Grants and Grant Recipients
Grant Year: 2021
Myelodysplastic syndrome (MDS) in children is a rare group of disorders in which blood-making
stem cells
stem cells:
Cells in the body that develop into other cells. There are two main sources of stem cells. Embryonic stem cells come from human embryos and are used in medical research. Adult stem cells in the body repair and maintain the organ or tissue in which they are found. Blood-forming (hemapoietic) stem…
in the
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
(BM) fail to work properly. As a result,…
Grant Year: 2020
Circulating low-density granulocytes (LDGs) are a subgroup of neutrophils with immunoregulatory functions which can spontaneously release extracellular web-like structures (NETs) and cytokines sustaining
T cell
T cell:
see lymphocyte
responses and dendritic cell activation. We hypothesize that LDGs and NETs might be impaired in functions and frequency during
myelodysplastic syndromes
myelodysplastic syndromes:
(my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the bone marrow does not work well, and the bone marrow cells fail to make enough healthy blood…
Myelodysplastic syndrome (MDS) is a pre-cancerous disease of the blood, which progresses to the more aggressive
acute myeloid leukemia
acute myeloid leukemia:
(uh-KYOOT my-uh-LOYD loo-KEE-mee-uh) A cancer of the blood cells. It happens when very young white blood cells (blasts) in the bone marrow fail to mature. The blast cells stay in the bone marrow and become to numerous. This slows production of red blood cells and platelets. Some cases of MDS become…
(AML) in approximately 30% of cases. MDS currently affects about 60,000 people in the US, and about 10,000 new cases are…
Grant Year: 2019
Aplastic anemia
Aplastic anemia:
(ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most…
is a rare but serious blood disorder that occurs when the body’s
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
cannot produce enough healthy blood cells to function properly.…
The National Academy of Sciences Engineering and Medicine’s (NASEM) Veterans and Agent Orange (AO) Committees have been performing biennial reviews of the literature examining the relationship between AO exposure and the risk for adverse health outcomes since 1996. In 2018, the committee concluded there are sufficient data to associate AO exposure with an increased risk of Hodgkin and non-Hodgkin lymphoma, chronic lymphocytic leukemia, monoclonal gammopathy of unknown significance, and hypertension. In 22 years of follow up, there is only one paper addressing myelodysplastic syndrome (MDS)…
Grant Year: 2018
Androgens
Androgens:
Androgens are natural male hormones that can cause your bone marrow to make more red blood cells. This can improve anemia. Androgens are sometimes used to treat aplastic anemia and PNH.
Androgens that may be used include:
Danazol (Danacrine®)
Fluoxymestrone (Halotestin®)
Oxymetholone
…
have been used alone or in combination with other
immunosuppressive therapy
immunosuppressive therapy:
Immunosuppressive drug therapy lowers your body's immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem…
Paroxysmal nocturnal hemoglobinuria
Paroxysmal nocturnal hemoglobinuria:
(par-uk-SIZ-muhl nok-TURN-uhl hee-muh-gloe-buh-NYOOR-ee-uh) A rare and serious blood disease that causes red blood cells to break apart. Paroxysmal means sudden and irregular. Nocturnal means at night. Hemoglobinuria means hemoglobin in the urine. Hemoglobin is the red part of red blood cells. A…
(PNH) is a stem cell disorder caused by a
mutation
mutation:
Any change or alteration in a gene. A mutation may cause disease or may be a normal variation. Paroxysmal nocturnal hemoglobinuria (PNH) occurs…
Recurrent change-of-function mutations in RNA splicing factors are frequent in patients with
myelodysplastic syndromes
myelodysplastic syndromes:
(my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the bone marrow does not work well, and the bone marrow cells fail to make enough healthy blood cells. Myelo refers to the bone marrow. Dysplastic means abnormal growth or development. People with MDS have low blood cell count for at…
(MDS) and related myeloid neoplasms. Splicing factor mutations most typically occur as heterozygous mutations at recurrent ‘hotspots’ along…
Aplastic anemia
Aplastic anemia:
(ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most…
is a disease in which the
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
gradually stops producing red and white blood cells and platelets. As a result, people with aplastic anemia…
Grant Year: 2017
Myelodysplastic syndrome (MDS) is a diverse group of
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
diseases, unified by poor blood counts and a propensity for development of acute leukemia. MDS is most often diagnosed in older adults, arising as part of aging and without a toxic exposure or predisposing medical condition. In rare cases, however, MDS develops as a complication of an inherited bone marrow disease, such as
dyskeratosis congenita
dyskeratosis congenita:
An inherited disease that may lead to bone…