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BrainStorm Cell Therapeutics Inc. |
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Technology
BrainStorm's core technology, NurOwn™, was developed through a collaboration between prominent neurologist, Prof. Eldad Melamed, former Head of Neurology of the Rabin Medical Center and member of the Scientific Committee of the Michael J. Fox Foundation for Parkinson's Research and expert cell biologist Prof. Daniel Offen, at the Felsenstein Medical Research Center of Tel-Aviv University.
The NurOwn™ technology is based on a novel differentiation protocol which reprograms adult human mesenchymal stem cells, present in bone marrow and capable of self-renewal, to mature into many other functional cell types. The research team is among the first in the world to have successfully achieved the in-vitro differentiation of adult bone marrow cells into neuron-like cells capable of releasing dopamine as well as into MSC-NTF cells capable of releasing several neurotrophic factors, including Glial-derived neurotrophic factor (GDNF) and Brain-derived neurotrophic factor (BDNF). The ability to induce autologous adult mesenchymal stem cells into MSC-NTF cells makes NurOwn technology highly attractive for treating ALS and MS as well as PD. The NurOwn technology is protected worldwide by a number of pending patents.
With a cGMP compliant production process for NurOwn in place, the company's efforts are currently focused on demonstrating safety and efficacy in ALS patients both in Israel and the US. Plans are also underway to begin pre-clinical testing of NurOwn in MS. BrainStorm is the first company to receive clearance from the MoH to carry out a differentiated stem cell based therapeutic clinical trial in Israel. Its current objectives are completing its ongoing Phase I/II clinical trial at the Hadassah Medical Center in Jerusalem; initiating a Phase II clinical trial in the US; submitting an IND to the FDA; and initiating pre-clinical studies for MS. The company intends to enter into strategic partnerships as it progresses towards advanced clinical development and commercialization.
BrainStorm’s commercialization strategy calls for NurOwn™ to be adopted by medical centers throughout the United States and Europe. Aiming to restrict access to its proprietary technology, BrainStorm intends to establish and maintain fully-equipped cGMP certified Cell-Processing Centers in strategic locations to support NurOwn production and distribution over the broadest geographic area. Each BrainStorm Cell-Processing Center will process bone marrow harvested from patients into the required biologically activated cells, which will then be transplanted back into the patient by neurosurgeons in leading medical centers.
Market
Neurodegenerative diseases (NDDs) present a great societal and economic burden and have generated a huge unmet medical need. NDDs are characterized by deterioration of neurons that lead to brain dysfunction, resulting in impairment of motor movement and/or cognitive abilities. Affecting primarily the growing aging population, these disorders are increasing in prevalence and significantly raising healthcare costs in the Western world. Innovative therapeutic approaches that provide safe and effective treatments have a multi-billion dollar potential. According to a recent report, the U.S. constitutes the world's largest market for NDD products, worth nearly $2.3 billion in 2004, or 40% of global sales. This figure is expected to rise to over $10 billion by 2013. According to the World Health Organization, NDDs will become the world's second leading cause of death by 2040, overtaking cancer.
