Neurodegenerative diseases and biomedical cell products: relevance, directions, and problems of development


Cite item

Full Text

Open Access Open Access
Restricted Access Access granted
Restricted Access Subscription or Fee Access

Abstract

Progress in clinical research and medicine has contributed to a significant reduction in the worldwide mortality rate. Currently, the strategy of developed countries is directed at increasing expectancy and improving life quality. As a consequence, the modern world is already faced the problems of aging and age-related diseases. Neurodegenerative diseases are one of the main medical problems suffered by an «aging» population of people. Stem cells as a part of biomedical cell products (preparations based on viable somatic human cells), which can have a regenerative function on the brain and spinal cord neurons or can be used for replacement therapy, are considered as one of the directions of the development of new approaches to the treatment for neurodegenerative diseases. The paper describes the features of using and development of stem cell-based drugs promising for the treatment of such neurodegenerative diseases as multiple sclerosis, Alzheimer’s disease, Parkinson’s disease, and lateral amyotrophic sclerosis. There are considered clinical studies conducted in the world with the use of mesenchymal stem cells from umbilical cord blood, bone marrow, neuronal and embryonic stem cells for the treatment of neurodegenerative diseases. Currently, clinical studies of preparations based on viable human cells for the treatment of neurodegenerative diseases are mainly characterized by the proven safety of their use and specific immunomodulatory activity. Given at the early stages of clinical trials involving a limited number of patients mainly in single-center localization, data on the efficiency of using the stem cells regenerative potential for the treatment of neurodegenerative diseases will be available in the future.

