Ultragenyx Pharmaceutical Inc. and Mereo BioPharma Group announced data from the dose-selection Phase 2 portion of the Phase 2/3 Orbit study, showing that setrusumab significantly  increased bone production in OI-affected patients after only 3 months. You can view the full press release here.

On June 5th, the Foundation received the following communication from Ultragenyx regarding the interim data of their Phase 2 study, Orbit.

 

Dear OI Advocates,
As our trusted partner in OI, we are excited to share with you an important update about the Orbit study, NCT05125809, on behalf of the Ultragenyx OI study team. As you may be aware, Ultragenyx is sponsoring Orbit, a global study to determine the safety and efficacy of setrusumab, UX143, in the treatment of OI. Setrusumab is a fully human monoclonal antibody that inhibits sclerostin. Sclerostin is a protein that causes decreased bone formation and increased bone resorption. By inhibiting sclerostin, the goal of setrusumab is to increase bone formation, strength and bone mineral density and, to a lesser extent, decrease bone resorption.

Orbit is a randomized double-blind placebo-controlled Phase 2/3 study in OI types I, III, IV patients aged 5 to <26 (N=219). Enrollment in the Phase 2 portion of the study was completed in February with 24 patients. Patients received setrusumab at one of two doses to determine the optimal dosing strategy for Phase 3. We are pleased to announce the interim results from Phase 2 portion of the Orbit study. Data from the pediatric cohort suggests a substantial benefit on bone formation within 3 months on setrusumab.

Across all patients evaluated, setrusumab significantly increased levels of serum P1NP, a marker of bone formation, and a significant and rapid bone-building effect in the lumbar spine. There have been no treatment-related serious adverse events observed in the study. Reported adverse events have been consistent with the anticipated safety profile for setrusumab based on previous trial and include infusion-related reactions, headache, and sinusitis. There were no safety-related differences observed between dosing groups or age groups. “It’s encouraging to see similar lumbar spine bone mineral density results at 3 months in children to what we saw in adults at 12 months using the same dose levels,” said Eric Crombez, M.D., chief medical officer at Ultragenyx. “We believe it suggests that growing bones are more dynamic, and we anticipate a greater effect on bone formation and strength in the younger subjects with immature skeletons.”

Having this data allows Ultragenyx to move forward to the pivotal Phase 3 portion of the global study. Phase 3 will include approximately 195 participants, randomized 2:1 to receive setrusumab or placebo. We are grateful to the patients, parents, and caregivers who contributed to this study. We couldn’t do any of this work without the support from the OI community! To learn more about Patient Advocacy at Ultragenyx and our commitment to the OI community, including the upcoming Phase 3 trial, please visit https://ultrarareadvocacy.com/conditions-we-study/osteogenesis-imperfecta-oi/ or reach out via patientadvocacy@ultragenyx.com.

Thank you all for your support and trust in this work.
Sincerely,
Jenny McCue
Jenny McCue, Executive Director OI patient advocacy and engagement

We are very excited to announce that long-time BBS member, Steven Hastings, has had another article published in The Clydesider magazine!

Steven begins by describing how he typically likes to spend his day, which can involve visiting cafes, attending group sessions ran by Stepping Stones, and volunteering for the Clydesider magazine.

Steven then goes on to outline some of the accessibility issues that he can be forced to deal with, such as unsuitable disabled access facilities and obstructions that can make it difficult for him to use pathways.

The article is very powerful in highlighting issues surrounding disabled access. As Steven says, ‘there is still a lot to be done to make everywhere truly accessible for all.’

You can read Steven’s article here.

 

 

Second Year Paramedic Students

 

Following on from our initial visit with the Students in March (which you can read more about here); Support Development Officer Coreen along with members Richard and  David returned to Glasgow Caledonian University on the 18^th April 2023 to hear presentations by the students based on the information we had previously shared with them and to find out what subsequent research they had done following this.

The students were tasked with producing a short presentation which introduced the individuals they had spoken with, the community, and the key points of learning they had taken from the discussions. The aim was to bring their findings together to form a service user care plan for use by paramedics to enhance care in the community.

