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Lorenzo Odone, whose story was told in the 1992 Hollywood movie "Lorenzo's Oil", starring Susan Sarandon and Nick Nolte, has died in Fairfax, VA, one day after his 30th birthday. Lorenzo was diagnosed with Adrenoleukodystrophy (ALD) in 1984, aged 6. Doctors told his parents that the neurological disease would swiftly deprive him of all his faculties and lead to his death withing a maximum of two years.
Odone's parents, Augusto and Michaela, despite having no scientific background, decided to research the rare genetic disorder. Their struggle and ultimate triumph, when they found an oil that stopped the disease in its tracks and, in pre-symptomatic boys, prevented the onslaught of ALD, captivated director George Miller, who went on to make 'Happy Feet' and 'The Witches of Eastwick'. He turned the true life story into a film which won Susan Sarandon an Oscar nomination as Best Actress.
(Sarandon insisted on bringing Michaela Odone with her to the Oscar ceremonies.)
The film met with criticism from the medical community, who condemned it for setting up false hopes in families of ALD sufferers. The Odones however were amply vindicated by a study published two years ago, which showed that Lorenzo's Oil did indeed stop ALD in pre-symptomatic boys.
The Odones also founded the Myelin Project, a mould-breaking medical research charity which as well as providing leadership in the search for a cure for ALD
In 1996, Phil Collins wrote the song "Lorenzo", based on lyrics written by Michaela.
Bedridden and unable to communicate from his seventh year, Lorenzo was kept at home by his parents. Nurses cared for him round the clock, as did his indefatigable parents. Lorenzo outlived his mother, who died of lung cancer in 2002. He is survived by his father, Augusto, his brother Francesco and sister and Cristina.
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|Source: The Myelin Project|
Thursday May 29, 8:00 am ET
The new Pharmacor report entitled Multiple Sclerosis estimates that Novartis/Mitsubishi Tanabe's oral agent, FTY-720 (fingolimod) will capture nearly $900 million by 2017 in the United States, France, Germany, Italy, Spain, United Kingdom and Japan, representing the greatest market share of any emerging multiple sclerosis therapy over the next decade. The report finds that, despite limited patient share, sales of FTY-720 will be driven by its improved efficacy, acceptable safety profile, and added convenience. The launch of nine emerging therapies-including five oral drugs-will drive moderate four percent annual growth through 2017 in the multiple sclerosis drug market. Rapid uptake of premium-priced oral drugs and the growing use of monoclonal antibodies, particularly in the United States, will spark this growth.
The report also finds that other strong contenders in the multiple sclerosis market over the next 10 years will include Merck Serono's oral cladribine (Mylinax), BioMS Medical/Eli Lilly's MBP-8298, and alemtuzumab, which is marketed in the U.S. as Campath for B-cell chronic lymphocytic leukemia by Bayer HealthCare and Genzyme Oncology. Oral cladribine's first-to- market status among emerging oral agents, MBP-8298's approval for secondary progressive multiple sclerosis and alemtuzumab's impressive efficacy will drive major-market peak-year sales for each drug to the range of $500 million to $1 billion.
"Oral cladribine, which will launch in 2010 and be the first emerging oral agent to market, will experience considerable initial uptake," said Bethany Kiernan, Ph.D., analyst at Decision Resources. "In contrast, while alemtuzumab's efficacy and its convenient once-yearly dosing have impressed interviewed experts, possible safety risks could limit its uptake. Additionally, MBP-8298, which will be approved for secondary progressive multiple sclerosis, will provide a much-needed therapeutic option for this niche population."
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Department of Neurology, Mount Sinai School of Medicine, New York, NY, USA.
New insights into the complex immunopathogenesis of multiple sclerosis have led to a proliferation of promising new therapeutic strategies. While the current armamentarium of immunomodulatory medications has demonstrated beneficial effects on the disease, more effective and tolerable therapies are needed. Several novel therapeutic strategies under investigation include oral therapies, monoclonal antibodies, symptomatic treatments, insights into neuroprotection and repair as well as combination regimens.
New therapies may prove more efficacious and tolerable than the available arsenal of treatments; however, decisions regarding first-line therapies will expectedly become more complicated, with greater influence if risk-to-benefit ratios in light of premature safety data. Biomarker profiles may help elucidate disease subtypes as well as therapeutic response in an effort to individualize treatment choice.
This review will highlight recent promising therapeutic strategies under investigation in the field of MS. Mt Sinai J Med 75: 157-167, 2008 (c) 2008 Mount Sinai School of Medicine.
Neuroimmunology Unit, Montreal Neurological Institute, McGill University, Montreal, Quebec, Canada H3A 2B4.
OBJECTIVE: To review the direct and indirect effects on the central nervous system (CNS) of systemically administered immuno-modulatory therapies in use or under evaluation for the relapsing forms of multiple sclerosis (MS).
