”All our efforts focused on a single objective – to cure Multiple Sclerosis”

PROJECT ACCELERATOR PROGRAMME

The Multiple Sclerosis Project Accelerator, a unique model in Europe among the MS organisations, which streamlines and reduces the risks involved in the transfer of potential products and innovative technologies from academia to companies, and from companies to society.

The input flow of the Accelerator is shown in the following diagram:

Originating from calls and contacts, basic science projects are presented to the GAEM’s Scientific Committee.

With the potential to be transformed into therapies, biomarkers or improvements in quality of life.

In the medium-term (3-7 years).
(3-7 anys).

Fundable with a biotech model.

With an entrepreneur team.

Creation of an entrepreneur team.

Mentoring.

Business plan.

Funding.

Creation of alliances.

PROJECTS IN THE PIPELINE

Project for Cellular Immune Tolerance Therapy “TolDec EM/NMO”

Esclerosis Múltiple y Neuromielitis ÓpticaProject in development at the Institut d’Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS) and the Hospital Clínic of Barcelona.

Incorporation to the GAEM Accelerator programme: in 2012

Cellular Immune Tolerance Therapy employs dendritic cells from patients pulsed with myelin peptides to regulate the autoimmune attack on myelin.

It is a pioneering technique worldwide that seeks to achieve greater efficiency and minimize side effects.

Specific antigen therapy with dendritic cells aims to:

  • Modulate the patient’s defenses in a very specific and selective way, to slow down the inflammation that causes multiple sclerosis without altering the rest of the defenses.
  • Minimize the adverse effects of the treatment (thanks to the use of the patient’s own cells) regarding the therapies currently available.

Clinical Phase:

Phase Ib was initiated in June 2015 with 12 patients with progressive and relapsing-remitting multiple sclerosis, and with Neuromyelitis Optica, in order of evaluating the safety of the treatment.

In 2018 an extension of the clinical phase Ib was started with 10 patients, the objective of which was to confirm the safety of the treatment with a single chosen dose and to evaluate certain relevant biochemical markers of neuroinflammation.

The extension of phase Ib is expected to be completed in 2019 and, based on the results obtained, Clinical Phase II would be started to determine the efficacy of the treatment in patients and to continue with the assessment of its safety.

Preclinical data show that myelin peptides are incorporated into 90% of the treated cells, resulting in improvements in the severity of Multiple Sclerosis in animal models, and have no toxic effects.

Data from clinical phase Ib, completed in May 2017, prove the safety of the treatment administered to individuals.

This treatment would prevent the progression of the disease, since the patient’s own cells, already treated, would not present the autoimmune attack reaction to the myelin sheath. Therefore, the deterioration of myelin and the consequent physical and cognitive effects caused by Multiple Sclerosis would be avoided.

The specific Scientific Committee that was specifically set up for this project it is formed by the following members:

Dr. Pablo Villoslada (President): Director of the MS pathogenesis and therapeutics group, IDIBAPS; National Coordinator of the Spanish Network of Excellence in Multiple Sclerosis (REEM), Carlos III Health Institute; member of the Scientific Advisory Board of the International Society of Neuroimmunology; member of the MS Scientific Advisory Board en el enfoque de las sociedades de Multiple Sclerosis International Federation; a member of the editorial board of the journal PlosOne and a member of the American Academy of Neurology, the International Society of Neuroimmunology, the American Association for the Advancement of Science, the Spanish Society of Neurology and the Catalan Society of Neurology. His research is focused on the system biology analysis of neurological diseases and the translational study of neurology: new therapies and biomarkers. Dr. Villoslada has received several awards and mentions and has written several publications in scientific journals.

Prof. Dr. Roland Martin: An internationally renowned expert in Multiple Sclerosis and Neuroimmunology, with a focus on the function and development of new immunological treatments. He has previously worked in the universities of Würzburg and Tubinga (Germany), the National Institutes of Health, Bethesda (USA), the Multiple Sclerosis Centre of Catalonia (Barcelona) and the University of Hamburg (Germany) and is currently working in the MS Neuroimmunology and Research department of the University of Zurich (Switzerland). The focus of his research is on obtaining a greater understanding of the heterogeneity of multiple sclerosis by using imaging techniques (magnetic resonance and optical coherence tomography), biological markers and also of motor function. He studies the mechanisms of multiple sclerosis, focusing particularly on cellular and biochemical immunology. He specializes in areas of unmet medical needs in MS, such as neuroprotection, tolerance induction and regenerative therapies.

Prof. Dr. Lawrence Steinman: Professor of Neurology, Neurological Sciences and Paediatrics at Stanford University, and also President of the Stanford Program in Immunology from 2001 to 2011. His research focuses on the causes of remissions and relapses in MS and NMO, and the search for a specific antigen therapy. He is also developing a small therapeutic molecule for use in Huntington’s disease trials. He was the lead author of the paper published in 1992 that led to the development of Tysabri, a drug approved for treating Crohn’s disease and MS. Prof. Steinman has numerous patents, more than 40. He was also the co-founder of several biotechnology companies. He was Director of Centocor from 1988 until its sale to Johnson & Johnson.