Chaim Lebovits has been at the forefront of mining and natural resource management in African regions for over a decade. He has spent years leading the exploration and development of resources in the Democratic Republic of Congo on behalf of world-leading firms. He has negotiated numerous, highly-successful deals on behalf of both African governments and global mining concerns. As an internationally known and respected business mogul and corporate manager, Lebovits has developed excellent personal relations with numerous African leaders, and has promoted the interests of their countries in the economic and political capitals of the world, both in official and unofficial capacities. Lebovits leads ACC HOLDINGS INTERNATIONAL and its many subsidiaries with his extensive experience and passion for the mining, oil, and energy industry, with intentions of taking ACC to a level of excellence and leadership in the resource mining sector. His commitment to the people that work with him, and to health, environmental and social issues, is evident in his projects in Africa. Originally from New York, Lebovits moved to Israel and now resides in Jerusalem. Lebovits has worked in senior positions for the worldwide Chabad Lubavitch organization and in the mining business, and launched his own private companies in 2005. Lebovits now splits his time between North America, Europe, Africa, and the Middle East. More Info
Dr. Adrian Harel joined BrainStorm in February 2011 as Acting CEO. Dr. Harel combines a broad scientific education with extensive industry experience. He holds a Ph.D. in Neurobiology, from the Weizmann Institute of Science and was a post-doctoral fellow at Washington University. He also has many years of experience in the pharmaceutical, biotech and medical device industry. In his last position, Dr. Harel was CEO of Meditor Pharmaceuticals. Dr. Harel has extensive regulatory know-how, and GMP manufacturing experience and is one of the most active entrepreneurs in the Israeli life sciences arena. He has been involved in the founding and management of more than ten ventures and brings to the company a highly developed network, as well as profound business development capabilities. More Info
Ms. Sossover has more than 15 years of experience as a financial officer in a wide range of international companies. Ms. Sossover previously served as VP of Finance for ForeScout Technologies and as VP of Finance and Secretary of Maximal Innovative Intelligence, which was acquired by Microsoft. Ms. Sossover has held positions as Chief Financial Officer at RT Set, which is now part of Vizrt and Financial Controller for BVR Technologies, which was later acquired by Esterline Technologies. Ms. Sossover has an MBA from Edinburgh University, and a Bachelor's degree in Accounting & Economics from Ben Gurion University. More Info
Prof. Melamed is a world-renowned expert in the field of neurodegenerative diseases, particularly on Parkinson’s disease. Prof. Melamed has served as head of the Neurology Department at the Rabin Medical Center and Tel Aviv University since 1987. He has held senior positions at the Hebrew University (Jerusalem), Bispebjerg Hospital (Copenhagen), National Hospital (London) and at the Laboratory of Neuroendocrine Regulation (Massachusetts). He is a past president of the Israel Neurological Association and former director of the National Parkinson Foundation (USA). Prof. Melamed is a member of the Scientific Committee of the Michael J. Fox Foundation for Parkinson's Research as well as a co-inventor named in the NurOwnTM technology patent. More Info
Prof. Offen has an internationally recognized reputation in neuroscience research. Since 1993 he has been head of the Neuroscience Laboratory at Tel Aviv University School of Medicine. He has lectured extensively, both at Tel Aviv University and at international scientific conferences, and has supervised many PhD students. Prof. Offen has published over 100 original scientific papers and several patents. Prof. Offen is a member of the Scientific Committee of the Israel Society for Neuroscience and a co-inventor named in the NurOwnTM technology patent. More Info
