UPDATE MAY 2021: Great Ormond Street Hospital have now given us an indication of the total costs of the compassionate use programme. We have had amazing fundraising efforts from families and have raised a total of £200,000 between different family groups. We are looking to raise another £50,000 to ensure we have enough money for this transformational compassionate use programme.

Here is the story..

We are very pleased to announce that Brineura© in the eyes was evaluated by the GOSH ethics committee and it was suggested that compassionate use of this drug for intravitreal injections is appropriate. This is a significant step forward towards the treatment being made available for children with CLN2 who are on enzyme replacement therapy. At the moment the costs of this compassionate use programme are a forecast and are not yet known precisely. The duration of the programme will be 18 months. There will be hospital costs associated with the intravitreal injections that will include operating theatre time including the costs of general anaesthesia as well as programme coordination.

We have been eager to share recent developments regarding our collaborative family fundraising campaign. We are looking to raise funds towards a potential compassionate use programme using Brineura ©, an enzyme replacement therapy, to help maintain eyesight in children with CLN2. Currently, this potential programme would only be for children who are already accessing Brineura © but offers potential hope to children diagnosed in the future.

Here is a bit of background..

Among the many cruel symptoms of Batten disease and one, which strikes fear into us all, is the loss of eyesight. The thought that our children will not be able to see our faces again is terrifying.

It is believed that giving Brineura ©(the drug that is given during the Enzyme Replacement Therapy ERT) into the brain will not help to save the eye sight because it is not known if it has the ability to cross through into the retina. Sadly there are some children who, despite receiving ERT, have lost their sight.

There are scientific advances being made every day and one of those incredible advances is the prospect of injecting the Brineura © drug directly into the eye to save the sight.

Early studies have been carried out which have shown this method is effective and so the next step is to establish a compassionate use programme which would give our children the opportunity to join, if they meet the eligibility criteria, and may benefit.

Clinical trials for this are already in the early phases in Hamburg and Ohio and the GOSH team (who currently ran the CLN2 ERT programme which is being transitioned to several UK centres) is in the process of exploring if it would be a possibility to conduct a similar programme in Great Ormond Street Hospital, London. This would ensure children in England would have the same opportunity to be part of this pioneering step to try and save their eyesight.

What is happening right now?

As a community of CLN2 Batten families, we want to come together to fundraise to ensure all of the children who are eligible (criteria are still to be drawn up) will have the chance of accessing this treatment at Great Ormond Street Hospital.

It may be that not all of our children will be able to access this treatment at this point in time, but we must think of the children who will be diagnosed in the future and what hope this treatment could bring to other families. That is a huge driving factor for all of our families, to help each other and those we do not yet know, who will join our Batten family in the months and years to come.

Imagine being diagnosed and being told there was a way of saving your childs sight, that would be quite the feeling to know that we have all, in some way, helped to make that a possibility for families in this country.

We want to be able to give our children every chance of retaining their sight and by joining our efforts as a collective, we are able to help make this a reality. It maybe that in order for GOSH to move things forward, the initial step would be to recruit a Trial Coordinator for example, so we could help to pay for that role.

We are delighted to be working together with the BDFA and will keep you updated on the fundraising efforts so you will know exactly how your valued contribution is helping!

As parents, we want to make sure we do everything we can to help our children so we will be working hard and asking for your support to help to raise funds to achieve this incredible advancement in the treatment of this cruel disease.

What are the next steps?

Because we will be working in conjunction with the BDFA, we will be able to use the BDFA charity number which will be an incredible opportunity to reach out to organisations where a charity number is a prerequisite for them to be able to support.

Important information regarding fundraising It is very important to the families and to the BDFA that the fundraising is transparent, so at this stage we don't know for certain, how much will be required to make this a reality.

If we begin raising funds through the BDFA and it is not possible for the programme to go ahead for any reason, the funds would be put towards alternative research and support activities which will benefit families living with Batten Disease in the UK.

How can you help?

It would be amazing if you could start to think of ways you may be able to help us reach these businesses and organisations and ask if they might be able to support this campaign in some way.

If you are a business owner, another way of helping is to have a fundraising bucket, (which we would supply) in your premises, where your lovely customers have the opportunity to pop a few pennies or spare change in and donate that way. Every single penny will help!

A Big Thank You for taking the time to read this and thank you in advance for anything you can do to support this family campaign. It means so much to all of the families involved, so thank you from our families to yours.

xxx