A new, experimental gene therapy demonstrated promising results for the treatment of cerebral adrenoleukodystrophy (CALD). The treatment, called LENTI-D, is being developed by bluebird bio (BLUE). Results from the study, called STARBEAM, were presented at the American Academy of Neurology meeting.
The primary endpoint of the study is freedom from "major functional disabilities, " such as the losing ability to communicate or wheelchair dependence (MFD-free), and will be assessed two years following treatment. In this interim report, all 17 treated patients remained free of major functional disabilities, with three patients completing two years follow-up and the remaining 14 patients completing at least six months of follow-up. While encouraging, the results are preliminary, and more follow up is needed prior to FDA approval.
This is the second therapy based on blood stem cell modification to make news in recent weeks. The first, called Strimvelis, received a positive opinion from the European CHMP, which recommended approval to the European Commission for the treatment of severe combined immunodeficiency.
Gene therapy has the potential to treat or even cure many chronic diseases, and as such, it has become an increasing area of focus for medical research. Researchers around the globe are investigating the application of gene therapy to HIV, diabetes, cancer, and many other serious conditions. A recent report by the market research firm RNCOS looked at the clinical applications of these treatments and how they could change the health care sector in the coming four years.
According to the report, titled “Global Gene Therapy Market Forecast to 2020,” the majority of research on gene therapy is done in the context of oncology, especially in relation to cancers that are traditionally resistant to treatment. However, the scope of gene therapy research is broad, encompassing developments in treating conditions from inherited blindness to cardiovascular disease.
So far, the vast majority of gene therapy products are still in the research and clinical development phase, with few therapies commercially available. The majority of financing in the field comes from funding, grants, and private investors. Promising clinical data, regulatory approvals, and commercial revenue are the key ingredients to fueling additional investment and excitement in the field.
