Website Search
ID 16954

Clinical Trials for New SMA Therapies

Dr. Charlotte Sumner discusses the clinical trial process for SMA antisense oligonucleotide therapy.
It's a long process to develop a new treatment and although there's enormous excitement about the treatments that have shown so much promise in SMA animal models, there are very, very important steps that have to occur before a treatment is really available and has been shown to be effective and safe for patients. So we still have a lot to do regarding some of these new very exciting therapies. Things are moving forward very rapidly and it's very exciting, but studies need to occur in a certain systematic manner and the first part of that is always what we call phase 1 clinical trials. And these are trials that are really centered on demonstrating the safety of a particular new drug. The other thing is the drug because it doesn’t pass the blood brain barrier, and we believe that it has to be in the CNS in order to be effective, we have to give the drug via an intrathecal route, via delivery to the CNS directly. And we have to make sure that that's going to be safe. We think it should, but these different parameters have to be evaluated first before subsequent phases of a clinical study can go forward. So the first step with the antisense oligonucleotide will be to do a phase 1 study in SMA patients to evaluate whether or not it's safe or not. If it is safe, then there will be phase 2 and hopefully subsequently phase 3 studies and these are studies that continue to evaluate safety of course, but also begin to look for evidence of efficacy of the compound whether or not the drug is really showing benefit for SMA patients.
Spinal muscular atrophy, SMA, RNA, mRNA, splicing, gene, genetic, DNA, antisense, motor neuron, splice, Therapy, translate, phase, Phase 1, Phase 2, Phase 3, Phase 4, clinical, clinical trial, efficacy, safety, model system, cell, animal, human, effective, safe, randomized, placebo, controlled, Hopkins, john Hopkins,
Creative Commons License This work by Cold Spring Harbor Laboratory is licensed under a Creative Commons Attribution-Noncommercial-No Derivative Works 3.0 United States License.

Related content:

16950. 2D Animation of Antisense Oligonucleotide Therapy for SMA
An animation shows how antisense oligonucleotide therapy for SMA utilizes RNA splicing.
16948. Alternative RNA Splicing Therapy for Spinal Muscular Atrophy
Drs. Sharp and Sumner describe how RNA splicing can be used as a therapy for SMA.
16952. 2D Animation of Intrathecal Drug Delivery for SMA
An animation shows how drugs for SMA may be delivered via infusion into the spinal cord.
16953. Gene Therapy for SMA
Dr. Sumner talks about gene therapy for SMA.
16939. RNA Splicing
A step-by-step 2D animation shows the details of RNA splicing.
16931. 2D Animation of Genes and Inheritance
An animation introduces DNA and the pattern of inheritance for SMA.
16943. Timing of SMA Therapies
Dr. Sumner describes how early intervention is crucial when treating SMA.
16946. An Overview of Future Therapies for SMA
Dr. Sumner provides an overview of targets for SMA therapies.
16941. 2D Animation of Alternative RNA Splicing
An animation shows alternate splicing of the SMN2 gene.
16947. Small Molecule Therapy for SMA
Dr. Sumner describes small molecule therapies for SMA.
Cold Spring Harbor Laboratory
CSHL HomeAbout CSHLResearchEducationPublic EventsNewsstandPartner With UsGiving