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The following article has been kindly provided by Hasan Çiçek
WASHINGTON, Jan. 23, 2009 (Reuters) — The U.S. Food and Drug Administration has cleared the way for the first trial to see if human embryonic stem cells can treat people safely, a company involved in the controversial research on Friday.
Geron Corp, a California biotechnology company, said it plans a clinical trial to try to use the stem cells to regrow nerve tissue in patients with crushed, but not severed, spinal cords.
The issue of human embryonic stem cell research has been a political touchstone, with anti-abortion forces backed by former president George W. Bush arguing the technique involves the destruction of human embryos. Advocates say it could transform medicine.
"For us, it marks the dawn of a new era in medical therapeutics. This approach is one that reaches beyond pills and scalpels to achieve a new level of healing," Geron Chief Executive Dr. Thomas Okarma said in a telephone briefing.
Shares of Geron rose more than 53 percent to $8 in mid-morning trading on Nasdaq after touching $8.38.
Geron will recruit eight to 10 recently injured patients and inject them with small numbers of human embryonic stem cells manipulated to become the oligodendrocyte cells that insulate nerves, and that produce compounds to stimulate the growth of nerve cells.
Okarma said there was no political significance to the announcement coming the same week as President Barack Obama took office. Obama has been widely expected to lift restrictions on federal funding and support of human embryonic stem cell research.
An FDA spokeswoman said, "Before FDA allowed the study to proceed, Geron worked with FDA to address important scientific questions."
CHEAP AND EASY
Okarma said the treatment should eventually become cheap and easy to mass produce because the cells can be grown in vats. He believes the cells may be useful for other diseases such as multiple sclerosis, in which nerve cells are stripped of their insulating sheaths, and perhaps strokes.
Financial analysts celebrated. Stephen Brozak and Daniel Mallin of WBB Securities LLC said it could "as important to drug therapy as the discovery of ... penicillin."
Dr. Robert Lanza of the rival Massachusetts-based Advanced Cell Technology, which is also seeking to create therapies using human embryonic stem cells, called the decision a "huge advance for the entire field". This sends a message that we're ready at last to start helping people," he said.
Stem cells are the body's master cells, giving rise to all the tissues, organs and blood. Embryonic stem cells are considered the most powerful kinds of stem cells, as they have the potential to give rise to any type of tissue.
Researchers are also trying to find ways to use so-called adult stem cells, taken from bone marrow and elsewhere in the body, and have learned how to transform ordinary skin cells into stem-like cells. But scientists argue that no one knows which route will work so all avenues must be pursued.
The Phase I trial will be designed to show that patients do not develop tumors, or damage to their nervous systems. But Okarma believes it will also indicate whether the stem cells might repair the damaged spinal cords.
"These are living cells that will divide, make more of themselves and migrate throughout the lesion after injection," he said.
While the patients will get low doses of immune-suppressing drugs for the first two months, Okarma is confident the cells will escape immune system recognition and patients will not have to endure the treatments that organ and tissue transplant recipients usually do. Treatment on the first patient should begin this summer.
Okarma said the company has a strong balance sheet, free of debt, and "ample to fund the company through this trial".
(Additional reporting by Susan Heavey, Doina Chiacu and Toni Clarke; Editing by Alan Elsner and Julie Steenhuysen)
By Maggie Fox, Health and Science Editor
If you feel like reading an unputdownable novel while collaborating with a just and solidary cause, "The Legacy of Marie Schlau" is your book! 100% of all funds raised will be dedicated to medical research to find a cure for Friedreich's Ataxia, a neurodegenerative disease that affects mostly young people, shortening their life expectancy and confining them to a wheelchair.
The life of Marie Schlau, a German Jewish girl born in 1833 hides great unsolved mysteries: accidents, disappearances, enigmas, unknown diagnoses, disturbing murders, love, tenderness, greed, lies, death ... alternatively a different story unfolds every time and takes us closer to the present. Thus, there are two parallel stories unravelling, each in a different age and place, which surprisingly converge in a revelatory chapter.
Paperback and Kindle versions for "The legacy of Marie Schlau" available for sale at Amazon now!
Currently, BabelFAmily is financing two promising research projects aimed at finding a cure for Friedreich's Ataxia. Whenever you make a donation to us or purchase a copy of "The legacy of Marie Schlau", this is where all funds raised will be devoted to:
1) Gene Therapy for Friedreich's Ataxia research project:
The project is the result of an initiative of Spanish people affected by this rare disease who are grouped in GENEFA in collaboration with the Spanish Federation of Ataxias and the BabelFAmily. The Friedreich’s Ataxia Research Alliance (FARA), one of the main patients’ associations in the United States now joins the endeavour.
2) Frataxin delivery research project:
The associations of patients and families Babel Family and the Asociación Granadina de la Ataxia de Friedreich (ASOGAF) channel 80,000 euros of their donations (50% from each organisation) into a new 18-month project at the Institute for Research in Biomedicine (IRB Barcelona). The project specifically aims to complete a step necessary in order to move towards a future frataxin replacement therapy for the brain, where the reduction of this protein causes the most damage in patients with Friedreich’s Ataxia.
The study is headed by Ernest Giralt, head of the Peptides and Proteins Lab, who has many years of experience and is a recognised expert in peptide chemistry and new systems of through which to delivery drugs to the brain, such as peptide shuttles—molecules that have the capacity to carry the drug across the barrier that surrounds and protects the brain. Since the lab started its relation with these patients’ associations in 2013*, it has been developing another two projects into Friedrich’s Ataxia.