LA ESPERANZA CON CELULAS MADRE...

UNA NOTICIA SOBRE CELULAS MADRE PARA STARGARDT Y DEGENERACION MACULAR POR LA EDAD,

 UN INTERESANTE COMENTARIO.

Aquí está el anuncio de Advanced Cell Technology del inicio del clinical trial con células madres para Stargardt y degeneración macular por la edad.

Los pacientes ( uno de cada patología) recibieron la dosis menor de las que van a probar el mes pasado, y de acuerdo con el artículo están muy bien.
Los pacientes serán monitoreados durante un año para verificar tolerancia y bioseguridad, mientras se insertan las células en el tejido retiniano.

 La expectativa mayor es que sea suficiente la dosis colocada para que esas células pigmentarias empiecen a funcionar y recuperen la función de los fotorreceptores latentes.

Estos pacientes están poniéndole el cuerpo a la investigación, con todas las dudas y miedos que esto significa.

Realmente esperamos que tengan el mayor de los éxitos recuperando la visión y abriendo el camino para que nosotros también estemos en condiciones de ser tratados. 

 

Poner el cuerpo implica una gran valentía y ya sea que se consiga el objetivo de que vuelvan a ver bien o no, su entrega merece el mayor de nuestros reconocimientos.

 

Son quienes están haciendo el camino más seguro para nosotros y para nuestros hijos.

 

Creo que debemos rezar por ellos a quien sintamos que debemos hacerlo, para que todo salga como esperamos.

UNA OPINION DE LA SRA. MARCELA CICCIOLI, GENETISTA, INTEGRANTE DE LA ASOCIACION STARGARDT APPNES.

GRACIAS POR LA INFORMACION MARCELA!!!

ESTA ES LA NOTA ORIGINAL EN INGLES:

New Human Embryonic Stem Cell Study For Dry AMD And Stargardt's Macular Dystrophy

14 july

Two patients have been treated using RPE (retinal pigment epithelial) cells derived from hESCs (human embryonic stem cells) in two Phase 1/2 clinical trials for dry age-related macular degeneration and Stargardt's macular dystrophy, Advanced Cell Technology Inc. has announced.

According to Dr. Steven Schwartz and Dr. Robert Lanza, the transplantation surgeries, which took place at the Jules Stein Eye Institute, California, were successful and both patients are recovering well.

Advanced Cell Technology says the two trials will enroll 12 patients in each one, with 3-patient cohorts in ascending dosage format. The primary endpoint is to establish safety and tolerability of hESC-derived RPE cells, after transplantation sub-retinally into patients with dry AMD and Stargardt's at 12 months. The investigators will also be looking for signs that the cells helped restore vision. In animal studies, rats experienced some recovery of vision.

Gary Rabin, interim chairman and chief executive officer of Advanced Cell Technology, said:

"This first treatment milestone is welcomed by scientists, stem cell advocates and patients hoping for cures. The two trials could not have started any smoother, and we are very pleased to announce that the procedures went well. The dosing of the first patients represents an important milestone for ACT and opens the doors to a potentially significant new therapeutic approach to treating the many forms of macular degeneration. We believe that these procedures represent a key step forward in therapeutic stem cell research, and the capacity to treat a variety of devastating diseases."

The studies' main investigator, Dr. Schwartz, said:

"One patient in each clinical trial, the Stargardt's trial and the dry AMD trial, has undergone surgical transplantation of a relatively small dose (50,000 cells) of fully-differentiated retinal pigment epithelial (RPE) cells derived from human embryonic stem cells. Early indications are that the patients tolerated the surgical procedures well. The primary objective of these Phase 1/2 studies is to assess the safety and tolerability of these stem cell-derived transplants. We will be carefully monitoring our patients over the course of the trials. We are privileged to be collaborating with ACT and honored to be working with these pioneering patients."

Stargardt's macular dystrophy, dry AMD and other types of atrophy-related macular degeneration are generally impossible to treat. These common forms of blindness urgently need safe and effective therapies.

Dr. Lanza said:

"Today -13 years after the discovery of human embryonic stem cells - the great promise of these cells is finally being put to the test. The initiation of these two clinical trials marks an important turning point for the field. While we will continue writing research papers and carrying out more research, it's time to start moving these exciting new stem cell therapies out of the laboratory and into the clinic

What are hESC-RPE Cells?

RPE (retinal pigment epitheliom) is an extremely thin, pigmented cell layer just below the retina. It nourishes the photoreceptors as well as carrying away waste products. Human embryonic stem cells can turn into (differentiate) into any type of cell, including RPE cells.

SMD, Dry AMD and Degenerative Diseases of the Retina

SMD (Stargardt's macular dystrophy), a common form of macular degeneration, causes progressive loss of vision. The disease usually starts when the patient is between 10 to 20 years old. Photoreceptor loss caused by degeneration in the pigmented layer of the retina (RPE cell layer) eventually leads to blindness.

Degenerative retinal diseases are one of the most common causes of untreatable blindness worldwide. Experts estimate that in the USA and Europe alone approximately 30 million people suffer from macular degeneration - a global market in excess of $25 billion. One in every ten individuals aged between 66 and 74 years is thought to have macular degeneration symptoms, most of them with dry AMD, which is untreatable. Three in every ten individuals aged between 75 and 85 are believed to be affected.

Written by Christian Nordqvist
Copyright: Medical News Today
Not to be reproduced without permission of Medical News Today