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Mesenchymal Stem cells (MSCs) derived from mouse muscle tissue and bone marrow were used in an experimental cell therapy for Duchenne Muscular Dystrophy.

Duchenne muscular dystrophy (DMD) is an incurable, progressively debilitating muscular-skeletal and cardiac disease caused by a mutation in the dystrophin gene. The encoded dystrophin protein is part of a larger protein complex involved in anchoring the muscle cytoskeleton to components of the extracellular matrix. Loss of dystrophin leads to muscle wasting, and heart disease is a major cause of death in patients with DMD. Therefore, the availability of effective treatments to address cardiac function in people with DMD is vital.

While there is no cure for acute kidney injury, a research team out of the University of Wisconsin-Madison may be shedding some new light on this condition.

Acute kidney injury (AKI) is characterized by a sudden decline in kidney function. This decline is marked by a significant rise in serum creatinine levels that may be accompanied by a reduction in urine output. Some cases of AKI require kidney transplantation or can lead to death. There are a number of causes of AKI, including injury from gadolinium-based contrast agents (nephrogenic systemic fibrosis), rhabdomyolysis, low blood volume, and vasculitis.

A collaborative team of scientists from Denmark, France, the UK, and the US recognized the potential to target CD163 macrophages for a novel approach to cancer immunotherapy.

Macrophages are one of many types of immune system cells of the body, and they function to phagocytize and digest invading substances including bacteria and other microorganisms; they also digest cellular debris and cancer cells. However, there are a subset of macrophages (CD163 macrophages) that actually supports tumor survival and impedes T-cell attack of tumors. Expression of CD163 by these macrophages is associated with a poorer prognosis for a number of human cancers, such as malignant melanoma.

The new FT516 immunotherapy is the first iPSC-derived therapy approved for clinical testing in the world and may provide a treatment for many different types of cancers.

A collaboration between the University of Minnesota and Fate Therapeutics will begin clinical trials for a novel “off-the-shelf” targeted natural killer (NK) cell cancer immunotherapy derived from induced pluripotent stem cells (iPSCs). iPSCs are adult cells that have been genetically reprogrammed so that they regain the ability to differentiate into any cell type, making them pluripotent (similar to embryonic stem cells). The new NK cell therapy, FT516, is developed from genetically engineered human iPSCs with enhanced anti-tumor activity.

A drug used to treat acetaminophen hepatotoxicity may give hope to an effective treatment for FVH caused by liver-impairing viruses, including hepatitis A.

Fulminant viral hepatitis (FVH) is a form of acute liver failure caused by a virus and characterized by severe impairment of liver function. Primarily, children and young adults are affected, and the condition is usually fatal unless liver transplantation is implemented early enough. FVH tends to occur in people who are healthy and without any history of previous liver disease and progresses rapidly leading to encephalopathy and death. Given the treatment challenges and lack of a full understanding of how FVH develops, a group of scientists from Rockefeller University in New York analyzed the DNA of an 11-year-old girl who died from FVH.