Mitochondrial Transplants from Mothers: A Controversial idea Evolves into a Potential Remedy for Mitochondrial Disease

 

Ten years ago, a biochemist was seeking investors for her new company that was developing mitochondrial-based medicines. The Israeli-born embryologist observed how the blending of mitochondria from one egg with another egg had the potential to prevent mitochondrial diseases. The technique is called three-parent IVF.

According to a recent article in StatNews, by turning nutrients and oxygen into chemical energy mitochrondria power the cells in our bodies, these organelles are responsible for metabolizing cholesterol and synthesizing neurotransmitters and hormones.

When Things Go Wrong

Deletions of human mitochondrial DNA are usually the cause of mitochondria diseases such as muscle weakness, cardiac and gastrointestinal issues, and premature death.

A child is born every thirty minutes with the potential to develop a mitochondrial disorder by age ten. One in every 4,300 people in the U.S. has a mitochondrial disorder which is a long-term, sometimes inherited, genetic disorder.

Children normally inherit one gene from the mother and one from the father. A child with a mitochondrial disease receives an abnormal (mutated) gene, usually from the mother. Mitochondrial diseases occur when the mitochondria do not produce sufficient energy and the body does not function properly.

In 2012, Natalie Yivgi-Ohana Ph.D. co-founded Minovia which develops mitochondria augmentation therapies for rare mitochondrial disorders. Professor Yivgi-Ohana acknowledges that the idea was controversial ten years ago.

Within the past seven years, Israeli researchers and doctors began to work on a possible treatment that utilizes mitochondria’s capacity to permeate cellular life forms. The patient’s hematopoietic stem cells, which form mature blood cells, are augmented with healthy mitochondria that are donated by the child’s mother.

The Israeli group reported in the journal Science Translational Medicine that the treatment administered to six children, not only proved to be safe but resulted in small yet significant improvements in the children’s symptoms.

After treatment, a six-year-old boy who had not been strong enough to walk unaided began running like a normal child his age.

A young girl who was bedridden and unable to sit up became strong enough after treatment to eat properly. She was able to stand up on her own for the first time.

Dr. Elad Jacoby, a pediatric hematologist in Tel Aviv, led the study. Dr. Jacoby noted that measures such as these are difficult to quantify, yet the team did observe improvement in all six children. A clinical trial is expected later in the year.

Dr. Jacoby further explained that mitochondrial transplantation has the potential to treat many rare genetic disorders and other conditions resulting from mitochondrial damage.

 

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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