First Clinical Trial Using Embryonic Stem Cells to Treat Dry AMD

This fall, the world’s first clinical trials of the use of cells derived from human embryonic stem cells began. Who needs it and why? The topic of the use of stem cells in medicine has long been boring for everyone, even the yellow newspapers no longer write about it. There is no longer any spam about rejuvenation, and for most serious journalists, stem cells are discredited. It’s funny, but at this moment scientists can provide real ground for exaggerated sensations and exaggerations. The fact is that in the fall of this year, two American biotechnological companies for the first time in the world were allowed to conduct clinical trials on the use of cells obtained from human embryonic stem cells (ESC). Why are ESCs remarkable for medicine? They can live almost indefinitely in a laboratory and theoretically act as a factory for all types of cells in the human body.

On September 22 this year, Northwestern University announced the beginning of the first clinical trials on the use of human ESC derivatives in medicine. Oligodendrocyte precursors will be injected in patients with severe thoracic spinal cord injuries. Once in the human body a week after the injury, they must help the nerve impulse pass through the spinal cord. Typically, in humans, oligodendrocytes play the role of “isolation” for the processes of nerve cells in the brain and spinal cord. They protect and are responsible for the rapid conduction of the impulse. In the case of severe spinal cord injuries, one of the paralysis factors is the violation of such “isolation”. It is logical to assume that if the precursors of these “auxiliary” cells are introduced into the spinal cord, the grown oligodendrocytes will close the “holes” in isolation. An endless source of these predecessors is ESCs. Anything can be grown from ESCs.

The technique was tested on rats back in 2005, but the launch of clinical trials in humans was delayed. First of all, because to launch you need to obtain permission from the US Food and Drug Administration (FDA), and this is very difficult. It is necessary to prove that the treatment will be successful, and, most importantly, safe, and the American government is constantly changing its opinion on this matter. The first launch of the project was announced at the end of January 2009, but the FDA froze clinical tests due to the appearance of seals at the site of the introduction of cells. It took a year and a half to prove that, at least in the case of rats, these cysts are harmless and do not degenerate into tumors. Although the first phase of clinical trials has now begun, these progenitor cells are far from ideal treatments. First, there is still a statistically small percentage of possible neoplasms, but the most annoying thing is that these cells, like any conventional transplant, can be rejected.

On November 22, another biotech giant received FDA approval to conduct clinical trials for the treatment of Stargardt’s Macular Dystrophy (SMD), a hereditary macular degeneration (destruction) of the macula. On November 30, they announced the beginning of clinical trials for the treatment of dry AMD (age-related macular degeneration) – one of the most common age-related eye diseases in the world. In both cases, it is proposed to surgically implant the cells of the pigment epithelium of the eye obtained from ESCs. The stumbling block remains the same – ethical issues, safety, efficiency.

The results are still long to come, and it is possible that the danger and complexity of the technology will be too high to be applied in practice. Again, embryonic stem cells, as an immortal source of all types of cells, are already crowding out iPS cells, their artificially created counterparts, which not only remove ethical questions but are also individual for each patient. We will talk about them in the following articles, but today, it seems to us, it is important that doctors have ceased to be afraid of the words “derivatives of human embryonic stem cells”, and began to look for approaches to the use of these promising cells in medicine.