atidarsagene autotemcel at a glance
Medicine class
Autologous, ex-vivo haematopoietic stem-cell gene therapy
Main use
Selected late-infantile or early-juvenile metachromatic leukodystrophy caused by ARSA mutations
Administration
Patient-specific intravenous infusion through a central venous catheter at a qualified treatment centre
Preparation
Requires stem-cell collection, manufacture, myeloablative conditioning and stored back-up stem cells
Follow-up
Regular blood, neurological and long-term gene-therapy safety monitoring
What is atidarsagene autotemcel used for?
UK-licensed use is restricted to genetically confirmed metachromatic leukodystrophy caused by biallelic ARSA mutations with reduced ARSA activity.
Children with late-infantile or early-juvenile disease who have no clinical manifestations.
Children with early-juvenile disease who have early manifestations, can still walk independently and have not developed cognitive decline.
How does atidarsagene autotemcel work?
The child’s CD34-positive blood-forming stem cells are collected and given a working ARSA gene using a lentiviral vector. After engraftment, their descendants produce ARSA and can enter nervous-system tissues. Nearby cells take up secreted ARSA, helping them break down harmful sulfatides.
How to take atidarsagene autotemcel
Atidarsagene autotemcel is prepared individually from the child’s collected stem cells and administered in hospital after myeloablative conditioning.
The treatment centre confirms that every infusion bag matches the intended patient.
The frozen cell dispersion is thawed only when the patient is ready and infused through a central venous catheter.
The child remains under specialist observation for infusion reactions, infection, low blood-cell counts and engraftment.
Keep the Patient Alert Card and show it whenever medical care is received.
atidarsagene autotemcel side effects
Common or expected effects
- A positive blood test for antibodies against ARSA may occur; in studies, these antibodies were generally low-level and resolved spontaneously or with treatment.
- The wider treatment pathway, particularly conditioning, frequently causes low neutrophils, fever with neutropenia, mouth inflammation or sores, vomiting, liver enlargement and impaired ovarian function.
- Other reported conditioning-related effects include infection, anaemia, low platelets, nausea, diarrhoea, headache, fluid retention, nosebleeds and bone or back pain.
Get urgent medical advice
- Severe allergic reaction, including anaphylaxis, associated with the preservative DMSO.
- Severe or prolonged reductions in blood cells, with risks of infection, anaemia and bleeding.
- Liver veno-occlusive disease, metabolic acidosis, serious infection or central-line complications.
- A theoretical long-term risk of leukaemia or lymphoma due to insertion of the modified gene.
atidarsagene autotemcel in pregnancy
Atidarsagene autotemcel is not intended for adults, and there are no human pregnancy data or reproductive animal studies. If pregnancy could be relevant in an exceptional case, the gene-therapy and conditioning teams must assess this before treatment.
atidarsagene autotemcel while breastfeeding
There are no human breastfeeding data because the treatment is not intended for adults. Any exceptional situation involving breastfeeding requires advice from the specialist treatment team before mobilisation or conditioning.
atidarsagene autotemcel interactions
Formal interaction studies have not been performed. The specialist team must review all medicines and vaccinations used around treatment.
Antiretroviral medicines
These must not be used during the specialist-defined period around stem-cell mobilisation and infusion; treatment may need to be delayed if antiretroviral therapy is required.
Live vaccines
Avoid before conditioning and until blood-cell and immune recovery because safety during this period has not been established.
Phenytoin
It may increase clearance of the conditioning medicine busulfan, so it is not recommended for seizure prophylaxis in this setting.
Common questions about atidarsagene autotemcel
Answers are fully visible for fast scanning and source review.
What is atidarsagene autotemcel used for?
It treats selected children with late-infantile or early-juvenile metachromatic leukodystrophy caused by faulty ARSA genes.
Can it be used after symptoms begin?
It may be used in early-symptomatic early-juvenile disease only while the child can still walk independently and before cognitive decline.
How is it made?
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.