Stem cells are special types of cells that have the ability to renew themselves and develop into different types of tissues. There are a variety of different types of stem cells but the general concept is that we all have a small reservoir of these cells in our bodies. These cells allow us to repair and replenish the cells that make up the different tissues in our body. For example, bone marrow stem cells allow our bone marrow to keep producing all the cells in our blood. Mesenchymal stem cells reside in the different tissues and help repair and replace damaged cells and tissues. Stem cells can give rise to progenitor cells. These cells have lost the ability to self-renew and are only able to develop into certain types of tissues.
The PHACet Study
To date we have had only a single study completed using stem cells to treat PAH. The PHACet Study included 7 patients who were given three infusions of stem cells into their heart (right atrium). This study did not look at long term changes in pulmonary artery pressure or right heart function and there was no control (or comparison) group. They were able to show increased six-minute walk distance at six months compared to the start of the study. One of the seven patients died shortly after receiving the stem cell infusion.
Cedars-Sinai in Los Angles has started a phase 1 clinical trial using stem cells derived from heart tissue. Dr. Marban who is the head of Cedars-Sinai’s Heart Institute developed the technology to create the stem cells and has licensed it to Capricor who is sponsoring the study.
Phase 1 studies are the very earliest clinical trials in humans. These early studies are not looking at efficacy but rather focused on whether the intervention is safe. Remember that there is no guarantee that stem cell therapy will be safe or effective. The mortality rate in the PHACet study was 14% (1/7 patients died during the study). We are very excited about this phase 1 trial but the journey from a phase 1 study to a proven effective therapy takes many years and the odds are long that any one therapy will become a successful approved treatment.
Beware of Scams
In contrast to the Cedars-Sinai study there are many scammers in the community preying on desperate PAH patients. Buying a “stem cell” infusion for a discount price of $10-15,000 is not a great deal. You are gambling with your health. The FDA has issued strong warnings against using these fake treatments. Not only are they not effective, they are potentially dangerous. In America, real medical treatments have to go through a long process showing efficacy and safety. Remember too that you will never be asked to pay to participate in a research trial.
Stay tuned for more information about Stem Cells and PAH.