Exploring Patient Access to NurOwn®:
Brainstorm is exploring the possible commencement of a program to make NurOwn® available to a limited number of patients with Amyotrophic Lateral Sclerosis (ALS, also known as motor neuron disease) through a regulatory pathway designated for an orphan drug with a good safety profile and strong efficacy signals. This program would potentially provide patients, who today face an unmet need, with access to this investigational treatment before it receives final marketing approval from the regulatory authorities.
The decision to proceed with this program will be based on responses collected from patients that demonstrate that there is interest in receiving treatment via this pathway. Making NurOwn® available in this way will require Brainstorm to invest in additional manufacturing facilities, recruit additional manpower and also to submit regulatory applications in various western countries.
NurOwn® uses proprietary culture conditions to induce mesenchymal stem cells (MSCs) to secrete high levels of neurotrophic factors known to promote the survival of neurons (nerve cells). The product is manufactured using a process that involves taking a sample of bone marrow from a patient and then sending the bone marrow to a manufacturing facility where MSCs are isolated and grown under carefully managed conditions. At the end of this process NurOwn® is harvested, placed into syringes and is ready to be delivered to the patient for an intrathecal administration.
Given the high costs associated with manufacturing, patients will be required to pay a fee in order to gain access to NurOwn®.
NurOwn® has been administered to approximately 70 patients with ALS in clinical trials conducted in the United States and Israel. In a randomized, double-blind, placebo-controlled clinical trial conducted in the U. S., a clinically meaningful benefit was demonstrated by higher response to NurOwn® compared with placebo. NurOwn® is not currently approved in the United States, Israel or elsewhere. There is no guaranty that this treatment will be effective or that there will not be any adverse effects for patients as a result of this treatment.
As previously announced, Brainstorm plans to conduct additional clinical trials with the goal of obtaining regulatory approval for NurOwn® in the United States and other major markets.
Patients interested in participating in this program to achieve early access to NurOwn®, should click on this link and provide their information.