First Embryonic Stem-Cell Trial Gets Approval From the FDA

Daily News Article   —   Posted on January 27, 2009

NOTE: The article below was first published in The Wall Street Journal on Jan. 23rd.

(by Ron Winslow & Alicia Mundy, WSJ.com) – In a watershed moment for one of the most contentious areas of science and American politics, the U.S. Food and Drug Administration cleared the way for the first-ever human trial of a medical treatment derived from embryonic stem cells.

Geron Corp., a Menlo Park, Calif., biotechnology company, is expected to announce Friday that it received a green light from the agency to mount a study of its [embryonic] stem-cell treatment for spinal cord injuries in up to 10 patients. The announcement caps more than a decade of advances in the company’s labs and comes on the cusp of a widely expected shift in U.S. policy toward support of embryonic stem-cell research after years of official opposition.

“This is the dawn of a new era in medical therapeutics,” said Thomas B. Okarma, Geron’s president and chief executive officer. The hope that [embryonic] stem-cell therapy will repair and regenerate diseased organs and tissue “goes beyond what pills and scalpels can ever do.”

Limits on [embryonic] stem-cell research, which prevented federal funding and were imposed by Congress and former President George W. Bush for ethical and religious reasons, have had a chilling effect on both academic and corporate research involving such cells. Proponents of stem-cell research say restrictions have delayed development of promising new treatments, while critics contend that harvesting stem cells from embryos destroys human life.

President Barack Obama said during his campaign that overturning research limits would be a top priority in his administration.

Both Geron and the FDA said the timing of the decision to approve the study was coincidental. “The FDA looks to the science on these types of issues, and we approve [such applications] based on a showing of safety,” said Karen Riley, an FDA spokeswoman. “Political considerations have no role in this process.”

Approval of the study is far from a guarantee that [embryonic] stem-cell treatments will work or make it to the market, but it is likely to be seen as an indication that opportunities for [embryonic] stem-cell research are poised to open and will fuel enthusiasm among academic and corporate researchers.

Mr. Obama’s plans for acting on the current research restrictions haven’t been finalized. Shortly after the election, Obama advisers thrilled biotech companies and investors when they suggested that the new president could use his executive authority to undo the Bush administration ban. But in a Jan. 18 interview on CNN, Mr. Obama said he might let Congress take the lead. “I like the idea of the American people’s representatives expressing their views on an issue like this,” he said.

Regulating [embryonic] stem-cell therapy is new turf for both industry and the FDA, a major reason why it took the agency nearly a year to review Geron’s 21,000-page application for the trial, which it filed last March. Approval came in a phone call Wednesday afternoon, Dr. Okarma said.

The study will focus on the safety of the treatment. At an FDA hearing in April, several firms’ executives and researchers complained that they were at a loss about what the FDA wanted in terms of clinical trials involving [embryonic] stem cells because the FDA itself wasn’t sure.

Embryonic stem cells are the building-block cells that help drive prenatal development. Geron has developed banks of embryonic stem cells and found a way to coax them into differentiating as they do in nature into progenitors of specific cells that make spinal-cord tissue, heart muscle, cartilage and other organs and tissues.

Spinal-cord injury is one of medicine’s most debilitating conditions, typically causing paralysis and other issues for which there are few, if any, effective treatments. The Geron study will enroll paralyzed patients who can be treated within 14 days of their injury. Patients will be evaluated for at least one year, after which, if the treatment proves safe, the company hopes to increase the dose and expand the potential candidates for the therapy.

In addition to safety, researchers will look for signs that the treatment is effective.

Write to Ron Winslow at ron.winslow@wsj.com and Alicia Mundy at alicia.mundy@wsj.com.

Copyright 2009 Dow Jones & Company, Inc.  All Rights Reserved.  Reprinted here for educational purposes only.  Visit the website at wsj.com. 

