Wiadomości

witam,

https://www.neals.org/for-people-with-als-caregivers/educational-webinars/results-from-the-immunosuppression-in-als-trial

pozdrawiam

Udało mi sie załatwic z Japonii.
Pozdrawiam 

Witam,

Ja mam SLA od trzech lat i mam niedowład czterokończynowy, opuszkowa forme choroby.
I ja biore od 2 tygodni nowy lek Edaravone i moja kinaza kreatynowa spadła z 500 do 180 czyli do normy z przed choroby. Badania aktywności kinazy kreatynowej w surowicy wykorzystuje się w diagnostyce medycznej. Podwyższony poziom CK świadczy przede wszystkim o uszkodzeniu tkanek, szczególnie mięśni. Moge przyjąć, że moja choroba sie zatrzymała

A czy jest ktos kto jeszcze probowal tego leku i badal sobie kinaze kreatynowa?
Pozrawiam

http://www.massgeneral.org/als/news/multimedia.aspx?id=970 pozdrawiam:)

Witam , mam artykuł , że witamina B3 a dokładnie jej formy pomagają w chorobach  neurodegeneracyjnych          

This is the main reason I take nicotinamide, Niagen and nicotinic acid (all types of vitamin B3). Overactivation of the enzyme called PARP-1 leads to rapid depletion of its substrate NAD. NAD depletion causes mitochondrial dysfunction and cell death. High dose B3 boosts NAD synthesis, thereby preventing neuronal cell death.

Semin Cell Dev Biol. 2016 Nov 17. pii: S1084-9521(16)30395-0. doi: 10.1016/j.semcdb.2016.11.007. [Epub ahead of print]
Poly(ADP-ribose)Polymerase-1 hyperactivation in neurodegenerative diseases: the death knell tolls for neurons.
Narne P1, Pandey V1, Simhadri PK1, Babu PP2.
Author information
Abstract
Neurodegeneration is a salient feature of chronic refractory brain disorders like Alzheimer's, Parkinsons, Huntington's, amyotropic lateral sclerosis and acute conditions like cerebral ischemia/reperfusion etc. The pathological protein aggregates, mitochondrial mutations or ischemic insults typifying these disease conditions collude with and intensify existing oxidative stress and attendant mitochondrial dysfunction. Interlocking these mechanisms is poly(ADP-ribose) polymerase (PARP-1) hyperactivation that invokes a distinct form of neuronal cell death viz., 'parthanatos'. PARP-1, a typical 'moonlighting protein' by virtue of its ability to poly(ADP-ribosyl)ate a plethora of cellular proteins exerts diverse functions that impinge significantly on cellular processes. In addition, its interactions with various nuclear proteins like transcription factors and chromatin modifiers elicit varied transcriptional outcomes that wield pathological cellular responses. Further, emerging leitmotifs like mitochondrial and nucleolar PARPs and the novel aspects of gene expression regulation by PARP-1 and poly(ADP-ribosyl)ation can provide a holistic view of PARP-1's influence on cell vitality. In this review, we discuss the pathological underpinnings of PARP-1, with a special emphasis on mitochondrial dysfunction and cell death subroutines, in the realm of neurodegeneration. This would provide a deeper insight into the functions of PARP-1 in neurodegenerative conditions that would enable the development of more effective therapeutic strategies.

pozdrawiam

In veterinary medicine, masitinib (animal medicine trade name Masivet® in Europe or Kinavet® in USA) was registered as a canine cancer therapy by the ...

I'm thinking about to use Masivet or Kinavet(Masitinib), it's a vet drug, although the same substance. To long to wait. For the record these news have been reposting for years, although it's hard to say about efficacy even from news.

https://alsnewstoday.com/2017/01/05/masitinib-protects-muscles-and-nerves-from-amyotrophic-lateral-sclerosis-damage/

Jest opcja weterynaryjna kinavet to to samo co masitinib ale nie w Polsce tylko w Niemczech i trzeba mieć znajomego weterynarza , ogólnie to jest to zrobienia
pozdrawiam

At week 30 SLA was stopped for 35% patients.
to oznacza  ze u 35% pacjentów  ALS zatrzymało sie po 30 tygodniu na stałe to jest powód do radości a nie do marudzenia bo ile można narzekać ? Weźcie się do kupy bo od narzekania  i marudzenia jeszcze nikt nie wyzdrowiał i nikomu to na dobre nie wyszło . Trochę pozytywnego myślenia, bo niedługo będzie metoda , która będzie cofała chorobę . Opracowywane są metody wydłużania neuronów za pomocą magnesów to się nazywa medycyna regeneracyjna

Witam

Poniżej pełny raport o Masitinibie
AB Science announces positive top-line results of final analysis from study AB10015 of masitinib in amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease

Primary analysis is a success and confirms interim analysis

Company to host webcast on masitinib in ALS

AB Science SA (NYSE Euronext - FR0010557264 - AB), a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), today announced that the phase 2/3 study AB10015 of masitinib in amyotrophic lateral sclerosis (ALS) has met its pre-specified primary endpoint. This is the first successful phase 3 trial of a tyrosine kinase inhibitor in the treatment of ALS, signifying masitinib as first-in-class for ALS, with a unique mechanism of action against microglia cells.

