The race to cure macular degeneration with stem cells


The major eye disease epidemic faced by an aging population is Macular Degeneration (AMD).  For the first time in history of civilization, the largest number of people will approach aging with the possible prospect of living indefinitely. They will also have to deal with losing their youthful energy, faculties and organs, before science and technology intervenes.  One such organ is the human eye with the function of sight.  The aged fear the loss of their independence and are looking to stem cell scientists for hope. For an overview read: “Stem cells in retinal regeneration: past, present and future“, June 2013 with lead author, Dr Peter Coffey, the pioneer in stem cell research. The journey to restoring eye health via stem cell therapy is discussed below. You may also enjoy reading the “Genes for AMD” below.

Stem Cell Retina Research
Here we will reveal to you the leaders in stem cell retina research in 2013, and the current status in academic – industry relationship to bring the laboratory discoveries to the clinic.  There is much cause for hope that soon this technology may be in your local clinic. We will also briefly discuss the genes that may predispose some of the people to age related macular degeneration (AMD).

The Eyes of the Young

The Eyes of the Young

A Hole in the Vision
AMD arises when damage occurs to the light-sensitive cells of the retina, at the back of the eye, leading to progressive loss of sight. All aging people do not lose vision to AMD. The University College of London (UCL) noted that about a quarter of the people above 60 in the United Kingdom were affected with either a wet form or a dry form of AMD. No cure for the dry AMD exists yet. UCL researchers today lead the way for cell replacement therapy research with a goal to replace the “sight” cells lost during aging. They are currently involved with using human embryonic cells.

UCL has entered a collaboration with biopharmaceutical company Pfizer, headquartered in New York city, USA. Pfizer will provide drug development and distribution experience. This UK_USA collaboration will enable stem cell based therapies for AMD and other retinal diseases. You may click here to read this announcement. They plan to be in clinics quickly.

The Eyes of those with years of Wisdom

The Eyes of those with years of Wisdom

The Countries at the Forefront of Pioneering Stem Cell Therapy
USA, Japan and Britain lead the race in stem cell therapy. Both the countries of Japan and Britain face a future of a large number of aging humans who may lose their eyesight from AMD and become dependent and their number of aged outnumber their new borns. In addition, in all three countries modern technology will keep the aging people alive until they are over 120 to 150 years old. Both countries have an incentive to use technology to keep their aging independent.

The Scientist Who Led the Retina Replacement with Stem Cell Effort
Dr. Pete Coffey, of the UCL School of Ophthalmology led the The London Project to Cure Blindness. He finds it great that Britain is at the forefront of this field of research. It has huge implications for the field of regenerative medicine as a whole.

Europe’s First Stem Cell Clinical Trial began with Retinal Cells
The first clinical trial in patients of a human embryonic stem cell therapy began in London’s Moor-fields Eye Hospital with 12 Stargardt disease patients (Click here to read article in Stem Cell Trans Med Jan 2012).  Stargardt is a type of macular degeneration currently untreatable.  It is an inherited disease causing progressive blindness with thinning of the cell on the retina called the epithelium.  The procedure, requiring the surgeon to inject several thousand cells behind the eye using a fine needle, takes up to an hour.  It is an out patient operation.  The cells will grow and replace the retina restoring sight which will require a follow up out patient check up to confirm. That’s all – two visits, both out patient and in about an hour the patient will have healthy eye sight.  Dr James Bainbridge will lead the clinical trial.  The retinal cells in the London trial were obtained from Advanced Cell Technology, that is developing similar trials in USA. Read about ground breaking transplantation of cells grown in a lab by clicking here.

Sights on a Cure for Age – Related Macular Degeneration?
Two clinical studies were started at University of California, USA to establish the safety and tolerability of human embryo derived stem cell transplantation in the retina. One patient had stargardt and the other had AMD. The cells grew and showed 99% normal retinal epithelial cell behavior. The eye is a great place to inject stem cells. It is contained in the eye cavity and there is no way for it to spread into the body. The only risk? Losing the blind eye. The future therapeutic goal is to treat patient in an earlier stage of retinal disease. Naturally, the first trial was with patients quite blind and their sight was restored favorably. Vision did improve although not perfected. You may click here to read the clinical trial report published on January 2012. Do be patient. It is a large 5.5MB file and takes time to load but is worth it for its clinical detail.

UCL School of Ophthalmology and Pfizer have requested permission for AMD treatment with stem cells. A patch of new retinal cells will be developed from human embryonic cells. The patch can be inserted into the eye through a slit in the retina where they will grow to cover the retina.

You may also enjoy reading “Stem cell scientists have macular degeneration on cross hairs“.
By the year 2020 there will be 450,000 Californians stuck with AMD unless modern technology intervenes. With negative population growth there may be fewer younger people to take care of the elderly so vision care becomes more practical an issue than simply a luxury point of care.