METHODS: We summarize data published by our own lab and by others that delineate the effects of such therapies on in vitro neural cell cultures and in animal model-based systems.
RESULTS: The long-approved therapies, interferon beta (IFNbeta) and glatiramer acetate (GA), do not readily access the CNS. These agents can still indirectly have an effect on disease-related immune regulatory and effector functions within the CNS by modulating the properties of systemic immune cells that migrate to this compartment. Such immune cells could interact with perivascular and innate immune cells that are involved in immune regulation and with cells that are either targets of the disease process (oligodendrocytes, neurons) and/or are involved with repair (progenitor cells). Newer agents reported to favorably impact on relapse frequency in MS include the sphingosine-1-phosphate agonist, fingolimod, and the lipophilic statin, simvastatin. Both agents access the CNS and thus represent examples of agents that could directly impact on disease-relevant injury and repair process within the CNS.
CONCLUSIONS: The observations reviewed in this report regarding indirect and direct effects of immuno-modulatory agents on the CNS indicate the need to understand and monitor the neurobiologic effects of such therapies.
Neuroimmunology Research Unit, School of Medicine, The University of Queensland, Brisbane, Qld 4072, Australia.
Myelin proteolipid protein (PLP) is the most abundant protein in central nervous system (CNS) myelin and plays a major role in maintaining the structural and functional integrity of myelin. Its abundance in, and restriction to, CNS myelin and its post-translational modification by acylation make PLP an effective autoantigen, which can induce experimental autoimmune encephalomyelitis in rodents and non-human primates and which is a target of pathogenic autoimmunity in people with multiple sclerosis, a chronic inflammatory demyelinating CNS disease.
Department of Neurology, Jiangxi Provincial People's Hospital, Nanchang 330006, P. R. China.
Baló's concentric sclerosis (BCS) is a rare demyelinating disorder usually considered a variant of multiple sclerosis (MS). However, its pathogenesis and its correlation with MS remains unclear and controversial. This report presents seven Hans Chinese subjects diagnosed as BCS on the basis of the pathognomonic MR (magnetic resonance) findings. Upon diagnosis, all the cases displayed good responses to corticosteroids and showed an overall benign prognosis during a follow-up period of 4-13.5 years, although three relapsed later. MR findings suggest that the characteristic concentric lesions of BCS frequently (5/7) coexist with multiple sclerosis-like lesions. During follow-up, the Baló-like lesions may either dissolve over time or transform into an MS-like lesion. Moreover, the Balóand MS-like lesions occurred one after another at the onset and relapse phases of the same patient in two cases. These clinical features suggest that Baló's disease showing benign clinical course and co-existence of multiple sclerosis (MS)-like lesion is not rare among the Chinese, and strengthens the notion that BCS correlates intrinsically with MS. Multiple Sclerosis 2008; 14: 418-424. http://msj.sagepub.com.
By Eva von Schaper and Dermot Doherty
Extavia, an interferon beta-1b, was approved for use in patients with early and relapsing forms of MS, Basel, Switzerland-based Novartis said today. Betaseron, also known as Betaferon, was first approved in Europe in 1996.
Novartis, Switzerland's second-biggest drugmaker, plans to start selling the injected treatment in the first half of 2009, giving it a foothold in the multiple sclerosis market before the planned introduction of its experimental drug fingolimod. Fingolimod, which could be the first oral MS disease medicine, may generate more than $1 billion in annual sales, analysts say.
Approval of Extavia ``is fundamentally positive because Novartis can now prepare and build up to the launch of their own product,'' Andrew Weiss, an analyst at Bank Vontobel in Zurich, said in an interview.
Weiss forecasts peak sales of $340 million annually for Extavia. Novartis' best-selling drug, the Diovan high-blood pressure treatment, had sales of more than $5 billion last year. The company is looking for new treatments to help it weather the loss of patent protection on Diovan and the Gleevec cancer medicine in the next few years.
Betaseron generated 1.03 billion euros ($1.62 billion) in global sales last year, about 3 percent of Bayer's total revenue of 32.4 billion euros.
Novartis fell 80 centimes, or 1.5 percent, to 53.45 Swiss francs at the close of Zurich trading. Bayer shares declined 18 cents, or 0.3 percent, to 54.52 euros in Frankfurt.
The introduction of Extavia ``was expected and we've had to reckon with it,'' Carsten Kunold, an analyst who follows Bayer at Merck Finck in Munich, said in an interview.
Novartis gained rights to Extavia through its acquisition of Chiron Corp. in 2005 and in agreement with Bayer, which gained Betaseron with the 2006 purchase of Schering AG. Bayer last year agreed to pay Novartis, which makes Betaseron under contract, $110 million for a California plant and another $90 million for inventories of the drug.
Betaseron competes with Biogen Idec Inc.'s Avonex, Merck Serono SA's Rebif, Teva Pharmaceutical Industries Ltd.'s Copaxone and Elan Corp. and Biogen's Tysabri for share of the MS market.