Scientists find vital clues that unravel the mystery behind the spread of a rare blood disease called myelodysplastic syndromes or MDS

By Syed Akbar
Hyderabad:  A US-based team of researchers including an Indian
doctor has found vital clues that unravel the mystery behind the
spread of a rare blood disease called myelodysplastic syndromes or MDS.

The team noticed that abnormal stem cells trigger the development and
progression of MDS and thus the disease can be tackled by wiping out
such abnormal cells. It could also help in finding therapies against
this serious blood disease and related cancers.

MDS has been in the news due to ABC-TV’s Robin Roberts’ diagnosis and
the death of writer/director Nora Ephron, who succumbed to a form of
leukemia that may have progressed from MDS. At present treatment such
as chemotherapy and bone marrow transplants are available, but they
help in only remission and they are not curative.

The study was published in the latest issue of science journal, Blood.
India-born Dr Amit Verma, who heads the Myelodysplastic Syndromes
Centre at the Montefiore Einstein Centre for Cancer Care, is part of
the research team.

Myelodysplastic syndrome is common among the elderly and can progress
to acute leukemia. “Researchers have suspected that MDS is a ‘stem
cell disease,’ and now we finally have proof,” said Amit Verma, who is
also an associate professor of medicine and
of developmental and molecular biology at Albert Einstein College of Medicine.

“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 do not 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,” Dr Amit Verma said

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 per cent of cases develop
into an aggressive disease called acute myeloid leukemia.

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.

The researchers analyzed bone marrow stem cells and progenitor cells
(cells formed by stem cells) from 16 patients with various types of
MDS and 17 healthy controls. The stem and progenitor cells were
isolated from bone marrow. 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.