Menkes International — Free Treatment Program
NPP-Menkes 2026
Named-Patients Program for Menkes Treatment with Elesclomol-Copper (ES-Cu) — coordinated by Menkes International Association, Copper Rare Foundation (MICu).
What is NPP-Menkes?
The Named-Patient Program (NPP-Menkes) is an experimental treatment program using Elesclomol-Copper (ES-Cu) for children with Menkes disease, launched in 2020 following the publication of treatment results in Science magazine (Guthrie et al., 2020).
This initiative was led by Aurora Mateos, the mother of the first child treated. It was organized within the framework of Menkes International (MIa), now a Foundation (MICu), in collaboration with James Sacchettini (Texas A&M), Denis Broun (MICu), Yusuf Hamied (CIPLA) and Francesc Palau from Hospital Sant Joan de Déu.
The program is coordinated by the Copperless Committee (MICu's Scientific Advisory Board). Treatment is provided completely free of charge. As of January 2026, nine children are being treated — 7 in Spain and 2 in Switzerland — authorized by AEMPS and Swissmedic respectively.
9
Children treated
2
Countries (ES + CH)
2020
Program launched
Key Milestones
May 2020
Publication of Es-Cu efficacy in Science (Guthrie et al., 2020) — research by V. Gohil and J. Sacchettini.
Nov 2020
Foundation of Menkes International by A. Mateos, MJ. Portillo and JV. Mateos.
Dec 2020
Set up of the 3 working groups (pharma, legal, clinical) for the NPP.
May 2021
Establishment of the Copperless Committee as MICu's Scientific Advisory Board. Therapeutic protocol drafted by M. Petris & C. Papageorgiou, calculated by J. Standing and V. Mangas.
Sep 2021
Synthesis of the ES-Cu molecule by Yusuf Hamied (CIPLA), donated to MICu and deposited at HSJD.
Oct 2021
Approval from AEMPS and clearance by HSJD Clinical Ethics Committee for the first child.
Jan 2022
Administration of the first dose of ES-Cu on January 30, 2022, at Hospital Sant Joan de Déu.
Jul 2025
First study showing efficacy of ES-Cu in two children published in the Journal of Clinical Investigation.
Jan 2026
Nine children treated in Spain (7) and Switzerland (2), authorized by AEMPS and Swissmedic.
Services Provided by Menkes International
1
Active Pharmaceutical Ingredient (API)
Donation of the API (not ready-to-use) free of charge.
2
Formulations
Facilitation of contact with authorized hospitals experienced in hospital-based preparation of ready-to-use subcutaneous formulations, where locally permitted (e.g., within the EU).
3
Dosing & Protocols
Scientific advice on dosing criteria and treatment approaches, provided by the Copperless Committee at the request of the local treating physician.
4
Scientific Support & Clinical Advice
Provided to the attending physicians by the Copperless Committee and the Core Team (medical and pharmacological team of MICu).
5
Collaboration with Authorized Stakeholders
Coordination with regulatory bodies, pharmacies and hospital teams across countries.
6
Support to Families
By the Menkes International Families Committee — from diagnosis to ongoing treatment.
Ten Steps for Accessing NPP-Menkes
How to access the Named-Patients Program with Elesclomol-Copper through Menkes International.
1Formal request to MICu by the patient's family and medical team
The attending physician evaluates the clinical situation and initiates a named-patient request. The family provides informed consent. MICu forwards the query to its Copperless Committee and Core Team.
2Approval by the ad hoc Ethics Committee
The Ethics Committee at the patient's hospital must approve the NPP-Menkes for the individual patient. MICu delegates all ethical aspects to this committee.
3Authorization by the national medicines agency
The request is formally submitted by the local treating hospital to the competent national authority (AEMPS in Spain; Swissmedic in Switzerland). Authorization is individual, not programmatic.
4Formulation arrangements
MICu provides the API in powder form. It must be formulated by an authorized hospital pharmacy before administration. Inside the EU, ready-to-use subcutaneous formulations are supplied; outside the EU, local preparation may be considered if authorized.
5Shipping
Inside the EU: the cooperating hospital pharmacy in Spain (Hospital of León or Hospital Sant Joan de Déu) ships the ready-to-use drug at 5°C after all legal procedures are completed.
6Basal tests
The clinical team administers the treatment with scientific support from the Copperless Committee and Core Team to ensure the highest level of therapeutic safety.
7Training of the patient medical team
Core Team members conduct an on-site visit to examine the patient and advise the medical team. Scientific exchange sessions are available remotely or during institutional meetings.
8Dosing plan
The Copperless Committee proposes an individualized dosing plan. The attending physician makes all final decisions on initiation, continuation, and discontinuation of treatment.
9Beginning the NPP
The family signs an informed consent and a liability release agreement. Clinical monitoring, follow-up, and pharmacovigilance are performed by the local medical team. Weekly contact from MICu.
10Information-sharing
Patient data, freely shared by families, is stored in the MICu repository and accessible to researchers after internal clearance. All data is anonymized under the EU GDPR framework.
Interested in the NPP-Menkes?
The full brochure (ISBN 978-84-09-83845-5) contains complete protocols, regulatory guidance, and references. For individual patient requests, contact our director.