Doxorubucin is an effective therapeutic in cancer treatment, but it can cause cardiotoxicity and damage the mitochondrial machinery. A new patient-derived and mitochondria-rich human induced pluripotent stem cell derived-cardiomyocyte (hiPSC-CM) model protected by dexrazoxane was developed.

Developing a therapy for an ultra-rare condition has its challenges, including finding enough patients for clinical enrollment and convincing regulatory authorities that limited data prove the candidate is safe and effective. For that reason, Stealth Biotherapeutics Inc. has faced numerous roadblocks getting its mitochondria-targeting elamipretide across the finish line for Barth syndrome, a condition that affects about 230 to 250 males worldwide, including fewer than 150 in the U.S.

Too much of a good thing, it turns out, is a concept that applies to oxygen. And researchers at the University of California at San Francisco are working on a small molecule, Hypoxystat, that can lower tissue oxygen levels and prevent damage when oxygen levels are too high. When administered to mice with the rare mitochondrial disorder Leigh syndrome, the molecule more than tripled their average lifespan.

The Annual Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) meeting in Copenhagen this week is celebrating its 40th edition. In recognition of this landmark, the plenary session and opening lecture were attended by Queen Margrethe of Denmark. Afterward, the hot topic session on neuroprotective therapies set the stage for the subsequent discussions on the latest trends in the management and treatment of multiple sclerosis (MS).

In repeated concussions, removing damaged mitochondria could prevent the neurodegeneration that occurs when pathology progresses in some patients. The key would be in the role of the p17 protein in restoring mitophagy, according to scientists from the Medical University of South Carolina (MUSC). “Brain injury is an extrinsic disease. It is not idiopathic. When the primary injury occurs, the secure mechanism only relies on an endogenous protection of the brain. If you have a good neuroprotective mechanism, then after the primary injury, basically you don’t see any symptomatic effect,” Onder Albayram told BioWorld.

Orsobio Inc., already in the clinic with three candidates, has completed its $60 million series A financing. The company, CEO Mani Subramanian told BioWorld, has taken its time to find the right programs, put them together and only raised capital when it saw the programs had legs. Even the series A is a measured step, as Subramanian called the financing “modest.”

Mitochondria regulate many processes that are altered in cancer cells, from metabolism to oxidative stress to apoptosis. The metabolic reprogramming in cancer cells promotes an immunosuppressive environment that drives cancer progression. However, in a recent study, researchers from the Salk Institute of Biological Sciences have identified succinate as a metabolite that accumulated due to specific disturbances in the mitochondrial electron transport chain.

Researchers have demonstrated that inhibiting mitophagy in ‘old’ hematopoietic stem cells (HSCs) completely restored their blood reconstitution capabilities, raising the prospect of addressing the age-related weakening of the immune system that stems from HSCs deteriorating over time.

Leukemic cells rely on excessive mitochondrial respiratory and energy metabolism. Therefore, targeting mitochondrial proteases has been proposed as a potential approach to improve therapeutic regimens for acute myeloid leukemia (AML). The mitochondrial caseinolytic protease P (ClpP), located in the mitochondrial matrix, maintains protein quality by mediating the proteolytic hydrolysis of damaged proteins. The chaperone ClpX regulates this hydrolysis and is overexpressed in AML, thus providing a rationale for using ClpP agonists to disrupt AML proliferation.

Researchers based at Northwestern University Feinberg School of Medicine have discovered that a lack of contact between mitochondria and lysosomes in the cells of patients with a genetic form of Parkinson’s disease likely contributed to their symptoms.