning America" Robin Roberts announced this week that she has myelodysplastic syndrome, or MDS, an uncommon blood and bone marrow disorder.

Myelodysplastic syndrome treated with deferasirox shows beneficial iron reduction

June 22, 2012
Researchers at Moffitt Cancer Center and colleagues at six other institutions have recently tested a treatment for patients with myelodysplastic syndrome, or MDS, a blood-related malignancy that involves the ineffective production ...

Discovery of genetic mutations better diagnose myelodysplastic syndromes

June 30, 2011
For patients with myelodysplastic syndromes (MDS), choosing the appropriate treatment depends heavily on the prognosis. Those patients at the highest risk of dying from their disease are typically offered the most aggressive ...

Recommended for you

Scientists find key to regenerating blood vessels

November 23, 2017
A new study led by researchers at Sanford Burnham Prebys Medical Discovery Institute (SBP) identifies a signaling pathway that is essential for angiogenesis, the growth of new blood vessels from pre-existing vessels. The ...

Researchers find infectious prions in Creutzfeldt-Jakob disease patient skin

November 22, 2017
Creutzfeldt-Jakob disease (CJD)—the human equivalent of mad cow disease—is caused by rogue, misfolded protein aggregates termed prions, which are infectious and cause fatal damages in the patient's brain. CJD patients ...

Surprising roles for muscle in tissue regeneration, study finds

November 22, 2017
A team of researchers at Whitehead has illuminated an important role for different subtypes of muscle cells in orchestrating the process of tissue regeneration. In a paper published in the November 22 issue of Nature, they ...

Study reveals new mechanisms of cell death in neurodegenerative disorders

November 22, 2017
Researchers at King's College London have discovered new mechanisms of cell death, which may be involved in debilitating neurodegenerative disorders, such as Alzheimer's disease and Parkinson's disease.

How rogue immune cells cross the blood-brain barrier to cause multiple sclerosis

November 21, 2017
Drug designers working on therapeutics against multiple sclerosis should focus on blocking two distinct ways rogue immune cells attack healthy neurons, according to a new study in the journal Cell Reports.

New simple test could help cystic fibrosis patients find best treatment

November 21, 2017
Several cutting-edge treatments have become available in recent years to correct the debilitating chronic lung congestion associated with cystic fibrosis. While the new drugs are life-changing for some patients, they do not ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.