NurOwn and the Therapy Development Process

The ALS Association, our partner ALS organizations, and the wider ALS community are all hopeful that several therapies currently in Phase 3 clinical trials will prove to be successful in slowing, halting, or reversing ALS. 

One of the therapies now in Phase 3 trials, NurOwn, has been the subject of considerable discussion on social media. Some of what is being stated on social media is unfortunately not accurate, and risks misleading people with ALS and their caregivers.

Below, we offer some clarifications about NurOwn and the therapy development process. 

NurOwn, a therapy developed by BrainStorm, showed promise in its Phase 2 trial and has now entered a Phase 3 clinical trial.  

Normally, access to a therapy is possible only after a successful Phase 3 Clinical trial and subsequent FDA approval. However, there are two legal pathways for NurOwn to be made available to people with ALS while it is undergoing clinical trials — Expanded Access and Right to Try. In both cases, the company that makes the experimental drug has to grant permission and supply the treatment. BrainStorm has indicated on multiple occasions that it will not provide NurOwn to individuals via Expanded Access or Right to Try. This is not uncommon, due to the costs and other factors that companies weigh when considering Expanded Access or Right to Try.

The ALS Association has been asked by some activists to demand that the FDA approve NurOwn or that President Trump sign an executive order approving NurOwn. Until BrainStorm applies for a biologics license for NurOwn, however, there is nothing the FDA or the President can do.  BrainStorm has said it will not seek early approval of NurOwn and that it intends to complete its Phase 3 trial of NurOwn. 

Although we do not have access to BrainStorm’s data, we do know that its trial is being run by excellent scientists. If BrainStorm is not ready to apply for approval, we have no choice but to respect its judgment. When Brainstorm does apply for a biologics license for NurOwn, The ALS Association stands ready to ask the FDA for rapid review. 

We will continue to work urgently on promising experimental treatments, whether we fund them directly or facilitate the work of others. Right now, we are funding over 120 grants in 13 countries, including 61 new grants awarded in 2018. There are 31 interventional drug trials that are currently enrolling patients. We have supported at least 12 of them during their development. We also continue to provide significant financial support for the infrastructure needed to ensure ALS trials run effectively. We support Certified Treatment Centers of Excellence around the country and the NEALS clinical trial consortium, where almost all ALS clinical trials are taking place. 

A webinar was held on December 18 which highlighted our research program in more detail and addressed additional questions about drug development, including NurOwn. You can watch the full webinar below.

8 thoughts on “NurOwn and the Therapy Development Process”

  1. I am begging the ALSA to please help make this treatment available and funded before it’s too late for me. It is apparent that it is much more effective than the other drugs that have been supported.

    Liked by 1 person

  2. Thank you for addressing what is an emotionally charged and confusing subject for pALS and their loved ones. Has the ALSA had discussions with Brainstorm or internally about supporting the Phase 3 study financially? Would additional funding potentially speed up the Phase 3 study and (hopefully) subsequent FDA approval?

    Like

  3. “…there are two legal pathways for NurOwn to be made available to people with ALS while it is undergoing clinical trials — Expanded Access and Right to Try. In both cases, the company that makes the experimental drug has to grant permission and supply the treatment.” Question: is this type of access available to ALS patients outside the USA?

    Like

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