There are currently several potential therapeutic products going through both pre-clinical and clinical development. Clinical development, or clinical trial, is a rigorously controlled test designed to examine the safety and/or effectiveness of medicines, devices, treatments, or preventive measures in humans. Clinical trials follow a strict protocol. The protocol describes the aims, design and organisation of a clinical trial. It also provides information on the background and reasons for a trial and outlines its study plan. The plan must be carefully designed to ensure the safety of the participants as well as answer specific research question(s).
Clinical trials for SMA
The identification, development and approval of drugs is a long, costly and difficult process. It is estimated that 9 out of 10 experimental drugs starting human clinical trials never gain final approval.
Researchers have identified four main different therapeutic approaches that show promise in treating SMA (please click on the approaches for more information):
- SMN1 gene replacement
- Modulation of the SMN2 ‘back-up’ gene
- Neuroprotection of motor neurons
- Muscle protection
Products in pre-clinical discovery and in clinical trials
Clinical trials in the UK and continental Europe
SMN2 gene enhancement
- Ionis/ Biogen Spinraza (aka Nusinersen; Ionis-SMNRX). On 7th October 2016, the companies filed an application to the European Medicines Agency (EMA) and the FDA in the US for marketing approval. Please read the companies’ community statement. On 23rd December 2016, the FDA approved Spinraza for SMA.
- PTC Therapeutics/ Roche – Moonfish (RG7800) is currently on hold.
- PTC Therapeutics/ Roche/ SMA Foundation – RG7916 . Recruitment for two phase 2 clinical trials has started in December 2016 in SMA types 1, 2 & 3 through trials called Firefish & Sunfish. It is anitcipated that a 3rd trial, Jewelfish will begin recruitment early in 2017. RG7916 is an orally available drug that aims to correct the splicing of SMN2, the “backup gene.” A community update was provided.
- Novartis’ LMI070. The recruitment to this trial is on hold. A statement has been provided by the company to update the community.
Motor neuron protection
- Roche (Trophos’) Olesoxime. The company filed an application with the European Medicines Agency in August 2015. The scientific advice received has led to Roche to decide to conduct a phase 3 clinical trial. A community statement was provided.
Clinical trials in the US
SMN2 gene enhancement
- PTC Therapeutics/ Roche – Moonfish (RG7800) – Patient recruitment currently suspended (see above).
SMN1 gene replacement
- Avexis – scAAV9.CB.SMN, AVXS-101, is being trialled at The Nationwide Children Hospital Research Institute (Ohio – USA).
- Cytokinetics – Tirasemtiv (CK-2127107) – This compound is a muscle activator and is the seventh SMA drug program to advance to clinical trials.