Professor Israeli has returned to his role as Head of the Health Policy, Health Care Management and Health Economics Department at the Hebrew University – Hadassah Faculty of Medicine. Prior to this he was the Director General of the Israel Ministry of Health (2003-2009) and the Director – General of Hadassah Medical Organization (1998 -2001). He is a Dr. Julian Rozan Professor of Family Medicine and Health Promotion Chair at the Hebrew University-Hadassah Medical School, Jerusalem (since 1996), and teaches there regularly. Professor Israeli chaired the national committee to update the Israeli national standard basket of health services. He received his medical degree and his master in Public Health from the Hadassah – Hebrew University Medical School. He completed residencies in Internal Medicine and in Health-Care Management at Hadassah University Hospital and received his Master's Degree from the Sloan School of Management at MIT, Boston. His scientific activities are related to applied, methodological and theoretical research in the fields of health policy, health care management, and the epidemiological, economic, social and cultural basis for decision-making. His publications deal with translation of academic knowledge and inputs from the field into policy setting and decision-making processes. More Info
Dr. Arbel was one of the founders of BrainStorm and served as its president until November 2004. Prior to joining BrainStorm, Dr. Arbel was the President and CEO of Pluristem, Inc., a stem cell research company traded on the OTCBB. From 1998 through 2002, Dr. Arbel served as national sales manager for Merck & Co. in Israel. She is a member of the Neurological Association and American Society of Hematology. Dr. Arbel is a certified chemical engineer and holds an MSc and a DSc in Medical Sciences from the Technion – Israel Institute of Technology. More Info
Mr. Friedman served as Chairman of the Board of The Israeli Electric Corporation from 2007 to 2010. He was Deputy Chairman and Chief Executive Officer of Deloitte Brightman Almagor Certified Public Accountants from 2005 to 2007. Mr. Friedman has been a partner and director in several business ventures and companies in Israel and abroad in the transportation, consumer business, telecommunication and energy industries. More Info
Mr. Pinkas is the Former Israeli Consul General to New York (2000-2004) and is an internationally respected foreign affairs analyst. Mr. Pinkas currently serves as an adviser at Tigris Financial Group and the Rhodium Group as well as a director for Ormat Industries Limited, B.G.I. Investments (1961) Ltd. and Agri-Invest Ltd. Mr. Pinkas has a Bachelors Degree in Political Science from The Hebrew University of Jerusalem (1986) and a Masters Degree in Politics from Georgetown University (1988). More Info
Mr. Schor is a global industry leader with vast experience in biotechnology, medical devices, business development and private equity. He led multiple licensing and M&A transactions valued over $2 billion with companies such as GSK, Amgen, Pfizer, Bayer, Merck-Serono, and OncoGeneX, and raised significant funds. Mr. Schor has a broad range of experience in multiple therapeutic areas including neurology, respiratory, oncology, auto-immune, genetic diseases, and women’s health. In addition to leading the global business development at Teva Pharmaceuticals, Mr. Schor played a key role in building early stage companies to receiving regulatory approvals, IPOs and M&As. Most recently, Mr. Schor served as VP Business Development, global branded products at Teva Pharmaceuticals. Prior to joining Teva, Mr. Schor was Chief Business Officer at Predix Pharmaceuticals, leading the formation of more than $1.5 billion collaborations with GlaxoSmithKline, Amgen and other pharmaceutical companies. Prior to joining Predix, Mr. Schor was a Partner at Yozma Venture Capital, managing the fund’s investments in biotechnology and medical device companies. Mr. Schor previously held positions at Arthur Anderson and BDO consultants and holds an MBA, B.A. in biology, B.A. in economics and is a Certified Public Accountant (CPA). More Info
Dr. Shorr's expertise ranges from technological innovation, to strategic planning and implementation of research and business development, to the founding, financing and guiding of full commercialization of biotechnology ventures. Currently, Dr. Shorr is President of Cornerstone Pharmaceuticals, a company engaged in clinical development of a novel drug delivery technology for cancer. Concurrently, as a member of the Department of Biomedical Engineering at SUNY Stony Brook, he acts as Director of Business Development for the University's Center for Advanced Technology. Since 1999 he has also served as a trustee of Tissue Engineering Charities, Imperial College, London. In addition, he has continued financial interests and investments as a Principal in Altira Capital and Consulting LLC. Dr. Shorr was also Founder, VP Technology and Chief Scientific Officer for AT Biochem (later sold to FMC) and Associate Director, Department of Molecular Pharmacology at SmithKline and French from 1982 to 1988. He has published over 100 scientific/technical articles and has more than 100 inventions, issued or patent-pending worldwide, many of them commercialized. He holds a B.Sc. in Biology from the State University of Buffalo, a D.I.C. and a Ph.D. from the University of London. More Info
