Grant Awarded to Find Affordable Treatments for Children

A researcher from Keele University School of Medicine in the U.K. was awarded a £99,959 ($138,633) research grant to identify and evaluate potential — and more affordable — treatments to improve the health of children with spinal muscular atrophy (SMA).

The two-year research grant of the Academy of Medical Science’s Springboard scheme was awarded to Melissa Bowerman, PhD, lecturer in Bioscience at the medical school.

Also collaborating in the research project is Kevin Talbot, PhD, professor of motor neuron biology and head of the Nuffield Department of Clinical Neurosciences at the University of Oxford.

“We are extremely pleased and honoured to have been awarded this prestigious Academy of Medical Sciences (AMS) Springboard Award, which will help us continue our research aimed at developing new and affordable treatment options for SMA patients,” Bowerman said in a press release. “Rare disease charities have been hit extremely hard by the pandemic and such support from AMS is incredibly important for the entire rare disease community.”

SMA is a neuromuscular genetic disorder caused by a mutation in the survival motor neuron 1 (SMN1) gene, which provides the instructions to make a protein involved in protecting nerve cells that control muscle movement.

Currently approved SMN gene replacement treatments are very expensive and their effectiveness is highly dependent on factors such as a patient’s age or disease severity.

With this new grant, Bowerman, Talbot and their research teams plan to evaluate the potential of three (undisclosed) commercially available drugs that can act as muscle-specific therapies to be combined with existing SMN gene replacement treatments.

Bowerman’s research has been focused on neurodegenerative and neuromuscular conditions such as SMA and amyotrophic lateral sclerosis (ALS). Her main objective is to identify new disease pathways that contribute to the development of these genetic conditions and to investigate therapeutic strategies to correct the identified defects.

The Academy of Medical Science’s Springboard research funding scheme offers a personalized package of career support to biomedical researchers at the start of their first independent post to help launch their research careers. This includes funding of up to £100,000 ($138,660) over two years and access to the academy’s acclaimed mentoring and career development program.