Blog Archives

Hope for patients with cutaneous squamous cell carcinoma (CSCC): Cemiplimab-Rwlc (Libtayo)

October 31, 2018 – The American Food and Drug Administration (FDA)  recently approved Cemiplimab-Rwlc (Libtayo) injection for intravenous use for the treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation.

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Patisiran (Onpattro): First targeted RNA-based therapy approved to treat hATTR

August 14, 2018 – The American Food and Drug Administration (FDA) just approved Patisiran (Onpattro) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients.

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Cancer is not like cancer: Ethnic background matters

November 24, 2017 – New research indicates that one-size treatment for one form of blood cancer likely does not fit all, particularly when it comes to ethnic differences of patients.  Thus, African-American men are three times more likely to be diagnosed with multiple myeloma,

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Besponsa: Is this really a viable new treatment for ALL?

August 18, 2017 – B-cell precursor acute lymphoblastic leukemia (ALL) is a aggressive and rapidly progressing type of cancer in which the bone marrow makes too many B-cell lymphocytes, an immature type of white blood cell. In the United States, the National Cancer Institute estimates that approximately 5,970 people will be diagnosed with B-cell ALL this year and approximately 1,440 will die from the disease.

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Eteplirsen (Exondys 51) for Duchenne Muscular Dystrophy (DMD) approved

September 19, 2016 – This post presents below a virtually unedited announcement by the American Food & Drug Administration (FDA) concerning Duchenne Muscular Dystrophy (DMD) therapy  option which gives much hope for at least some of the affected patients.

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The HCV saga goes on: Epclusa has been approved for treatment of chronic Hepatitis C virus (HCV) infection

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Hope for patients with urothelial carcinoma: Atezolizumab (Tecentriq) approved

May 18, 2016 –  Over the last two years, biologics  targeting the PD-1/PD-L1 pathway (proteins found on the body’s immune cells and some cancer cells), notably Nivolumab (Opdivo) and Pembrolizumab (Keytruda), have come to the clinic for the treatment of a selection of oncologic indications.

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Venetoclax (Venclexta) for the treatment of patients with chronic lymphocytic leukemia (CLL)

April 12, 2016 – The American Food and Drug Administration (FDA) just approved Venetoclax (Venclexta) for the treatment of patients with chronic lymphocytic leukemia (CLL) who have a chromosomal abnormality called 17p deletion and who have been treated with a least one prior therapy.

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Theragenomic Medicine: Gefinitib (Iressa) targeted therapy for first-line treatment of patients with a type of metastatic lung cancer approved

July 13, 2015 – This post persents a press announcement by the U.S. Food and Drug Administration (FDA).  The text has slightly been edited by the autor of this blog. Thus, FDA today approved Gefinitib (Iressa) for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors harbor specific types of epidermal growth factor receptor (EGFR) gene mutations,

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Theragenomic Medicine: Targeted Therapies and Associated Toxicity

March 22, 2015 – This post addresses a topic which often gets lost in the current hype on targeted and precision (theragenomic) medicine. The issue is that targeted therapies are not only very precise and highly effective (in most patients) but they often also come with considerable clinical toxicity.

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  • Mapping genetic variants driving toxicity to leukemia therapy February 25, 2020
    Scientists at St. Jude Children's Research Hospital, alongside collaborators around the world, have created a comprehensive reference of functional variants in an important drug-metabolizing enzyme called NUDT15. This thorough understanding of NUDT15 variants provides an invaluable resource for predicting which patients being treated with thiopurine drugs for acute lymphoblastic leukemia (ALL) are likely to experience […]
  • Releasing brakes: Potential new methods for Duchenne muscular dystrophy therapies February 24, 2020
    Researchers identified a group of small molecules that may open the door to developing new therapies for Duchenne muscular dystrophy (DMD), an as-yet-uncured disease that results in devastating muscle weakening and loss. The molecules tested by the team from the Perelman School of Medicine at the University of Pennsylvania eased repression of a specific gene, […]
  • Extra chromosomes in cancers can be good or bad February 24, 2020
    Cancer cells are notorious for their genetic disarray. A tumor cell can contain an abundance of DNA mutations and most have the wrong number of chromosomes. A missing or extra copy of a single chromosome creates an imbalance called aneuploidy, which can skew the activity of hundreds or thousands of genes. As cancer progresses, so […]
  • Researchers show that DNA topological problems may cause lymphoma February 24, 2020
    Researchers from the Spanish National Cancer Research Centre (CNIO), Madrid, and the Andalusian Molecular Biology and Regenerative Medicine Centre (Cabimer), Seville, published a paper in Nature Communications that shows that DNA topological problems may cause endogenous DNA breaks that have a causal relationship with cancer.
  • New DNA test that reveals a child's 'true age' has promise, but ethical pitfalls February 24, 2020
    Epigenetic clocks are a new type of biological test currently capturing the attention of the scientific community, private companies and governmental agencies because of their potential to reveal an individual's "true" age.
  • Regular walnut consumption linked to health and longevity in women, according to new study February 25, 2020
    According to a new epidemiological study, women in their late 50s and early 60s who consumed at least two servings of walnuts per week had a greater likelihood of healthy aging compared to those who did not eat walnuts. After accounting for various factors that could impact health in older adults, such as education and […]
  • Cynicism and disrespect: A vicious cycle February 25, 2020
    An international study with data from Europe and the USA has found that disrespectful treatment leads to cynical beliefs about human nature. These cynical beliefs, in turn, again lead to disrespectful treatment.
  • New study shows significant increase in weight after breast cancer February 25, 2020
    New study findings suggest that weight gain after breast cancer is a greater problem than previously thought. The first national survey on weight after breast cancer in Australia, published in BMC Cancer journal, found close to two-thirds (63.7%) of women reported weight gain at an average of nine kilograms after a breast cancer diagnosis, and […]
  • Study finds gender disparities in hematology research success February 25, 2020
    Hematologists who complete a mentored training program experience greater levels of academic success than those who do not; however, a study published today in Blood Advances suggests a slight discrepancy in success levels between male and female hematologists.
  • NUS-led study suggests mangrove forests provide cause for conservation optimism, for now February 25, 2020
    An international team of researchers led by Associate Professor Daniel Friess and Dr Erik Yando of the National University of Singapore has found that globally, mangrove loss rates have reduced by almost an order of magnitude between the late 20th and early 21st century -- from what was previously estimated at one to three per […]
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