Accelerated Access for Motor Neurone Disease (MND / ALS) treatment in the UK - NurOwn

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Motor Neurone Disease (MND / ALS)

Six lives lost daily to MND in the UK and treatments already exist.

There are over 5,000 people suffering from Motor Neurone Disease (MND) in the UK. MND is also known as Amyotrophic Lateral Sclerosis (ALS) for which the Ice Bucket Challenge raised awareness in 2014.  A third of people with MND die within a year and more than half within two years of diagnosis. Six people a day are diagnosed with MND in the UK. Six people a day die from MND in the UK. The impact of MND on patients, their families and the economy cannot be overestimated. Previously, the only drug available was one not much better than a placebo.  For the first time in history patients need not die from MND as treatments now exist. However, patients don't yet have access to such treatments. 

When it comes to cancer, other diseases and historically HIV / AIDS lifesaving drugs have been granted accelerated pathways through the clinical trial and drug approval processes. Furthermore, the EU drug approval process through the European Commission / European Medicines Agency (EMA) and/or the review by MHRA/NICE in the UK to approve a new treatment for NHS patients may each lag behind the US FDA approval process by 9 months or longer. While there is a movement that is gaining momentum in the US to accelerate completion of phase 3 clinical trials and approval of drugs that have been deemed safe and effective for MND patients, there is no similar effort in the UK.  This could result in a situation where a treatment is available in the US but not in the EU or UK for a year or more until approved by the EU and/or MHRA/NICE, resulting in over 2,000 lives lost in the UK during the interval. The UK has its own accelerated process known as Accelerated Assessment / Access which the UK could potentially apply to new MND treatments before or immediately after ongoing phase 3 clinical trials are complete in the US.

https://www.gov.uk/guidance/guidance-note-on-new-assessment-routes-in-a-no-deal-scenario#accelerated-assessment-pathway

https://www.nice.org.uk/aac

NurOwn

1-  Stem cells are taken from the patient's own bone marrow (BMMSCs).

2-  The BMMSCs are harvested and enhanced using the NurOwn process to produce a two year supply.

3-  The enhanced BMMSCs are injected into the spinal fluid to preserve nerve cells and slow or halt progression of MND.  This injection is repeated more than once annually.

Effective treatments currently in phase 3 trials:

1-      NurOwn   - Stem cell therapy (Brainstorm, US)

2-      Cu-ATSM  - A basic copper compound that crosses the blood brain barrier (CMD, US/Australia) - (Phase 2/3)

The anticipated schedule in the US without an accelerated process there is listed below.

- December 2020, Completion of phase 3 clinical Trial (NurOwn only)
- June 2021, FDA review and drug approval
- March 2022, EU and MHRA/NICE approval (without Accelerated Assessment / Access in the EU or UK)

The first likely to be approved is NurOwn. It may be possible to combine both treatments potentially increasing efficacy.

[ Tregs is still in early clinical trials but is also emerging as an effective treatment for MND. The above excludes a platform trial of 5 drugs due to commence in the US in 2020.]

Unnecessarily, waiting, without an Accelerated Assessment / Access Review, for many more months to March 2022 at the earliest is another 4,250 lives lost in the UK despite two different safe and effective treatments being available. Current health / social care and economic costs relating to MND may well offset the cost of new treatments. There is certainly no reason for UK patients to wait beyond the date of FDA approval of NurOwn. A UK government initiative could potentially result in early approval before the FDA as clinical trial data is available and the same stem cell procedure is being carried out at hospitals abroad.

If the process is accelerated in the US the FDA review and drug approval processes may commence early and/or drug approval may be granted while phase 3 clinical trials are still ongoing but with sufficient data released. The disease is fatal so approving a drug that has been shown to be safe and effective can be no more fatal than time lost without treatment.The UK government does not have to rely on the US process but can take action under its Accelerated Assessment / Access Review to approve before mid 2021.

The above treatments are not a cure for MND but transform it into a disease that can be managed similar to how drugs for HIV allow sufferers to live long productive lives today.  We are at the same point in time of the drug discovery process for MND that we were in when new effective lifesaving drugs were approved to treat HIV. The only difference is that there aren’t as many influential voices calling for early access to treatment for MND patients.

What can the UK government do to save over 2,000 lives per annum and reduce the cost of MND to the economy?

1- Announce a policy on MND with the objective of providing patients with treatment at the earliest possible date by actively accelerating the approval process where possible and not simply waiting for the US to approve before taking action.