A webcast will be hosted on 20 March 2017 at 6 pm (CET). To participate please email at linda.carlet@ab-science.com.

Study AB10015 was a double-blind, placebo-controlled phase 2/3 study to compare the efficacy and safety of masitinib in combination with riluzole, versus placebo in combination with riluzole in the treatment of patients suffering from ALS.

In accordance with study protocol, the final analysis was performed based on 394 patients treated for 48-weeks and randomly allocated to three different treatment arms: masitinib at 4.5 mg/kg/day, versus masitinib at 3 mg/kg/day, versus placebo, each administered as an add-on to riluzole. The primary endpoint was based on the change from baseline to week 48 in the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R). The ALSFRS-R score is a validated rating instrument for monitoring the progression of disability in patients with ALS, which correlates significantly with quality-of-life and survival. This endpoint is recommended by EMA and FDA guidelines for registration in ALS. Also consistent with EMA guidance, Progression Free Survival (PFS) was included as a key secondary endpoint for registration, with progression being defined as ALSFRS-R deterioration of more than 9 points or death. A stepwise sequence of analysis was predefined to first test masitinib at 4.5 mg/kg/day versus placebo, and then masitinib at 3 mg/kg/day versus placebo.

For masitinib at 4.5 mg/kg/day:

Primary analysis on the change in ALSFRS-R score at week 48 (mLOCF methodology) is statistically significant with a P-value of 0.014.
Sensitivity tests on the primary analysis consisted in two models to impute a value at week 48 for any patients who discontinued treatment before week 48. Those sensitivity analyses are also significant with a P-value of 0.020.
The key secondary analysis on PFS was statistically significant with a P-value of 0.016.
Quality-of-life measured by change in ALSAQ score was also statistically significant with a P-value<0.01.
For masitinib at 3 mg/kg/day:

There was a trend in favor of masitinib versus placebo for change in ALSFRS score at week 48 (LOCF methodology) and likewise for the two imputation models (sensitivity analyses) and in PFS (secondary analysis).
The change in quality-of-life was statistically significant (p-value<0.01) in favor of masitinib.
The adverse events observed for masitinib in study AB10015 were consistent with its known safety profile. There were no new safety events at final analysis as compared with interim analysis.

The final analysis confirms the interim analysis, which was performed with 50% of patients.

AB Science filed an application for marketing authorization of masitinib in ALS at EMA in September 2016.

Full efficacy and safety data will be submitted for presentation at the European Network for the Cure of ALS (ENCALS) annual meeting in Ljubljana, Slovenia (18 - 20 May, 2017).

Alain Moussy, CEO of AB Science said: "This is a very good news for the patients. These final data confirm findings from the study's interim analysis and proves that masitinib is capable of slowing down motoneuron degenerative disease such as ALS, which is a devastating condition with an urgent unmet medical need."

Professor Olivier Hermine, President of AB Science scientific committee declared: "Perhaps the most impressive finding from this study is that masitinib has generated a significant difference in progression free survival with respect to the placebo treatment-arm. Similar to cancer studies when PFS is significantly improved, this indicates a clear clinical benefit in favor of masitinib."

Dr. Jesús Mora Pardina, international coordinator of study AB10015 and neurologist expert in ALS declared: "Masitinib is one of the rare drugs developed for the treatment of ALS that has proved its efficacy through validated endpoints. These results are truly encouraging and can be considered as a major breakthrough for ALS treatment, a condition that has proved extremely challenging in the development of new effective medication. Masitinib may be the long-awaited addition to the therapeutic armamentarium of ALS."

The mechanism of action of masitinib in ALS is based on the targeting of neurotoxic aberrant glial cells via CSF1R inhibition, providing a neuroprotective effect and slowing down neurodegeneration.

Amyotrophic lateral sclerosis is a rare degenerative disorder that results in progressive wasting and paralysis of voluntary muscles. There are approximately 50,000 people with ALS in the European Union and in the US, with more than 16,000 new cases diagnosed each year in Europe and in the US. Almost 80% of ALS patients die within 5 years and 90% die within 10 years.

Masitinib received orphan drug designation for ALS from both EMA and FDA.

About masitinib

Masitinib is a new orally administered tyrosine kinase inhibitor that targets mast cells and macrophages, important cells for immunity, through inhibiting a limited number of kinases. Based on its unique mechanism of action, masitinib can be developed in a large number of conditions in oncology, in inflammatory diseases, and in certain diseases of the central nervous system. In oncology due to its immunotherapy effect, masitinib can have an effect on survival, alone or in combination with chemotherapy. Through its activity on mast cells and microglia and consequently the inhibition of the activation of the inflammatory process, masitinib can have an effect on the symptoms associated with some inflammatory and central nervous system diseases and the degeneration of these diseases.

About AB Science

Founded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a class of targeted proteins whose action are key in signaling pathways within cells. Our programs target only diseases with high unmet medical needs, often lethal with short term survival or rare or refractory to previous line of treatment in cancers, inflammatory diseases, and central nervous system diseases, both in humans and animal health.