Since 1995, Mr. Taub has been the Managing Member of Malcom S. Taub LLP, a law firm which practices in the areas of commercial litigation, among other practice areas. Mr. Taub also works on art transactions, as an attorney and a consultant. Mr. Taub has also served as a principal of a firm which provides consulting services to private companies going public in the United States. He has acted as a consultant to the New York Stock Exchange in its Market Surveillance department. Mr. Taub acts as a Trustee of The Gateway Schools of New York and The Devereux Glenholme School in Devereux, Connecticut. Mr. Taub has served as an adjunct professor at Long Island University, Manhattan Marymount College and New York University Real Estate Institute. Mr. Taub holds a B.A. from Brooklyn College and a J.D. from Brooklyn Law School. He has served on the Board of Directors of Safer Shot, Inc. (formerly known as Monumental Marketing Inc.), a company which trades on the Pink Sheets. More Info|
Year Ended December 31 |
Period from Inception*
through December 31 |
||
|
Income Statement Data ($K) |
2011 |
2010 |
2011 |
| Total Revenues |
0.0 |
0.0 |
0.0 |
| Operating Cost and Expenses |
3,894 |
2,589 |
41,422 |
| Financial Income (Expense), net |
-151 |
189 |
-2,547 |
| Pretax Loss |
3,913 |
2,400 |
43,837 |
| Net Loss |
3,918 |
2,419 |
44,078 |
| Diluted Loss Per Share ($) |
0.03 |
0.03 |
- |
| *Inc eption = September 22, 2000 |
|
|
|
| Balance Sheet Data ($K) |
12/31/11 |
12/31/10 |
|
Total Cash & Cash Equivalents |
1,923 |
93 |
| Current Assets |
2,304 |
579 |
| Total Assets |
2,744 |
1,089 |
| Current Liabilities |
1,135 |
1,423 |
|
Total Equity |
1,488 |
-459 |
|
|
|
| Key Financial Ratios |
12/31/11 |
12/31/10 |
| Current Ratio |
2.0x |
0.4x |
| L-T Debt/Equity |
NA |
NA |
|
|
|
| Dividend Policy |
| Company does not currently pay dividends. |
| Main Shareholders |
|
As of February 24, 2012, there were approximately 93 holders of record of our common stock. |
|
NurOwn™ In February 2011, the U.S. Food and Drug Administration (“FDA”) granted Orphan Drug designation to the Company’s NurOwn autologous adult stem cell product candidate for the treatment of ALS. Orphan status entitles BrainStorm to seven years of marketing exclusivity for NurOwn upon regulatory approval, as well as the opportunity to apply for grant funding from the U.S. government to defray costs of clinical trial expenses, tax credits for clinical research expenses and potential exemption from the FDA's application user fee. In June 2011 BrainStorm initiated a Phase I/II clinical trial for the treatment of ALS with NurOwn at the Hadassah University Medical Center after receiving approval from the ethical committee and the Israeli Ministry of Health (MoH). In January 2012, BrainStorm reported initial results from the first four patients in its ALS trial, demonstrating safety, no adverse side effects, and some clinical benefit. |
BrainStorm Cell Therapeutics Expands Pipeline with the Initiation of a Study for Multiple Sclerosis More Info
Nature: BrainStorm's NurOwn™ Stem Cell Technology Offers Hope for Treating Huntington Disease More Info
Experimental Neurology Journal: BrainStorm's NurOwn™ Stem Cell Technology Shows Promise for Treating Huntington's Disease More Info
BrainStorm Announces Clinical Data Supporting Safety and Efficacy of NurOwn™ Based on Initial Patient Results More Info
Professor Karussis: BrainStorm's Study Offers the Biggest Hope to Stop Disease Progression in ALS Patients More Info
All Announcements
BrainStorm Cell Therapeutic ist ein Biotechnologie-Unternehmen, das sich auf die Entwicklung von therapeutischen Anwendungen auf der Basis von adulten Stammzellen spezialisiert hat. Die Stammzellen werden aus autologen (eigenen) Knochenmarkzellen gewonnen und dienen zur Behandlung von neurodegenerativen Erkrankungen. Das Unternehmen konzentriert sich auf die Entwicklung von Stammzelltechnologien, die das patienteneigene Knochenmark nutzen, um Krankheiten wie Parkinson, amyotrophe Lateralsklerose (ALS) sowie Verletzungen des Rückenmarks zu behandeln. BrainStorm besitzt durch eine exklusive, weltweite Vereinbarung mit Ramot, dem Technologietransferzentrum der Tel-Aviv Universität, die Rechte zur Entwicklung und Vermarktung dieser Technologie.
