“New research has nudged scientist’s closer to one of regenerative medicine’s holy grails: the ability to create customized human stem cells capable of forming blood that would be safe for patients.”
In a recent article published in the LA Times entitled, ‘A Window on A Wellspring of Life’ by Melissa Healy, Healy touches on the topic of how blood stem cells could be modified to directly treat blood diseases such as cancer, as well as gain understanding of the specific genetic architecture of such blood diseases. Scientists are researching ways to allow patient’s own blood stem cells to be reprogrammed to fight diseases and in turn allowing for patient graft transplants to take, reducing the risk of complications.
Though the use of stem cells in medicine has been a common practice since 1950, the current approach leaves much to be desired. Currently, patients suffering from blood cancers, are put through a combination of chemotherapy and radiation to fight the disease. The results leave patients with a weakened immune system and often in need of a bone-marrow transplant. When patients undergo a graft transplant, there is a high chance of the patient developing “graft-vs-host disease”, in which the bone marrow from the host, contains blood stem cells that can include the host’s genetic signature and in turn the recipient’s immune system rejects the transplant, resulting in a fatality.
As science advances, researchers are looking to change this current approach by customizing stem cells.
“Rather than hunt for a donor who’s a perfect match for a patient in need of a transplant — a process that can be lengthy, ethically fraught and ultimately unsuccessful — doctors would like to use a patient’s own cells to engineer the hematopoietic stem cells.”
This advancement in science toward understanding blood stem cells, along with the ability to reprogram the blood cells, allows scientists to gain insight into how blood cancers develop as well as develop specific treatments to combat. The advancement of being able to reprogram blood stem cells would also eliminate the rate of rejection found in transplant recipients. Reducing or eliminating the risk of “graft-vs-host disease”, could allow researchers to pursue related-medical advancements in other blood diseases such as Sickle Cell.
Healy continues that, “The patient’s mature cells would be ‘reprogrammed’ to their most primitive form: stem cells capable of becoming virtually any kind of human cell. Then factors in their environment would coax them to become the specific type of stem cells capable of giving rise to blood.” Using research found by Dr. George Q. Daley of Harvard Medical School and The Dana-Farber Cancer Institute in Boston, which included turning “pluripotent stem cells”, primitive cells that can be turned into any type of mature cell, into “endothelial cells”, which are blood cells that are located inside specific blood vessels. Daley and his team were able to achieve this by creating a specific genetic protein mixture and adding it to the pluripotent stem cells during the programming process.
The ability for scientists to be able to program blood stem cells to allow patients to fight diseases by using their own blood stem cells; allows for many medical advancements not just in fighting blood diseases but in understanding how blood diseases begin and evolve. “’From a research point of view, you could now actually begin to model diseases,’ Greenberger said. ‘If you were to take the cell that’s defective and make it revert to a stem cell, you could effectively reproduce the disease and watch its progression from the earliest stages.’ That, in turn, would make it easier to narrow the search for drugs that could disrupt that disease process early. And it would speed the process of discovering which genes are implicated in causing diseases.”
As stem cell research continues, the advancement of science and medical benefits continue to be unearthed.
“But Daley cautioned that significant hurdles remain before studies like these will transform the treatment of blood diseases.”
The ability to maintain and achieve reprogrammable blood stem cells in humans requires more research and trials, but as scientist continue, new advancements are being contemplated and discovered to address, treat and prevent blood diseases using stem cells.