Cecilia Götherström, BBC

My goal is to develop cell therapies that will have an impact on the health of persons with severe disorders. An evidence of this is that I have brought a cell therapy from the bench to the bedside.

Cecilia Götherström

Researcher

My goal is to develop cell therapies that will have an impact on the health of persons with severe disorders. An evidence of this is that I have brought a cell therapy from the bench to the bedside.

About me

I am driven by challenges, which has led me to initiate, lead and successfully fulfill several complex projects and educations in the field of regenerative medicine in the academia, the health care sector and in the industry. My motto in life and work is courage and passion, courage for daring to discover new paths, and passion, which is to always go the extra mile.

Research description

Cells derived from the fetal and perinatal tissues have more potential than cells derived from adult tissues. Promising candidates for clinical translation are mesenchymal stem cells (MSC).

My interest in these cells is based on their multipotentiality, low immunogenicity and tissue repairing effects in combination with a minimal oncogeneic risk.

1. BOOSTB4 and BOOST2B: Developing MSC as a therapy for Osteogenesis Imperfecta

In the BOOSTB4 and BOOST2B clinical trials fetal MSC are investigated as a therapy for severe types of Osteogenesis Imperfecta (inherited brittle bone disease). Previous case studies performed by us at KI have demonstrated a potential positive effect. The manufacturing of the MSC is led by Dr Götherström and is performed at Karolinska Center for Cell Therapy at Karolinska University Hospital.

Boost Brittle Bones Before Birth (BOOSTB4) is a European consortium lead by Dr Götherström. The trial is investigating the safety and efficacy of this innovation in the treatment of Osteogenesis Imperfecta in the pediatric population. In the project we will also evaluate ethical standards and risks, acceptability and stakeholders views and experiences and health economics.

Four doses of fetal MSC will be administered after birth (group 1, <18 months of age) or 1 dose before and after 3 doses after birth (group 2). The phase I/II clinical trial included the first participant in 2020. 

The BOOSTB4 consortium includes 12 partners in Sweden, the United Kingdom, Germany and the Netherlands. Dr Götherström at KI is the coordinator for the consortium. The BOOSTB4 project has received funding from the European Union's Horizon 2020 research and innovation programme under grant agreement 681045 and from the Swedish Research Council. See www.BOOSTB4.eu and www.ClinicalTrials.gov (ID: NCT03706482) for more information.

The Boost to Brittle Bones (BOOST2B) is a collaborative project between KI and Christian Medical College in Vellore, India. In this project we investigate the safety and efficacy of fetal MSC in the treatment of severe types of Osteogenesis Imperfecta in children less than 4 years of age. Different delivery routes (intravenous and intraosseous) will also be investigated in this phase I/II clinical trial. The first participant was included in 2020.

The BOOST2B project has received funding from Sweden's innovation agency (Vinnova) and the Department of Biotechnology, Government of India. See www.BOOST2B.in for more information.

In both the BOOSTB4 and BOOST2B projects, the effect and mechanism of fetal MSC will be further investigated in models in vitro and in vivo.

2. Characterizing and comparing MSC from different perinatal sources

We work with many fetal and perinatal sources: fetal tissues (first trimester), amniotic fluid, chorionic villus, biopsies of muscle and skin (second trimester), umbilical cord blood, placenta, Wharton´s Jelly (term).

These cells are evaluated for their potential in perinatal regenerative medicine in clinical application. We also evaluate how maternal and fetal microchimerism, pregnancy, and mode of delivery affect the isolated cells.

3. Developing new clinical treatment options using perinatal MSC for:

  • Severe structural defects diagnosed before birth
  • Life threatening bronchopulmonary dysplasia and necrotizing enterocolitis in premature babies

4. MSC and endometriosis

In this project we examine the involvement of MSC in the chronic inflammatory disease endometriosis.

Teaching portfolio

  • Director of Studies, Doctoral Program in Regenerative Medicine, Karolinska Institutet 2008-2015.
  • Organizer of 5 doctoral courses and 9 scientific conferences in the regenerative medicine field.
  • 250+ hours of teaching on all levels.
  • PhD thesis examiner >10 times, halftime examiner 6 times, and defence chair 5 times.
  • Main supervisor for 2 graduated PhD students 2018, and co-supervisor for 3 PhD students 2009-2014.
  • Supervisor of >10 Master, Bachelor, foreign exchange and summer project students 2005-2020.

Education

Associate Professor (Docent) 2014, Karolinska Institutet, Stockholm, Sweden.

Subject: Stem Cell Science

PhD 2004, Karolinska Institutet, Stockholm, Sweden.

Subject: Allogeneic stem cell transplantation & transplantation research Thesis: Characterization of human fetal mesenchymal stem cells

MSc 2001, Södertörns University College, Stockholm, Sweden.

Subject: Molecular Cell Biology Degree thesis: Clinical aspects on maternal cells in the fetus

Academic honours, awards and prizes

  • Established and lead Sweden’s altruistic Umbilical Cord Blood Bank at Karolinska University Hospital 2010-2016.
  • Secured > 73 million SEK as main Principle Investigator & > 30 million SEK as co-Principle Investigator.
  • Established an approved GMP-production of mesenchymal stem cells.
  • Initiated and lead the European Horizon 2020 BOOSTB4 project and clinical trial.
  • Established two approved and ongoing clinical trials investigating MSC transplantation as a treatment of brittle bone disease (Osteogenesis Imperfecta) in children.
  • Initiated the first clinical trial on prenatal stem cell transplantation in the world.
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