Technologie
BrainStorms Kerntechnologie, NurOwn™, wurde in Zusammenarbeit mit dem renommierten Neurologen Prof. Eldad Melamed entwickelt. Er ist der ehemalige Institutsleiter des Rabin Medical Centers und Mitglied des wissenschaftlichen Ausschusses der Michael J. Fox Stiftung für Parkinsonforschung. Des Weiteren war der erfahrene Zellbiologe Dr. Daniel Offen vom Felsenstein Medical Research Center der Tel-Aviv University maßgeblich an der Entwicklung beteiligt.
Die NurOwn™-Technologie basiert auf einer neuen Zellteilungsmethode, mit der adulte humane mesenchymale Stammzellen (MSC) neu programmiert werden. Diese befinden sich im Rückenmark und besitzen die Fähigkeit zur Selbsterneuerung sowie zur Umwandlung in viele weitere funktionelle Zellarten. Dem Forscherteam ist es weltweit erstmalig gelungen, eine erfolgreiche In-vitro-Umwandlung von adulten Knochenmarkzellen (sowohl tierischen als auch menschlichen Zellen) in neuronenartige Zellen durchzuführen. Die somit hergestellten Zellen sind in der Lage, Dopamin freizusetzen. Ebenso gelang die Teilung in MSC-NTF-Zellen, die verschiedene neurotrophe Faktoren (NTF) freisetzen können, darunter das Molekül GDNF (Glial Cell-Line-derived Neurotrophic Factor) und BDNF (Brain-derived Neurotrophic Factor). Die Fähigkeit, adulte humane mesenchymale Stammzellen zur Umwandlung in MSC-NTF-Zellen anzuregen, macht die NurOwn™-Technologie zu einer vielversprechenden Möglichkeit zur Behandlung von ALS, Parkinson und multipler Sklerose sowie von Verletzungen des Rückenmarks. Die NurOwn™-Technologie ist weltweit durch eine Vielzahl von Patentanmeldungen geschützt.
Die Kerntätigkeit des Unternehmens liegt darin, die Weiterentwicklung dieser Technologie vom Laborstadium in die klinische Anwendung zu betreiben. Der primäre Fokus dieser Tätigkeit liegt momentan auf der Behandlung von ALS und Parkinson sowie von Verletzungen des Rückenmarks. BrainStorm entwickelt zurzeit einen cGMP-gerechten Produktionsprozess für die vorklinischen ALS-Sicherheitsstudien. Die klinischen Studien für ALS wurden im Juni 2011 begonnen. Die Hauptziele des Unternehmens sind die Entwicklung von Zellteilungsverfahren gemäß den FDA-Richtlinien, die Darlegung von Sicherheit und Wirksamkeit zuerst bei Tieren und dann bei Patienten sowie die Errichtung von Anlagen, die die therapeutischen Produkte von NurOwn™ für den Einsatz bei Patienten herstellen können. Das Unternehmen beabsichtigt, im Laufe der fortschreitenden klinischen Entwicklung und Kommerzialisierung strategische Partnerschaften einzugehen.
Geschäftsstrategie
Die Kommerzialisierungsstrategie von NurOwn™ soll dazu führen, dass die Produkte von medizinischen Einrichtungen in den USA und in Europa eingesetzt werden. Auf Basis eines eingeschränkten Zugangs zur firmeneigenen Technologie möchte BrainStorm voll ausgestattete und cGMP-zertifizierte Zellverarbeitungszentren an strategisch bedeutsamen Orten errichten. Diese sollen die NurOwn™-Produktion unterstützen und die Distribution in den jeweiligen Absatzmärkten ermöglichen. Jedes BrainStorm-Zellverarbeitungszentrum wird das geerntete Knochenmark der Patienten in die benötigten biologisch aktivierten Zellen umwandeln. Diese können dann dem Patienten von Neurochirurgen in führenden medizinischen Einrichtungen rücktransplantiert werden.