Dr. Christian Von Buedingen: Winner of the Helmut Bauer Prize for Research in Engineering at the University of Göttingen (Germany). His work was funded by NIH / NINDS, the National Multiple Sclerosis Society, the Nancy Davis Foundation for MS Research, Small Ventures USA / Brass Family Foundation, through a donation from the Rachleff Family Foundation, and through scientific collaborations with the industry (Roche, Genentech, and Pfizer). He is a physician-scientist and was Distinguished Professor of Neurology at UCSF III between 2009 and 2016. His research focuses on B cells and antibody-mediated immunity in disorders related to multiple sclerosis. He was a clinical provider for the Multiple Sclerosis Centre of the UCSF from 2009 to 2014. Since 2016 he has held a position in translational medicine (Neuroscience) at the USCF (Switzerland).

Prof. Dr. Heinz Wiendl: Studied psychology and medicine and was a researcher at various centres and universities in Germany, in different departments, including Immunology and Neurology. He was professor of Neurology and Head of the Clinical Research Group for Multiple Sclerosis and Neuroimmunology at the University of Würzburg (Germany), Professor and Head of Neurology of Inflammatory Diseases of the Nervous System and Neuro-Oncology at the University of Münster (Germany) and was Dean of Research and Young Scientists at the Medical Faculty in Münster. His research focuses on leukocyte penetration of the blood-brain barrier, the functional interactions between the immune system and the nervous system and the mechanism of TREK1-mediated T lymphocyte transmigration via the blood-brain barrier.

This project is financed by:

Developed by:

clinicidibaps

Project AP-1

Ankar Pharma is a spin-off of the CSIC (Spanish National Research Council). Its goal is the development of new therapies for healing or improving the life quality of patients with neurodegenerative diseases.

Incorporation to the GAEM Accelerator programme: in 2014.

♦ Ankar Pharma website

AP-1 is a first-in-class molecule with an innovative dual action mechanism which favours the replacement of oligodendrocytes – myelin producing cells – and, therefore, the remyelination and neuroprotection.

Currently, the project is in the preclinical phase (animal model trial), having been proved his effectiveness in all the key components of the disease, such as: decrease of clinical symptoms, reduction of inflammation, neuroprotection, increase of remyelination, oligodendrocytes protection and, finally, prevention of the progression of multiple sclerosis.

The results obtained in animal models show a reduction in neurological symptoms and inflammation, as well as neuroprotection. Both effects would prevent the development of the disease.

This drug is capable of replacing the oligodendrocytes destroyed by the immune system, thus enabling myelin production to start again and reversing the effects of MS.

This project is financed by:

Project mentored by:

Project BN201

Bionure is a start-up emerged from the Hospital Clínic of Barcelona / IDIBAPS, based in Barcelona and San Francisco (USA).

Incorporation to the GAEM Accelerator programme: in 2016.

♦ Bionure website

BN201 is a first-in-class molecule with the dual ability of protecting neurons and regenerating the damaged myelin through a new mechanism of action. These gives it the potential to prevent axonal injury and to promote the survival of neuronal cells.

The preclinical development (in animal models) and the preclinical regulatory phase of the project were completed in 2017.

The clinical phase I has been started in 2018, during which its safety will be assessed in humans.

The results obtained in animals show a significant reversal of neuron loss, demyelination and axonal damage in various types of MS.

 

This drug would have neuroprotective effects, as well as the ability to restore and remyelinate the cell population. Therefore it could halt some effects of the disease.

This project is financed by:

Project mentored by:

3D Neuroimaging Platform

QMENTA is a start-up specialized in science and engineering, pioneering the creation of 3D neuroimaging platforms for medical diagnosis.

Incorporation to the GAEM Accelerator: in June 2017

♦ QMENTA website

(hacer clic en imagen para ver vídeo)

QMENTA is an image-based diagnosis start-up that uses the latest technologies in the image treatment. Its objective is to detect new markers that could allow to improve the diagnosis of Multiple Sclerosis and the segmentation of patients for a better treatment.

Matching the images of different diagnostic techniques, QMENTA elaborates detailed three-dimensional maps of the human brain that allow to:

  • Identify the damaged regions and assess the degree of demyelination
  • Detect if there is a relationship between affected regions
  • Track the evolution of disease symptoms and side effects of treatments

Some preliminary studies have already been performed and the technology is being validated and refined for its future application in MS patients.