Full Text

Restricted Access

About the authors

E. O Kozhevnikova

Scientific Centre for Expert Evaluation of Medicinal Products

E. V Melnikova

Scientific Centre for Expert Evaluation of Medicinal Products

O. A Rachinskaya

Scientific Centre for Expert Evaluation of Medicinal Products

I. S Semenova

Scientific Centre for Expert Evaluation of Medicinal Products

V. A Merkulov

Scientific Centre for Expert Evaluation of Medicinal Products

References

  1. Commission Regulation (EC) No. 507/2006.
  2. Иллариошкин C.H. Ранняя диагностика нейродегенеративных заболеваний. Нервы. 2008; 1: 11-3.
  3. Сайт European Medicine Agency [Электронный ресурс]. Defining unmet medical need. 2019. URL: https://www ema.europa.eu/en/documents/presenta-tion/presentation-defining-unmet-medi-cal-need-jstokx_en.pdf.
  4. Сайт World Health Organization [Электронный ресурс]. New WHO guidelines promote healthy lifestyle to reduce risk of dementia. 2019. URL: http://www. euro.who.int/ru/health-topics/Life-stages/ healthy-ageing/news/news/2019/05/new-who-guidelines-promote-healthy-lifestyle-to-reduce-risk-of-dementia.
  5. Summary basis for regulatory action -YESCARTA [Электронный ресурс]. Food and Drug Administration. 2017. URL: https:// www.fda.gov/downloads/BiologicsBlood-Vaccines/CellularGeneTherapyProducts/ ApprovedProducts/UCM584335.pdf (дата обращения 17.10.2018).
  6. Summary of product characteristics. Holoclar (EMA/6865/2015) [Электронный ресурс]. European Medicines Agency 2015. URL: http://www.ema.europa.eu/ docs/en_GB/document_library/EPAR_-_ Product_Information/human/002450/ WC500183404.pdf (дата обращения 06.02.2018).
  7. Summary basis for regulatory action -KYMRIAH [Электронный ресурс]. Food and Drug Administration, 2018. URL: https:// www.fda.gov/downloads/BiologicsBlood-Vaccines/CellularGeneTherapyProducts/ ApprovedProducts/UCM606836.pdf (дата обращения 17.10.2018).
  8. Васильев Р.Г., Грицык В.Ф., Литвинова Л.С., Родниченко А.Е., Губарь О.С., Шуплецова В.В. Постнатальные мультипотентные стволовые/прогениторные клетки - производные нервного гребня: трансляция в клиническую практику Гены и Клетки. Материалы III национального конгресса по регенеративной медицине. Москва, (ноябрь 2017 г.); XII (3): 35.
  9. Summary of product characteristics. Strimvelis (EMA/CHMP/249031/2016) [Электронный ресурс]. European Medicines Agency 2016. URL: http:// www.ema.europa.eu/docs/en_GB/docu-ment_library/EPAR_-_Product_Information/ human/003854/WC500208199.pdf (дата обращения 29.08.2018).
  10. Федеральный закон от 23.06.2016 № ФЗ-180 «О биомедицинских клеточных продуктах».
  11. Сайт Национального медицинского исследовательского центра детской гематологии, онкологии и иммунологии имени Дмитрия Рогачева (Москва). URL: http://www.fnkc.ru/index.jsp?load=news& year=2018&monfh=7&id=153
  12. BIOCAD инвестирует $26 млн в инфраструктуру создания генно-терапевтических препаратов [Электронный ресурс]. 2018. URL: https://gmpnews. net/2018/05/biocad-will-invest-26-million-in-infrastructure-for-gene-therapies-development/
  13. Пономарева А.С., Сургученко В.А., Можейко Н.П., Ильинский И.М., Севастьянов В.И. Использование мезенхимальных стромальных клеток жировой ткани человека и биополимерных матриксов для тканеинженерной конструкции хряща. Цитология. 2011; 53 (9): 743
  14. Dauphinot V, Delphin-Combe F., Mouchoux C., Dorey A., Bathsavanis A., Makaroff Z., Rouch I., Krolak-Salmon P. Risk factors of caregiver burden among patients with Alzheimer's disease or related disorders: a cross-sectional study. J. Alzheimers Diseases. 2015; 44 (3): 907-16
  15. Hardy J. The amyloid hypothesis for Alzheimer's disease: a critical reappraisal. J. Neurochemistry. 2009; 110 (4): 1129-34.
  16. Harper P., Bates G., Jones L. Huntington's desease. New York: Oxford University Press, 2002; 558.