Stem Cells in Retinal Development: Past, Present and Future
Stem cell therapy trials are now actively recruiting for treating various visual disorders. Future directions and challenges for the field are also discussed. Do share your thoughts on this with all our readers by leaving a comment below. Would you be willing to participate in a clinical trial if you were almost blind? If yes, click here for details of such a trial in progress summarized June 2013

Take Hope from First Human Trials
Stem cells that do not originate from patients own body:
Human trials have been conducted in Britain. Very brave, very old and almost blind British volunteers asked to be guinea pigs for retina replacement stem cell research, for the sake of all aging people. The procedure took less than an hour and each of two visits. The transplanted stem cells settled into the back of the volunteers’ eyes. The retina stem cell transfer was generally successful and the aging volunteers reported eye sight better than that of an infant, which was probably an exaggeration since they were so greatful to be regain eyesight. One patient could even drive to the clinic independently for follow up studies. Prior to this procedure, he was dependent. The British are brimming with pride and joy: even their seniors know more about the advantages of stem cell biology than the average high school or college student in other countries. After all, the seniors learnt that donating their own cells to be coaxed to produce stem cells that could regenerate retina cells would benefit them while serving a scientific cause. Britain had led one of the earliest human trials in stem cell research for an important human organ.

The Eyes of a person who has lived several decades and has perfect eyesight

The Eyes of a person who has lived several decades and has perfect eyesight

Stem cells that originate from patients own body:
In 2006, Shinya Yamanaka of Kyoto University, Japan, winner of the Nobel Laureate in medicine in 2012, was the first to successfully harvest stem cells from adult tissue. He heralded the era of regenerative medicine in which iPS cells can grow into cells of any tissue. An ancient country ethically opposed to human embryo research, has embraced induced pluripotent stem cell(iPS) research or adult tissue stem cell generation.

In July 2013, the Japanese Health Minister gave permission to two Japanese research institutes to treat age-related Macular Degeneration using induced pluripotent stem cells (iPS). The Riken center for developmental biology will harvest the tissue stem cells while The Institute of Biomedical Research and Innovation will transplant six Japanese patients with the retinal cells created by targeted regeneration of the harvested stem cells (click here to read).

Eye of a person who has seen one decade

Eye of a person who has seen one decade

Japan has a disprotionate number of more older people to younger people and faces the impending problem of elderly dependent care. AMD affects 700,000 adults in Japan alone and the numbers will rise as the elderly continue to live indefinitely. By giving the elderly the choice to regain their vision with the clarity of youth, the Japanese government reduces the number of elderly dependents. This condition is incurable with available chemical medicinal approaches.

Eye of person who has seen two decades

Eye of person who has seen two decades

The Genes for AMD
Researchers at Columbia University absolutely confirm the significant roles of two genes, and consequently, the central role of a specific immune response pathway, in the development of AMD. Their genetic discovery explains 75% of the cases of AMD. You may read the original article by clicking here.
It is estimated that more than 50 million people worldwide will suffer from irreversible blindness from AMD It is the most common cause of blindness for those over sixty, but occasionally it is diagnosed in younger people who possibly had a very stressful event.

Several variants of a factor H gene help shut down an immune response against common infections, once the infection is eliminated. People with these risk-increasing variations of factor H are less able to control inflammation caused by infection triggers, which may spark AMD later in life. However, one third of people with a factor H risk variant have not been diagnosed with AMD. Looking for additional culprits, the researchers discovered factor B as the major modifier of the disease. While factor H is the inhibitor of the immune response to infection, factor B is an activator. They complement each other. A protective factor B variation protects against AMD, and vice versa. What about those who have a protective factor B and a non-risk variation of factor H? They may never need to fear AMD.

The researchers predict:

It’s estimated that 30 percent of the population will have some form of AMD by the time they reach the age of seventy-five. The disease is marked by a progressive loss of central vision due to degeneration of the macula-a region of the retina and the area responsible for fine, central vision.

Long Road Ahead
Clearly, losing eyesight with aging is no longer an option scientists are willing to accept. Do encourage them if you believe that choosing to fight blindness with modern research tools is a goal you fully aim to support. If not, do suggest how a society should arrange for one in every four elderly persons going blind and living with blindness for several decades. Who will take care of them? The number of aging far out number children. The number of young are fewer than the number of aging for the first time in history of mankind. Giving eyesight to the almost blind will make them independent longer.

The following articles provide relevant information on iPS therapy for AMD
http://www.cirm.ca.gov/our-progress/feature-sights-cure
http://dev.biologists.org/content/140/12/2576.abstract
http://www.theguardian.com/science/2012/jun/04/stem-cell-first-human-trials
http://www.ucl.ac.uk/news/news-articles/0904/09042301
http://japandailypress.com/worlds-first-human-stem-cell-clinical-trial-approved-by-japanese-government-1932608/

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