Families of SMA Awards $150,000 to Drs. Hua and Krainer at CSHL
Families of SMA is dedicated to creating a treatment and cure for Spinal Muscular Atrophy by funding and advancing a comprehensive research program, including drug discovery programs to make practical new therapies. This is the fourth drug discovery project funded by Families of SMA in 2013 with a total investment of $550,000.
How Does the ASO Approach for SMA Work?
This therapeutic approach for SMA involves the use of small pieces of genetic material, called oligonucleotides, to increase the production of a missing protein. The methodology is often called Antisense Oligonucleotide (ASO) technology. In SMA, the SMN protein is reduced due to the loss of the SMN1 gene. A second closely related back-up gene called SMN2 exists that normally produces a truncated and low-functioning form of SMN protein.
How can Backbone Chemistry Influence Effectiveness?
A factor that influences the effectiveness of an ASO is the backbone chemistry used to hold the nucleotides together. The different ASO chemistry can influence many factors, including: 1) how the ASO distributes throughout the body; 2) the length of time the ASO persists in a given tissue; 3) the efficiency of ASO uptake into cells; 4) how strongly a particular ASO binds to the target RNA; and 4) the likelihood of non-specific interactions with other RNAs.
“As a Research Investigator in my lab at Cold Spring Harbor Laboratory, and as part of our long-standing collaboration with Isis, Dr. Hua has been doing pioneering work towards the development and characterization of the mode of action of ISIS-SMNRx. I am grateful for the support from Families of SMA, which will allow Dr. Hua to study key aspects of ASO efficacy in SMA mouse models, with relevance to the clinic,” said Dr. Adrian Krainer, Ph.D., Professor at Cold Spring Harbor Laboratory.
“We have a long term collaboration with Drs. Adrian Krainer and Yimin Hua to identify an antisense drug to treat spinal muscular atrophy. This has been a very productive collaboration resulting in the identification of ISIS-SMNRX, which is currently in clinical trials. We would be delighted to continue to work with Dr. Hua to investigate the mechanistic differences between the different olignucleotide chemistries. Dr. Hua has a strong track record of successfully completing projects and making important contributions to our understanding of SMA and antisense drugs,” said Dr. C. Frank Bennett, Ph.D., Senior Vice President of Research, Isis Pharmaceuticals
The SMA Drug Pipeline
There are now 15 new SMA drug programs in development, including 3 in clinical trials. This pipeline has expanded from just 4 programs 5 years ago. Families of SMA has funded over half of all the ongoing drug programs for SMA.
About Families of Spinal Muscular Atrophy
Families of SMA is the world’s leader focused on funding SMA research to develop a treatment and cure for the disease. The successful results and progress that the organization has delivered, from basic research to drug discovery to clinical trials, provide real hope for families and patients impacted by the disease. The charity has invested over $55 million in research and has been involved in funding half of all the ongoing novel drug programs for SMA. Families of SMA is a nonprofit 501(c)3 organization, with 31 Chapters and 90,000 members and supporters throughout the United States. The organization’s work has produced major discoveries, including identification of the underlying cause and a back-up gene for the disease, which provides a clearly defined target for disease altering therapies. The organization is also dedicated to supporting SMA families through networking, information and services and to improving care for all SMA patients. www.curesma.org
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