reallyreally wrote: I have actually been excepted into the Dr Burt trial! Wish me luck
HarryZ wrote:reallyreally wrote:The point was that she used chemo. I was only offered FDA approved treatment. (which were available in '98 when she was diagnosed). Maybe I am mistaken, but I believe I have to be in a clinical trial (which I am going to be) for a DR. to try a non approved therapy? Or, at least failed every FDA approved therapy and getting worse. Again, maybe I am wrong.
And with a little look around. The drug could have been Rituximab a "B cell" therapy. There are some articles with Dr Weiner and research with the drug. This is the drug that made it with huge success into phase 2 clinical trials. I think though, sadly, it will become generic in the next couple years. So, there will not be a phase 3.
Any time a patient uses a drug that has not been approved for his/her specific disease, then its use is called "off label". As long as a doctor prescribes the use of the drug, then it is OK and does not need FDA approval.
FDA approval is required if you want to advertise a drug for a specific disease. Clinical trials are required to prove to the FDA that the particular drug meets certain criteria in the disease's treatment.
Over the years we have seen many drugs that have shown tremendous promise in the treatment of MS. Even during some clinical trials, the results look promising but once the medication starts getting used in the general population, the results often become far less effective. The CRAB drugs are but one example.
The challenge of multiple sclerosis: how do we cure a chronic heterogeneous disease?
SourcePartners Multiple Sclerosis Center, Center for Neurologic Diseases, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA. firstname.lastname@example.org
Multiple sclerosis is (MS) a T-cell autoimmune disease characterized by a relapsing-remitting followed by a progressive phase. Relapses are driven by the adaptive immune system and involve waves of T helper cell 1 (Th1), Th17, and CD8 cells that infiltrate the nervous system and provoke a attack. These cells are modulated by regulatory T and B cells. Infiltration of T cells into the nervous system initiates a complex immunological cascade consisting of epitope spreading, which triggers new attacks, and activation of the innate immune system (microglia, dendritic cells, astrocytes, B cells), which leads to chronic inflammation. The secondary progressive phase is due to neurodegeneration triggered by inflammation and is driven by the innate immune system. Why a shift to the progressive stage occurs and how to prevent it is a central question in MS. Effective treatment of MS must affect multiple disease pathways: suppression of proinflammatory T cells, induction of regulatory T cells, altering traffic of cells into the nervous system, protecting axons and myelin, and controlling innate immune responses. Without biomarkers, the clinical and pathological heterogeneity of MS makes treatment difficult. Treatment is further hampered by untoward adverse effects caused by immune suppression. Nonetheless, major progress has been made in the understanding and treatment of MS. There are three definitions of cure as it applies to MS: (1) halt progression of disease, (2) reverse neurological deficits, and (3) prevent MS. Although the pathways to each of these cures are linked, each requires a unique strategy.
Curing Multiple Sclerosis
Immunotherapy to Halt Disease Activity and Progression
One could argue that a cure is a treatment that eradicates MS. This may be true for an infection or tumor but not for MS in which there is an inherent defect of
the immune system and chronic inflammation of the brain. If, however, one treated MS at the clinical onset and prevented disease progression for the remainder of
the patient’s life, it would be considered a cure. There may be patients who are being “cured” with current therapy, though they may have less severe forms of the
disease. Interferons and glatiramer acetate are only partially effective in MS, with stronger effects observed with natalizumab and alemtuzumab.This raises a
central question: Will aggressive and early immunotherapy prevent the secondary progressive form of the disease? With the strong antiinflammatory effects of a
drug such as alemtuzumab, this question could be addressed in the future, though widespread testing may be prohibited by adverse effects. Other approaches of
aggressive immunotherapy at disease onset to test this hypothesis include bone marrow transplantation, nonablative chemotherapy utilizing cyclophosphamide, or
induction with a drug such as cyclophosphamide or mitoxantrone followed by maintenance immune modulation. We found that a short course of cyclophosphamide
was very effective in shutting down disease activity in early aggressive MS, but less effective in older patients who had entered the later progressivestages. This
phenomenon was also observed in trials of beta interferon in progressive MS and with other anti-inflammatory drugs such as alemtuzumab and rituximab.
Patients most likely to respond to drugs with anti-inflammatory actions are those with active disease progression and adaptive immune activity as evidence
by continued relapse activity.
georgegoss wrote:Except that immune dysfunction has already been scientifically proved as the root of MS pathology, regardless of the funding source to develop the data
Further, if the immune system were not at the pathological root of MS, then clearly several hundreds-of-thousands of patients around the world would not have their disease progression slowed by immunomodulator therapies which the population studies have already conclusively demonstrated as real. (And it is also known that immunomodulaor drugs don't work for everyone because of the immune system's spreading epitope, the reason that HSCT has a superior curative effect for most MS patients compared to any/all other theraputic approaches.)
Although the specific role of T-regs is not yet completely elucidated (I'm sure it will be someday), this is far FAR more plausible of an explanation as compared with any speculation about non-immune mediated pathology of MS.
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