Advancements Made in Treating Colorblindness

Scientific advancements are helping people with colorblindness to see the world more vividly.

Researchers are working on several fronts to combat color blindness, a genetic condition that primarily affects men, reducing color vision to as few as two colors. People with colorblindness typically have photopigments in their eyes that are less sensitive to certain colors and cannot translate those hues to the brain. 

Husband-and-wife researchers Jay and Maureen Neitz of the University of Washington have used retinal surgery and gene therapy to treat color blindness in squirrel monkeys. Their method, which is being developed for humans as the result of a partnership with the company Adverum (formerly Avalanche Biotechnologies), replaces a defective gene with one that functions correctly. 

Currently, there are no treatments available to help people with color blindness. Although some claim that spectacles can help, they have been tested in clinical trials, and no available data supports their use.

Gene Therapy’s Potential for Treating Neuroblastoma

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.

The Financial Future of the Gene Therapy Market

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.

Gene Therapy Offers Hope for Patients with Eye Diseases

Ophthalmic research in gene therapy could lead to treatments for a number of diseases that have hitherto been untreatable, Avalanche Biotechnologies CEO Thomas Chalberg recently told Retina Today. The field is seeing a great deal of interest and innovation, said Chalberg, which could usher in a new generation of medical therapy and a new class of biologics.

Avalanche's current trials focus on treatment for wet age-related macular degeneration. So far, participants in the trials have not exhibited any serious adverse events related to the medication. Avalanche Biotechnologies, a leading developer of ophthalmic gene therapy, also is investigating the potential for gene therapy to treat vascular diseases such as diabetic macular edema and retinal vein occlusion.

“The idea of using DNA as a pharmaceutical is a sort of marvelous and wondrous proposition,” Chalberg said. “But why now? Because there are positive clinical data emerging that demonstrates that gene therapy is working.”

Gene therapy may not be available in clinics for a few years, but research increasingly shows that it could significantly change how retina specialists treat certain ocular diseases.                            

Technology Pioneer 2015 by Thomas W Chalberg

Thomas W Chalberg of Avalanche Biotech discusses gene therapy to improve and preserve the sight of people with serious eye diseases with an unmet medical need.