Overcoming Obstacles to Stem Cell Use

Researchers from the University of California, San Francisco, recently overcame the obstacles that have prevented the success of stem cell efforts in the past.

Initial test results show the design of a universal type of stem cell, meaning stem cells did not need to be collected from the individual needing them. Instead, stem cells were designed which could be used in any individual.

These new stem cells were able to be developed into the type of cell desired. For example, in the test case, the stem cells successfully turned into heart tissue.

The researchers published their ideas and results in the journal Nature Biotechnology.

Historically, there have been two fundamental obstacles to effective stem cell therapy.

  1. A particularly frustrating obstacle is the fact that even stem cells collected from the particular individual needing a transplant, known as induced pluripotent stem cells, often face rejection. In the words of Dr. Tobias Deuse, who led the current research effort, “There are many issues with induced pluripotent stem cells technology, but the biggest hurdles are quality control and reproducibility. We don’t know what makes some cells amenable to reprogramming, but most scientists agree it can’t yet be reliably done. […] Most approaches to individualized induced pluripotent stem cell therapies have been abandoned because of this.”
  2. Even when a rejection response is not triggered, new cells become targets of a specific set of immune cells called natural killer (NK) cells.

Using advanced gene editing techniques, the researchers modified several genes in stem cells to protect them both from rejection and from NK cells. More specifically, the researchers deleted two specific genes in their stem cells. These genes cause particular proteins to send a signal to the immune system, a signal that can begin the process of rejection. They also enhanced a particular surface protein, known as CD47, in order to block the activity of the NK cells.

After making these modifications, the scientists engineered a group of mice to have human immune system elements that were likely to cause a negative immune system response. Nevertheless, the altered stem cells were successfully transplanted into the mice without rejection or destruction.

The researchers’ final step was to induce their new stem cells to transform into the specific cells they desired. In the case reported in their paper, the new stem cells were transplanted into mice with human-like immune systems, and successfully transformed into heart cells, including heart muscle tissue and essential heart blood vessels.

Again quoting Dr. Deuse, “Our technique can benefit a wider range of people with production costs that are far lower than any individualized approach. We only need to manufacture our cells one time, and we’re left with a product that can applied universally.”

This advance may indeed be the breakthrough that we’ve been waiting for, a breakthrough that would negate the difficulties of organ transplants: the shortage of organs needed, the difficulty in finding a suitable match, the likelihood of rejection or destruction, and the need to take immunosuppressant medications.

Laurel Bay Health and Rehabilitation Center, in the scenic beach town of Keansburg, NJ, specializes in pulmonary care, and welcomes the day when stem cells can be used to help those with lung diseases. In the meantime, our program, headed by leading pulmonary specialist Dr. Avtar Parhar, is the most highly advanced pulmonary rehabilitation program in Monmouth County. At Laurel Bay, we focus on restorative and preventive care for those who suffer with chronic respiratory disease.

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