According to the results of a first-in-human study published in Pharmaceutics, 5-aminolevulinic acid-based extracorporeal photopheresis (ALA-ECP) appears to be safe and tolerable in patients with chronic graft vs host disease (cGVHD).
ALA-ECP could serve as a more selective alternative to 8-methoxypsoralen-ECP, which is used to induce apoptosis of T cells for modulation of immune responses in patients with cGVHD .
To evaluate the safety and tolerability of ALA-ECP in patients with cGVHD, the investigators conducted a prospective, open-label, single-center phase 1(-2) study (ClinicalTrials.gov Identifier: NCT03109353).
The study included 5 adult patients (3 female; 2 male) with cGVHD who previously experienced inadequate responses to 8-MOP-ECP after a minimum of 3 months of treatment at the study center. The mean age of patients at study entry was 48 years (range, 32-60). Patients had been diagnosed with acute myeloid leukemia (n=4) and high-risk myelodysplastic syndrome (n=1) prior to undergoing allogeneic bone marrow transplantation.
Patients received a total of 82 single treatments (42 treatment cycles) of ALA-ECP. Due to disease progression, 1 patient was discharged from the study. No clinically significant changes in patients’ vital signs or laboratory values were observed.
A total of 62 adverse events (AEs) were reported. Mild symptoms (43 of 62) were most common, followed by moderate (17 of 62) and severe (2 of 62) symptoms. An internal safety review committee deemed that none of the AEs were considered to be likely related to the study medication.
The primary limitations of the study were the open-label design and small patient sample size.
“This study met the aim to evaluate safety and tolerability after multiple treatments with ALA-ECP in patients with cGvHD and lays the basis for designing future clinical ALA-ECP studies,” the authors wrote in their report. “Further research is needed to assess the safety and for the optimization of the treatment.”
Disclosure: Some study authors are co-inventors of patent number 10695371. Please see the original reference for a full list of authors’ disclosures.
Christensen E, Foss OA, Quist-Paulsen P, et al. Application of photodynamic therapy with 5-aminolevulinic acid to extracorporeal photopheresis in the treatment of patients with chronic graft-versus-host disease: a first-in-human study. Pharmaceutics. 2021;13(10):1558. doi:10.3390/pharmaceutics13101558