Markt
Neurodegenerative Erkrankungen (NDE) sind eine bedeutende gesellschaftliche und ökonomische Belastung. Sie erzeugen einen immensen, bis jetzt noch nicht verfügbaren medizinischen Bedarf. NDE werden durch die Zerstörung von Neuronen charakterisiert und verursachen Dysfunktionen des Gehirns. Dies führt zu einer Verschlechterung der Motorik und/oder der kognitiven Fähigkeiten. Da die Erkrankungen hauptsächlich die stetig zunehmende alternde Bevölkerung betreffen und somit immer häufiger eintreten werden, führen sie zu einer signifikanten Erhöhung der Gesundheitsausgaben in den westlichen Gesellschaften. Innovative therapeutische Ansätze, die sichere und wirksame Behandlungen bieten, haben ein Umsatzpotential von vielen Milliarden US-Dollar. Gemäß einer kürzlich veröffentlichten Studie verfügen die USA über den weltweit größten Markt für NDE-Produkte. Im Jahr 2004 betrug das dortige Marktvolumen fast 2,3 Milliarden US-Dollar. Dies sind 40 % des globalen Umsatzes. Laut Auskunft der Weltgesundheitsorganisation werden NDE bis zum Jahr 2040 die weltweit zweithäufigste Todesursache werden und damit sogar häufiger als Krebs auftreten.
BrainStorm Cell Therapeutics est une entreprise de biotechnologie qui développe des produits fabriqués à partir de cellules souches adultes dérivés de cellules autologues de la moelle épinière dans le but de traiter des maladies neurodégénératives. L’entreprise se consacre plus particulièrement au développement de technologies cellulaires qui utilisent la moelle épinière du patient pour traiter des maladies telles que la maladie de Parkinson, la sclérose latérale amyotrophique ou les lésions de la moelle épinière. L’entreprise détient les droits lui permettant de développer et de commercialiser cette technologie par le biais d’un contrat de licence internationale exclusive conclu avec Ramot, la société de transfert technologique de l’université de Tel-Aviv. La technologie est actuellement dans sa phase I/II d’essais cliniques relatifs à la SLA.
Technologie
La technologie de base de BrainStorm, le NurOwn™, a été développée grâce à la collaboration entre un célèbre neurologue, le Professeur Eldad Melamed, ancien directeur du département de neurologie du Centre médical Rabin et membre du Comité scientifique de la Fondation Michael J. Fox pour la recherche sur la maladie de Parkinson, et un spécialiste de la biologie cellulaire, le Docteur Daniel Offen, au Centre de recherche médical Felsenstein de l’université de Tel-Aviv.
La technologie NurOwn™ se base sur un protocole de différenciation novateur qui consiste à reprogrammer les cellules souches humaines mésenchymales des adultes présentes dans la moelle épinière et capables de se régénérer pour donner naissance à de nombreux autres types de cellules fonctionnelles.
L’équipe de recherche est parmi les premières au monde à avoir pratiqué avec succès la différenciation in-vitro de cellules de moelle épinière adulte (tant animales qu’humaines) pour isoler des cellules de type neuronales capables de libérer de la dopamine ainsi que des cellules MSC-NTF capables de libérer divers facteurs neurotropes, dont le facteur neurotrophique dérivé des cellules gliales (GDNF) et le facteur neurotrophique dérivé du cerveau (BDNF). La possibilité de produire des cellules MSC-NTF à partir de cellules souches mésenchymales autologues adultes rend la technologie NurOwn™ particulièrement intéressante dans le traitement de la SLA, de la maladie de Parkinson et des lésions de la moelle épinière. La technologie NurOwn™ est protégée au niveau international par un certain nombre de brevets.
