Here you can find the text of the PETITION 0635/2021

"Amyotrophic Lateral Sclerosis CALL FOR ACTION"

Petition addressed to:

• European Commission

• European Parliament

• European Council

• President of the European Commission (Ms Ursula von der Leyen)

• President of the European Parliament (Mr. David Maria Sassoli)

• President of the European Council (Mr. Charles Michel)

• EMA Executive Director (Ms Emer Cooke)

The Amyotrophic Lateral Sclerosis - ALS - also known as Lou Gehrig's Disease and Charcot's disease, is a relentlessly progressive neurodegenerative disease which attacks motor neurons in the brain - Upper or First Motor Neuron - and in the spinal cord - Lower or Second Motor Neuron - resulting in the wasting away of muscle and loss of movement.

Progress is generally rapid, with an average life expectancy of between 2 and 5 years from the onset of symptoms.

After 150+ years from first diagnosis, there is no cure at all for ALS! This is astonishing. This is inhuman and simply unacceptable. ALS is 100% lethal.

ALS Community too deserves the level of creativity, cooperation, sense of urgency and sense of responsibility shown by the various Ministries of Health of the different countries, the EMA, the FDA, the EU, the Local Regulatory Agencies and the pharmaceutical industry in the COVID19 pandemic.

ALS Community is in desperate need of help and EC & EP have the duty and the power to change the outcome of a story that already seems to be written.

THE REQUEST is based on 4 pillars

1. Clinical Research

   1. Investments to boost Scientific Research - As part of the EU4Health 2021-2027 & Horizon 2020 Project, there must be a dedicated chapter of expenditure to fund scientific research for ALS. The annual budget, owned and coordinated by the European Commission, has to be € 350 M until a cure is found. The European Commission must engage cutting-edge companies to fund & develop drugs as done for COVID19 Vaccines. ALS is not incurable, it is just underfunded - and time is ticking ineluctably.

   2. Centre of Excellence for Genetic Research - EU must create a clinical site aimed at sequencing the entire Human Genome. The scope for this initiative is simply invaluable as the potential to discover the pathogenesis and etiology of several diseases is concrete. All inherited forms are genetic but not all genetic forms are inherited. Genome sequencing could open up to the solution not only of ALS but also of multiple diseases such as Frontotemporal Dementia, Alzheimer's, Parkinson, Diabetes and many more. CRISPR based approach could then be key.

   3. Centre of Excellence for Regenerative Medicines - EU must create a clinical site aimed at repairing damaged tissues through Stem Cells use. Restoring/reverting damage can give back full functionality. Also in this case, the potential to restore from damages caused by disease or trauma is enormous. Bioengineering and biotechnology approaches are key.

2. Drugs Early Access

   1. Allow Use of Experimental Drugs - Considering there is no valid therapeutic alternative, EMA has to grant early/accelerated access - under Conditional Marketing Authorization, for: (i) drugs not yet authorized but undergoing clinical trials. Medicinal products not yet authorized, can still be in clinical trial phase and have completed studies, at least in Phase 2, that demonstrate adequate efficacy with an acceptable risk profile to support the requested indication. Early/Accelerated Access does NOT jeopardise any trial.

   2. Compassionate Use Take-Over - Considering there is no valid therapeutic alternative, given the unique nature of this disease, grant early access - under Compassionate Use, fund by EC for: (i) drugs authorized outside the EU; (ii) drugs to be used for a therapeutic indication other than that authorized; (iii) orphan drugs.

3. Process Simplification

   1. Shorten Drugs Evaluation Time (EMA/EC) - Create a dedicated/accelerated pathway for the regulatory process of drugs related to ALS to speed up the overall review and approval. The dedicated pathway for the drugs addressing neurodegenerative diseases has to be managed by a dedicated team (as strengthening of “therapies for neurological and psychiatric disorders” part) and the overall evaluation and approval by EMA must take no longer than 45 days. The following approval by EC must take no longer than 15 days.

   2. Streamline Drug Approval Process (with Member States) - EC, EP and EMA must work with member states in order to smooth the adoption of the new drugs, reducing the time-to-market and avoiding redundant steps. After the EMA approval and the EC one, there is very little need to have another end-2-end validation process from the National Regulatory Agency (including a review from the Ethics Committee) and Country Ministry of Health.

4. Family Support

   1. Patient’s Family Contribution - In order to support family expenses for the caregiving and the daily tasks, it is fundamental to receive a monthly contribution from the EC. This kind of lump sum is vital to guarantee an adequate standard of life and should be in the range of € 1.500 - € 2.000 per month.

The POWER OF 1

The European Commission must be in charge of centralized investments for coordinated Scientific Research (point 1.1) & Centers of Excellence (points 1.2 and 1.3) in order to establish both a Standardized & Common Framework and an Efficient & Effective end-2-end Workflow, maximizing the return on investments and the researchers’ efforts and reducing the waste of TIME, money as well as redundant studies.

The European Commission must consider exploiting and leveraging the existing European Network (e.g. ENCALS, TRICALS) to boost the research and bring it to the next level in order to find a definitive solution.