Pioneer’s 25-year struggle to treat blindness

Being a pioneer is never easy. You are charting unknown territory, tackling problems that have defeated others before you. You have to overcome so many obstacles that at times the challenge can seem insurmountable. But for those who succeed in reaching their goal, the rewards can be extraordinary.

Graziella Pellegrini, Center for Regenerative Medicine, University of Modena, Italy

Graziella Pellegrini, Center for Regenerative Medicine, University of Modena, Italy

Last month Italian researcher Graziella Pellegrini saw 25 years of work pay off when a treatment she developed to cure a form of blindness was given approval for sale by the European Commission.

This is quite an achievement as this means her treatment, called Holoclar, is among the first commercial stem therapies in the world (the first was Prochymal, which has been approved in Canada and New Zealand for the treatment of pediatric GVHD. This drug was developed by Osiris, which was led by our current President & CEO, Dr. Randy Mills.)

Holoclar uses stem cells to help stimulate the regrowth of a cornea. It can only be used for certain rare conditions, but that in no way diminishes its importance for patients or significance for the regenerative medicine field as a whole.

Nature recently sat down with Dr. Pellegrini to talk about her work, her struggle, and the many obstacles she had to overcome to get market approval for her work.

The interview makes for fascinating reading, and is a timely reminder why this kind of groundbreaking research never goes quite as quickly, or smoothly, as one would hope.

CIRM currently has a number of projects focused treating different causes of blindness on limbal cells (the kind Dr. Pellegrini worked on) and other forms of blindness; including a project to treat macular degeneration that has been approved for a clinical trial, and a therapy for retinitis pigmentosa that we hope will be approved for a clinical trial later this year.

Stem cell stories that caught our eye; progress toward artificial brain, teeth may help the blind and obesity

Here are some stem cell stories that caught our eye this past week. Some are groundbreaking science, others are of personal interest to us, and still others are just fun.

More progress toward artificial brain. A team at the RIKEN Institute in Japan has used stem cells in a 3-D culture to create brain tissue more complex than prior efforts and from an area of the brain not produced before, the cerebellum—that lobe at the lower back of the brain that controls motor function and attention. As far back as 2008, a RIKEN team had created simple tissue that mimicked the cortex, the large surface area that controls memory and language.

shutterstock_93075775

The Inquisitr web portal wrote a feature on a wide variety of efforts to create an artificial brain teeing off of this week’s publication of the cerebellum work in Cell Reports. The piece is fairly comprehensive covering computerized efforts to give robots intelligence and Europe’s Human Brain Project that is trying to map all the activity of the brain as a starting point for recapitulating it in the lab.

The experts interviewed included Robert Caplan of Tufts University in Massachusetts who is using 3-D scaffolding to build functional brain tissues that can process electrical signals. He is not planning any Frankenstein moments; he hopes to create models to improve understanding of brain diseases.

“Ideally we would like to have a laboratory brain system that recapitulates the most devastating diseases. We want to be able to take our existing toolkit of drugs and understand how they work instead of using trial and error.”

Teeth eyed as source of help for the blind. Today the European Union announced the first approval of a stem cell therapy for blindness. And already yesterday a team at the University of Pittsburg announced they had developed a new method to use stem cells to restore vision that could expand the number of patients who could benefit from stem cell therapy.

Many people have lost part or all their vision due to damage to the cornea on the surface of their eye. Even when they can gain vision back through a corneal transplant, their immune system often rejects the new tissue. So the ideal would be making new corneal tissue from the patient’s own cells. The Italian company that garnered the EU approval does this in patients by harvesting some of their own cornea-specific stem cells, called limbal stem cells. But this is only an option if only one eye is impacted by the damage.

The Pittsburgh team thinks it may have found an unlikely alternative source of limbal cells: the dental pulp taken from teeth that have be extracted. It is not as far fetched at it sounds on the surface. Teeth and the cornea both develop in the same section of the embryo, the cranial neural crest. So, they have a common lineage.

The researchers first treated the pulp cells with a solution that makes them turn into the type of cells found in the cornea. Then they created a fiber scaffold shaped like a cornea and seeded the cells on it. Many steps remain before people give up a tooth to regain their sight, but this first milestone points the way and was described in a press release from the journal Stem Cells Translational Medicine, which was picked up by the web site ClinicaSpace.

CIRM funds a project that also proposes to use the patient’s own limbal stem cells but using methods more likely to gain approval of the Food and Drug Administration than those used by the Italian company.

Stem cells and the fight against obesity. Of the two types of stem cells found in your bone marrow, one can form bone and cartilage and, all too often, fat. Preventing these stem cells from maturing into fat may be a tool in the fight against obesity according to a team at Queen Mary University of London.

The conversion of stem cells to fat seems to involve the cilia, or hair-like projections found on cells. When the cilia lengthen the stem cells progress toward becoming fat. But if the researchers genetically prevented that lengthening, they stopped the conversion to fat cells. The findings opens several different ways to think about understanding and curbing obesity says Melis Dalbay one of the authors of the study in a university press release picked up by ScienceNewsline.

“This is the first time that it has been shown that subtle changes in primary cilia structure can influence the differentiation of stem cells into fat. Since primary cilia length can be influenced by various factors including pharmaceuticals, inflammation and even mechanical forces, this study provides new insight into the regulation of fat cell formation and obesity.”