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Boost Brittle Bones Before Birth

Objective

Osteogenesis imperfecta (OI) is, in its severe forms, a devastating inherited disorder characterised by brittle bones. A person with severe OI is affected throughout their lifetime with repeated, multiple fractures, considerable pain and handicap. There is no curative or effective treatment for OI. Our preclinical studies and initial clinical cases have demonstrated that transplantation of fetal mesenchymal stem cells (MSC) is a promising approach for treatment of OI. We receive regular requests for MSC transplantation from patients and their physicians; patient organisations support our approach.
The principal objective of the BOOSTB4 project is to conduct a Phase I/II clinical trial of the safety and efficacy of pre- and/or postnatal MSC transplantation in the severest forms of OI (type III, severe type IV). Transplantation before birth at the onset of disease should lead to greater efficacy and engraftment with less rejection than transplantation after birth. Postnatal transplantation will be evaluated in cases where prenatal diagnosis was not made. The trial’s primary outcome is safety; secondary outcomes relate to efficacy (fracture frequency, growth, bone mineral density and quality of life). All patients will undergo molecular diagnosis to confirm OI before inclusion in the trial. Non-invasive prenatal diagnosis will be developed and validated.
The BOOSTB4 consortium is led by experts in MSC, prenatal therapy and OI at the Karolinska Institutet (KI), which will also lead the international multicentre trial; five additional EU centres of excellence are included. Ethical and regulatory applications are underway to conduct this clinical trial. These are facilitated by the ethical and regulatory approvals for prenatal MSC transplantation in 10 cases of OI that have already been granted at KI.
Successful prenatal transplantation represents a major step forward in the management of patients with severe OI, and beyond, to a range of other inherited birth defects.

Field of science

  • /medical and health sciences/medical biotechnology/cells technologies/stem cells
  • /medical and health sciences/clinical medicine/obstetrics and gynaecology/obstetrics/postnatal
  • /medical and health sciences/clinical medicine/transplantation
  • /social sciences/sociology/demography/fertility

Call for proposal

H2020-PHC-2015-single-stage_RTD
See other projects for this call

Funding Scheme

RIA - Research and Innovation action

Coordinator

KAROLINSKA INSTITUTET
Address
Nobels Vag 5
17177 Stockholm
Sweden
Activity type
Higher or Secondary Education Establishments
EU contribution
€ 1 827 061,50

Participants (12)

UNIVERSITY COLLEGE LONDON
United Kingdom
EU contribution
€ 648 543,75
Address
Gower Street
WC1E 6BT London
Activity type
Higher or Secondary Education Establishments
ACADEMISCH ZIEKENHUIS LEIDEN
Netherlands
EU contribution
€ 408 395
Address
Albinusdreef 2
2333 ZA Leiden
Activity type
Higher or Secondary Education Establishments
KLINIKUM DER UNIVERSITAET ZU KOELN
Germany
EU contribution
€ 703 132,50
Address
Kerpener Strasse 62
50937 Koeln
Activity type
Higher or Secondary Education Establishments
GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST
United Kingdom
EU contribution
€ 1 410 493,25
Address
Great Ormond Street
WC1N 3JH London
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
LUNDS UNIVERSITET
Sweden
EU contribution
€ 241 156
Address
Paradisgatan 5C
22100 Lund
Activity type
Higher or Secondary Education Establishments
XNK THERAPEUTICS AB
Sweden
EU contribution
€ 549 781,25
Address
Halsovagen 7 Novum
141 57 Huddinge
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
NORDIC HEALTH ECONOMICS AB
Sweden
EU contribution
€ 106 000
Address
Medicinaregatan 8B
413 46 Goteborg
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
MEDSCINET AB
Sweden
EU contribution
€ 121 093,75
Address
Luntmakargatan 71
11351 Stockholm
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
EURAM LIMITED

Participation ended

United Kingdom
EU contribution
€ 77 275
Address
Lucy Tower Street Tower House
LN1 1XW Lincoln
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
UNIVERSITY OF LEICESTER
United Kingdom
EU contribution
€ 50 000
Address
University Road
LE1 7RH Leicester
Activity type
Higher or Secondary Education Establishments
UNIVERSITAIR MEDISCH CENTRUM UTRECHT
Netherlands
EU contribution
€ 262 097,50
Address
Heidelberglaan 100
3584 CX Utrecht
Activity type
Higher or Secondary Education Establishments
A2F ASSOCIATES LIMITED
United Kingdom
EU contribution
€ 203 725
Address
7 Laureate Paddocks
CB8 0AP Newmarket
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)