Long COVID Stem Cell Therapy Expanded Access Programme Wins FDA Approval

Key details

• Life-limiting condition: Long COVID’s most common symptoms, fatigue and brain fog, can have a strong negative impact on sufferers’ ability to carry out day-to-day activities, reducing quality of life and placing limits on what they are able to accomplish [1-2].
• Positive early trial results: Results from a recent phase 2 trial of REGENECYTE, an investigational stem cell therapy derived from umbilical cord blood, suggest that the therapy was generally well tolerated and may reduce fatigue in some long COVID patients [3].
• Expanded access programme approval: Following these early findings, the FDA has cleared an expanded access programme for the therapy in the US. This may allow certain patients with long COVID who have limited treatment options to access the therapy while further research is ongoing.
• Further trial planned: A phase 3 trial of REGENECYTE has been planned and is expected to begin in the near future [4].

What is long COVID?

Long COVID, also known as post-COVID syndrome or post-COVID-19 condition, is a group of health issues that persist or develop after an initial infection with COVID-19.

Based on data from November 2023 to March 2024, around 2 million people in the UK (3.3%) reported experiencing long COVID symptoms lasting at least four weeks, and most of those affected (74.7%) said their symptoms were having a noticeable impact on their day-to-day lives [5]. While estimates vary between countries due to differences in how long COVID is defined and measured, some analyses suggest that roughly 2–7% of the population may experience the condition in some form [2,6-9].

Research also suggests that the risk of developing long COVID is not the same for everyone. It may be influenced by factors such as how severe the initial infection was and whether someone was vaccinated. Systematic reviews have identified a range of clinical and demographic risk factors that can increase or reduce this likelihood [8-10].

The condition itself can look very different from person to person, but common symptoms include ongoing fatigue, muscle or joint pain, breathlessness, and difficulties with memory and concentration, often described as “brain fog.” Large-scale global studies have consistently found these symptom patterns, particularly fatigue and cognitive and respiratory issues, to be among the most frequently reported [4,5, 9-10].

There is also growing evidence that long COVID may share similarities with other post-viral conditions such as myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS), especially in relation to persistent fatigue and worsening of symptoms after exertion [11].

The wider impact of long COVID is still being fully understood, but evidence increasingly shows that ongoing symptoms can significantly affect a person’s ability to function in daily life and may limit their capacity to work. This contributes to broader economic and societal pressures [1-2]. Healthcare systems are also affected: a UK analysis of electronic health records found that people with long COVID had higher levels of healthcare use and higher per-person healthcare costs compared with several matched control groups, including people who had COVID-19 but did not go on to develop long COVID [12].

Some groups appear to be more affected than others. For example, individuals with pre-existing disabilities may experience greater functional limitations and a higher burden of symptoms compared with the general population [13].

At present, there is no definitive cure for long COVID. Instead, care focuses on managing symptoms and supporting recovery through approaches such as pacing and fatigue management, physiotherapy, cognitive rehabilitation, and personalised support plans tailored to individual needs [14].

Could cell therapies help with long COVID?

Research into potential treatments for long COVID is ongoing. While the biological mechanisms underlying the condition are still being investigated, current evidence suggests a complex interplay of immune, inflammatory, and vascular factors [2,15].

Early findings from a small phase 2 trial suggest that an investigational haematopoietic stem cell therapy, REGENECYTE, may have the potential to improve fatigue symptoms in some patients. However, these findings are preliminary and require confirmation in larger studies.

The therapy is derived from umbilical cord blood. While cord blood-derived stem cells are approved in the United States for certain blood and immune system disorders, their use in long COVID remains investigational [16-17].

The trial enrolled 30 patients with long COVID whose symptoms had persisted for 6–18 months. Participants were randomly assigned to receive either the stem cell therapy (administered as three IV infusions at three-week intervals) or placebo saline infusions [3].

The primary objective of the trial was to assess safety, with secondary objectives including evaluation of fatigue and other health outcomes. The therapy was reported to be generally well tolerated in this small group, with no serious treatment-related adverse events observed. Some participants experienced mild to moderate side effects, including localised allergic reactions and transient symptoms such as shortness of breath or anxiety.

Fatigue was assessed using the Chalder Fatigue Scale (CFQ-11). The treatment group showed improvements in fatigue scores compared with baseline, with the greatest gains seen in physical fatigue. Participants also reported improvements in quality of life. However, there were no statistically significant differences between the treatment and control groups in frailty or cognitive measures [3].

Overall, while the results are encouraging, the small sample size means they should be interpreted with caution.

Expanded access and future trial steps

Following these early findings, the FDA has cleared an expanded access programme for REGENECYTE in the United States. Expanded access (also known as compassionate use) allows certain patients with serious or life-impacting conditions to access investigational therapies outside of clinical trials, where appropriate, while further evidence is gathered [18].

In parallel, a phase 3 clinical trial is planned for the near future to further evaluate the safety and effectiveness of the therapy in a larger patient population [4].

Although this therapy is still at an early stage of clinical development and is not currently part of standard care, the trial findings and expanded access programme highlight the ongoing scientific interest in the potential clinical applications of umbilical cord–derived stem cells.

To learn more about stem cells found in a baby’s umbilical cord and current research into their potential future medical uses, you can request our free guide by completing the form below.

