ELK GROVE VILLAGE, Ill. - June 7, 2016 - PRLog -- 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, attacking SMA from all sides, and providing the best chance of a comprehensive, effective treatment. This is particularly important in order 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 Cure SMA's 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.
At past several SMA Researcher Meetings, Cure SMA has explored this important issue with the research community. More background on developing combination therapies can be found in the 2014 Researcher Meeting Summary and the 2015 SMA Researcher Meeting Summary.
Drug Discovery Funding
This grant to Dr. Sumner is part of $704,000 in new drug discovery funding that Cure SMA is currently announcing. Drug discovery converts what Cure SMA has learned about the causes and biology of SMA through basic research into new drug candidates that can be tested in clinical trials.
About Cure SMA
Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA) - a disease that takes away a person's ability to walk, eat, or breathe. It is the number one genetic cause of death for infants.
Since 1984, we've directed and invested in comprehensive research that has shaped the scientific community's understanding of SMA. We are currently on the verge of breakthroughs in treatment that will strengthen our children's bodies, extend life, and lead to a cure.
We have deep expertise in every aspect of SMA - from the day-to-day realities to the nuances of care options - and until we have a cure, we'll do everything we can to support children and families affected by the disease.
Learn more about how you can help us reach a treatment and cure at www.cureSMA.org.
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Cure SMA Awards $300,000 Drug Discovery Grant to Charlotte Sumner, in Collaboration with Imago Pharmaceuticals
June 07, 2016 at 10:38 AM EDT