Home » Clinical Trials » Taking actionable steps together to find a cure for motor neurone disease

Professor Martina Hennessy

Director of the Wellcome-HRB Clinical Research facility at St James’s Hospital and Trinity College, Dublin

Mr Michael Campbell

Participant in the Motor Neurone Clinical Trial at the Wellcome-HRB Clinical Research facility at St James’s Hospital

Photographer – Gabrielle Masterson

Irish patient participates in trial informing development of new drugs for motor neurone disease.

While on stage at the Dublin Fringe festival, actor and writer Michael Campbell tripped and fell. Eight months later, he was diagnosed with a rare type of inherited motor neurone disease (MND), for which there is no cure.

Motor neurone disease

Motor neurone disease affects the nerves and brain. Symptoms include muscle weakness, slurred speech and difficulty swallowing, progressively increasing over time; most cases are sporadic with around 10% of cases inherited. Campbell’s case was especially rare, stemming from a fault in his FUS gene, occurring in approximately 300 people worldwide.

Campbell’s father died from the same disease. Although stunned by the diagnosis, he immediately contacted Professor Orla Hardiman, Ireland’s leading neurologist for MND and began seeking clinical trials of new drugs. He found one just beginning at St James’s Hospital.

MND clinical research facility

“The standard of care has been nothing short of amazing. I am constantly being checked in on, and I feel like part of the team,” Campbell says. Although the outcome is not guaranteed, he knows this is an “actionable step forward to identifying a potential treatment,” he says.

Provide greater access to innovative medicines.

Research centres improving outcomes

Opened in May 2013, the Wellcome-HRB purpose-built clinical research facility (CRF) has grown from strength to strength, supporting a wide variety of clinical trials and studies and specialising in early-phase and advanced therapy trials. Professor Martina Hennessy, its Director, shines a light on how research improves clinical outcomes.

She says: “Evidence shows that research-performing hospitals, including clinical trials, provide greater access to innovative medicines, make better use of evidence, offer more precise care, attract more highly qualified staff, have better facilities and greater capacity to develop novel approaches to the least well-served diseases.”

Patient engagement for progress

“With patients and clinicians working in partnership … we can design better trials that begin to unlock the potential of new medicines. Irish patients are positive about engaging with research and about contributing to the quality of the data, which informs the development of new drugs worldwide,” she says.

“They do it for themselves but also their families and society. The outcome is unknown, but CRF staff are with them every step … to make it as safe as possible,” explains Prof Hennessy.

She stresses the importance of working with patients with rare diseases. “With patients like Campbell as part of the team, there is always hope,” she insists. Today, Campbell is continuing to write and act — showcasing that life perseveres despite the challenge of disability, thanks to today’s research landscape.

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