  17. Balestrino R., Schapira A.HV. Parkinson Disease. Eur J. Neurol. 2019. https://doi. org/10.1111/ene.14108. (Epub ahead of print).
  18. Benbrika S., Desgranges B., Eustache F., Viader F. Cognitive, Emotional and Psychological Manifestations in Amyotrophic Lateral Sclerosis at Baseline and Overtime: A Review. Front Neurosci. 2019; 13: 951.
  19. Owens B. Multiple sclerosis. Nature. 2016; 540 (7631): 1.
  20. Santiago J., Potashkin J. A network approach to clinical intervention in neurode-generative diseases. Trends in Molecular Medcine. 2014; 20: 694-703.
  21. Singh S., Srivastava A., Srivastava P., Dhuriya Y.K., Pandey A., Kumar D., Rajpurohit C.S. Advances in Stem Cell Research- A Ray of Hope in Better Diagnosis and Prognosis in Neurodegenerative Diseases. Front Mol. Biosci. 2016; 3: 72.
  22. Zhang F.-Q., Jiang J.-L., Zhang J.-T, Niu H., Fu X.-Q., Zeng L.-L. Current status and future prospects of stem cell therapy in Alzheimer's disease. Neural Regen Res. 2019; 15 (2): 242-50.
  23. Mason A.R., Ziemann A., Finkbeiner S. Targeting the low-hanging fruit of neurodegeneration. Neurology. 2014; 83 (16): 1470-3.
  24. Yagi T., Kosakai A., Ito D., Okada Y., Akamatsu W., Nihei Y., Nabetani A., Ishikawa F., Arai Y., Hirose N., Okano H., Suzuki N. Establishment of induced pluripotent stem cells from centenarians for neurodegenerative disease research. PLoS One. 2012; 7: e41572.
  25. Yuewen Tang, Pei Yu, Lin Cheng. Current progress in the derivation and therapeutic application of neural stem cells. Cell Death Dis. 2017; 8 (10): e3108
  26. Qu T., Brannen C.L., Kim H.M., Sugaya K. Human neural stem cells improve cognitive function of aged brain. Neuroreport. 2001; 12 (6): 1127-32
  27. Chia-Yu Chang, Hsiao-Chien Ting, Ching-Ann Liu, Hong-Lin Su, Tzyy-Wen Chiou, Horng-Jyh Harn, Shinn-Zong Lin. Induced Pluripotent Stem Cells: A Powerful Neurodegenerative Disease Modeling Tool for Mechanism Study and Drug Discovery. Cell Transplant. 201; 27 (11): 1588-602.
  28. Fu-Qiang Zhang, Jin-Lan Jiang, Jing-Tian Zhang, Han Niu, Xue-Qi Fu, Lin-Lin Zeng. Current status and future prospects of stem cell therapy in Alzheimer's disease. Neural regeneration research. 2019; 15 (2): 242-50.
  29. Wang Y.K., Zhu W.W., Wu M.H., Wu Y.H., Liu Z.X., Liang L.M., Sheng C., Hao J., Wang L., Li W., Zhou Q., Hu B.Y. Human Clinical-Grade Parthenogenetic ESC-Derived Dopaminergic Neurons Recover Locomotive Defects of Nonhuman Primate Models of Parkinson's Disease. Stem Cell Reports. 2018; 11 (1): 171-82.
  30. Sugarman J. Human stem cell ethics: beyond the embryo. Cell Stem Cell. 2008; 2: 529-33.
  31. Hyun I., Lindvall O., Ahrlund-Richter L., Cattaneo E., Cavazzana-Calvo M., Cossu G., De Luca M., Fox I.J., Gerstle C., Goldstein R.A., Hermeren G., High K.A., Kim H.O., Lee H.P., Levy-Lahad E., Li L., Lo B., Marshak D.R., McNab A., Munsie M., Nakauchi H., Rao M., Rooke H.M., Valles C.S., Srivastava A., Sugarman J., Taylor P.L., Veiga A., Wong A.L., Zoloth L., Daley G.Q. New ISSCR guidelines underscore major principles for responsible translational stem cell research. Cell Stem Cell. 2008; 3: 607-9.
  32. Subcommitte on Motor Neuron Diseases of World Federation of Neurology Research Group on Neuromuscular Diseases, El Escorial «Clinical limits of ALS» Workshop Contributors. El Escorial World Federation of Neurology criteria for the dignosis of amyotrophic lateral sclerosis. Journal of the Neurological Sciences. 1994; 124: 96-107.
  33. Сайт World Federation of Neurology. Fostering quality neurology and brain health worldwide (Электронный ресурс). 2019. URL: https://wfneurology.org/.
  34. McKhann G., Drachman D., Folstein M., Katzman R., Price D., Stadlan E.M. Clinical diagnosis of Alzheimer's disease: report of the NINCDS-ADRDA Work Group under the auspices of Department of Health and Human Services Task Force on Alzheimer's Disease. Neurology. 1984; 34 (7): 939-44.
