Final Dosing in BOOSTB4 Trial
We are very happy to announce that the BOOSTB4 trial has completed its final dosing. All doses of mesenchymal stem cells (MSCs) have been successfully administered to the participating children, suffering from Osteogenesis Imperfecta (OI).
A massive thank you to everyone that has worked so hard and contributed so much to making the BOOSTB4 trial a success, and a special heartfelt recognition to all the children and families that have taken part in the trial!
BOOST Pharma awarded RPD
On October 14th 2022, FDA awarded the Rare Paediatric Disease (RPD) designation to BOOST Pharma for its novel cell therapy for the treatment of children suffering from Osteogenesis Imperfecta (OI). OI is a serious rare genetic disease that can lead to severe physical and functional disability, and for which there is currently no approved therapy. The RPD designation allows BOOST Pharma to receive a priority review voucher (at the time of marketing authorization) that can expedite product approval. RPD designation follows the recently awarded Orphan Drug Designation in both Europe and USA.
Dr Cecilia Götherström, Associate Professor in Stem Cell Science at Karolinska Institute and CSO of BOOST Pharma, is presenting at at the OIFE’s Annual Educational Webinar, giving an update on the ongoing BOOSTB4 clinical trial.
Evert Kueppers appointed CEO and co-founder Dr Lilian Walther Jallow transitioned to Chief Development Officer. Significant progress seen for ongoing BOOSTB4 clinical study.
Orphan Drug Designation for OI
BOOST Pharma receives an Orphan Drug Designation (ODD) for Osteogenesis Imperfecta (OI) in Europe, strengthening the company’s position in the OI field.
BOOST Pharma receives Innobooster Grant
BOOST Pharma have received an additional €200K funding from the the Danish Innovation Foundation in the form of an Innobooster Grant. The funding is dedicated to develop the IP around BOOST Cells, stem cells with unique bone forming capabilities.
Additional funding for BOOST Pharma
BOOST Pharma have received an additional €800K funding from the Danish Growth Foundation (Vækstfonden). This funding will allow BOOST Pharma to strengthen CMC development and manufacturing of the bone forming BOOST Cells.
PreSeed funding received from BII
BOOST Pharma has been granted PreSeed funding from BioInnovation Institute in Copenhagen. The funding of €470K will facilitate the establishment of BOOST Pharma as an active player in the development of Stem Cell Therapies. The funding will be used to increase the team behind BOOST Pharma and further develop BOOST Cell treatment.
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.
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.
- 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
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
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
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.
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.