Skip to main content

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.

Call for proposal

H2020-PHC-2014-2015

See other projects for this call

Sub call

H2020-PHC-2015-single-stage_RTD

Coordinator

KAROLINSKA INSTITUTET
Net EU contribution
€ 1 605 091,75
Address
Nobels Vag 5
17177 Stockholm
Sweden

See on map

Region
Östra Sverige Stockholm Stockholms län
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 1 769 996,50

Participants (13)

Third-party

Legal entity other than a subcontractor which is affiliated or legally linked to a participant. The entity carries out work under the conditions laid down in the Grant Agreement, supplies goods or provides services for the action, but did not sign the Grant Agreement. A third party abides by the rules applicable to its related participant under the Grant Agreement with regard to eligibility of costs and control of expenditure.

REGION STOCKHOLM
Sweden
Net EU contribution
€ 1 750 688,00
Address
Hantverkargatan 45
104 22 Stockholm

See on map

Region
Östra Sverige Stockholm Stockholms län
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
Non-EU contribution
€ 0,75
UNIVERSITY COLLEGE LONDON
United Kingdom
Net EU contribution
€ 416 336,00
Address
Gower Street
WC1E 6BT London

See on map

Region
London Inner London — West Camden and City of London
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,25
ACADEMISCH ZIEKENHUIS LEIDEN
Netherlands
Net EU contribution
€ 239 392,00
Address
Albinusdreef 2
2333 ZA Leiden

See on map

Region
West-Nederland Zuid-Holland Agglomeratie Leiden en Bollenstreek
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,50
KLINIKUM DER UNIVERSITAET ZU KOELN
Germany
Net EU contribution
€ 343 462,00
Address
Kerpener Strasse 62
50937 Koeln

See on map

Region
Nordrhein-Westfalen Köln Köln, Kreisfreie Stadt
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,50
GREAT ORMOND STREET HOSPITAL FOR CHILDREN NHS FOUNDATION TRUST
United Kingdom
Net EU contribution
€ 1 088 078,00
Address
Great Ormond Street
WC1N 3JH London

See on map

Region
London Inner London — West Camden and City of London
Activity type
Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
Non-EU contribution
€ 0,75
LUNDS UNIVERSITET
Sweden
Net EU contribution
€ 241 156,00
Address
Paradisgatan 5C
22100 Lund

See on map

Region
Södra Sverige Sydsverige Skåne län
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,25
XNK THERAPEUTICS AB
Sweden
Net EU contribution
€ 209 522,00
Address
Halsovagen 7 Novum
141 57 Huddinge

See on map

SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
Östra Sverige Stockholm Stockholms län
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,50
PHARMALEX SWEDEN AB
Sweden
Net EU contribution
€ 106 000,00
Address
Medicinaregatan 8B
413 46 Goteborg

See on map

SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
Södra Sverige Västsverige Västra Götalands län
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00
MEDSCINET AB
Sweden
Net EU contribution
€ 121 093,75
Address
Luntmakargatan 71
11351 Stockholm

See on map

SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
Östra Sverige Stockholm Stockholms län
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00
EURAM LIMITED

Participation ended

United Kingdom
Net EU contribution
€ 77 275,00
Address
Lucy Tower Street Tower House
LN1 1XW Lincoln

See on map

SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00
UNIVERSITY OF LEICESTER
United Kingdom
Net EU contribution
€ 50 000,00
Address
University Road
LE1 7RH Leicester

See on map

Region
East Midlands (England) Leicestershire, Rutland and Northamptonshire Leicester
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 0,00
UNIVERSITAIR MEDISCH CENTRUM UTRECHT
Netherlands
Net EU contribution
€ 101 835,00
Address
Heidelberglaan 100
3584 CX Utrecht

See on map

Region
West-Nederland Utrecht Utrecht
Activity type
Higher or Secondary Education Establishments
Non-EU contribution
€ 1,25
A2F ASSOCIATES LIMITED
United Kingdom
Net EU contribution
€ 258 825,00
Address
7 Laureate Paddocks
CB8 0AP Newmarket

See on map

SME

The organization defined itself as SME (small and medium-sized enterprise) at the time the Grant Agreement was signed.

Yes
Region
East of England East Anglia Suffolk
Activity type
Private for-profit entities (excluding Higher or Secondary Education Establishments)
Non-EU contribution
€ 0,00