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 doesnâ€™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.
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- ID: 16954
- Source: DNALC.SMA
- Download: MPEG 4 Video
An animation shows how antisense oligonucleotide therapy for SMA utilizes RNA splicing.
Drs. Sharp and Sumner describe how RNA splicing can be used as a therapy for SMA.
An animation shows how drugs for SMA may be delivered via infusion into the spinal cord.
Dr. Sumner talks about gene therapy for SMA.
A step-by-step 2D animation shows the details of RNA splicing.
An animation introduces DNA and the pattern of inheritance for SMA.
Dr. Sumner describes how early intervention is crucial when treating SMA.
Dr. Sumner provides an overview of targets for SMA therapies.
An animation shows alternate splicing of the SMN2 gene.
Dr. Sumner describes small molecule therapies for SMA.