Tuesday 29 November 2011
Collaboration offers new hope for potential spinal muscular atrophy treatment
Roche has signed an agreement with PTC Therapeutics to develop drugs for spinal muscular atrophy (SMA). This will provide the crucial injection of cash needed to bring promising potential treatments being developed by PTC Therapeutics to clinical trial.
Under the terms of the agreement, pharmaceutical giant Roche gains an exclusive worldwide license to PTC Therapeutic's SMA drug development programme, which includes three compounds currently in preclinical development, as well as potential back-up compounds. In return, PTC receives US$30 million as an upfront payment, up to US$460 million upon successful completion of certain milestones, and up to double-digit royalties on commercial sales.
Individuals with SMA have two non-functional copies of the SMN1 gene in their DNA (one inherited from each parent). The SMN1 gene is responsible for the production of a protein called "survival of motor neuron" or SMN which is critical for motor neuron survival. However, scientists developing therapies for SMA can take advantage of the fact that everybody has at least one copy of a gene that is closely related to the SMN1 gene - called SMN2. This gene mostly produces shortened versions of the SMN protein that do not work but scientists are looking for drugs that increase the amount of full-length, working SMN protein produced from this gene.
A representative from PTC Therapeutics gave a presentation at the recent World Muscle Society conference about their SMA drug development programme. Researchers at the company have screened thousands of potential drugs for any that increased levels of SMN protein in cells grown in the laboratory. So far they have found three positive hits. Testing in a mouse model of SMA provided further encouraging data: SMN protein was produced and a positive effect was seen on the lifespan of the mice. Further work is now ongoing to optimize these drugs before a lead compound is identified to go into clinical trial.
Further information and links
Read PTC Therapeutics' press release.
More information about spinal muscular atrophy.
Other SMA research news.
Find out about the SMA patient registry.
Read about the SMA research we fund.
If you have any questions about this or any other research please contact us: research@muscular-dystrophy.org
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