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Stem Cell Research
The Front Line Of The Fight
Against Stargardt's'â
by Helen Reynolds

The field of human knowledge is relentlessly advancing, and, thankfully, one of the fastest areas of knowledge-increase is in medicine. Those of us with Stargardt's Disease will be intrigued to hear about the various trials and findings related to the disease which have occurred over the last few years, and the progressive research leading from these. One of the most exciting and promising areas of study relating to Stargardt's involved Human Embryonic Stem Cells. While it is not without its controversies and opponents [1], stem cell research seems set to revolutionize the field of medicine, and provides hope for future sufferers of Stargardt's Disease and other such degenerative conditions.

Human Embryonic Stem Cells

When we are embryos, our cells have the ability to grow, change their function, and develop new tissue. We lose this ability as we age. Were we to be able to retain and control our ability to generate and regenerate tissue in this manner, a great many human medical problems would be wiped out at a stroke. The science behind stem cell research is complex, but it involves working at a genetic level with the very blueprints of humanity, and saving lives through the base-level repair of faulty elements within that blueprint. Initially, the stem cells used in this research were extracted from human embryos, but they can now be synthesised in a lab. This not only provides enormous potential for the use of stem cells in actual healing, but allows unprecedented ease of research. Our knowledge of the neurobiological effects of substance addiction [2], for example, has been greatly enhanced by the ability to grow human cells within a lab [3] rather than undertaking the ethically and practically dubious process of studying a living human addict.


Gene therapy and stem cell research are leading the way in the progress towards a cure for Stargardt's Disease. It was through such genetic research that the gene responsible for the majority of cases of Stargardt's Disease was identified – gene ABCA4. Genetic testing on mice [4] has improved our knowledge of the genetic factors behind the disease itself, clearing the way towards reaching a cure.

Human Trials

Indeed, so advanced is our knowledge of the factors behind Stargardt's Disease that researchers feel confident enough to test stem cell treatments on human patients. There have been several clinical trials involving stem cells and gene therapy upon human subjects for macular degenerative diseases, some of which are still ongoing, and many of which are under review. A few years ago, an international project set up the first gene therapy trial for Stargardt's Disease in particular. Carried out at the Oregon Health And Science University's Casey Eye Institute and the Hospital Nationale des Quinze-Vingt in Paris, a trial of a treatment developed by British company Oxford BioMedica got underway. Early results were positive, with patients reporting improvements in vision over time. BioMedica are now following this up with further gene therapy trials, for which patients can sign up if they so wish [5]. More recently, a Massachusets company named Advanced Cell Technology (now Octa Therapeutics) conducted trials in the US and the UK which involved placing healthy stem cells which had been transformed into retinal pigment epithelial cells into the affected retina. It is hoped that this will prevent further vision loss, and initial results are encouraging.

Progressing Towards A Cure

Building upon this research, a collaboration in Europe between the French Institut de la Vision and the Friedrich Meischer Institute in Switzerland is working on a project which aims to revitalize and regenerate failing cone cells within the eye. If successful, this project would provide a symptomatic aid rather than tackling the underlying genetic condition, but could be of enormous help for those with degenerating eyesight nonetheless. Simultaneously, Octa Therapeutics (formerly Advanced Cell Technology) are planning to build upon their ground breaking work with stem cell retinal implants [6], continuing to observe the results of their trials and collating what they have learned in order to potentially develop medical improvements for the treatment of Stargardt's Disease in the not-too distant future. All very promising – but it is important to note that this research is still in relative infancy. If you wish to speed the process, volunteering for trials (check the websites of Oxford BioMedica and Octa Therapeutics) or donating to funds which support such research is always welcome!

[1] Jessica Reaves, The Great Debate Over Stem Cell Research, Time, Jul 2001

[2] MentalHelp.net, Outpatient and Inpatient Drug Treatment Centers In New York

[3] Jim H. Smith,Using Stem Cells to Study Alcohol Addiction, University of Connecticut, Mar 2010

[4] Robert K Koenekoop, The gene for Stargardt's disease, ABCA4, is a major retinal gene: A mini-reviewâ€, McGill University

[5] Oxford BioMedica, Clinical Trials

[6] Susan Young, Stem-Cell Treatment for Blindness Moving Through Patient Testing, MIT Technology Review, Apr 2014