$300,000 Cure SMA Grant Awarded for Preclinical Study of Potential Therapy LDN-5178

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by Ashraf Malhas |

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Spotlight Innovation announced that the nonprofit Cure SMA has awarded $300,000 to Prof. Kevin Hodgetts, of Brigham and Women’s Hospital in Boston and a Spotlight research collaborator, for the preclinical development of the potential therapy LDN-5178 for spinal muscular atrophy (SMA).

Hodgetts’ project is titled “Pre-Clinical Development of LDN-5178 for the Treatment of SMA.

Spotlight Innovation is a pharmaceutical company that licensed the worldwide development and commercialization rights for LDN-5178 from the Indiana University Innovation and Commercialization Office.

Individuals with two faulty copies of the survival motor neuron 1 (SMN1) gene will develop SMA. The SMN protein that is normally encoded by the SMN1 gene is involved in the function of nerve cells that carry signals from the brain to move certain muscles.

Although our bodies have another gene that produces SMN, the SMN2 gene — often called the SMA backup gene — it is not enough to make up for the loss of normal SMN1 gene function in individuals with SMA.

The grant awarded to Hodgetts will be applied to the study of small molecules that can increase the amount of accumulated SMA protein produced by SMN2 to compensate for the loss of SMN1.

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This is expected to result in increased SMN protein in the brain and normalize the function of nerve cells that are affected in SMA.

Hodgetts and his team have previously “identified lead compounds that increased SMN protein in brain and extended survival and motor function in a mouse model of SMA,” Hodgetts said in an interview with Cure SMA.

He said the SMA grant will help the teams to “continue the development of … lead series of compounds, performing preclinical pharmokinetics [drug properties] and toxicity studies, to ensure that they are as safe and effective as possible prior to entering clinical trials.”

The research funded by the grant will be carried out by Hodgetts and his team at the Laboratory for Drug Discovery in Neurodegeneration (LDDN) at Brigham and Women’s Hospital, an affiliate of Harvard Medical School, and Prof. Elliot Androphy and his team at Indiana University.

“Cure SMA’s support of Professor Hodgetts to work with Dr. Elliot Androphy and other collaborators on SMA drug candidate LDN-5178 represents an important validation of Spotlight Innovation’s preclinical SMA pipeline,” said Geoffrey Laff, PhD, Spotlight Innovation’s senior vice president of business development.

“If we can translate our test tube finding, that our compounds work synergistically with SMN increasing agents such as Spinraza, to first mice and then humans, it potentially would reduce the needed quantity and frequency of administration of Spinraza, resulting in greater therapeutic response and reduced occurrence of side effects,” Hodgetts said.