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Treating liver diseases with mitochondria-enriched stem cells

August 26, 2021
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Stem cells enriched with mitochondria can be used in effective and long-term therapies for liver diseases, according to a patent application from Minovia Therapeutics.

Minovia is an Israeli clinical-stage company specializing in mitochondrial cell therapies. Diseases of the mitochondria occur when the powerhouse of the cell fails to produce enough energy for the body to function properly. These disorders are long-term, genetic, and often inherited.

Minovia’s proposed treatment is particularly for liver diseases, also known as hepatopathies, and their symptoms. The company says the target diseases may or may not be associated with mitochondrial dysfunction. Mitochondrial hepatopathies can be characterized by liver inflammation or scarring, if not the outright failure of the organ. Current medical therapy of mitochondrial hepatopathies is largely ineffective, and the prognosis is usually poor.

A form of mitochondria augmentation therapy is being patented. It involves administering, either systemically or directly to the liver, human stem cells enriched with healthy and functional human mitochondria. The stem cells would be derived from bone marrow, adipose tissue, oral mucosa, skin fibroblasts, blood, or umbilical cord blood. The mitochondria would be obtained from a donor’s placenta, placental cells grown in culture or blood cells.

Minovia says the invention is partly informed by findings from a single round of treatment of juveniles with Pearson Syndrome (PS), a congenital disease caused by a mutation in mitochondrial DNA. It is a bone marrow failure disorder in which the body is unable to produce enough red blood cells, white blood cells, and platelets. PS usually starts in infancy, and affected children typically feel weak and tired, are sick more often, bruise more easily, and take a longer time to stop bleeding when cut.

The patent application says stem cells enriched with healthy functional mitochondria delivered significant improvements across a variety of parameters, including those relating to liver function.

Minovia cited the case of one patient, a 6-year-old boy with PS who had not gained weight in three years. The patient received, via IV infusion, a single round of treatment of stem cells enriched with healthy mitochondria. The therapy resulted in weight and height gains after 9 and 15 months, respectively.

Additionally, doctors measured the amount of lactic acid in the patient’s blood, an indicator of mitochondria dysfunction. Blood lactate is a result of anaerobic metabolism when mitochondria are damaged or when oxygen delivery to the tissues is insufficient to support normal metabolic demands. Following treatment, the patient’s blood lactate level decreased to normal values.

A line graph illustrating the standard deviation score of the weight and height of a PS patient treated by the methods provided in the present invention as a function of time before and after therapy.
A line graph illustrating the standard deviation score of the weight and height of a PS patient treated by the methods provided in the present invention as a function of time before and after therapy.

The pharmaceutical composition described in the patent application has the potential to become a viable approach to a host of liver-related health complications. Around the world, approximately 2 million individuals die from liver disease each year; there are 4.5 million people diagnosed with liver disease in the U.S. alone. What Minovia has outlined is a way to make stem cells—“master cells” capable of turning into every other cell in the human body—even more powerful. This therapy may offer a new chance of recovery to liver disease patients, simply by giving the body the power it needs to fix itself.

The featured patent application, “Mitochondrial Augmentation Therapy of Liver Diseases”, was filed with the USPTO on July 22, 2019 and published thereafter on August 19, 2021. The listed applicant is Minovia Therapeutics Ltd. The listed inventors are Natalie Yivgi Ohana, Uriel Halavee, Shmuel Bukshpan, Noa Sher, and Moriya Blumkin.

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