JUN 09, 2017 07:39 AM PDT

Stem Cell Treatment is Both Disappointing and Promising

WRITTEN BY: Carmen Leitch

There is a tremendous amount of interest in using stem cells for the treatment of a wide variety of diseases, and for good reason. It has already proven an effective treatment for some diseases that affect the blood, like leukemia, and shows a lot of promise in regenerative medicine as well. Another area in which researchers have decided to test out stem cells is the treatment of a genetic disease that results in what is called a gain of function mutation. In those instances, a mutated gene creates a protein that has new characteristics and does different things than intended in cells.

This is one of many phenotypes associated with this potentially lethal mutation. / Credit: Satoshi Okada

Success has been seen using this methodology in a single patient, and investigators decided to expand on that work. A trial of patients aged 13 months to 33 years carrying a gain of function mutation in the STAT1 gene was performed after an international call to consortiums and transplant centers. The investigators confirmed the mutation in each patient and traced their affliction to the dysfunction in the gene.

The researchers then used chemotherapy to wipe out the patient’s bone marrow cells. Stem cell cultures obtained from healthy donors were then transplanted into the patients with the hope that their bone marrow would be regenerated without mutation or disease. Of the 15 patients, in the end only six survived the regimen. However, of those six, five were totally cured of their ailments.

This image shows the prognosis of 15 patients at completion of treatment. / Credit: Satoshi Okada

The work was performed by a team that included Dr. Satoshi Okada of Hiroshima University, Professor Jennifer Leiding from the University of Florida, Professor Tomohiro Morio of Tokyo Medical and Dental University, and Professor Troy Torgerson at the University of Washington.

Dr. Satoshi Okada of Hiroshima University was the first to find the gain of function mutation in STAT1 in 2011 and is an author of the report on this trial, which was published in the Journal of Allergy and Clinical Immunology. "Overall, this result is disappointing - but the fact five patients were cured proves that treatment with stem cells can work, and we now need to learn from these 15 individual cases,” he said.

STAT1-GOF can cause a range of problems with varying degrees of severity, and primarily affects the immune system. Because of infections from viral and fungal sources, it can cause life-threatening complications in roughly ten percent of cases.

Good treatments for this disorder have been hard to come by and usually involve just dealing with the symptoms by using anti-fungal drugs. Immunosuppressant treatments are not a good option because the immune system is already overburdened.

The scientists have some hypothesis about the forty percent success rate seen in this new trial of stem cells. It was found that younger patients had a higher rate of success in this study, but whether it’s because they respond better to the therapy or have had the disease for less time is unknown. Following the transplantation, the healthy cells gradually decreased over time, giving the patient’s own bone marrow a chance to regrow. In addition, the types of chemotherapy used on the patients was different and some were so harsh that they did serious damage to the patient organs. Infections were then able to gain a foothold in those organs, and the patients did not recover.

The researchers have suggested improvements; in the future, only patients with the worst cases of STAT1-GOF be selected, and a milder chemotherapy regimen should be utilized. They add, however, that the chemotherapy still must be harsh enough to properly eradicate the diseased marrow. A final suggestion is that selected patients should be as young as possible.

 

Learn about stem cell treatments from the TedxTalk with Timothy Henry, MD, FACC, the Chief of Cardiology at Cedars Sinai Heart Institute in Los Angeles, California.

 

 

Sources: AAAS/Eurekalert! via Hiroshima University, NIH, Journal of Allergy and Clinical Immunology

About the Author
  • Experienced research scientist and technical expert with authorships on 28 peer-reviewed publications, traveler to over 60 countries, published photographer and internationally-exhibited painter, volunteer trained in disaster-response, CPR and DV counseling.
You May Also Like
NOV 03, 2018
Cell & Molecular Biology
NOV 03, 2018
New Insight Into the Regulation of Sleep
Once thought of as supporting structures, researchers are uncovering more roles for astrocytes, a type of cell in the brain with a shape like a star....
NOV 05, 2018
Neuroscience
NOV 05, 2018
How does Brain's GPS work?
Understanding the brain cells involved in decoding and encoding naviagtional information....
NOV 05, 2018
Genetics & Genomics
NOV 05, 2018
Trying to Repair Age-related Gut Damage
As we age, our intestinal lining gets more permeable; sometimes called leaky gut, and it can wreak havoc on our bodies....
NOV 22, 2018
Cell & Molecular Biology
NOV 22, 2018
Stopping Cancer Cells in Their Tracks
Researchers at the University of Minnesota have developed a technique to stop the movement of cancer cells....
NOV 26, 2018
Neuroscience
NOV 26, 2018
Ventromedial Prefrontal Cortex Central For Imagination Mitigated Fear
Extinction; presenting fear in a neutral setting where the negative consequences are negated is one of the successful fear conditioning mechanisms...
DEC 04, 2018
Genetics & Genomics
DEC 04, 2018
The Fallout From the First CRISPR Babies Continues
Last week the world was stunned to hear that twin babies had been born after a gene-editing experiment was conducted on human embryos....
Loading Comments...