Huntington's Disease
Ceregene is developing CERE-120 (AAV-neurturin) with the goal of reducing the symptoms of Huntington’s disease (HD), while also delaying disease progression. HD is a devastating genetic disorder characterized by severe, abnormal, debilitating movements, as well as significant personality changes and cognitive impairments. There is currently no truly effective treatment for HD, which is fatal within 15 years of symptom onset. Ceregene is currently conducting nonclinical studies to establish the necessary data required to eventually advance this program into human clinical trials of CERE-120.
Treatment of Huntington’s disease
There are currently no effective treatments for HD. While some pharmacological treatments mildly attenuate the severe, abnormal movements (referred to as choreas), the benefit is small and is directed toward a single clinical symptom of the disease (i.e., the movement disorder), while ignoring the other important deficits. HD is caused by a genetic mutation involving an expanded stretch of amino acids on a single gene. HD is passed on from generation to generation so that if either parent has HD, each offspring has a 50 percent chance of inheriting the disease. In the brain, this genetic defect causes a loss of neurons in specific brain regions such as the striatum and cerebral cortex. The loss of these nerve cells is responsible for the severe, abnormal movements, dementia, and personality disorders that are characteristic of HD.
Ceregene’s Huntington’s Disease Treatment Approach
Ceregene has performed a series of studies that have demonstrated that neurturin (NTN) can prevent the loss of vulnerable neurons and prevent or attenuate motor abnormalities displayed in animal models of HD. We have demonstrated in nonclinical studies that delivery of CERE-120 (AAV-NTN) prevents motor deficits and attenuates striatal cell loss in several models of HD.
Because treatment of HD with CERE-120 involves injecting the same brain region (striatum) as Parkinson’s disease, the same preclinical data used for that program, coupled with the excellent safety and tolerability profile seen in our CERE-120 Phase 1 and Phase 2 clinical trials for Parkinson’s disease, may be leveraged to facilitate and accelerate a clinical program for HD. We are currently performing a final series of nonclinical studies aimed at determining whether treatment with CERE-120 following the onset of symptoms, reverses functional decline, prevents the death of striatal and cortical neurons and extends the life of mice carrying the gene for HD. If these data are positive, then CERE-120 could be advanced into human testing in a Phase 1 trial for HD.
Ceregene is currently evaluating future development of CERE-120 for Huntington’s disease.