The presentations were broken up into two 2 hour sessions.  There were 9 groups of students presenting in total, 2 of whom presented on OI. Other presentations we heard were from Turning Point, Visual Impairment, Carers Scotland, Papyrus Scotland and Deaf/Blind Scotland.

All students did an amazing job and it was great to see so much enthusiasm.

We have shared the Care Plan they designed  with the BBS Care Pathways writing group and hope we will be able to develop this further.

Fourth Year Dental Students, Dundee University

 

The 4^th year Dental Students at Dundee University had been doing some research work with local groups as part of their course work. They had reached out to various charities in the area including the Salvation Army, Advocating Together and the Brittle Bone Society.

CEO Patricia Osborne and Trustee Yvonne Grant and long standing member Mark Ross travelled to the lecture theatre in the nearby university to hear the students presentations on the 18th April 2023. They also listened to a number of presentations by other groups and were very impressed with the level of commitment by the students to reach out to groups that clearly needed some assistance with their oral health.

 

BBS Project group

 

The group focussing on the BBS project had previously come along to our offices in Guthrie Street and met with a number of members, this was of course the group which we were most excited to hear what their final conclusions and outcomes were.  (You can read about their previous visit here.)

They broke up into a few groups and had some focussed questions about dental concerns and trying to find out ways to improve dental care for people with OI.

To get their presentation off to a flying start the students opened up with a quiz – testing the knowledge of the other students in the audience. The questions were about amalgam restorations being used on OI and DI patients and when should an orthodontist examine a child with OI and at what age. It was amazing to hear some of the gasps when some of the delegates had answered incorrectly. It was really just so marvellous to see an entire room of healthcare students so engaged in wanting to learn and understand more about OI and what they could do to make it a reality.

We enjoyed all the presentations immensely.  Its so uplifting to hear all those knowledgeable students who will all make fine dentists.

 

Awareness

 

The students had mocked up a trial data info leaflet.  We have already pledged that we would be really grateful if they wanted to discuss further with us.

We were also blown away that the students are considering further awareness raising of OI around Wishbone Day. They have pledged to wear yellow and challenged themselves to speak to as many other students across the faculty that they can possibly manage on the day.

 

 

Communications Officer

 

We’re looking for an experienced Communications professional to lead our external campaigns and content at Brittle Bone Society (BBS).

The person will be responsible for leading our busy external communication activity on a day-to-day basis including  communications calendar management, campaign delivery, all digital media and press office management. This means first understanding the needs of the community our Charity support. We will expect the individual to develop external communication plans and content that supports the goals/and mission of our UK Charity to reach our target audiences.

Communication is key to the smooth running of our support work. There is a requirement involved to achieve effective and consistent organisational planning and performance across the Charity. This is a great opportunity to develop those skills.

 

The full job description can be downloaded here

To apply please send a CV with covering letter to coreen@brittlebone.org.

CV’s should be no more than 2 sides of A4 to coreen@brittlebone.org

 

Closing date is 16th April 2023

 

CLOSED

 

Role

The successful candidate will have a good knowledge of general office procedures, excellent IT and literacy skills, and the ability to accurately manage databases and spreadsheets.

This post is part-time (minimum 15 hours/per week)  –  hours to be negotiated.

 

Downloads

 

Job Description

 

To Apply – Now closed for applications

Please submit CV with covering letter to coreen@brittlebone.org

 

 

Brittle Bone Society (BBS) Westminster Parliament reception event (OI Awareness)

 

13^th June 2023 at Westminster, House of Commons, London 2 to 4 pm

 

The BBS have secured sponsorship from the local constituency MP Chris Law to support this event at the Parliament. The initial project commenced 1 year ago; seeking greater awareness of OI and calling for the month of May to be recognized. Generally  supporting the needs of the OI community. We encouraged our members to write to their elected representatives to bring their attention to the OI community. At this planned event we will also be presenting the results of our patient charter highlighting the shortfalls in patient care. We are also grateful that Mr Law raised an early day motion in parliament to call for greater awareness and a month of focus on OI.