References

[1] Ford, N.D., Slaughter, D., Edwards, D. et al. (2023) ‘Long COVID and significant activity limitation among adults, by age — United States, June 1–13, 2022, to June 7–19, 2023’, Morbidity and Mortality Weekly Report, 72, pp. 866–870. http://dx.doi.org/10.15585/mmwr.mm7232a3

[2] Al-Aly, Z., Davis, H., McCorkell, L. et al. (2024) ‘Long COVID science, research and policy’, Nature Medicine, 30, pp. 2148–2164. https://doi.org/10.1038/s41591-024-03173-6

[3] Huang YW, Chen YC, Lun Lau EY, Su YC, Tai LK, Rosenthal J, Wang J, Lee TY. REGENECYTE cord blood cell therapy in post-COVID syndrome: a phase IIa randomized, placebo-controlled trial. EClinicalMedicine. 2026 Jan 9;91:103737. doi: 10.1016/j.eclinm.2025.103737. PMID: 41625963; PMCID: PMC12853356.

[4] Clinicaltrials.gov. A Study of Human Umbilical Cord Blood (REGENECYTE) Infusion in Patients With Post-COVID Condition. https://clinicaltrials.gov/study/NCT07184385

[5] Self-reported coronavirus (COVID-19) infections and associated symptoms, England and Scotland – Office for National Statistics. https://www.ons.gov.uk/peoplepopulationandcommunity/healthandsocialcare/conditionsanddiseases/articles/selfreportedcoronaviruscovid19infectionsandassociatedsymptomsenglandandscotland/november2023tomarch2024

[6] Vos, T. et al. (2022) ‘Estimated global proportions of individuals with persistent fatigue, cognitive, and respiratory symptom clusters following symptomatic COVID-19 in 2020 and 2021’, JAMA, 328, pp. 1604–1615.

[7] Greenhalgh T, Sivan M, Perlowski A, Nikolich JŽ. Long COVID: a clinical update. Lancet. 2024 Aug 17;404(10453):707-724. doi: 10.1016/S0140-6736(24)01136-X. Epub 2024 Jul 31. PMID: 39096925.

[8] Tsampasian, V., Elghazaly, H., Chattopadhyay, R., Debski, M., Naing, T.K.P., Garg, P., Clark, A., Ntatsaki, E. and Vassiliou, V.S. (2023) ‘Risk factors associated with post-COVID-19 condition: a systematic review and meta-analysis’, JAMA Internal Medicine, 183(6), pp. 566–580. https://doi.org/10.1001/jamainternmed.2023.0750

[9] World Health Organization. Post COVID-19 condition (long COVID). https://www.who.int/news-room/fact-sheets/detail/post-covid-19-condition-(long-covid)

[10] Byambasuren, O., Stehlik, P., Clark, J. et al. (2023) ‘Effect of COVID-19 vaccination on long COVID: systematic review’, BMJ Medicine, 2, e000385. https://doi.org/10.1136/bmjmed-2022-000385

[11] Dehlia A, Guthridge MA. The persistence of myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) after SARS-CoV-2 infection: A systematic review and meta-analysis. J Infect. 2024 Dec;89(6):106297. doi: 10.1016/j.jinf.2024.106297. Epub 2024 Sep 29. PMID: 39353473.

[12] Mu Y, Dashtban A, Mizani MA, Tomlinson C, Mohamed M, Ashworth M, Mamas M, Priedon R, Petersen S, Kontopantelis E, Horstmanshof K, Pagel C, Hocaoğlu M, Khunti K, Williams R, Thygesen J, Lorgelly P, Gomes M, Heightman M, Banerjee A; CVD-COVID-UK/COVID-IMPACT Consortium. Healthcare utilisation of 282,080 individuals with long COVID over two years: a multiple matched control, longitudinal cohort analysis. J R Soc Med. 2024 Nov;117(11):369-381. doi: 10.1177/01410768241288345. Epub 2024 Nov 27. PMID: 39603265; PMCID: PMC11603423.

[13] Hall, J.P., Kurth, N.K., McCorkell, L. and Goddard, K.S. (2024) ‘Long COVID among people with preexisting disabilities’, American Journal of Public Health, 114, pp. 1261–1264. https://doi.org/10.2105/AJPH.2024.307794

[14] NHS. Long COVID. https://www.nhs.uk/conditions/long-covid/

[15] Peluso, M.J. and Deeks, S.G. (2024) ‘Mechanisms of long COVID and the path toward therapeutics’, Cell, 187(20), pp. 5500–5529. https://doi.org/10.1016/j.cell.2024.07.054

[16] USFDA Approves StemCyte BLA for RegeneCyte. StemCyte. https://www.stemcyte.com/usfda-approves-stemcyte-bla

[17] U.S. Food and Drug Administration. REGENECYTE. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenecyte

[18] PRNewsWire. StemCyte Announces FDA Clearance of Expanded Access Program for RegeneCyte® (HPC, Cord Blood) to Address Unmet Need in Long COVID. https://www.prnewswire.com/news-releases/stemcyte-announces-fda-clearance-of-expanded-access-program-for-regenecyte-hpc-cord-blood-to-address-unmet-need-in-long-covid-302669830.html