Shinya Yamanaka---Embryo-like stem cells enter first human trial--- Enjoy the future Medicine

It will be the first clinical study to put induced pluripotent stem (iPS) cells into humans — and where more fitting than in Japan, where Shinya Yamanaka garnered a Nobel prize last December for showing how to take bodily cells and return them to an embryo-like pluripotent state.

STEM CELL RESEARCH 

Masayo Takahashi of the Center for Developmental Biology in Kobe just cleared the second and, observers say, most difficult hurdle in starting her iPS cell trial to treat age-related macular degeneration, a condition that affects the retina and can lead to blindness.

On Wednesday an institutional review board (IRB) at the Institute for Biomedical Research and Innovation (IBRI), which is going to sponsor the trial, gave conditional approval. The team needs now only to notify the IRB of the final results of some preclinical safety trials now underway (see story in Japanese).

Having already received IRB approval at her home institution, Takahashi can now move towards the final step before patient recruitment: getting health ministry approval. She’s expected receive that in time for starting the trials during this fiscal year, ending March 2014.

The trial will enrol six patients older than 50 years of age. The researchers will remove damaged pigment epithelium and then implant a small sheet of new epithelium. The sheet will be created by coaxing iPS cells, created from the patient’s body cells, to become epithelium cells.

Takahashi says that apart from a small risk of damage from the surgical procedure itself (sticking a needle in the eye) and a much smaller risk of the stem cells themselves going awry, preclinical studies suggest the procedure to be safe.

The clinical trial, meant to demonstrate the safety of the procedure in humans, is not expected to reverse the damage of macular degeneration, but researchers hope that it will at least slow its progress.

Nobel Prize in Adult Stem cell Research

Adult Stem Cell Researchers Win Nobel 

"We can’t keep destroying embryos for our research," said Shinya Yamanaka of his motivation.

The Nobel Assembly has awarded the 2012 Nobel Prize in Physiology or Medicine to John B. Gurdon and Shinya Yamanaka, researchers whodiscovered a way to develop stem cells from adult skin cells—thus removing the need to use and destroy human embryos.

The Nobel Assembly states that Gurdon and Yamanaka's discoveryrevolutionizes "the dogma" that stem cells could only be derived from immature cells, such as those of human embryos.

The pair jointly received the prize for their "discovery that mature cells can be reprogrammed to become pluripotent." These pluripotent cells are able to develop into any tissue of the human body.

Embryonic stem-cell research "has long been controversial—which is one reason why Yamanaka's discovery of an alternate way to obtain human stem cells, without the use of embryos, is so important."

Pro-life groups such as National Right to Life are trumpeting the award, lauding the discovery as the "ethically acceptable (and far more promising) alternative to harvesting stem cells from human embryos."

But the "paradigm-shifting discovery" is almost 40 years in the making. In 1962, Gurdon, a British researcher, discovered that mature "specialized" cells can reverse themselves, still containing all the necessary information to develop all immature cells. In 2006, Yamanaka, a senior investigator at the Gladstone Institutes, reprogrammed the first adult cells to become immature human stem cells.

In 2007, Yamanaka told the New York Times he was inspired to pursue alternatives to embryonic stem-cell research when he was invited to view a stored human embryo during a visit to a friend's fertility clinic.

“I thought, we can’t keep destroying embryos for our research," he said. "There must be another way.”

CT previously noted Yamanaka's discovery in 2008, and mentioned his work in coverage of President Barack Obama's decision not to fund embryonic stem cell research in 2009.