L’entreprise concentre ses efforts sur le développement de cette technologie, du laboratoire jusqu’à l’application clinique, en se consacrant, dans un premier temps, à la SLA, pour ensuite aborder la MP et les lésions de la moelle épinière. Le processus de production de Brainstorm conforme à la norme cGMP concernant la sécurité des études précliniques sur la SLA est en cours d’élaboration et les premiers essais cliniques ont été lancés en juin 2011. Ses principaux objectifs sont le développement d’un processus de différenciation cellulaire conforme aux directives du FDA ; la démonstration de l’innocuité et de l’efficacité de ses produits d’abord sur les animaux puis sur les patients ; la création d’établissements de production des produits thérapeutiques NurOwn™ à transplanter sur les patients. L’entreprise envisage de conclure des partenariats stratégiques à mesure qu’elle progressera vers le développement clinique avancé et la commercialisation.
Stratégie d’entreprise
La stratégie commerciale de BrainStorm vise à faire adopter le NurOwn™ par les établissements de santé partout aux États-Unis et en Europe. Dans le but de restreindre l’accès à sa technologie brevetée, BrainStorm envisage d’établir et de maintenir des Centres de traitement des cellules entièrement équipés et certifiés cGMP dans des endroits stratégiques afin que la production et la distribution du NurOwn™ couvrent le plus grand territoire possible. Chaque Centre de traitement des cellules BrainStorm produira, à partir de moelle épinière prélevée sur des patients, les cellules activées biologiquement nécessaires qui seront ensuite transplantées sur ces mêmes patients par les neurochirurgiens des meilleurs établissements hospitaliers.
Marché
Les maladies neurodégénératives (NDD) représentent un fardeau social et économique ; elles ont généré un immense besoin médical qui n’a pas encore reçu de réponse. Les NDD se caractérisent par la détérioration des neurones qui entraîne le dysfonctionnement du cerveau et provoque une déficience des mouvements moteur et/ou des capacités cognitives. Affectant surtout la population grandissante des personnes âgées, ces maladies sont en augmentation et entraînent un accroissement substantiel des dépenses de santé dans les pays occidentaux. Les approches thérapeutiques innovantes proposant des traitements sûrs et efficaces représentent des bénéfices potentiels de plusieurs milliards de dollars. Selon une enquête récente, les États-Unis constituent le plus grand marché au monde en ce qui concerne les produits liés aux NDD avec un coût estimé à 2,3 milliards de dollars en 2004, soit 40 % des ventes globales. Ce chiffre devrait encore augmenter pour atteindre 10 milliards de dollars en 2013. Selon l’Organisation mondiale de la Santé, les NDD vont devenir la plus importante cause de mortalité dans le monde d’ici 2040 et dépasser ainsi le cancer.
BrainStorm Cell Therapeutics es una compañía de biotecnología que desarrolla productos terapéuticos a partir de células madre de adultos derivadas de células de médula ósea autólogas (de uno mismo), para el tratamiento de enfermedades neurodegenerativas. La compañía se centra en desarrollar tecnologías de células madre que utilicen la propia médula ósea del paciente para tratar enfermedades como el Parkinson, la Esclerosis lateral amiotrófica y daños en la espina dorsal. La compañía posee derechos para desarrollar y comercializar la tecnología mediante un acuerdo de licencia mundial exclusivo con Ramot, la compañía de transferencia de tecnología de la Universidad de Tel-Aviv. La tecnología está actualmente en las fases I/II de ensayos clínicos para la ELM.
