Stem Cells & Cystic Fibrosis: What Are the Possibilities?

My adopted niece was diagnosed with cystic fibrosis (CF) when she was 3 years old. My family and I try to stay up-to-date on any research or new treatment that develops, including the recent studies into stem cell research.

Stem cell research is a highly controversial topic. Stem cell research originally involved working with embryonic stem cells. Although these stem cells were from spare embryos, I was among the population that believed that even though these embryos were created in the laboratory, it was still "killing babies." I still believe that. I am highly against destroying these "babies" for research!

However, the stem cell research being done for CF research uses adult stem cells taken from bone marrow. This process involves very little risk to the donor. This kind of stem cell research is something I can easily live with. I would even volunteer to donate bone marrow, especially if it leads to a better life for my niece.

To begin to understand how stem cells work, you need to know that stem cells are produced in the bone marrow and develop into different cell types. Adult stem cells can be coaxed to copy and grow into a certain type of cell. This may offer a source of replacement cells to treat diseases including the damaged lung cells in CF patients.

CF is believed to be caused by a defective gene, CFTR, which filters the chloride out of the lung cells. The filtering of the chloride is very vital for controlling the liquid in the lungs. Due to this defective gene in CF patients, there is not enough liquid in the lungs, which then causes an excessive amount of mucus to build up. This mucus traps bacteria, causing inflammation and infections in the lungs, which leads to lung damage and eventually lung failure - the main cause of death for CF patients.

With this knowledge, researchers are trying to perfect a method of growing healthy new cells that can be injected into CF patients in the hope that these new cells will become fully functioning cells. So far, the results have been promising. Using ex vitro stem cell trial and error tests, they have been able to duplicate a healthy gene to replace the defective one. When tested, the corrected CF stem cells secreted significantly more chloride. This proves that at least in principle, the CFTR-corrected stem cells work and may one day be a treatment for CF. However, in vitro studies would need to be done, so stem cell transplantation in CF patients could be a long way off.

Maybe one day, CF patients can be treated using their own stem cells which have been given a boost of something to fix the faulty gene. The advantage of using a patient's own stem cells to repair or replace tissue is that there is less chance of the body rejecting the cell transplant.

With enough people getting involved with stem cell research, maybe a cure will be found before time runs out for little Sara and all the other children suffering with CF.