The first trial has been approved by the FDA using retinal cells derived from human embryonic stem cells to treat patients with SMD (Stargardt’s Macula Dystrophy), a common form of macular degeneration that affects young people. The FDA had a clinical hold on the commencement of Phase I/II clinical trial at multiple centers – this has now been lifted, the company Advanced Cell Technologies announced today.

Stargardt’s Macular Dystrophy, sometimes called Juvenile Macular Dystrophy, is an inherited condition in which the macula – the central area of the retina – is affected. The macula is responsible for what we see directly in front of us, the vision required for detailed activities, such as writing and reading, and the detection and appreciation of color. In SMD, which tends to first appear when the patient is aged between ten and twenty years, some of the macula cells stop working, resulting in problems with central vision, detailed vision, and possibly color perception – specifically, the cells of the pigmented layer of the retina, called the retinal pigment epithelium (RPE), deteriorate and die. It is a progressive disease which eventually leads to blindness.

Dr. Robert Lanza, Chief Scientific Officer, Advanced Cell Technologies, said:

    “There is currently no treatment for Stargardt’s disease. Using stem cells, we can generate a virtually unlimited supply of healthy RPE cells, which are the first cells to die off in SMD and other forms of macular degeneration. We’ve tested these cells in animal models of eye disease. In rats, we’ve seen 100% improvement in visual performance over untreated animals without any adverse effects. Our studies showed that the cells were capable of extensive rescue of photoreceptors in animals that otherwise would have gone blind. Near-normal function was also achieved in a mouse model of Stargardt’s disease. We hope to see a similar benefit in patients with various forms of macular degeneration.”

The Prospective Phase I/II trial will be an open-label study with the main aim of determining the safety and tolerability of the retinal pigment epithelium cells following sub-retinal transplantation on twelve patients with advanced Stargardt’s Macular Dystrophy.

Because the FDA granted the RPE cells Orphan Drug designation, Advanced Cell Technologies will also be eligible for tax credits, grant funding, accelerated approval, and marketing exclusivity up to seven years after approval.

Raymond Lund, Ph.D., a respected retinal cell physiology and vision restoration expert, said:

    “The study results of ACT’s RPE cells implanted in the various animal models of macular degeneration was phenomenal. If ACT observes even a fraction of that benefit in humans, it will be nothing short of a home run.”

William M. Caldwell IV, Chairman and CEO of Advanced Cell Technologies, said:

    “Initiating our macular degeneration clinical trial represents a significant milestone in the progress of developing human embryonic stem cell-based therapies aimed at large worldwide markets. I think generations will look back at this time as one of the most exciting in the history of medicine. With the initiation of this clinical trial, and that of Geron’s earlier this fall, the field of regenerative medicine is poised to take embryonic stem cell therapies from the realm of nebulous potential to that of tangible and real treatments that will make a significant difference in the lives of millions of people worldwide. This is truly a ‘game changer’ for the medical community.”

Thirty million people in Europe and the USA are estimated to be affected by macular degeneration. This $25 to 30 billion global market has not yet been properly addressed, the company explains. About 1 in every ten individuals aged 66 to 74 has symptoms of macular degeneration, most of them in the dry form of AMD, a condition for which there is no current treatment. 30% of 75 to 85 year-olds have symptoms.

Edmund Mickunas, Vice President of Regulatory, Advanced Cell Technology, said:

    “These patient numbers are staggering in size, and when the impact on health and quality-of-life is considered in that context, macular degeneration represents one of the more significant unmet medical needs in our society. With the momentum of this Stargardt’s trial, and the unique experience we have gained as one of the few companies in the world having succeeded in taking an hESC program into the clinic, we are preparing to extend our lead with an IND in the use for treating age-related macular degeneration, as well as filing to begin clinical trials in Europe, in the very near future.”

Source: Advanced Cell Technology

Written by Christian Nordqvist