Is it drug manufacturing or the practice of medicine? Stem cell therapy debate just getting started

Aug 03, 2012   

By: Stephanie Baum
August 3, 2012

The latest round of a stem cell debate may have concluded, but as far as the lawyers representing an orthopedics company that has offered stem cell treatments are concerned, it’s a long way from over.

The lawyers representing Colorado-based Regenerative Science said they were filing a notice of appeal this week after a U.S. District Court judge’s ruling in favor of the U.S. Food and Drug Administration that its stem cell therapy is a drug. The case is being closely watched by everyone with a stake or an interest in the use of stem cells in medicine and could have significant ramifications for stem cell entrepreneurs who don’t believe their procedures amount to a drug or biologic.

Regenerative Science

Andrew Ittleman of Fuerst Ittleman is part of the legal team representing Regenerative Science. Although he acknowledged that the company has had to suspend its cell culture process, he said the ruling doesn’t really change anything in the big picture of stem cell policy. “If anything, [the decision] preserves the status quo, but the problem with that is that there’s so much uncertainty regarding what doctors can do and can’t do.”

Colorado-based Regnerative Science’s Regenexx procedure is presented as an alternative to traditional surgery that can treat fractures that have failed to heal, joint cartilage problems, partial tears of tendons, muscles, or ligaments, among other problems. It works like this: A physician takes a small bone marrow sample from the back of the patient’s hip through a needle. Blood samples are taken from a vein in the patient’s arm. The samples are sent to the Regenerative laboratory where the mesenchymal stem cells are isolated from the bone marrow and grown to greater numbers using growth factors in the patient’s blood. The stem cells are injected back into the relevant area in the patient.

The FDA initially sent a warning letter to Regenerative in 2008 after seeing its website. The company turned around and sued the FDA, and two years ago the FDA sought an injunction to shut it down.

The companies that occupy the stem cell treatment landscape are as diverse a group as you could hope to find in medicine. Some offer age-defying beauty solutions, others provide life-saving technology, others have quality-of-life-improving treatments for joints and muscles. There are entrepreneurs leading startups and Big Pharma companies too.

In 2010, the stem cell therapy market was $139.6 million and it was projected to grow to $1 billion by 2015, according to data compiled by Robin Young, the publisher of Orthopedics Week and the CEO of medical data mining company PearlDiver Technologies.

“This is on the very cutting edge of medicine and the FDA needs to move forward with it by making sure they have the best information on this science available to inform their decision-making,” said Michelle Hart Yeary, counsel with Dechert’s life science practice.

Minimally manipulated

The source for the debate comes from the regulatory framework the FDA set up in 2001 in light of the development of research and medical treatments using human cells, tissues and cellular or tissue-based products (human cell or tissue products) “to improve protection of the public health without imposing unnecessary restrictions on research, development or the availability of new products,” according to court documents. One point in this framework is that human cells or tissue can only be minimally manipulated. Minimal manipulation is defined as “processing that does not alter the relevant biological characteristics of cells or tissues.”

“There are lots of doctors out there using stem cell treatments,” said Arnie Friede of Arnie I Friede & Associates, a New York-based food and drug lawyer who has served as an associate chief counsel in the FDA chief counsel’s office. “I think you have to distinguish between people in traditional drug development and others engaged in different procedures that amount to the practice of medicine.”

Yeary says the regulator should evaluate cases on an individual basis. “Given the ‘minimally manipulate’ standard hasn’t been fully defined by the FDA or fully interpreted by the courts, I think decisions will need to be made on a case-by-case basis. This follows from the evolving state of the law.”

The case for stem cell treatments as drug manufacturing

Drug manufacturers that have invested millions in ensuring their facilities meet good manufacturing practices meeting FDA requirements and carrying out clinical trials to prove the efficacy and safety of their products have welcomed the ruling.

Christopher Scott, the director of Stanford University’s stem cell program for its center for biomedical ethics, told New Scientist he hoped it would lead to more FDA investigations of unproven stem cell treatments at other clinics.

Fibrocell is developing a platform technology to produce mesenchymal stem cells that are being used in research to develop osteoblasts, or bone cells; chondrocytes, or cartilage cells; and adipocytes, or fat cells. David Pernock also said he welcomed the judge’s decision.

Juventas Therapeutics recently raised $22 million for two phase 2 trials for chronic heart failure and for critical limb ischemia testing its stem cell treatment, JVS-100, which uses a protein naturally produced by the heart (stromal cell-derived factor 1) that attracts stem cells to damaged tissue, keeps cells from dying and restores blood flow. Medical device company Histogenics Corp. is raising $49 million in part to get CE Mark approval for its VeriCart  product — a single-step, cell-free collagen scaffold  designed to be used with the patient’s own stem cells to repair small cartilage defects associated with knee injuries.

If Regenerative Science ultimately loses, it could lead to a significant decrease in the number of stem cell companies unprepared to fork out more for the years of development needed to bring a drug or biologic to the FDA approval finish line. Perhaps having members of the stem cell industry work with the FDA to hammer out some more coherent regulations could better fill the perceived gaps between the medical industry and drug and medical device companies.

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