Tissue Implant as Alternative to Gene Therapy

Using a patient's own tissue as the site of production and delivery of precise amounts of protein is a new alternative to gene therapy and other protein injections.

A tiny strip of skin is taken from the patient and then genetically engineered in vitro by the introduction of specific genes via viral vectors. The small bit of tissue is transformed into a "factory”, called Biopump, which will produce a therapeutic protein for the treatment of diseases such as anemia, muscle wasting, hepatitis and other disorders. After being implanted under the skin, this modified tissue begins to deliver a measured supply of protein. Protein production can last for many months, and the therapy can be safely and immediately stopped by simply removing the implant.

"Biopump is intended as an alternative to intravenous protein injections, gene therapy and slow-release protein technologies,” said Dr. Andrew Pearlman, CEO of Medgenics, the developer of Biopump. "Injections are an inefficient way to deliver therapeutic proteins because there is an initial spike in the concentration of the proteins created at the time of intravenous injection, which can result in toxic side effects. Conversely, there is a rapid decline in protein concentration to levels that are suboptimal in therapeutic terms, and the patient must wait until the next injection until the level of therapeutic protein can be restored.”

The shape of the tissue strip is critical to the BioPump method. It has been selected to allow the tissue to live outside of the body for up to several months while it is being altered genetically. It is substantial enough to comprise a viable tissue but sufficiently thin so that all cells are close enough to the support medium to receive nutrients through diffusion. Only minimal medium is needed and there is no use of animal by-products or serum.

"This is a critical advantage over gene therapy, which is an attempt to produce a drug inside the body by injecting a virus with a desired gene that will initiate the production of a certain protein by body cells. However, it cannot be known how much protein will be produced by protein therapy or from which cells, and there is no practical way to halt gene therapy if needed,” explained Dr. Pearlman. "If you want our treatment to stop, you simply pull it out,” said Dr. Pearlman.




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