Tecnología
La tecnología central de BrainStorm, NurOwn™, fue desarrollada mediante una colaboración entre el prominente neurólogo, prof. Eldad Melamed, anterior responsable de Neurología del Centro Médico Rabin y miembro del Comité científico de la Fundación Michael J. Fox para la investigación del Parkinson, y el experto biólogo celular, el dr. Daniel Offen, del Centro de Investigaciones Médicas Falsenstein de la Universidad de Tel-Aviv.
La tecnología NurOwn™ se basa en un nuevo protocolo de diferenciación que reprograma las células madres mesenquimatosas humanas adultas presentes en la médula espinal y capaces de autorenovación, para hacerlas madurar y convertirlas en muchos otros tipos de células funcionales. El equipo de investigación está entre los primeros del mundo en haber logrado con éxito la diferenciación in-vitro de células de médula espinal de adultos (tanto animales como humanas) en células similares a las neuronales capaces de liberar domapina, al igual que en células MSC-NTF capaces de liberar varios factores neurotróficos, incluyendo el factor neurotrófico derivado del glial (GDNF) y factores neurotróficos derivados del cerebro (BDNF). La capacidad para inducir células madre mesenquimatosas adultas autólogas en células MSC-NTF, hace que la tecnología NurOwn™ sea muy atractiva para tratar ELM y Parkinson al igual que la esclerosis múltiple y el daño en la espina dorsal. La tecnología NurOwn™ está protegida en todo el mundo por numerosas patentes pendientes.
Los esfuerzos de la compañía se dirigen al desarrollo de esta tecnología desde el laboratorio a la clínica, con un enfoque inicial en la ELM y, posteriormente en el Parkinson y el daño en la espina dorsal. El proceso de producción de Brainstorm cumpliendo la cGMP para estudios de seguridad preclínicos de la ELM está en funcionamiento, habiéndose iniciado los ensayos clínicos de la ELM en junio de 2011. Sus principales objetivos son desarrollar el proceso de diferenciación celular según las normas de la FDA; demostrar seguridad y eficacia primero en animales y después en pacientes y crear instalaciones para proporcionar los productos terapéuticos de NurOwn™ para su transplante en pacientes. La compañía se propone asociarse estratégicamente según vaya progresando hacia el desarrollo clínico avanzado y la comercialización.
Estrategia de negocios
La estrategia de comercialización de BrainStorm pretende que NurOwn™ sea adoptado por centros médicos en todo Estados Unidos y Europa. Con el objetivo de restringir el acceso a su tecnología patentada, BrainStorm establecerá y mantendrá Centros de procesamiento celular con la certificación de cGMP completamente equipados en ubicaciones estratégicas para asistir a la producción y distribución de NurOwn™ en el área geográfica más extensa posible. Cada Centro de procesamiento celular de BrainStorm procesará médula ósea extraída de pacientes dentro de las células requeridas biológicamente activadas, las cuales serán transplantadas de nuevo en el paciente por neurocirujanos en centros médicos punteros.
Mercado
Las enfermedades neurodegenerativas (NDD por sus siglas en inglés) suponen una gran carga social y económica, y han generado una enorme necesidad médica no satisfecha. Las NDD se caracterizan por el deterioro de las neuronas, lo que conduce a un malfuncionamiento del cerebro, dando como resultado una discapacidad de las destrezas cognitivas y/o el movimiento motor. Afectando principalmente a la creciente población mayor, estas afecciones están incrementándose en preponderancia y elevando significativamente los costes sanitarios en el mundo occidental. Los innovadores enfoques terapéuticos que proporcionan tratamientos seguros y efectivos tienen un potencial de muchos miles de millones de dólares. Según un informe reciente, Estados Unidos constituye el mayor mercado del mundo para productos contra NDD, con un valor cercano a 2.300 millones de dólares en 2004, o el 40% de las ventas globales. Se espera que esta cifra se eleve a más de 10.000 millones de dólares en 2013. Según la Organización Mundial de la Salud, las NDD se convertirán en la segunda causa de muerte en el mundo en 2040, sobrepasando al cáncer.
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