The 3D neuroimaging platform is a valuable tool and help for medical experts, enabling them to:

  • Improve the diagnosis of brain diseases in a non-invasive way
  • Optimize the process of drug development and accelerate clinical trials
  • Interpret information about patients more quickly and with more reliability, therefore making possible to improve and customize the treatments

3D Neuroimaging Platform

The final objective of the collaboration between QMENTA and GAEM Foundation is to improve the diagnosis of MS affected people and to follow in detail the evolution of the brain lesions caused by the disease, as well as the side effects of the treatments.

In this way, the early diagnosis of the disease is facilitated and therapies can be customized to the maximum, depending on how the disease is expressed in each person.

This project is developed by:

Project mentored by:

Research for detecting blood biomarkers of multiple sclerosis

IDIBGI – Institut d’Biomedical Research of Girona

Incorporation to the GAEM Accelerator: in April 2019

IDIBGI website

There is scientific evidence that both genetic and environmental factors influence the pathogenesis of multiple sclerosis. Epigenetics is a key factor in understanding the gene-environment relationship and consequently the origin of MS. Our research focuses on the epigenetic study of homogeneous cell types (B and T lymphocytes) at the time of diagnosis of the disease and in more progressive stages. Our working hypothesis is based on the fact that epigenetic changes in these cell types can facilitate the clinical manifestation and progression of this disease. This study has 3 main objectives:

  • To determine the epigenetic profile of these cell types from peripheral blood in patients newly diagnosed with MS and in more progressive stages of the disease.
  • Validate the results obtained in blood at the level of the cerebrospinal fluid and the Central Nervous System.
  • Determine biomarkers of the disease at the time of diagnosis and in the progressive phases of the disease in order to predict the evolution of the disease and potential therapeutic targets.

This is a basic research project which studies lymphocyte populations from 10 newly diagnosed patients and 10 healthy controls matched by age, sex and education level. We will also study blood B and T lymphocytes from 10 subjects with secondary progressive MS. The validation of the results will be done both in cerebrospinal fluid and in brain tissue from deceased donors with MS. For the epigenetic study of lymphocyte populations, avant-garde techniques based on massive sequencing will be used.

To be able to predict by a simple extraction of blood how the patient will evolve, how many outbreaks it will have, how often it will suffer these outbreaks in the course of the disease. It will allow us to advance in the design of personalized pharmacological treatments that reverse the epigenetic alterations observed in patients with MS .

We believe that research on epigenetic biomarkers will provide more tools in clinical practice to predict the evolution of the disease. The design of drugs aimed at correcting the epigenetics of the cells of the immune system will make it possible to avoid the autoimmune reaction, neurodegeneration and therefore the progression of this disease.

https://fundaciongaem.org/biomarcadores-diagnostico-gaem/

This project is developed by:

Project mentored by:

Research to develop imaging biomarkers

Research to develop imaging biomarkers, which allow the detection, control and clinical monitoring of people affected by Multiple Sclerosis, as well as quantify the efficacy of drugs that promote remyelination and neuroprotection.

Fractalmed® by Health Engineering

Incorporation to the GAEM Accelerator: in July 2019

Web page of Health Engineering

The Fractalmed® biomarker, when analyzing the fractal dimension of brain magnetic resonance, is able to classify between patients and controls, in addition to being based on the severity of the disease with good precision.

 

The fractal dimension of brain MRI can become a useful biomarker for disease monitoring and prognosis.

This is a basic research project which studies lymphocyte populations from 10 newly diagnosed patients and 10 healthy controls matched by age, sex and education level. We will also study blood B and T lymphocytes from 10 subjects with secondary progressive MS. The validation of the results will be done both in cerebrospinal fluid and in brain tissue from deceased donors with MS. For the epigenetic study of lymphocyte populations, avant-garde techniques based on massive sequencing will be used.

The aim is to cover these needs in neurology:

  • To have biomarkers that are capable of quantifying the efficacy of drugs that promote remyelination and neuroprotection.
  • To provide an early detection tool before injuries are irreversible and early intervention can be allowed, which is very relevant in neurodegenerative diseases.
  • To provide a biomarker to monitor and predict the clinical course of brain diseases, a tool thatcan be very helpful in therapeutic decision making. The biomarker would allow predicting the response to different therapies in terms of efficacy.
  • To improve the patients quality of life, to reduce the costs of medical care and to facilitate the development of new therapies with the use of biomarkers of images.

The new imaging biomarkers will allow for more tools for earlier diagnosis and prediction of disease progression.

 

They will also allow to quantify more quickly the efficacy of drugs that promote remyelination and neuroprotection.

This project is developed by:

Project mentored by:

OUR CALLS

If you would like to be part of our Project Accelerator Programme please see the terms and conditions and application forms:

Download the files

Terms and Conditions

Application Form

Evaluation Criteria

Draft Contract

Information reviewed and validated by: Dra. Marta Príncep.

Latest update: 19 Nov 2019

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