Cell therapy in degenerative retinal diseases: expectations and threats Review article

Main Article Content

Anna Machalińska


Currently, for many retinal degenerative diseases there is no effective treatment based on disease etiology. Neurotrophic factors secreted by the applied cells are responsible for improved morphology and function of degenerative retina. Moreover, these cells present immunomodulatory effect and reduce the inflammatory response of the damaged tissue. The best effect is obtained at the initial stage of the disease. Despite promising preliminary results, cell therapy requires further investigation to evaluate its efficacy and long-term safety. It is worth to underline that cell therapy should be conducted only as part of free clinical trials in certified research centers after obtaining the patient's informed consent for treatment.

Article Details

How to Cite
Machalińska A. Cell therapy in degenerative retinal diseases: expectations and threats. Ophthatherapy [Internet]. 2020Mar.31 [cited 2020Nov.30];7(1):30-5. Available from: https://journalsmededu.pl/index.php/ophthatherapy/article/view/972
Conservative treatment


1. Mandai M, Watanabe A, Kurimoto Y et al. Autologous Induced Stem-Cell-Derived Retinal Cells for Macular Degeneration. N Engl J Med. 2017; 376(11): 1038-46.
2. Machalińska A, Karczewicz D, Machaliński B. Stem cells – new perspectives in the treatment of retinal disorders. Klin Oczna. 2006; 108(10-12): 471-4.
3. Machalińska A, Baumert B, Kuprjanowicz L et al. Potential application of adult stem cells in retinal repair – challenge for regenerative medicine. Curr Eye Res. 2009; 34(9): 748-60.
4. Machalińska A, Zuba-Surma EK. Stem cells in adult retina – current state of research, future therapeutic prospects. Klin Oczna. 2009; 111(7-9): 253-7.
5. Machalińska A, Kłos P, Baumert B et al. Stem cells are mobilized from the bone marrow into the peripheral circulation in response to retinal pigment epithelium damage – a pathophysiological attempt to induce endogenousregeneration. Curr Eye Res. 2011; 36(7): 663-72.
6. Machalińska A, Lubiński W, Penkala K et al. Functional improvement of injured retina following the adjuvant stem cell-based therapy. Preliminary report. Klin Oczna. 2011; 113(4-6): 117-21.
7. Machalińska A, Kawa M, Pius-Sadowska E et al. Long-term neuroprotective effects of NT-4-engineered mesenchymal stem cells injectedintravitreally in a mouse model of acute retinal injury. Invest Ophthalmol Vis Sci. 2013; 54(13): 8292-305.
8. Sobieniecki A, Machalińska A, Rogińska D et al. Slow retinal degeneration in a mouse model: morphological and functional aspects. Pomeranian J Life Sci. 2015; 61(1): 81-9.
9. Machalińska A, Rogińska D, Pius-Sadowska E et al. Neuroprotective and antiapoptotic activity of lineage-negative bone marrow cells after intravitrealinjection in a mouse model of acute retinal injury. Stem Cells Int. 2015; 2015: 620364.
10. Paczkowska E, Kaczyńska K, Pius-Sadowska E et al. Humoral activity of cord blood-derived stem/progenitor cells: implications for stem cell-based adjuvant therapy of neurodegenerative disorders. PLoS One. 2013; 8: e83833.
11. Lejkowska R, Kawa MP, Pius-Sadowska E et al. Preclinical Evaluation of Long-Term Neuroprotective Effects of BDNF-Engineered MesenchymalStromal Cells as Intravitreal Therapy for Chronic Retinal Degeneration in Rd6 Mutant Mice. Int J Mol Sci. 2019; 20(3): 777.
12. Kerschensteiner M. Neuroprotective approaches in the animal model. Drug Res (Stuttg). 2013; 63(suppl 1): S7.
13. Wenzel A, Grimm C, Samardzija M et al. Molecular mechanisms of light-induced photoreceptor apoptosis and neuroprotection for retinal degeneration. Prog Retin Eye Res. 2005; 24(2): 275-306.
14. Siqueira RC, Messias A, Gurgel VP et al. Improvement of ischaemic macular oedema after intravitreal injection of autologous bone marrow-derived haematopoietic stem cells. Acta Ophthalmol. 2015; 93(2): e174-6.
15. Siqueira RC, Messias A, Voltarelli JC et al. Resolution of macular oedema associated with retinitis pigmentosa after intravitreal use of autologous BM-derived hematopoietic stem cell transplantation. Bone Marrow Transplant. 2013; 48(4): 612-3.
16. Park SS, Bauer G, Abedi M et al. Intravitreal autologous bone marrow cd34+ cell therapy for ischemic and degenerative retinal disorders: Preliminary phase 1 clinical trial findings. Investig Ophthalmol Vis Sci. 2015; 56(1): 81-9.
17. Cotrim CC, Toscano L, Messias A et al. Intravitreal use of bone marrow mononuclear fraction containing CD34+stem cells in patients with atrophic age-related macular degeneration. Clin Ophthalmol. 2017; 11: 931-8.
18. Weiss JN, Levy S. Stem Cell Ophthalmology Treatment Study: bone marrow derived stem cells in the treatment of Retinitis Pigmentosa. Stem Cell Investig. 2018; 5: 18.
19. Weiss JN, Levy S. Stem Cell Ophthalmology Treatment Study (SCOTS): bone marrow derived stem cells in the treatment of Usher syndrome. Stem Cell Investig. 2019; 6: 31.
20. Kim JY, You YS, Kim SH et al. Epiretinal Membrane Formation After Intravitreal Autologous Stem Cell Implantation in a Retinitis Pigmentosa Patient. Retin Cases Brief Rep. 2017; 11(3): 227-31.
21. Boudreault K, Justus S, Lee W et al. Complication of Autologous Stem Cell Transplantation in Retinitis Pigmentosa. JAMA Ophthalmol. 2016; 134(6): 711-2.
22. Kuriyan AE, Albini TA, Townsend JH et al. Vision Loss after Intravitreal Injection of Autologous “Stem Cells” for AMD. N Engl J Med. 2017; 376(11): 1047-53.
23. Souied E, Pulido J, Staurenghi G. Autologous Induced Stem-Cell-Derived Retinal Cells for Macular Degeneration. N Engl J Med. 2017; 377(8): 792.