September 2017

MESSAGE FROM OUR CHAIRMAN

WELCOME to our new-look newsletter.

The CGD Society exists to support families with Chronic Granulomatous Disorder from around the world. 2016 marked the organisation’s 25th anniversary and I hope you will agree that the charity has achieved an extraordinary amount over the years.

As newly-appointed Chairman I would like to know what you think of our work. Please do email me at hello@cgdsociety.org with feedback on any aspect of our services to families.

In this newsletter you will read about the research into CGD that the Society both funds and monitors; the information that we provide for affected families on topics such as bone marrow transplantation and health guidance for X-linked CGD carrier females. You can also find out how you can contribute to the global body of knowledge on CGD by sharing your stories with us.

This charity relies heavily on income from the UK Jeans for Genes fundraising day, so please get behind this year’s event on September 22nd. The details are below. We also need members to fundraise for us so that we can continue to provide the services that you value so highly. Whatever you choose to do, let us know and your place in a future newsletter is guaranteed.

If there are any particular topics that you would like to see covered in future newsletters, please do let me know.

With best wishes, Geoff Creamer (pictured with sons Adam (left) and Alex (right))

INTERNATIONAL RESEARCH

CGD GENE THERAPY TRIALS

Gene therapy will become increasingly important to CGD patients. This is because gene therapy uses the patient’s own cells so unlike BMT, a donor is not required and rejection or graft-versus-host disease is not a problem. Two trials are currently underway in Europe and the US that involve CGD patients.

In Europe, a new gene therapy clinical trial for X-linked CGD is open for recruitment in the UK, Switzerland and Germany. The study is coordinated by GENETHON, a not-for-profit French biotechnology laboratory and funded through the Net4CGD program. If you are interested in knowing more, take a look at our Q&A about this trial here.

In the US, stem cell researchers at the University of California have received $7.4 million (£5.9 million) to fund a phaseI/II clinical trial enrolling 10 patients with CGD at three different locations across the States.

 CGDS AWARDS £50,000 GRANT TO ESTABLISH QUICKER GENE THERAPY TEST FOR CGD PATIENTS

CGD Society-supported research into gene therapy has achieved its first results. Professor Janine Reichenbach and her team at the University Children’s Hospital, Zurich, have developed a new cellular model that enables better testing of the effectiveness of new gene therapies for p47phox deficiency, which affects 25 to 30 per cent of patients and is the second most common form of CGD.

Professor Reichenbach (pictured) says: "The standard way of testing has been to use patient’s skin cells that are reprogrammed into stem cells, a time consuming and expensive process. The new testing system will be faster. We have used Crispr/Cas9 technology, so-called 'gene scissors', to change a human cell line model so that the blood cells show the genetic change typical of the p47phox form of CGD". Support from the CGD Society helped fund the development of the new cell model and means that new gene therapies for affected patients will be able to be screened more efficiently.

£14,000 GRANT GIVEN FOR RESEARCH INTO PREVENTION AND TREATMENT OF ASPERGILLOSIS

The CGD Society has awarded £14,141 to Professor Adilia Warris (pictured) who is researching prevention and treatment of the invasive aspergillosis (IA) fungus, which can be a serious issue for CGD patients.

The study will test a combination of treatments in a laboratory model. Professor Warris, is deputy director of the Medical Research Council (MRC) Centre for Medical Mycology at the University of Aberdeen.

EXPERT INFORMATION

The CGD Society collaborates with medical experts and affected families to provide up-to-date and relevant information on a whole range of topics associated with CGD.

In countries where BMT is accessible, the decision to go ahead with a transplant is a very difficult one for individuals and parents to make. Our website is full of information about the BMT process and includes films, diaries and interviews with parents and patients about the experience. We are always looking to add to these resources so if you are willing to share your story with us to help others, we would love to hear from you.

In March of this year, ten-year-old Jonathan (pictured) became the first person in the world to undergo an unrelated cord stem cell transplant to treat his X-linked CGD. Click here to read what his dad, Ning, says about their journey. 

X-LINKED CGD FEMALE CARRIERS

Until recently, very little was understood about the impact that being a carrier of X-linked CGD had on a woman’s health. 

We have a dedicated section on our website for X-linked CGD female carriers that provides information on a wide range of issues. We have also developed a leaflet that you can download here.

YOUR CHANCE TO SHAPE THE WORK OF THE CGD SOCIETY

We have two exciting opportunities at the CGD Society and are looking for individuals from our community in the UK who are interested in working with us.

MEMBERSHIP COORDINATOR

We want to strengthen our links with the membership by recruiting a coordinator who will become the membership hub of the charity. This person will work part time from their home office and will liaise closely with our members and the rest of the team to promote fundraising and communication.

TRUSTEES

The Society is steered by a small group of enthusiastic trustees who come from families affected by CGD. We are looking for two new UK-based trustees to join us. If you would like to help shape the future of the charity and you have some time and expertise to spare, please do get in touch.

If you are interested in either position, please email hello@cgdsociety.org for the attention of the Chairman of Trustees.  In return we will send further details of the roles and how to apply.

YOUR STORIES

CGD SOCIETY HELPS SEARCH FOR A DONOR FOR ALLY KIM

The CGD Society has reached a global audience in its search for a stem cell donor for CGD patient Ally Kim, three (pictured with mum, Judy), who lives in Oxford, England.

Ally’s family is Korean so we signed up Tottenham Hotspur and Korean international footballer Son Heung Min to make videos appealing to the Korean community to register as donors.

The Premier League soccer star posted his videos (filmed in English and Korean) on his personal Facebook page and they have been viewed over 125,000 times to date. The story has also been picked up by Korean and Chinese TV. Ally’s dad, Andrew, said: “Son's involvement has had a huge impact worldwide, especially in Korea and the Asian community, resulting in many more international bone marrow donor registrations. We are still waiting for Ally's match, but excited to know that many others will be saved through these efforts".

Ally is currently undergoing an experimental gene therapy treatment at Great Ormond Street Hospital in London. We wish him well.

FUNDRAISING

RideLondon

Huge thanks to Emma Waller for raising an incredible £771 for CGDS by completing the RideLondon cycle challenge in July. Londoner Emma (pictured with her medal) who has a close friend with CGD, finished the 100-mile (160km) route that winds through the capital city, out to the countryside and back to Buckingham Palace, in seven hours and 34 mins.

Emma, a marketing executive, said: "CGD is so rare, and the charity has made great progress in effecting awareness and new treatments over the last 20 years. Exciting new research is under way, and more people than ever around the world are discovering the CGDS community. It must continue, and I hope that other people are encouraged to fundraise for this amazing charity". Emma’s highlight of the ride? "Seeing so many people, of all ages, pouring blood, sweat and tears to raise money for charity". And her low point? "Being overtaken by a man in his seventies".

JEANS FOR GENES DAY 22nd September

Jeans for Genes Day is a one-day UK fundraising event that was created by the CGD Society over twenty years ago. Children and adults wear their jeans to school and work in exchange for a small donation. The majority of the CGD Society’s activity is funded from money raised on Jeans for Genes Day.


So if you know of a school or workplace that would like to get involved, please do encourage them to sign up.

YOUR FUNDRAISING STORIES

If you are fundraising for the CGD Society we would love to see your pictures and hear your story. Email them to sam.carlisle@causecommunications.co.uk

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Email us at: hello@cgdsociety.org

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