BOOST Pharma
Treating the untreatable
BOOST Pharma – Treating the untreatable
BOOST Pharma is based on many years of collaborative research from Karolinska Institute in Stockholm with the focus on a novel cell therapy treatment for Osteogenesis Imperfecta. The research teams of associate professor Cecilia Götherström and professor Magnus Westgren have shown that that treatment with BOOST Cells greatly enhanced the quality of life for patients suffering from this otherwise extremely debilitating disease.
Osteogenesis Imperfecta
Brittle Bone Disease
The greatest wish of every parent, every family, is to have their children grow up with a bright future, the ability to pursue their dreams, and life in perfect, or at the very least modest, health.
Unfortunately, for parents with children suffering from Osteogenesis Imperfecta, this is not the case. This devastating inherited disease, which is usually abbreviated as OI, leaves patients bound to a life filled with pain, fear, and disability. This disease is due to mutations, often in the collagen gene, that cause collagen to either not be present in sufficient amounts or not be of sufficient quality.
Patients have:
- Very fragile bones.
- Experience constant fractures and breakage of their bones.
- Deformities of long bones, spinal curvature, and deformed ribs.
There are very few therapies available and those that exist, such as physiotherapy, rodding surgery, and bisphosphates (BPs), are merely palliative and fail to reduce the frequency of fractures. Generally, OI sufferers have an almost normal life span with severe disabilities due to bone defects and hundreds of painful bone fractures, even during fetal life, causing irreversible damage.
OI affects more than 56.000 people in the United States of America (USA) and the European Union (EU) with an annual incidence rate of 1.000 newborns globally.
There is no cure, no treatment.
BOOST Pharma was founded based science of the Karolinska Institutet, which is a world-leader in cell therapy research. BOOST Pharma is developing a cell therapy treatment for Osteogenesis Imperfecta (OI) using mesenchymal stem cells (MSC).
Boost Cell Therapy
Technology
BOOST Pharma is developing a first-in-class therapy to treat Osteogenesis Imperfecta. This treatment is based on novel Mesenchymal Stem Cells (MSC), with high bone-forming capabilities – dubbed BOOST Cells.
In mice models, BOOST Cells has shown that cell therapy leads to higher calcium deposition, higher alkaline phosphatase activity, and a high ectopic bone formation.
Once injected, these BOOST Cells will migrate to the bone of patients with OI where they will engraft, and start bone formation. BOOST Pharma has human proof-of-concept studies from four children with Type III and IV that were treated with BOOST Cell therapy.
This treatment showed great promise in the effectiveness of treating children with OI; the children followed their own growth curve, they had increased lengthwise growth compared to contemporary OI patients, and a significant reduction of fractures was observed. The cells showed great safety, with no adverse reactions and no immune responses towards the donor MSC.
BOOST Cell Therapy start already at the prenatal stage, when OI is first diagnosed, or as early as possible after the child is born. By treating this early, BOOST Pharma are addressing the disease at the earliest possible stage and increasing the benefits for the patient in later years. The boost cells are given as one prenatal infusion dose. Postnatally, treatment schemes will vary from patient to patient, but a typical treatment scheme will involve postnatal infusions of BOOST Cells every 4 month during childhood and early adolescence. Patients will be monitored to see if they follow their normal growth curve and monitored for fracture rates.
BOOST Pharma major milestones
The next steps for BOOST Pharma are to further develop our treatment cell therapy in the ongoing clinical trials sponsored by Karolinska Institute in Sweden, prepare for a pivotal trial, and upscale our manufacturing capabilities and develop our CMC package. We have ongoing fundraising activities to support these developments.
Key focus areas
- Finalizing European BOOSTB4 trial
- Finalizing Indian BOOST2B trial
- Obtaining orphan drug designation for OI in US
- Upscaling manufacturing capabilities
- Start of pivotal clinical trial
Clinical Trials
The two ongoing clinical studies – one in Europe (BOOSTB4) and one in India (BOOST2B), are funded under a Horizon 2020 academic grant, Vinnova, and the Swedish Research Council. Both of these clinical trials started in the first half of 2020, with expected positive outcomes in 2022.
The BOOSTB4 phase I/II clinical trial is a multicentre, open-label, and multiple doses for safety and efficacy of postnatal or pre-and/or postnatal dosing. The treatment regime will be four doses of allogeneic MSC every 4th month during the duration of the trials. The inclusion age is ≤18 months. For the non-treatment arm, we will use historical and untreated perspective controls. The BOOST2B phase I/II clinical trial is a single-center, open-label, and multiple doses for safety and efficacy postnatal dosing. In this trial, older children will be treated (1-4 years of age). We will also examine another type of administration; direct injection into the bone, in addition to intravenous injection.
