Additional funding for BOOST Pharma
BOOST Pharma are pleased to announce that we have received additional funding from the Danish Growth Foundation (Vækstfonden). This funding will allow us to strengthen our CMC development plan.
In the spring 2020 BOOST Pharma was part of a Business Accelerator Academy facilitated by BioInnovation Institute in Copenhagen. Read our interview here. – April 2020
BOOST Pharma – Treating the untreatable
BOOST Pharma is based on years of collaborative research from Karolinska Institute in Stockholm with the focus on novel cell therapy treatments 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 fetal mesenchymal stem cells (fMSC).
Boost Cell Therapy
BOOST Pharma is developing a first-in-class therapy to treat Osteogenesis Imperfecta. This treatment is based on Fetal Mesenchymal Stem Cells (fMSC), which is provided from abortive material. When thawed and injected, the cells will migrate to the bone of patients with OI where they will engraft, and start bone formation.
In mice models, BOOST Pharma has shown that cell therapy leads to higher calcium deposition, higher alkaline phosphatase activity, and a high ectopic bone formation. Furthermore, BOOST Pharma has human proof-of-concept studies from four children with Type III and IV that was 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 fMSC.
How does it work:
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:
- Milestone 1: Finalizing the BOOSTB4 Trial
- Milestone 2: Finalize BOOST2B Trial.
- Milestone 3: Approved Orphan Drug Designation
- Milestone 4: Manufacturing capabilities upscaled.
- Milestone 5: Commencement of pivotal 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 fMSC 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.
European study: BOOSTB4
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.
WELCOME TO BOOST TO BRITTLE BONES - India
Osteogenesis imperfecta is a hereditary disorder characterized by bone fragility and often severe deformities.
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.