2- There is nothing stopping the UK government and MHRA/NICE approving treatments before the FDA in the US, based on existing and future clinical trial data.

3- A fast track approval process for the treatment/drug in the UK under the Accelerated Assessment / Access Review or an executive order/decree that approves the treatment/drug.

4- Instruct MHRA/NICE to open discussions with the drug companies NOW to agree supply arrangements and prices to avoid having the same discussions well after the treatment is on the market in the US while people die in the UK as treatment is either too expensive for the NHS or is yet to be approved.

5- Explore the possibility of commencing new large scale (up to 5,000 patients) clinical trials at UK hospitals. One interesting future trial could be the efficacy of combining NurOwn and Cu-ATSM.

6- In the event of a likely delay to the approval of NurOwn and/or Cu-ATSM, explore the possibility of funding patient treatment abroad, where feasible, until approved and until MHRA/NICE completes its review for the NHS to provide treatment.

7- Cu-ATSM is a simple compound to produce.  An agreement with CMD in Australia/US may achieve the objective of agreeing early availability of the drug through centres that specialise in MND such as Sheffield MND Care and Research Centre.  This drug could be administered through a large scale clinical trial.

8- The UK government and NICE should contact Brainstorm Cell Therapeutics and CMD inviting the companies to submit applications for approval / early approval.

9- The MND Association and leading MND treatment centres in the UK can be included in discussions with Brainstorm and CMD as an introduction to the treatments and to plan and prepare for patients receiving treatment.

10- MND treatment centres in the UK can start clinical trials performing the stem cell procedure without using the NurOwn platform for harvesting of bone marrow MSC until NurOwn is available in the UK. This Bone Marrow Mesenchymal Stem Cells (BMMSCs) procedure is already available in many counties.

What can you do?  Write to the Prime Minister and your MP highlighting the issue so that the government can review and approve access to MND treatments early.

While the UK excels in medicine and healthcare this does not apply to either stem cell procedures or those using Regulatory T cells (Tregs) in neurology and for treatment of MND. It therefore follows that effective treatments for MND in the UK may have to be provided in cooperation with other specialists or hospital departments other than neurology until there is change.

There is no reason for clinical trials not to be commenced in the UK using Stem Cells and Tregs to treat MND.  It may also be possible to host trials using Cu-ATSM with CMD's permission.  Either way government, MHRA, NICE, NHS and treatment centres need to start preparing NOW.

The MND Association perhaps could do more to advocate for the early introduction of such treatments in the UK and/or ensure that there is no delay upon FDA approval. Hence start planning NOW.

Every life counts!

https://thehill.com/opinion/healthcare/467580-the-real-horror-this-halloween-is-the-fdas-drug-approval-process

https://n.neurology.org/content/neurology/early/2019/11/17/WNL.0000000000008620.full.pdf?fbclid=IwAR2t-h0fav4unoep3Fas_AqLGt8ERvZ4rGMj_s6cLFCbu4zZZDF0zJY4sIA

https://alsnewstoday.com/2019/11/01/nurown-gets-good-safety-review-by-phase-3-trial-monitoring-board/

https://www.youtube.com/watch?v=bruX2CzQAgw

https://www.youtube.com/watch?v=3FI4mHtYESw

https://www.youtube.com/watch?v=AuDxBQYeiS4

https://www.youtube.com/watch?v=Ezj5iJCU5K4

https://www.youtube.com/watch?v=jrYb4mQAHFU&feature=emb_logo   [Tregs]

https://alsadotorg.wordpress.com/2019/06/24/challenge-me-to-halt-the-progression-of-als/  [Tregs]

https://alsnewstoday.com/2019/10/16/first-patient-enrolled-in-phase-2-3-trial-of-cuatsm/

https://www.als.ca/blogs/whats-the-story-with-cuatsm/

https://clinicaltrials.gov/ct2/show/NCT03280056?term=nurown&draw=2&rank=1

https://clinicaltrials.gov/ct2/show/NCT04082832?term=cuatsm&draw=2&rank=4

https://clinicaltrials.gov/ct2/show/NCT04055623?cond=als&draw=2&rank=11

https://www.massgeneral.org/neurology/als/research/platform-trial

https://www.bloomberg.com/news/articles/2019-02-05/right-to-try-law-namesake-gets-als-therapy-says-it-s-working

https://philadelphia.cbslocal.com/2019/03/11/als-patients-fighting-fda-for-experimental-drug-nurown/

https://www.facebook.com/6209609/posts/10107753561682093/