These studies will ensure the investigation of the safety and efficacy of multiple (dosing) to show a clear clinical effect of BOOST Cells on bone development. Additional safety will be assessed to support findings from the human proof-of-concept case studies as well as the effect. Evaluation of the safety and effect of prenatal treatment will also be determined.
Click Logo for European study: BOOSTB4
Click Logo for India study: BOOST2B
Evert Küppers
Corporate Development - MBA & M.Sc. Biomedical Sciences
Serial biotech CEO ao. Pieris Pharmaceuticals (NASDAQ: PIRS)
Lilian Walther Jallow
Co-founder & CDO - PhD
Project manager of Drug Development and Advanced Therapy Medicinal Products, CMC/RA experience
Cecilia Götherström
Co-founder - PhD
Research Leader and Associate Professor in Stem Cell Science at Karolinska Institutet. Manages the BOOSTB4 consortium and trial.
Carlos Camozzi
Med. Adv. - MD
Expert on Orphan Drugs and Pediatric Clinical Development
Team
Board of Directors
Ingelise Saunders
Chairman & CEO - M.Sc. Pharm.
More than 35 years experience from biotech and pharmaceutical industry at C-level positions as well as chairman/member of boards both in SME and large pharma companies
Magnus Westgren
Co-founder and Member of BoD and SAB - MD & PhD
Senior Professor at Karolinska Institutet. Board Member in several biotech companies. Established fetal therapies in Sweden and performed the first prenatal MSC transplantation in the world
John Öhd
Member of BoD - MD PhD
Board representative for Karolinska Development, Sweden
Jonathan Ilicki
Member of BoD - MD MSc. MBA
Board representative for Industrifonden, Sweden
Scientific Advisors
Magnus Westgren
Co-founder and Member of BoD and SAB - MD & PhD
Senior Professor at Karolinska Institutet. Board Member in several biotech companies. Established fetal therapies in Sweden and performed the first prenatal MSC transplantation in the world
Jörg Oliver Semler
Member of SAB - MD & PhD
One of the world’s foremost OI experts. Head of the Department of Skeletal Dysplasias and associate professor of pediatrics at University Hospital in Cologne. Chair of the medical advisory board of the national and the European patient organization for OI
Willem E. Fibbe
Member of SAB - MD & PhD
Chairman of the Department of Immunohematology and Blood Transfusion at Leiden University. An expert within Stem Cell Therapeies and has vast experience with clinical program using Advanced Therapy Medicinal Products (ATMP) for a variety of clinical indications
General
Publications
Docs try new stem cell therapy to help children with ‘brittle bones’
Chennai: When Devi’s baby was just 45 days old, he fractured ..
Svenska Dagbladet, SvD, article about the first child treated in the European study. The article is in Swedish and requires payment.
Varje gång Tellef börjar gråta får hans mamma en klump i magen. Har han brutit något? Föräldrarna uppmanades att ta farväl när sonen föddes med en ovanlig bensjukdom och knappt kunde andas.
Scientific
Publications
Clinical trial article on treatment in children with OI:
Götherström C, Westgren M, et.al Pre- and postnatal transplantation of fetal mesenchymal stem cells in osteogenesis imperfecta: a two-center experience. Stem Cells Transl Med. 2014 Feb;3(2):255-64. doi: 10.5966/sctm.2013-0090. Epub 2013 Dec 16. PMID: 24342908; PMCID: PMC3925052.
Case report on stem cell engraftment in OI patient:
Le Blanc, Katarina et al. Fetal mesenchymal stem-cell engraftment in bone after in utero transplantation in a patient with severe osteogenesis imperfecta. Transplantation vol. 79,11 (2005): 1607-14. doi:10.1097/01.tp.0000159029.48678.93
Review article on prenatal stem cell therapies:
Ekblad-Nordberg Å, Walther-Jallow L, Westgren M, Götherström C. Prenatal stem cell therapy for inherited diseases: Past, present, and future treatment strategies. Stem Cells Transl Med. 2020;9(2):148-157. doi:10.1002/sctm.19-0107
Review article on stem cell therapy for OI:
Götherström C, Walther-Jallow L. Stem Cell Therapy as a Treatment for Osteogenesis Imperfecta. Curr Osteoporos Rep. 2020 Aug;18(4):337-343. doi: 10.1007/s11914-020-00594-3. PMID: 32710427; PMCID: PMC7419362.
About BOOST Pharma
BOOST Pharma is based on years of collaborative research from Karolinska Institute in Stockholm with a focus on novel cell therapy treatments for Osteogenesis Imperfecta.
