Cure SMA Awards $300,000 Drug Discovery Grant to Charlotte Sumner, in Collaboration with Imago Pharmaceuticals

Cure SMA has awarded a $300,000 drug discovery grant to Charlotte Sumner, MD, at Johns Hopkins University, in collaboration with Imago Pharmaceuticals. The award is for the project, “Preclinical Development of a JNK Drug Candidate to Alter Disease Progression in SMA.”

The Importance of Combination Therapies

As the SMA drug pipeline has grown in breadth, depth, and sophistication, the need for combination therapies has become increasingly apparent.

Individuals with SMA don’t produce survival motor neuron (SMN) protein at high enough levels due to a mutation in the survival motor neuron 1 (SMN1) gene. Much of the early research into SMA has focused on increasing SMN production, either by replacing or correcting SMN1 or by modulating SMN2, the low-functioning SMA “backup gene.”

Many of these SMN-based approaches, such as gene therapy and antisense oligonucleotides, are already being tested in clinical trials. Furthermore, research has also revealed that a number of systems, pathways and processes are affected in SMA, and there may be additional ways to treat SMA that work on these other areas.

And perhaps most crucially, these other approaches could be used in combination with approaches that work on SMN levels, allowing us to attack SMA from all sides and giving us the best chance of a comprehensive, effective treatment. This is particularly important as we seek to develop treatments for all ages, stages and types of SMA.

About the Sumner/Imago Project

Jun N-terminal kinase (JNK) is a stress-activated enzyme (an enzyme is a special kind of protein capable of producing specific chemical changes in cell) that is known to be activated in many neurodegenerative diseases, perhaps including SMA. When activated, JNK may cause motor neurons to function improperly and die. It may also cause muscles to atrophy in SMA.

Imago Pharmaceuticals has developed compounds that inhibit JNK and therefore protect neurons and muscle. The goal of this project is to test these compounds in SMA animal models to see if they improve survival, motor function, reduce neuron loss and/or improve muscle function, both alone and in combination with SMN enhancers. The safety of these compounds will then be tested.

If these JNK inhibitors are safe and work in SMA animal models to protect either neurons and/or muscle, they will progress into studies required by the FDA to support human clinical trials.

Driving Research to Attack SMA from All Sides

Recently, the Cure SMA Board of Directors developed a strategic plan for our SMA research funding, in collaboration with leading experts in the field. One outcome of this plan is a greater focus on developing combination drugs for SMA, in order to treat all stages and types of the disease. SMN enhancing approaches may be accentuated by combining them with drugs that target pathways that contribute to motor neuron death and/or muscle dysfunction, but act independently of SMN.

Drug Discovery Funding

This grant to Dr. Sumner is part of $704,000 in new drug discovery funding that we’re currently announcing. Drug discovery converts what we have learned about the causes and biology of SMA through basic research into new drug candidates that can be tested in clinical trials.

Pictured above: Inhibitor compounds binding the JNK Kinase.

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