  35. DSM-IV. American Psychiatric Association. Diagnostic and Statistical Manual of Mental Disorders (4th ed.). Washington, DC. 1994.
  36. Poser C.M., Paty D.W., Scheinberg L., McDonald W.I., Davis F.A., Ebers G.C., Johnson K.P., Sibley W.A., Silberberg D.H., Tourtellotte W.W. New diagnostic criteria for multiple sclerosis: guidelines for research protocols. Ann Neurol. 1983; 13 (3): 227-31.
  37. McDonald W.I., Compston A., Edan G., Goodkin D., Hartung H.P., Lublin F.D., McFarland H.F., Paty D.W., Polman C.H., Reingold S.C., Sandberg-Wollheim M., Sibley W., Thompson A., van den Noort S., Weinshenker B.Y., Wolinsky J.S. Recommended diagnostic criteria for multiple sclerosis: guidelines from the International Panel on the diagnosis of multiple sclerosis. Annals of neurology. 2001; 50 (1): 121-7.
  38. Kim H.J., Seo S.W., Chang J.W., Lee J.I., Kim C.H., Chin J., Choi S.J., Kwon H., Yun H.J., Lee J.M., Kim S.T., Choe Y.S., Lee K.H., Na D.L. Stereotactic brain injection of human umbilical cord blood mesenchymal stem cells in patients with Alzheimer's disease dementia: A phase 1 clinical trial. Alzheimers Dement (N Y). 2015; 1 (2): 95-102.
  39. Mazzini L., Gelati M., Profico D.C., Sgaravizzi G., Projetti Pensi M., Muzi G., Ricciolini C., Rota Nodari L., Carletti S., Giorgi C., Spera C., Domenico F., Bersano E., Petruzzelli F., Cisari C., Maglione A., Sarnelli M.F., Stecco A., Querin G., Masiero S., Cantello R., Ferrari D., Zalfa C., Binda E., Visioli A., Trombetta D., Novelli A., Torres B., Bernardini L., Carriero A., Prandi P., Servo S., Cerino A., Cima V, Gaiani A., Nasuelli N., Massara M., Glass J., Soraru G., Boulis N.M., Vescovi A.L. Human neural stem cell transplantation in ALS: initial results from a phase I trial. J. Transl Med. 2015; 27: 13-7.
  40. Nabavi S.M., Arab L., Jarooghi N., Bolurieh T, Abbasi F., Mardpour S., Azimyian V, Moeininia F, Maroufizadeh S., Sanjari L., Hosseini S.E., Aghdami N. Safety, Feasibility of Intravenous and Intrathecal Injection of Autologous Bone Marrow Derived Mesenchymal Stromal Cells in Patients with Amyotrophic Lateral Sclerosis: An Open Label Phase I Clinical Trial. Cell J. 2019; 20 (4): 592-8.
  41. Camilleri E.T, Gustafson M.P., Dudakovic A., Riester S.M., Garces C.G., Paradise C.R., Takai H., Karperien M., Cool S., Sampen H.J., Larson A.N., Qu W, Smith J., Dietz A.B., van Wijnen A.J. Identification and validation of multiple cell surface markers of clinical-grade adipose-derived mesenchymal stromal cells as novel release criteria for good manufacturing practice-compliant production. Stem Cell Res Ther. 2016; 7 (1): 107.
  42. Riordan N.H., Morales I., Fernandez G., Allen N., Fearnot N.E., Leckrone M.E., Markovich D.J., Mansfield D., Avila D., Patel A.N., Kesari S., Paz Rodriguez J. Clinical feasibility of umbilical cord tissue-derived mesenchymal stem cells in the treatment of multiple sclerosis. J. Transl Med. 2018; 16 (1): 57.
  43. Harris V.K., Stark J., Vyshkina T., Blackshear L., Joo G., Stefanova V, Sara G., Sadiq S.A. Phase I Trial of Intrathecal Mesenchymal Stem Cell-derived Neural Progenitors in Progressive Multiple Sclerosis. EBioMedicine. 2018; 29: 23-30.
  44. Karussis D., Karageorgiou C., Vaknin-Dembinsky A, Gowda-Kurkalli B, Gomori JM, Kassis I, Bulte JW, Petrou P., Ben-Hur T, Abramsky O., Slavin S. Safety and immunological effects of mesenchymal stem cell transplantation in patients with multiple sclerosis and amyotrophic lateral sclerosis. Arch Neurol. 2010; 67 (10): 1187-94.

Supplementary files

Supplementary Files
Action
1. JATS XML
Download

This website uses cookies

You consent to our cookies if you continue to use our website.

About Cookies