 

Please contact your MP about the event

 

We ask members to get in touch with their elected reps and make them aware of the event and encourage them to attend. The host MP Mr Chris Law will also seek to foster support from (the house) elected members to attend.

 

OI army of ambassadors

 

All the individuals and their close relatives involved in ‘the BBS year long campaign’ and who feature on our literature about it will be invited to attend. Shani Dhanda, Monique Jarrett, Yvonne Grant, Eva Kelly and her mum Silean, Lucy MacConnell and her mum Dr. Niamh MacConnell, Kieran Dube and mum Rachel Dube, Kat Watkins, Max Edney and dad Steve Edney, Richard Toner, Amna Hammad and more!!!

Other OI ambassadors who have helped us in other campaigns and given talks and presentations representing the Charity will be invited and will include representatives of the Charity’s Trustee board, BBS medical advisory board and scientific advisory board representation. Key BBS supporters, healthcare professionals either involved or interested in our work and other NHS personnel and sponsors. Various funders will be invited to attend. Friends from the wider rare bone disease network. E.g. HPP, XLH, Fibrous Dysplasia. The aim is to raise awareness of OI and foster greater support for care in rare bone.

 

Speakers

 

We have an impressive line up of speakers Host MP Mr Chris Law, The Lord Kevin Shinkwin, CEO Patricia Osborne, Vice Chairman and Trustee Mr John Phillips, Professor Richard Keen – Chairman of our Scientific Advisory Board, Dr Judith Bubbear (standing in for Chair of our MAB, Assoc. Prof Kassim Javaid) and Steve Edney (dad to Max, advocate of OI and champion fundraiser for the BBS).

 

Whats on the Agenda

 

Welcome address from MP  about the charity’s standing in the community and hopes to raise more awareness. (with input about the UK Rare Disease Plan).

We will address our important work to develop care pathways referencing to any network care work BBS have contributed to. We will reference any published papers we have achieved eg. Impact of Covid on the OI community and provide an update on any new treatments/exciting prospects including research in OI care. There will also be a talk from Steve Edney on his, and his family’s own OI Journey.

 

Sign the BBS Charter

 

There is still time to sign the #OICANMayCampaign charter! You can sign the charter here. 

 

Brittle Bone Society (BBS) Scottish Parliament reception event (OI Awareness)

 

23^rd May 2023 at the Scottish Parliament, Edinburgh 6 to 8 pm 

 

The BBS secured sponsorship from the local constituency MSP to support this event at the Scottish Parliament. The initial project commenced 1 year ago; seeking greater awareness of OI and calling for the month of May to be recognized. Generally supporting the needs of the OI community. We encouraged our members to write to their elected representatives to bring their attention to the OI community. At this planned event we presented the results of our patient charter highlighting the shortfalls in patient care.

 

MSP Support

 

We asked members to get in touch with their elected reps to make them aware of the event and encourage them to attend. The host, MSP Bob Doris also fostered support from (the house) elected members to attend.

 

OI army of ambassadors

 

All the individuals and their close relatives involved in ‘the BBS year long campaign’ and who feature on our literature about it were invited to attend. Shani Dhanda, Monique Jarrett, Yvonne Grant, Eva Kelly and her mum Silean, Lucy MacConnell and her mum Dr. Niamh MacConnell, Kieran Dube and mum Rachel Dube, Kat Watkins, Max Edney and dad Steve Edney, Richard Toner, Amna Hammad and more!!!

 

Other OI ambassadors who previously helped us in other campaigns by giving talks and presentations representing the Charity were invited, which included representatives of the Charity’s Trustee board, BBS medical advisory board and scientific advisory board representation. Key BBS supporters, healthcare professionals either involved or interested in our work and other NHS personnel and sponsors. Various funders and friends from the wider rare bone disease network. e.g. HPP, XLH, Fibrous Dysplasia, were also invited to attend.  The aim of the evening was to raise awareness of OI and foster greater support for care in rare bone diseases.

