On 16 November, the US Food and Drug Administration (FDA) issued new guidelines to encourage growth in the emerging field of stem cell therapy (SCT) – and simultaneously, protect patients from dangerous products used by unregulated clinics.

The carefully crafted guidelines define the narrow boundaries between allowing or hastening the approval of potentially life-changing cellular therapies, and preventing providers from selling unproven and possibly dangerous cell products directly to patients.

The stem cell community – given the rapid and unchecked growth of therapies – has eagerly anticipated the announcement of these guidelines. Clinics around America have been carelessly promoting SCT to treat all sorts of conditions, from autism to erectile dysfunction (ED).

The criteria set out by the agency, states that SCTs entering the drug approval process are only allowed if all the cells or tissues come from the patient being treated; they are used only for the same function they perform naturally in the body; and they must be only “minimally manipulated.” Only under these conditions, the FDA believes, are the products considered low-risk.

Proven promises of stem cell therapies

The bright spots in this human eye scan are signs of macular degeneration. Photo credit: National Eye Institute
The bright spots in this human eye scan are signs of macular degeneration. Photo credit: National Eye Institute

Scientists fear new regulations would limit the potential of SCTs.

Two clinical trials testing the use of ESC in treating the dry form of macular degeneration, have been successful. One was conducted by Hadassah-Hebrew University Medical Centre in Jerusalem, and the other, in the University of Miami’s Bascom Palmer Eye Institute. In both trials, researchers injected ESC behind the human retina.

The ESC transformed into retinal pigment epithelial cells, which support and carry nutrition to the retina and remove waste. This prevented degeneration of the cells, and even improved patients’ vision.

In both trials, there were no adverse side effects and the transplanted cells seemed to have survived. The cells were also almost identical to the ones found in the eye. The transplant site also had a minimal risk of the immune system rejecting the implants.

European regulations still a grey area

Many scientists believe that traditional methods of regulation should not apply to SCTs, since they were too expensive and time-consuming, hindering promising progress. But, the technology demonstrates potential to create humans from somatic stem cells (SSC), All European Academies (ALLEA) warned. This means that limitations can still apply, with European courts hampering the patenting of such innovations - where despite allowing stem cells to be used in research, it cannot be commercialised.

This is a position that was “absolutely inconsistent” and “not very encouraging” for European science, said Joseph Straus, an intellectual property expert and chair of ALLEA’s working group on IP rights.

There is “certainly good evidence that human pluripotent stem cells can replicate some aspects of early embryonic development in a culture dish,” says Martin Pera, a professor at the Jackson Laboratory, a biomedical research centre in US.

But, Julian Hitchcock, a life sciences specialist at the law firm Marriott Harrison assures it is “extremely unlikely” that scientists could “just leave them [the stem cells] and they turn into a human.”

They would not have the “inherent capacity” do so themselves. He did however, note that, “the nature of science is that nothing is certain” and was always “contingent on the next experiment.”

The issue may always be ethically fraught

Scientists have already produced mouse clones from induced pluripotent stem cells (IPSC).
Scientists have already produced mouse clones from induced pluripotent stem cells (IPSC).

The possibility of creating – and then destroying life – either with SSC or embryonic stem cells (ESC) is the biggest debate of SCT. Arguments of what stage a group of cells is considered life, whether it is moral to ‘play God’ in this way and the value of human life are very dividing.

Research has also showed that deriving sperm and eggs from skin cells are possible, making reproduction at any age, using tissue from living or dead individuals possible. Already scientists have produced mouse clones from induced pluripotent stem cells (IPSC).

This presents more pressing concerns on who has control over donated IPSC obtained from donated tissue. Already, biobanks full of donated tissue and data for research exist globally and in many cases, consent is not needed if the material cannot be traced back to the donor.

In the future, it is expected that consensus will be needed but according to surveys, there is much disagreement on whether consent will be needed each time tissue is used or whether blanket consent should suffice. In addition, what are the rules should a donor wish to withdraw their samples from research; and should donors be given a share of the commercial potential of their tissues as disease models or drug testing platforms?

“The most vulnerable resource,” said Jan Helge Solbakk, head of research at the Centre for Medical Ethics at the University of Oslo, “is trust.” MIMS

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