Background

Adult stem cells and embryonic stem cells. (from njfpc.org/Articles/AdultStem.asp)

• Adult stem cell research has been on-going for 20-30 years, is not under any government restriction, and does not require the destruction of human life. These stem cells have already been used to treat spinal cord injuries, Leukemia, and even Parkinson's disease . Adult stem cells are derived from umbilical cords, placentas, amniotic fluid, various tissues and organ systems like skin and the liver, and even fat obtained from liposuction.
• In contrast, embryonic stem cells are obtained by harvesting living embryos generally 5 to 7 days old, which are destroyed in the process. Most importantly, embryonic stem cells have never yet been successfully used to help cure disease. In fact, in animals they have caused tumors and other complications. Embryonic stem cells are also being touted by some as a possible treatment for repairing the brains of Alzheimer's patients, but stem cell researchers confess that this is a distortion that is not being aggressively corrected by scientists.
• A new poll, conducted by International Communications Research, reveals that once Americans understand the difference between adult and embryonic stem cells, Americans strongly prefer funding adult stem cell research that does not destroy human life, by a margin of 61% to 23%. So, what is driving the biotech industry and many government officials to press for government money to subsidize embryonic stem cell research? Free money, and research without ethical limitations.
• Private industry has not been willing to put up any large sums of money on their own for embryonic stem cell research, because they are not sure it will yield the results they hope for. However, some drugmakers are getting into the field of research utilizing adult stem cells from umbilical cord blood and bone marrow . Investors are now taking notice that adult cells are actually working with human patients, and researchers are finding that these cells appear to be as flexible as the embryonic type.

Background on Adult Stem Cells vs. Embryonic Stem Cells: (from pbs.org, a Newshour Extra report on the Stem Cell Research Debate by Lisa Prososki)
Stem cells are universal cells that have the ability to develop into specialized types of tissues that can then be used throughout the body to treat diseases or injuries. [Embryonic] Stem Cell Research is a topic embroiled in much controversy. Scientists are hopeful that one day stem cells will be used to grow new organs such as kidneys or spinal cords as well as different types of tissues such as nerves, muscles, and blood vessels. The controversy sparked by the use of [embryonic] stem cells and research in this area comes from the fact that...these cells are taken from embryos that are just days old. As a result of this, the embryo, which is a developing human life, is destroyed. Many people feel it is immoral and unethical to destroy embryos for the sake of science. To further the debate, while these cells are easily cultured, replicate quickly, and have a relatively long life, embryonic stem cells have not yet been successfully used to provide any kind of therapy for humans and pose risks such as tumor growth and rejection by the body.

On the other side of the issue is the use of adult stem cells for research. Adult stem cells are available from a variety of sources including blood from the umbilical cord, the placenta, bone marrow, and even human fat. ....they may have some limitations in the type of tissues they are able to form. For many years, adult stem cells have been used to provide a number of different therapies to people with a relatively high rate of success. Recent research has shown that adult stem cells taken from one area of the body are able to regenerate and form tissues of a different kind. In addition to the proven therapies and research, the use of adult stem cells from a patient's own body decreases the risk of rejection because the cells are not seen as foreign invaders.

All in all, many scientists believe that the use of adult stem cells should be the primary focus of stem cell research based on past success, lower chances of patient rejection, and the idea that adult stem cell research does not spark the moral, ethical, and political debate seen so frequently when the use of embryonic stem cells is considered.

The Adult stem cell technique named as Science Magazine's "2008 Scientific Breakthrough of the Year" is a technique that allows stem cells to be created without the need to destroy embryos.
--The advance, which involves turning back the clock on adult tissue and "reprogramming" it with the properties of stem cells, could lead to new treatments for diseases including Parkinson's and diabetes.
--The process allows for a potentially limitless numbers of "induced pluripotent stem" (IPS) cells to be made to order from a sick patient's cells, meaning they do not risk rejection from the immune system when transplanted.
--The technique does not require stem calls to be harvested from embryos, making it more acceptable to religious groups.
--Dr Robert Coontz of the journal Science, which placed cellular reprogramming top of its list of the biggest scientific breakthroughs of 2008, said it "opened a new field of biology almost overnight and holds out hope of life-saving medical advances". Three teams working in Japan and the United States made major advances with the technique over the last 12 months.