Research News – May and June 2018 – Ataxia

Research News – May and June 2018

Research News – May and June 2018

AMRC Releases Annual Research Expenditure Statistics

The Association of Medical Research Charities (AMRC), of which Ataxia UK is a member, has published their annual research expenditure infographic and briefing. New figures show that the medical research charity sector has maintained a collective spend of £1.6bn on research in the UK in 2017 despite a tough Brexit environment. This is nearly half of all publicly funded medical research nationally (47%).

200,000 people in the UK were recruited into over 1,300 AMRC charity-funded clinical studies or trials in 2017 so the AMRC is making a difference for patients too. It is funding essential research in all areas of health and disease and 8% of its funding goes towards neurological conditions such as ataxia.

You can find out more about charity impact in this infographic.

Posted on 15/06/2018

FDA Orphan Drug Designation for Spinocerebellar Ataxia

Biopharmaceutical company, IntraBio, has received an Orphan Drug Designation from the US Food and Drug Administration (FDA) for its lead compound (IB1000) for the treatment of spinocerebellar ataxias (SCA). Although they have only investigated the clinical efficacy of IB1000 in a small number of the over 40 known SCA subtypes, the data was so compelling that the compound was granted a single designation covering all SCAs.

IntraBio have also recently filed this application in the EU, and expect to receive the designation between July and September 2018.  The designation has been posted on the FDA website, you can read it here. Read our full article here.

Posted on 15/06/2018

Raising awareness and understanding of ataxia among healthcare professionals

Ataxia UK was invited to attend two conferences to talk about ataxia.

The first was a training day on rare diseases in Birmingham aimed at junior doctors and GPs. We are grateful to Dr Rajith de Silva, ataxia specialist neurologist and member of Ataxia UK’s Medical Advisory Panel for attending this event in March and to Ataxia UK Trustee Richard Brown who agreed to give a talk about how ataxia affects him.

The second event was in St Asaph in Wales a Study Day for health and social care professionals from across North Wales and North West England working in the field of movement disorders. Dr John Ealing, another member of Ataxia UK’s Medical Advisory Panel and a Friend of Ataxia UK Martin McCudden, who has ataxia, attended the day and helped raise awareness of the ataxias.

Posted on 23/05/2018

Government commits to align as closely as possible with EU rules on clinical trials following Brexit

Following some uncertainty due to Brexit, the government has committed to ensuring that the UK is as aligned as possible with the new EU Clinical Trials Regulation (CTR) when it comes into force.

The commitment was made following a debate on a proposed Bill amendment which was supported by a range of science and research organisations including AMRC (the Association of Medical Research Charities), who provided us with this information.

Alignment of the clinical trials landscape across the EU is vital to allow patients to take part in pioneering pan-EU trials. Ensuring full alignment with the CTR is one of AMRC’s key priorities for patients and medical research post-Brexit.

Ataxia UK is a member of the AMRC. You can read their position statement on exiting the European Union here.

Posted on 23/05/2018

GoFAR reinforces partnership with Powell Gene Therapy Center at University of Florida (UF) to develop a gene therapy for Friedreich’s ataxia

University of Florida (UF) researchers have received a $450,000 grant from the Italian patient advocacy group GoFAR to continue developing a gene therapy for Friedreich’s ataxia (FA).

GoFAR originally began a collaboration with the UF Powell Gene Therapy Center in 2016 by awarding a grant of $750,000. The total award of $1.2Ml has been staged to reach milestones toward the clinical implantation of the therapeutic strategy for FA.

The most recent award will be used to complete preclinical studies and prepare data for submission to the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Italian regulatory authority, Instituto Superiore della Sanità (ISS). It is hoped that trials will begin later this year.

You can find and read the full press release from UF here.

Posted on 23/05/2018

Data released from preclinical study into gene therapy for Friedreich’s ataxia

Voyager Therapeutics have created a gene therapy vector for Friedreich’s ataxia that has been undergoing pre-clinical testing in mice and non-human primates. They announced their latest findings of their study at the American Society of Gene and Cell Therapy 2018 annual meeting.

In mice, the therapy vector for Friedreich’s ataxia was shown to prevent central and peripheral disease progression up to one year after a single administration. The treatment additionally demonstrated good tolerability in non-human primates, along with widespread gene transfer to the brain and spinal cord after a single intravenous administration.

Voyager plans to continue to advance their preclinical gene therapy programmes and is focussed on developing life-changing treatments for severe neurological diseases, including Friedreich’s ataxia.

You can find and read the full press release here.

Posted on 23/05/2018


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