Hadassah

Clinical Trial of Hadassah-Developed Stem Cell Treatment for Macular Degeneration Gets Go-Ahead from FDA

Thursday, Nov 6 2014

Jerusalem’s Cell Cure Neurosciences, whose mission is to become a leading supplier of human cell-based therapies for the treatment of retinal and neurodegenerative diseases, has received approval from the United States Food and Drug Administration (FDA) to conduct a clinical trial to treat the dry form of age-related macular degeneration (AMD) with OpRegen®, a first-of-its kind animal product-free, retinal pigment epithelial (RPE) cell formulation derived from human embryonic stem cells (hESCs).

“This is a significant milestone for our company and in the broader development of therapies based on human embryonic stem cells for the treatment of major diseases,” notes Prof. Benjamin Reubinoff, Director of the Hadassah Medical Center’s Human Embryonic Stem Cell Research Center and Chief Scientific Officer of Cell Cure. “We look forward to initiating this first-of-its-kind study and to continuing the clinical development of OpRegen®.”

“Cell Cure’s Phase I/IIa study of OpRegen® has been designed to provide preliminary, objective functional and structural data on the ability of hESC-RPE cell transplantation to slow the progression of geographic atrophy (dry-AMD), in addition to safety data,” explains Prof. Eyal Banin, head of Hadassah’s Center for Retinal and Macular Degeneration. Prof. Banin, together with Prof. Reubinoff, helped develop this novel treatment over the last decade. “We are truly excited that this unique, hESC-based therapy will finally be tested in patients with dry-AMD, which severely impacts the quality of life of the elderly, and for which no approved therapy yet exists,” Dr. Banin added.

Dry AMD is the leading cause of visual impairment and blindness among the elderly in the US and other developed countries. While treatment options exist for the treatment of the wet form of AMD, 90 percent of all patients with AMD have the dry form. As Cell Cure’s website explains: “The root cause of dry-AMD is believed to be the dysfunction of RPE cells. Therefore, one of the most exciting new therapeutic strategies for dry-AMD is the transplantation of healthy young RPE cells to support and replace those lost with age. Pluripotent stem cells, such as hESCs, can potentially provide a means of manufacturing such healthy RPE cells on an industrial scale.” Cell Cure will conduct this clinical trial with 15 patients at the Hadassah Medical Center, where OpRegen® will be transplanted as a single dose into the subretinal space of the patient’s eye to test the product’s safety and efficacy. The trial will evaluate three different dose regimens of 50,000 to 500,000 retinal pigment cells. Patient enrollment is expected to begin following approval of the trial by Israel’s Ministry of Health.

Following stem cell transplantation, the patients will be followed for 12 months at specified intervals to evaluate the safety and tolerability of OpRegen®. A secondary objective of the clinical trial will be to examine the ability of transplanted OpRegen® to engraft, survive, and induce changes in visual acuity. In addition to thorough characterization of visual function, a battery of defined ophthalmic imaging modalities will be used to quantify structural changes.

Preclinical studies at Hadassah with mice have shown that following a single subretinal injection of OpRegen® as a suspension of cells, the cells can rapidly organize into their natural monolayer structure and survive throughout the animal’s lifetime. Cell Cure reports that OpRegen® is anticipated to be an “off-the-shelf” product that would be provided to retinal surgeons in a final formulation ready for transplantation. Unlike treatments that require multiple, frequent injections into the eye, such as currently marketed products like Lucentis and Eylea for wet-AMD, it is expected that OpRegen® would be administered in a single procedure.

More information about the trial is available at the US National Institutes of Health

Comments

From: Barbara on July 18, 2016
I was thrilled to learn of your research. I have Dry ARMD in my right eye and Wet ARMD in my left. My left eye was trated with Eyelea which was like a miracle as my sight literally returned in days. My eyes are deteriorating and reading is becoming difficult. I am 70 years old and would love to participate as a patient in your study. I live in Canada and would come to Israel as necessary. I wish you every success with your research.
From: James on September 21, 2015
Am writing from Nairobi , Kenya on behalf of a friend suffering from MACD and in desperate need of medical intervention . Will greatly appreciate knowing progress of your clinical trials.. Many thanks, James.
From: Roger on January 14, 2015
Wonderful. Sure hope this works. I am 78 and diminishing vision due to my dry MacD has alarmingly excellerated in the past 12 month. Lens perscriptions become less effective within months. I would appreciate being informed of your progress from time to time if possible. Thank you. Roger Whitcomb, Juno Beach, FL USA
First Name
Email
Comment
Enter this word:

Related Stories

alt_text

Tuesday, Jun 20 2017

Send in the Clowns: A Hadassah UK Dispatch

This week Hadassah UK (HUK) brought in one of the Hadassah Medical Organization's best and more unusual, head medical clown David Barashi (DuSH).

READ MORE ›
alt_text

Friday, Jun 16 2017

Hadassah’s Director General Receives Honorary PhD from Cyprus Medical School

For sharing his knowledge and experience with senior officials in the Cyprus medical system, Prof. Zeev Rotstein, Director General of the Hadassah Medical Organization, was awarded an honorary PhD from the St. George University of London’s Medical Program at the University of Nicosia Medical School in Cyprus.

READ MORE ›
alt_text

Thursday, Jun 8 2017

Hadassah Doctors Discover Bullet
in 12-Year-Old’s Arm

Muhammad Baduan, age 12, had been experiencing pain in his arm on and off for months. Hadassah Hospital Ein Kerem Emergency Room physicians put an end to the boy’s pain when they solved the mystery: Muhammad had a 22-millimeter bullet hidden in that arm--a bullet that had been shot into the air as part of a post-wedding ceremony celebration.

READ MORE ›
alt_text

Monday, Jun 5 2017

Hadassah Cutting-Edge Neuropsychiatrist Keynotes Alzheimer’s Conference

“The key is to give medicines much earlier--before the brain material has been destroyed by Alzheimer’s disease,” Dr. Shahar Arzy, head of the Hadassah Medical Organization’s Computational Neuropsychiatry Laboratory, told his audience at the Alzheimer’s Foundation of America (AFA)’s New York Educational Conference on May 5, 2017.

READ MORE ›

Donation Questions

donorservices@hadassah.org

(800) 928-0685

Membership Questions

membership@hadassah.org

(800) 664-5646

Missions Department

missions@hadassah.org

(800) 237-1517

Contact Us

40 Wall Street

New York, NY 10005

support@hadassah.org

More ›

Show More