Positive Phase 2a Trial Supports FDA Clearance of StemCyte’s HPC Therapy for Long COVID Patients

The FDA has cleared an expanded access program for hematopoietic progenitor cell (HPC) therapy (RegeneCyte; StemCyte Inc), indicated for patients suffering from severe effects of post-acute sequelae of SARS-CoV-2, also recognized as long COVID.^1,2

"The FDA's clearance of this program is a significant validation of the safety and therapeutic potential of RegeneCyte, our HPC-cord blood product," Tong-Young Lee, PhD, CEO of StemCyte, said in a news release. "With our RMAT (Regenerative Medicine Advanced Therapy) designation already in place, this program allows us to bridge the gap for patients in need as we continue advancing RegeneCyte through the clinical development and regulatory pathway."¹

RegeneCyte is an allogeneic cord blood-derived hematopoietic progenitor cell therapy used in unrelated donor transplants. It is administered with an appropriate preparative regimen to restore blood and immune function in patients with inherited or acquired blood disorders or in those whose hematopoietic system has been damaged by myeloablative treatment.²

Safety and Efficacy Outcomes From a Phase 2a Study of HPC Cord Blood in Long COVID

In the 2-arm, single-blind, randomized, placebo-controlled phase 2a study (NCT05682560), researchers aimed to assess the safety and efficacy of HPC infusion in patients with long COVID. StemCyte announced positive trial results showing that its HPC cord blood therapy was successful in treating patients with long COVID symptoms.^1,3,4

The company received FDA approval to initiate the phase 2a clinical trial. Researchers conducted the trial in the US with 30 participants. A total of 22 participants received the HPC cord blood treatment while 10 received a placebo. The Chalder Fatigue Questionnaire (CFQ-11) was used to measure fatigue severity.^1,3

Results showed significant improvement in overall fatigue symptoms, fatigue severity, and physical fatigue among long COVID patients treated with the HPC. Specifically, 85% of patients treated with the HPC reported complete relief from fatigue, compared with only 20% of those receiving placebo, meeting the trial’s secondary end points.^1,3

The trial met its primary safety end point based on treatment-emergent adverse events (TEAE). In the HPC cord blood group, 2 of 20 participants experienced mild TEAEs with no severe adverse events, all of which resolved within 1 hour, resulting in a 10% incidence rate. In the placebo group, 2 of 10 participants experienced TEAEs with no severe adverse events, for an incidence rate of 20%, and all events were unrelated to treatment and resolved with appropriate care. Overall, the results indicate that HPC cord blood therapy was safe and well tolerated in post-COVID patients.³

The PGI-S (Patient Global Impression of Severity) scale showed significant improvements in fatigue symptoms among patients treated with HPC cord blood at weeks 12, 18, and 26 compared with placebo, consistent with findings from the CFQ-11 scale. Cognitive function, assessed using the MoCA (Montreal Cognitive Assessment), improved slightly after cord blood treatment, with average scores increasing from 24 at baseline to 26 at weeks 12 and 26, though these changes were not statistically significant compared with placebo.³

The study authors noted that the phase 2a trial results showed that HPC cord blood therapy was safe and effective, with significant improvements in fatigue symptoms among long COVID patients. Based on these promising findings, StemCyte plans to accelerate the development of RegenCyte and is in ongoing discussions with the FDA to advance to the next phase of clinical trials.^1,3

Understanding Long COVID

Long COVID is defined as a chronic condition triggered by the virus that causes COVID-19. More than 200 symptoms have been linked to long COVID, which could stay the same over time, get worse, or go away and come back. Common symptoms of long COVID include extreme tiredness, brain fog, lightheadedness, problems with taste or smell, sleep problems, shortness of breath, cough, headache, a fast or irregular heartbeat, and digestive problems.⁵

Some individuals with long COVID may also have other underlying illnesses or develop conditions caused or worsened by the syndrome, including migraine, lung disease, autoimmune disease, and chronic kidney disease. Long COVID can lead to diagnoses such as heart disease, mood disorders, anxiety, stroke, postural orthostatic tachycardia syndrome, myalgic encephalomyelitis/chronic fatigue syndrome, mast cell activation syndrome, fibromyalgia, diabetes, and hyperlipidemia.⁵

Long COVID symptoms can occur weeks or months after an individual seems to be recovered from COVID-19, even if they never experienced COVID-19 symptoms.⁵

REFERENCES

1. StemCyte Announces FDA Clearance of Expanded Access Program for RegeneCyte® (HPC, Cord Blood) to Address Unmet Need in Long COVID. News release. StemCyte. January 26, 2026. Accessed January 27, 2026. 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

2. REGENECYTE. FDA. Cellular & Gene Therapy Products. Accessed January 27, 2026. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenecyte

3. StemCyte Announces Successful Phase IIa Clinical Trial Results for HPC, Cord Blood in Treating Post-COVID Syndrome: 85% of Patients Experience Complete Relief from Fatigue Symptoms. StemCyte. December 20, 2024. Accessed January 27, 2026. https://www.stemcyte.com/long-covidclinicaltrialresults

4. Human Umbilical Cord Blood (RegeneCyte) Infusion in Patients with Post-COVID Syndrome. Updated March 3, 2025. Accessed January 27, 2026. https://clinicaltrials.gov/study/NCT05682560

5. Mayo Clinic Staff. Long COVID: Lasting effects of COVID-19. News release. Mayo Clinic. August 23, 2024. Accessed January 27, 2026. https://www.mayoclinic.org/diseases-conditions/coronavirus/in-depth/coronavirus-long-term-effects/art-20490351