Cell therapy developed by a team of Japanese researchers led the Aurion Biotech IOTA trial, and the company recently published the first results of the study.
The treatment, corneal endothelial cell therapy, is an innovative biological therapy developed by Shigeru Kinoshita, MD, PhD, professor and chair of ophthalmology at Kyoto Prefecture Medical University in Japan, and his colleagues and materialized with the spread of healthy cornea. cells from 2 donors used to treat 50 patients with corneal endothelial disease.
According to the company, preliminary results from the IOTA trial showed improvements in visual acuity and central corneal thickness.
Corneal edema is the result of the death or breakdown of corneal endothelial cells. Since these cells do not regenerate, vision loss can occur.
So far, transplantation procedures such as Descemet membrane endothelial keratoplasty (DMEK) and Descemet stripping automated endothelial keratoplasty (DSAEK) have been performed but are limited by their drawbacks, namely that they are complex surgeries. that few surgeons perform and that the supply of donor corneas. is limited.
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In contrast, corneal endothelial cell therapy has several potential advantages:
- The supply of corneal endothelial cells is abundant. The endothelial cells of
a donor can replicate to treat up to 100 eyes.
- The procedure is simple, can be performed relatively quickly, and is less complex than the DMEK and DSAEK procedures.
- The procedure is patient friendly, with postoperative recovery in several hours versus several days for DSAEK and DMEK procedures.
The corneal endothelial cell procedure has 3 steps.
First of all, the diseased endothelium is removed from the recipient cornea.
Second, the cultured cells are loaded into syringes and the cell suspension is injected into the patient’s anterior chamber, where the cells rapidly self-align.
Third, the patient recovers for about 3 hours in a supine position.
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To date, Kinoshita and her team have performed cell therapy procedures in 65 patients in Japan, with excellent clinical results.
The main results of these studies were published in the New England Journal of Medicine1 and a 5-year follow-up study in Ophthalmology.2
In the IOTA trial, conducted from November 2020 to May 2021, at ClÃnica Quesada in San Salvador, El Salvador, researchers injected the cells into the affected eyes with the goal of reducing corneal edema and restoring vision.
The IOTA trial was conducted as a proof of concept for earlier studies of the cell therapy procedure developed by Kinoshita and colleagues.
The results showed that the procedure was safe, well tolerated and effective.
This is the first trial of the corneal endothelial cell therapy procedure to be conducted outside of Japan.
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Investigators are Gabriel Quesada, MD, and Rodrigo Quesada, MD, El Salvador and Edward Holland, MD; Elizabeth Yeu, MD; John Berdahl, MD; Matt Giegengack, MD; John Vukich, MD; and Kevin Waltz, MD, in the United States.
âThis is a historic moment for the treatment of corneal endothelial disease,â said Holland, who is chief medical advisor and chairman of the medical advisory board of Aurion Biotech.
Holland reported that in the first part of the trial, investigators treated 16 patients with cells from a single donor. In the second part of the trial, they treated 34 patients from a single donor.
Additionally, Holland pointed out that the study confirmed that the procedure is accessible for surgeons and minimally invasive for patients.
âThe initial reading of this proof of concept essay is promising. The patients demonstrated improvements in visual acuity and central corneal thickness, âsaid Holland.
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Greg Kunst, CEO of Aurion Biotech, said the IOTA trial has provided information and confirms that the procedure can restore vision in patients with blindness resulting from corneal endothelial disease.
âUntil Professor Kinoshita’s astonishing innovations in corneal endothelial cell therapy, vision loss caused by corneal edema remained largely under-served,â he said. âThis is due to a chronic shortage of donor corneas and current standards of care that involve unusually complex procedures. Because we have acquired this intellectual property, we look forward to advancing this cell therapy through regulatory approval in the United States and elsewhere to restore vision to patients around the world. “
Patients will be followed for 12 months to assess the safety and effectiveness of the treatment long term and after the end of the study.
Edward Holland, MD
E: [email protected]
Holland is Chief Medical Advisor and Chairman of the Medical Advisory Board of Aurion Biotech.
1. Kinoshita S, Koizumi N, Ueno M, et al. Injection of cells cultured with a ROCK inhibitor for bullous keratopathy. N English J Med. 2018; 378: 995-1003. doi: 10.1056 / NEJMoa1712770
2. Numa K, Imai K, Ueno M, et al. Five-year follow-up of the first 11 patients having undergone an injection of corneal endothelial cells in culture for corneal endothelial insufficiency. Ophthalmology. 2020; 128 (4): 504-514. doi: 10.1016 / j.ophtha.2020.09.002