FDA Approves Australian Spinal Disease Adult Stem Cell Trial

Australian Associated Press, December 20, 2006

A world-first Australian medical therapy that uses stem cells to treat degenerative spinal disease has been approved for testing on patients in the U.S.

Using the treatment to replace painful bone grafts is the goal and hope of researchers.

The U.S. government awarded approval to an Australian biotechnology company for its adult stem cell treatment to be used in a major trial.

The U.S. Food and Drug Administration allowed it to bypass the small phase-one human safety trial and move directly into a larger phase-two trial since the preliminary animal trials were so promising.

This marks the first time that adult stem cells will be used to treat spinal disc disease in a genetically unrelated patient “off-the-shelf”.

“In 40 patients who suffer severely from degenerative disc disease, the stem cells will be used to create a spinal fusion,” said mesoblast chief scientific adviser Professor Silviu.

Spinal fusion is a procedure in which two vertebrae are fused together to prevent them from moving and causing pain.

Researchers expect the stem cells will grow into a bony bridge between a patient's vertebrae.

A section of the patient's hip bone is grafted onto their spine in the current spinal fusion treatment known as an autograph. The trial will establish if the technology is as safe and effective as this presently favored treatment.

Professor Itescu said the existing treatment could lead to long-term pain at the site of the procedure, a high rate of complications such as infection, and of course required a separate operation.

"If we can show that our cells give a spinal fusion that is equivalent to an autograph, we've got an off-the-shelf product that prevents the need for a second procedure," Professor Itescu said.

He estimated about 15,000 of the 300,000 patients who undergo spinal infusion each year in the United States, have their procedures conducted in Australia.

Using donor cells that are genetically different from the patient, the technology is unique.

The company uses a rare patented form of adult stem cells that are not recognized by the immune system, thus eliminating the problem of rejection.

Before the product can be sold commercially, the trial must be successful and the company then has to run an even larger phase-three trial.

Hopefully, patients with early-stage could have their discs regenerated with the same stem cells to avoid the need for spinal fusion.

"I don't think it's that long in the future, we are talking the next couple of years," Professor Itescu said.

"If it works it will change the paradigm of treating these chronic degenerative conditions."