Second Chance at Life for Adult Stem Cell Transplant Patient

by David Prentice

An update on the City of Hope’s 10,000th bone marrow adult stem cell transplant. The patient, now identified as 51-year-old William Fuller, was released from the hospital last week. The father of three, a small-business owner, was born in Belize and came to the U.S. in 1982. When he had his adult stem cell transplant on Jan. 13, 2011, his nurse wished him “Happy Birthday,” signaling the beginning of his new life.

According to Dr. Stephen J. Forman at City of Hope:

“Mr. Fuller is the poster child for what we do. There are thousands of other people like him who have been helped because a donor came forward to provide lifesaving stem cells that allowed us to do a transplant and hopefully cure the disease. Every patient who gets through a transplant here is the beneficiary of a lot of laboratory work and hard thinking that’s gone into trying to solve the problem – how to best cure the cancer in the safest way possible.”

Dr. Forman noted many patients view their adult stem cell donors as new members of their family, and often develop lifelong relationships. “They are ‘blood relatives,’” he said.

Mr. Fuller credited his sister, Karen Hyde, as being instrumental in arranging bone marrow drives in California, Florida and New York with the help of “Be the Match,” the national marrow donor program.

Adult stem cells continue to save thousands of lives every year.

Making Buckets of Adult Stem Cells

by David Prentice

Growing lots of adult stem cells in the lab, for study or for a patient treatment, has been difficult in the past.

While some groups have successfully grown large numbers of adult stem cells, many labs have difficulties keeping the cells growing for more than a few days. Now scientists at Weill Cornell have shown that culturing adult stem cells with endothelial cells, the cells that compose the innermost linings of blood vessels, is the key to growing unlimited amounts of adult stem cells.

The research group reasoned that because endothelial cells line blood vessels and are often in contact with adult stem cells, these cells might play a significant role in the growth and maintenance of stem cells. Using a mouse model, the scientists were able to grow adult stem cells for weeks at a time and increase the number of cells over 400-fold. They also showed that even after one year, there was no indication of tumor formation from the adult stem cells. Senior author, Dr. Shahin Rafii, noted:

“This study will have a major impact on the treatment of any blood-related disorder that requires a stem cell transplant.”

Previous work from Dr. Rafii’s lab had demonstrated that endothelial cells are not “passive conduits” for delivery of oxygen and nutrients but also produce novel stem-cell-active growth factors.

The breakthrough promises broad clinical benefits, from bone marrow transplantation to therapies for heart, brain, skin and lungs. If the system continues to be validated, physicians could use any source of hematopoietic (blood-forming) stem cells, grow large numbers, and bank the adult stem cells for transplantation into patients.

The paper is published in the journal Cell Stem Cell.

Scientists Create Heart Cells from Skin Cells

( Israeli scientists have discovered a way to create beating heart cells using human skin cells reprogrammed to become stem cells. The findings could lead to advances in disease research, and could in theory be used to repair damaged or diseased tissues.

Lior Gepstein photo

Published in the latest issue of Circulation, the findings by Professor Lior Gepstein of the Technion-Israel Institute of Technology could make it possible to clinically repair damaged human hearts.

Such an application is at least 10 to 20 years away, says Gepstein, but the process can already be utilized for in-depth study of genetic diseases and the development of personalized drugs for irregular heartbeats and other inherited disorders.

Transforming our cells through reprogramming

The team’s work is based on the research of Japanese scientists followed by other groups, who generated “induced pluripotent stem cells” (iPSCs) from adult mouse and human skin cells. The iPSCs can be turned into almost any type of body cell – something that experts previously thought possible only with embryonic stem cells – and could, in theory, be used to repair damaged or diseased tissues.

Taking a patient’s own cells and turning them into iPSCs for use in tissue repair and regeneration would also eliminate the risk of rejection by the body.

Gepstein and his team from Technion’s Rappaport Faculty of Medicine and Rambam Medical Center used reprogrammed iPSCs derived from healthy human subjects’ skin cells with the characteristics of pluripotent embryonic stem cells. They were then able to convert them into heart cells with all the necessary properties such as expression of heart-related genes, spontaneous electrical activity, mechanical contraction, and response to various hormones such as adrenaline.

According to Gepstein, the rejuvenation of human cells and their transformation into iPSCs can be accomplished with almost any human cell.

Making heart headlines

Nearly eight years ago, Gepstein and colleagues made headlines by creating beating cardiac tissue in the lab from human embryonic stem cells. In 2007, he teamed with the Technion’s Dr. Shulamit Levenberg to create tiny blood vessels within the tissue. This breakthrough could eventually make it possible to implant the tissue in a diseased human heart.

The findings could also someday lead to advances in research on diseases caused by single-gene mutations. The list of these diseases includes familial arrhythmogenic syndromes leading to irregular heartbeat and sudden cardiac death, cardiomyopathies that weaken the heart muscle, and several neurodegenerative disorders.

Certain challenges exist, however. One hazard of using iPSCs as well as ordinary embryonic stem cells is the possibility that the cells will begin to divide wildly and turn cancerous. As a result, “it will be years before they are used clinically,” says Gepstein. While animal studies could eventually lead to clinical work, scientists would first have to learn how to make large amounts of the iPSC-derived heart cells, he concludes.