In short
PEI 15-216 is an investigational cell therapy being studied in patients with newly diagnosed diffuse intrinsic pontine glioma (DIPG), a rare and aggressive brain tumour that occurs mainly in children. It is a cell suspension for injection given into the skin (intradermally). The substance is under investigation in one authorised Phase 1 clinical trial with 15 planned participants, which is testing its safety in combination with another cell therapy.
Key points
- PEI 15-216 is an investigational cell therapy, not an authorised medicine.
- It is being studied in newly diagnosed diffuse intrinsic pontine glioma (DIPG), a rare brain tumour.
- The only clinical trial is a Phase 1 study with a planned enrolment of 15 participants.
- The trial is authorised and is testing the safety of a combination immunotherapy approach.
- PEI 15-216 is given as a cell suspension for intradermal injection.
- The main measure is the number of Grade 3–4 serious adverse events during the study.
What is PEI 15-216?
PEI 15-216 is an investigational cell therapy. It is classified in the registry as a structurally diverse substance of cell therapy origin, meaning it is a product made from living cells rather than a chemically synthesised medicine. In the product records, the substance is listed under the product name DIPG-lysate. It has no marketing authorisation in the product records, so it is not yet an approved medicine.
The therapeutic area for this substance is neoplasms, which are abnormal growths of tissue that can form tumours. The product is supplied by Fundació de Recerca Clínic Barcelona–Institut d'Investigacions Biomèdiques August Pi i Sunyer.
How does PEI 15-216 work?
PEI 15-216 is a lysate derived from a pool of eight DIPG K27M-positive tumour cell lines. A lysate is a preparation made by breaking open cells so that their contents are released. In the clinical trial, this lysate is used to pulse dendritic cells, which are immune cells that help the body recognise foreign or abnormal material. The pulsed dendritic cells are then given back to the patient as an immunisation, with the aim of helping the immune system recognise and respond to the tumour.
Which conditions is PEI 15-216 studied for?
- Newly diagnosed diffuse intrinsic pontine glioma (DIPG) — a rare and aggressive brain tumour that arises in the brainstem, most often in children. The trial is studying the substance in patients who have just received this diagnosis.
How is PEI 15-216 given?
PEI 15-216 is a cell suspension for injection, and it is given by intradermal injection, which means it is injected into the skin. In the trial, it is not given on its own but as part of a combination: dendritic cells pulsed with the DIPG-lysate are given intradermally, while a separate CAR-T cell product called ARI0008 is given into the fluid-filled spaces of the brain (intrathecally).
What do the clinical trials show?
There is one clinical trial of PEI 15-216 in the registry. It is a Phase 1 study, which is the earliest stage of testing in people and focuses mainly on safety. The trial is authorised, meaning it has received regulatory approval to proceed.
The trial plans to enrol 15 participants with newly diagnosed DIPG. The main outcome being measured is the number of Grade 3–4 serious adverse events, graded according to Common Toxicity Criteria, from the first administration of dendritic cells until the end of the study for each patient.
The study is a first-in-human trial, meaning it is the first time this combination of treatments is being given to people. It evaluates the safety of dendritic cell immunisation with DIPG-DC pulsed with DIPG-lysate, together with anti-IL13Ra2 CAR-T cells (ARI0008) that are derived from T cells previously stimulated by the dendritic cells.
What side effects are known?
The registry data for this trial does not list a separate side effect profile for PEI 15-216. Because this is a first-in-human Phase 1 study, the main goal is to find out what side effects occur. The primary outcome measure is the number of Grade 3–4 serious adverse events, which are severe or life-threatening side effects, recorded from the start of treatment until the end of the study.