Cecilia Götherström

Cecilia Götherström

Developing pioneering fetal stem cell therapies and leading first‑in‑human clinical trials to create disease‑modifying treatments for children with severe congenital disorders.

Telephone: +46852483395
Visiting address: Hälsovägen, Enheten för obstetrik och gynekologi K65, 14186 Stockholm
Postal address: H9 Klinisk vetenskap, intervention och teknik, H9 CLINTEC Obstetrik o gynekologi, 141 52 Huddinge
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About me

  • I am a senior researcher and Associate Professor in Stem Cell Science at Karolinska Institutet, dedicated to translating fetal mesenchymal stem cells (MSC) into disease‑modifying therapies for severe congenital disorders. My work spans basic stem‑cell biology, stem cell transplantation, GMP‑grade manufacturing, and leadership of first‑in‑human clinical trials, including the world's first prenatal MSC transplantation for Osteogenesis Imperfecta (OI).

    I lead the Horizon 2020 funded BOOSTB4 consortium and clinical trial and head the Reproductive Medicine Laboratory at Karolinska University Hospital that perform highly specialized fertility treatments. In 2010 I established and headed Sweden’s altruistic Umbilical Cord Blood Bank at
    Karolinska University Hospital until 2016. My work is characterized by translational impact, multi‑stakeholder collaboration, and development of advanced therapy medicinal products (ATMPs).

    I have secured more than 110 million SEK as Principal Investigator and co‑founded BOOST Pharma ApS, developing BT‑101, the first disease‑modifying MSC therapy for OI.

Research

  • My research focuses on fetal and perinatal mesenchymal stem cells (MSC), which show strong proliferative, immunomodulatory, and osteogenic capabilities. The goal is to develop ATMPs for rare pediatric skeletal and connective‑tissue disorders.

    Key research areas:

    • Stem cell biology, mechanisms of osteogenesis, MSC engraftment, collagen repair, and immune interactions in early life.
    • GMP manufacturing and clinical use of fetal MSCs across multiple international sites.
    • Translational development of fetal MSC therapy for OI.
    • BOOSTB4 & BOOST2B – prenatal and postnatal MSC therapy for severe OI, demonstrating significant reductions in fractures and improvements in early childhood outcomes.
    • Ethical, societal, and health‑economic considerations related to early‑life ATMP interventions.

Teaching

  • I have extensive experience in teaching and supervision within regenerative medicine and translational cell therapy.

    • Director of Studies for the Doctoral Program in Regenerative Medicine (2008–2015) that I established and headed, and established more than 25 courses.
    • More than 270 hours of teaching across doctoral, medical, and clinical training levels.
    • Supervisor or co‑supervisor for multiple PhD and MSc/BSc students.
    • Examiner at numerous PhD defenses, halftime reviews, and dissertation events.

Articles

All other publications

Grants

  • VINNOVA
    12 January 2026 - 31 December 2028
  • Developing fetal mesenchymal stem cells into a treatment for Osteogenesis Imperfecta.
    Karolinska Institutet
    1 October 2025 - 30 September 2029
  • Swedish Research Council
    1 January 2025 - 31 December 2027
    Our aim is to harness the full potential of our recently developed fetal mesenchymal stem cell (fMSC) therapy. We are currently about to change the lives of people living with Osteogenesis Imperfecta (OI), by the demonstrated 76% reduction in fracture frequency in 18 children with severe OI in the VR-supported BOOSTB4 trial. OI is a devastating congenital disease, resulting in hundreds of fractures, some already before birth, deformed long bones, short stature and an chronic pain.The unmet medical need is striking, and we are determined to pursue our quest to take this very promising therapy to the patients. For this to materialize some topics must be addressed, including 1) identifying the true bone-forming stem cell in the starting material, 2) investigate cell stress and interactions between fMSCs and OI-osteoblasts and, 3) evaluating efficacy of the enriched fMSCs in vitro and in an OI zebrafish model. The project will be performed at Karolinska Institutet over 3 years.The benefits of this novel stem cell treatment are vast
    it is the only treatment being developed for OI that addresses the underlying molecular defect (it supplements healthy collagen and bone), it does not require pre- or post-conditioning and treatment can be started before or at birth. In conclusion, we have performed a successful clinical trial with impressive results and need to fill the gaps as described in this application before making this a therapy helping children with OI to get better lives.
  • Developing fetal mesenchymal stem cells into a treatment for Osteogenesis Imperfecta.
    Center for Innovative Medicine
    1 January 2025 - 31 December 2027
  • Foundation Freemason Child House
    1 January 2024 - 31 December 2024
  • Boost Brittle Bones Before Birth – a clinical trial to treat severe brittle bone disease before and after birth.
    Region Stockholm
    1 January 2023 - 31 December 2025
  • Swedish Research Council
    1 January 2022 - 31 December 2023
  • BOOSTB4 – en klinisk stamcells-studie för behandling av svår medfödd benskörhet hos små barn.
    Foundation Freemason Child House
    1 January 2022 - 31 December 2022
  • Swedish Research Council
    1 January 2021 - 31 December 2023
  • BOOSTB4: Boost Brittle Bones Before Birth – a clinical trial to treat severe OI before and after birth
    Center for Innovative Medicine
    1 January 2021 - 31 December 2023
  • Boost Brittle Bones Before Birth – a clinical trial to treat severe OI before and after birth.
    Region Stockholm
    1 January 2021 - 31 December 2022
  • VINNOVA
    1 May 2017 - 30 June 2022
  • European Commission
    1 January 2016 - 31 December 2022
  • Challenges in collection of umbilical cord blood for treatment of leukemia.
    Region Stockholm
    1 January 2015 - 31 December 2015
  • Swedish Research Council
    1 January 2014 - 31 December 2017
  • Wait a minute? Challenges in altruistic donation of UCB.
    Barncancerfonden
    1 January 2012 - 31 December 2014
  • Challenges in collection of UCB for leukemia.
    Dr Åke Olsson Foundation for Haematological Research
    1 January 2012 - 31 December 2013
  • How can collection of UCB to Sweden’s national UCB bank be improved?
    Region Västra Götaland
    1 January 2012 - 31 December 2012
  • In utero transplantation of human fetal MSC.
    Swedish Society for Medical Research
    1 January 2007 - 31 December 2010
  • In utero transplantation of human fetal MSC.
    Karolinska Institutet
    1 January 2006 - 31 December 2006

Employments

  • Director of Studies, Department of Clinical Science, Intervention and Technology, Karolinska Institutet, 2008-2008
  • Postdoctoral Researcher, Department of Laboratory Medicine, Karolinska Institutet, 2007-2007
  • Researcher, Department of Laboratory Medicine, Karolinska Institutet, 2006-2006
  • Phd Student, Department of Laboratory Medicine, Karolinska Institutet, 2003-2004
  • Clinical Position, ME Gynekologi och Reproduktionsmedicin, Karolinska Universitetssjukhuset

Degrees and Education

  • Docent, Karolinska Institutet, 2014
  • Doctor Of Philosophy, Department of Laboratory Medicine, Karolinska Institutet, 2004

Visiting research fellowships

  • Post Doc, Imperial College London, 2005-2006

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