Neurotrophic Factors
Neurotrophic factors provide the opportunity to enhance the vitality and function of dying neurons in the brain and retina, while also enabling the neurons to withstand further degeneration. They are naturally occurring proteins which are essential during the early development of the brain and eye. While several different neurotrophic factors have been discovered, each typically provides benefit for specific neuronal populations. They have long been known to have powerful beneficial effects on damaged or dying neurons, in that elevating the levels of the appropriate neurotrophic factor can help restore the health and vitality of injured neurons. However, until now the inability to specifically deliver them to degenerating neurons has precluded their use as therapies for human diseases. Our gene delivery technology has solved these long-standing problems and is intended to provide targeted, long-term exposure of neurotrophic factors to specific neurons. Thus, our product candidates offer the opportunity to restore the health of neurons whose death is linked to serious neurological diseases. This should not only substantially improve the serious symptoms, but also partially reverse and further delay the progression of diseases such as Parkinson’s disease (PD), Alzheimer’s disease (AD), amyotropic lateral sclerosis (ALS), Huntington’s disease (HD) and several ocular diseases that cause blindness (e.g., retinitis pigmentosa, age-related macular degeneration and glaucoma). We are currently conducting human clinical trials with the hope of gaining approval for products using neurotrophic factors for PD and AD and have programs intended to advance into human testing in ALS, Huntington’s disease and several blinding eye diseases.
Therapeutic Targets
Improving function, while protecting dying neurons from further damage, represents a revolutionary approach to treating neurodegenerative diseases. It also represents a very difficult medical challenge. The causes of neurodegeneration range widely and include injury, hereditary defects, exposure to toxins, and age-related changes. The effects are often devastating and include paralysis, loss of motor skills, blindness, serious loss of mental function and even death. How then might it be possible that a single treatment may provide such benefit for the lifetime of a patient when the diseases are so complicated.
Treatment
Advances in biology have uncovered a host of naturally occurring biological proteins (called neurotrophic factors) that are responsible for maintaining the viability of cells in the brain and the retina. All these neurotrophic factors are crucial to the survival of neurons early in neuronal development. Without them, developing neurons would fail to make proper connections and would die. In the adult brain, these neurotrophic factors take on a maintenance function, keeping neurons healthy. While different neuronal cell types use different neurotrophic factors, in essence, the appropriate neurotrophic factors, if delivered in sufficient concentrations to the appropriate cells, can have truly remarkable effects. In all cases they can rejuvenate the cells, restore lost function, protect against further degeneration and even delay cell death.
Collectively, neurotrophic factors provide a remarkable opportunity to treat degenerating neurons. However, because neurotrophic factors are large proteins, they cannot be taken orally, nor can they be injected into systemic (i.e. blood) circulation, for they are neither sufficiently stable nor can they access the brain or retina where the degenerating cells exist. Because of these problems, no neurotrophic factor product has yet been successfully developed, and the lack of an effective means of delivering the growth factors to the targeted sites of degeneration has continued to obstruct their development as effective therapeutics.
However, we have developed a proprietary gene delivery technology as a practical means to deliver growth factors, with the added benefit of prolonged, sustained effects from merely a single treatment. By delivering the neurotrophic gene to a highly specific targeted area where the major neurodegeneration is occurring, a constant source of the neurotrophic factor is provided precisely to the tissues most damaged by the disease, for the lifetime of the patient.
Ceregene Core Technology
We are employing several neurotrophic factors that can restore the vitality of neurons in different brain and ocular diseases, as well as help protect these neurons from further degeneration. We have also developed proprietary gene delivery technology that allows delivery of neurotrophic factor genes to the degenerating cells that could benefit from them. This approach may thus revolutionize the treatment of a host of tragic diseases as varied as Parkinson’s disease, Alzheimer’s disease, ALS, Huntington’s disease and several ocular diseases that cause blindness, by providing the means to heal, strengthen and protect degenerating neurons.