Scientists partly funded by NHLBI identified two proteins in cancer cells that make them resistant to chemotherapy. They discovered that blocking those proteins during therapy eliminated human leukemia in mice. According to the authors of the study, published in Nature Medicine, this molecular mechanism is present in almost all cancers driven by enzymes known as tyrosine kinases, such as lung, breast, colon and brain tumors. “We anticipate that this study opens a new avenue to develop curative therapy not only for leukemia but solid tumors, as well,” said lead investigator Mohammad Azam, Ph.D.
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Researchers funded by NHLBI found that noninvasive CT and stress tests can help predict heart attacks without the complications associated with more invasive procedures. "The traditional approach with invasive catheterization requires that patients go to the hospital, get a catheter inserted into their leg and go in for the nuclear SPECT study on a different day," said NHLBI researcher and coauthor Marcus Chen, M.D. The findings appear in Radiology.
A group of NHLBI-funded researchers have found that the corticosteroids that people with severe asthma are prescribed might be harming them. Ten to 15 percent of people with asthma have a severe form of the condition that doctors try to control unsuccessfully by increasing the dosage of the medications. In a study published in Science Immunology, the researchers found that these patients’ immune systems present differences that explain why the higher doses don’t help and could be harming the patients by worsening the inflammation.
Researchers funded by NHLBI have discovered that mutations in ABL1, a well-known gene involved in the Philadelphia chromosome of leukemia cells, cause congenital heart defects and skeletal malformations. The findings, published in Nature Genetics, suggest that ABL1 has an important role during organism development.
In an important advance in the stem cell field, researchers are reporting that they were able to use a patients’ own cells to create cells similar to those in the bone marrow and then use them to identify potential treatments for a rare blood disorder. The researchers obtained the so-called blood progenitor cells from the skin of two patients with Diamond-Blackfan anemia (DBA), a rare blood disorder in which the bone marrow cannot make enough oxygen-carrying red blood cells. They converted the skin cells into blood progenitor cells, which they then processed through a drug screening system. The researchers identified one chemical in particular, called SMER28, during this screening process that showed promise in animal studies for producing red blood cells. The finding could lead to new treatments for DBA, they say. The study, which appeared in Science Translational Medicine, was partly funded by NHLBI.
In a clinical trial partly funded by NHLBI, researchers are reporting early success in treatment of sickle cell disease with gene therapy. The findings of the report, published in the New England Journal of Medicine, come from a single patient who has been free of symptoms for over a year. Researchers caution, however, that much more research is needed before gene therapy can become an option for treatment or cure of this condition.
Research partly funded by NHLBI links heart disease risks such as diabetes, heart blood pressure or smoking in middle age with higher dementia risk later in life. Researchers presented their findings at the American Stroke Association’s International Stroke Conference 2017.
The study Modulation of mTOR signaling as a strategy for the treatment of Pompe disease partly funded by NHBLI made the cover of the March issue of the journal EMBO Molecular Medicine. The findings might pave the way to some therapeutic interventions by identifying profound signaling defects of the mTOR gene.
There is something to be said for ‘eating your own,’ at least at the cellular level. Researchers funded by NHLBI demonstrate that autophagy, a process of cellular self-consumption, is essential for the healthy metabolism and function of haematopoietic stem cells, the rare adult stem cells, found in the bone marrow, that produce all blood cell types. The findings, published in Nature, show how autophagy, which is associated with health and longevity, contributes to maintaining the stem cells’ ability to regenerate the blood system.
Researchers partially funded by NHLBI warned that seniors sleeping more than nine hours each night could be at higher risk of dementia in later years. The study, published in the journal Neurology, shows the increased risk in people who were not used to sleep that much before. The researchers say that the need for extra sleep might be a sign of the risk of dementia, not the cause.