 

Speakers

 

We had an impressive line up of speakers, such as Host MSP Mr Bob Doris, CEO Patricia Osborne, Vice Chairman and Trustee Mr John Phillips, Professor Stuart Ralston – member of our Scientific Advisory Board and Laura Pilkington (young member, advocate and Disability Equality Lead within the Scottish Student Sport Equality Steering Group.).

 

Whats on the Agenda

 

Welcome address from MSP about the charity’s standing in the community and the hopes to raise more awareness (with input about the UK Rare Disease Plan – Scottish slant). We addressed our important work to develop care pathways referencing to any network care work BBS have contributed to. We also made reference to published papers we have achieved eg. Impact of Covid on the OI community and gave updates on new treatments/exciting prospects including research in OI care as well as a talk from Ms Pilkington on her own OI Journey.

 

Reflecting on the Event: BBS Goes to Holyrood & Getting in the Mood for Conference

 

 

BBS staff and trustees made their way to Edinburgh to visit the Scottish Parliament on Tuesday 23rd May for their highly anticipated ‘OI CAN’ Awareness Event.

 

The evening event was held in the formal Holyrood Room, with sponsorship from Mr Bob Doris MSP, who is convenor of the cross party working group on rare and undiagnosed conditions. We were delighted to have senior members of the Scottish Government attending, including Minister Jenni Minto, for Public Health, Sport & Wellbeing, Joe Fitzpatrick, Minister for local Government Empowerment and planning and other MSP’s. Our guest list of BBS members, healthcare professionals, academics, funders and sponsors was extensive. We had a packed and very happy room on the night!

 

The meeting featured an overview of our year long campaign delivered by our CEO Patricia, with talks from Professor Ralston who gave an update on the TOPAZ trial and research at the BBS, Ms Laura Pilkington who gave a passionate overview on her journey in swimming/sport, Mr Alasdair Bachell, University of Dundee recapped on his progress with the History Bones Archive project. The outright highlight for most of us in the room – was the Poetry Recital by Trustee Yvonne Grant (‘School Worries’ written by the late Margaret Grant) and one (by unknown) on issues around access, beautifully delivered with NO notes by young Mila Perry. It was a perfect end to a marvelous night.

 

Vice chairman John Phillips hosted the evening and trustee Margaret Grant offered up a vote of thanks to all concerned. Ordinary members we spoke to said how much they enjoyed the event and it that it genuinely put them in the mood for Conference.

 

Sign the BBS Charter

 

There is still time to sign the #OICANMayCampaign charter! You can sign the charter here. 

 

In line with the BBS’ initiative ‘What you need to know about my OI’ BBS staff at Dundee HQ were delighted to  welcome fourteen 4^th year dental students from the Dundee Dental School to their offices to meet with members Ingrid Brodie, Jamie Abbot and Trustee Yvonne Grant.

The students, as part of the Year 4 undergraduate curriculum cover a unit which involves community engagement where they meet with different groups to better understand different social, general and oral health needs of individuals. We were delighted to be able to share specific aspects of OI with the group.

The dental students visited for the afternoon on the 13^th March, and were split into 3 groups where they had the opportunity to interact with staff and members to understand the needs of the OI Community.

Following on from these discussions and as part of their assessment, the students will develop their ideas and present what they have come up with at a session in the Dental school on the 18^th April.  The members and staff who participated will be able to attend and give feedback, which we will report on in due course. BBS are delighted to forge ahead with the mutual exchange and will keep our members up to speed on exciting developments.

 

Coreen attended the first face to face POINT (Paediatric OI National Team) meeting since pre Covid Pandemic on the 9^th February in Birmingham.

There were Allied Healthcare professionals in attendance from GOSH, Sheffield, Birmingham, Bristol, Manchester and Cardiff Childrens Hospitals.

The healthcare professionals provided updates during the morning session on each of their centres and  various projects they are working on. Highlights and learning from recent medical conferences anyone had attended were discussed as well as poster presentations which had submitted on various research topics.

Coreen had an opportunity to provide updates on BBS activity covering topics such as Events, Care Pathways, Research and surveys, factsheets and new collaborations.

In the afternoon the various specialities divided up into breakout groups to discuss topics relevant to them.

If you want to find our more information about POINT see here