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FDA approves third human stem cell trial

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The US Food and Drug Administration has given the green light for a third human trial using human embryonic stem (hES) cells to proceed.

Advanced Cell Technology (ACT), which in November won approval to use hES cells to treat a rare form of juvenile blindness called Stargardt’s disease, announced on January 3 that it had the go-ahead to use the cells in a trial of adults with a related but far more common condition: age-related macular degeneration (AMD).

The Santa Monica, California-based biotechnology company said it would enroll twelve subjects as soon as possible in a phase one trial to test the safety of the therapy, which uses hES cells to recreate retinal pigment epithelium (RPE) cells. These essential cells support the centrally located photoreceptors needed for high acuity vision, and it is their degeneration that causes AMD, the leading source of blindness in people over age 55, according to Robert Lanza, ACT’s Chief Scientific Officer. (The company’s therapy is aimed at “dry” AMD, which accounts for roughly 90 percent of all AMD cases.)

The company said that it is initially considering trial sites at the Stanford University School of Medicine in Palo Alto, California and at the Jules Stein Eye Institute at the University of California, Los Angeles.


Lanza says that ACT is able to generate “a virtually unlimited and reproducible” supply of healthy RPE cells. He adds that, because only 50,000 to 200,000 cells — a small number in the world of hES cell therapy — are needed to treat each patient, manufacturing and distribution of the cells looks readily achievable.

ACT also said it plans to “aggressively” pursue approval for a similar trial with the European Medicines Agency. It estimates that over ten million Europeans suffer from AMD, along with ten to fifteen million Americans.

Geron, the Menlo Park, California-based biotechnology firm, won the first FDA approval for a clinical trial of an hES cell therapy – in that case, aimed at treating spinal cord injury – two years ago this month. However, the launch of the trial was delayed and the first subject was not injected with cells until October, 2010.

PHOTO COURTESY OF THE NATIONAL EYE INSTITUTE

Comments

  1. Report this comment

    Murugan Vetrivel said:

    Good way..i wish to Lanza and his team to get right results……

  2. Report this comment

    Cornelius Lukas Jacobsz said:

    I would like to inquire about the Stargardt’s trial. I am very interested in the results as i am a sufferer of this disease.

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    Sarah Clay said:

    My husband, age 45, was diagnosed with Stargardt’s as a teenager. I have been following these news releases closely, but I was wondering if there is some sort of mailing list or newsletter that automatically updates people on the progress of this trial.

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    charlie medeiros said:

    I wish to be subject for testing.

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    Duane Werner said:

    I have a son that is 15 years of age and is really wanting to go ahead and be a candidate for this stem cell replacement, he prays every night for a break through so he can be a normal kid. So please let us know what we have to do to get in line for this. Thanks

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