Two patients receiving embryonic stem cells for their blinding retinal diseases reported improvement in their vision. The results were released just last week online in the medical journal Lancet.
Each patient received retinal epithelial pigment cells (aka RPE cells) grown from human embryonic stem cells. RPE cells are the faulty cells in macular diseases such as ARMD and Stargardt’s Disease. In healthy patients, these cells are normal and function appropriately.
Dysfunctional, sick or degenerated retinal epithelial cells lead to progressive loss of vision. As the healthy RPE cells begin to dwindle, so does the vision. The RPE cells are a key component to translating light to vision.
Researchers have been hopeful to replace sick RPE cells with the healthy replacements derived from the stem cells.
Hence, the excitement. Two individuals, one with dry ARMD and the other with Stargardt’s underwent RPE cell transplantation and the reports at 4 months after the operation are encouraging. Both noted improvement and, according to the report, no complications occurred either to the eye or to the patient.
Both patients underwent surgery at UCLA. The research was funded by Advanced Cell Technology, the same company conducting stem cell research on patients with Stargardt’s Disease.
What Does This Mean?
At most, this is encouraging news. This does NOT mean that stem cell transplantation works.
There are lots of flaws; only 2 patients involved, inability to objectively measure improvement in vision, funding company (Advanced Cell Technology) is involved and adds bias to the study results, etc.
On the other hand, I am very excited that the research seems to be moving forward. Yes, the results are tainted, but it does demonstrate several “successes” in solving some of the technical challenges of stem cell research;
- This is pioneering work. Regardless of visual outcomes, researchers have designed techniques to deliver the RPE cells to the correct anatomic location. That is, retinal surgeons have figured out how to transplant the cells effectively.
- The cells may indeed remain “alive” when transplanted. How long? We don’t know, but it seems to be at least 4 months!
- This is great news in the face of recent failures of stem cells used in spinal cord injury.
- There were apparently no complications.
This certainly is enlightening and exciting. With time, we will need more extensive research and objective data. This is no different than a new “drug” fighting for FDA approval.
There is still many questions to answer the ultimate questions; Is the technique safe? Is the technique effective?