Monday, July 03, 2017

I thought the Brits had a stem cell treatment for mitochondrial disease.

3 July 2017
By Patrick Greenfield

Pontiff asks that they be allowed to do everything possible to treat their critically ill 10-month-old son.

Pope Francis (click here) is calling for the parents of critically ill baby, Charlie Gard, to be allowed to do everything possible to treat their child.
In a statement on Sunday evening, the Vatican said the pope was following the case of the 10-month-old, who has a form of mitochondrial disease, “with affection and sadness” and was close to his parents: “For this he prays that their wish to accompany and treat their child until the end isn’t neglected.”...

Most people involved with this disease know about the British breakthrough and this goes back long before this article was published. I realize all mitochondrial disease is not the same and there are also ways to identify "carriers" of the disease. I am surprised Charlie Gard is having a problem. I don't pretend to know everything the doctors know.

It is genetics. He was somewhat healthy at birth, but, over time the disease began to cause problems.

July 17, 2015
By Patricia Silvia

A new study recently published (click here) in the renowned journal Nature revealed a breakthrough on combined stem cell and gene therapies for the treatment of mitochondrial diseases. The study is entitled “Metabolic rescue in pluripotent cells from patients with mtDNA disease” and was led by researchers at Oregon Health & Science University.

Mitochondria are small organelles considered to be the powerhouses of the cells where the energy for the body is produced in the form of adenosine triphosphate (ATP). This ATP is crucial for cellular metabolism and proper body functioning. Mitochondrial diseases are rare disorders caused by DNA mutations in mitochondrially-expressed genes. They include a vast range of lethal or severely debilitating conditions. It is estimated that 1,000 to 4,000 children are born with a mitochondrial DNA disease every year in the United States. Treatment options for these diseases are quite limited.

In the study, researchers focused on gene and stem cell therapies for mitochondrial diseases. The team was able to genetically correct pluripotent stem cells (PSCs; cells with the potential to differentiate into several specialized cell types) from patients with mitochondrial DNA disease, namely mitochondrial encephalomyopathy and stroke-like episodes (MELAS) and Leigh syndrome. They collected skin cells from patients with mitochondrial DNA mutations and through a technique based on mitochondrial replacement via nuclear transfer, they generated embryonic stem cells with healthy mitochondria....

Back in 2004, I believe, there was a Virginia mother that took her seven year old daughter to Great Britain for treatment, but, was then investigated by the "W" White House. Remember? There was no such stem cell treatment allowed under "W." Not even receiving it outside the country. This is one of the diseases that can be brought under control and in the case of the seven year old some of the disease processes were reversed with stem cell treatment.

The stem cell therapies are exactly that, regenerative medicine. It does not create life, but, restores it by biological functions that work rather than biological functions that have, for some reason, taken a turn in an unnatural direction.

...The research team believes that this technique represents the start of a new kind of regenerative medicine. “Regenerative technologies offer the prospect of transformative solutions to correct tissue defects in disease. Current care for mitochondrial diseases is limited to addressing patient symptoms, but falls short from providing a definitive cure,” explained the study’s co-author Dr. Andre Terzic from the Center for Regenerative Medicine at Mayo Clinic. “Resetting or replacing disease-corrupted mitochondria to produce healthy patient derived stem cells paves the way towards targeting the root cause of the problem.”

People, like the extremist right to life folks, like to deprive people of hope by stating it is an unnatural treatment and deprives possible infants to be born. Well, here you have their paradox. Charlie Gard is 10 months old and still viable. IF he has a form of treatable genetic disorder with stem cells should he receive it and live OR should the stem cells be preserved for POSSIBLE babies in the future. The ethical and so called religious debate is hideous or better yet ludicrous.. They rather see Charlie suffer and die than give him a chance at life. I am assuming he can be treated. 

Even experimentally treated.