AB Science has developed a proprietary portfolio of molecules and the Company's lead compound, masitinib, has already been registered for veterinary medicine in Europe and in the USA. The company is currently pursuing thirteen phase 3 studies in human medicine in metastatic prostate cancer, metastatic pancreatic cancer, relapsing metastatic colorectal cancer, relapsing metastatic ovarian cancer, GIST, metastatic melanoma expressing JM mutation of c-Kit, relapsing multiple myeloma, relapsing T-cell lymphoma, mastocytosis, severe asthma, amyotrophic lateral sclerosis, Alzheimer's disease and progressive forms of multiple sclerosis. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB).

Further information is available on AB Science's website: AB SCIENCE - Pharmaceutical Company.

Forward-looking Statements - AB Science

This press release contains forward-looking statements. These statements are not historical facts. These statements include projections and estimates as well as the assumptions on which they are based, statements based on projects, objectives, intentions and expectations regarding financial results, events, operations, future services, product development and their potential or future performance.

These forward-looking statements can often be identified by the words "expect", "anticipate", "believe", "intend", "estimate" or "plan" as well as other similar terms. While AB Science believes these forward-looking statements are reasonable, investors are cautioned that these forward-looking statements are subject to numerous risks and uncertainties that are difficult to predict and generally beyond the control of AB Science and which may imply that results and actual events significantly differ from those expressed, induced or anticipated in the forward-looking information and statements. These risks and uncertainties include the uncertainties related to product development of the Company which may not be successful or to the marketing authorizations granted by competent authorities or, more generally, any factors that may affect marketing capacity of the products developed by AB Science, as well as those developed or identified in the public documents filed by AB Science with the Autorité des Marchés Financiers (AMF), including those listed in the Chapter 4 "Risk Factors" of AB Science reference document filed with the AMF on November 22, 2016, under the number R. 16-078. AB Science disclaims any obligation or undertaking to update the forward-looking information and statements, subject to the applicable regulations, in particular articles 223-1 et seq. of the AMF General Regulations.

pozdrawiam

Witam , poniżej artykuł

https://alsnewstoday.com/2017/02/27/study-shows-meditation-improves-qualtity-of-life-and-psychological-well-being-of-als-patients/

Pozdrawiam

Bardzo dziękuję za odpowiedź spróbuję odswieżyć temat na tych forach i dam znać pozdrawiam.

Witam
Pomaga na objawy opuszkowe poniżej link z trialem


http://link.springer.com/article/10.1007%2Fs13311-016-0508-5

Nuedexta
kapsułki twarde, 0,023g+9mg, 60 kaps.

Nazwa międzynarodowa:Quinine sulphatePodmiot odpowiedzialny:JENSON PHARMACEUTICAL SERVICESDostępność:Pełna odpłatność, na receptę

Pozdrawiam

Od paru dni zaczalem czytac o propofolu jest to srodek usypiajacy stosowany podczas operacji i malych zabiegow.
Pacjenci z SLA ktorzy mieli podawane podczas tych zebiegow ten srodek mieli poprawe i objawy choroby sie im cofnely na pewien czas, ale nigdy nie bylo zadnych trialow potwierdzajacych, jedynie opis pacjentow i ich wlasnych doswiadczen i badan.
Znalazlem badania naukowe o dzialaniu tego leku i jego wplywie na organizm i cos jest na rzeczy.
Dziwne jest to, ze nigdy nie slyszalem o tym leku a wy?

Ponizej zalaczam to co znalazlem:

Pozdrawiam

http://link.springer.com/content/pdf/10.1007%2Fs005400050040.pdf

http://pvangels.com/news/1525/affordable-safe-propofol-treatments-for-als-patients-in-puerto-vallarta

http://www.tandfonline.com/doi/pdf/10.3109/21678421.2013.826469

http://www.alsforums.com/forum/als-research-news/22130-propofol-reverses-symptoms.html

http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2438270/


Here is some BioChemical evidence of why this molecule/compound might be working on PALS.

Quote:
Note that the existence of defective inhibitory GABA-A alpha-1 receptor in ALS patients is not a hypothesis. It is an actual research finding. The inhibitory glycine alpha-1 receptors are also known to be defective in ALS patients. What really makes this interesting is that those receptors are used for inhibition by both the CNS and the immune system. In other words they are responsible for both hypnosis (sleep) and the suppression of inflammation. This is the reason that Propofol is such a powerful anti-inflammatory substance: it directly activates those specific inhibitory receptors and suppresses the elevated inflammatory response in the brain and spinal cord.

But Propofol appears to be doing much more than that. It seems to be able to restore the receptors to their normal inhibitory function and this reversal of symptoms can last a few weeks if the abnormal inflammation is completely suppressed in an ALS patient, the patient may see a complete remission of symptoms for a longer period, possibly years.

As an aside, why does Propofol reduce ALS-caused inflammation in the brain and spinal cord and not some of the other powerful anti-inflammatory drugs? The answer is that there are many types of inflammation. It all has to do with activating the correct receptors. This is why Propofol works and why the arthritis drugs do not.

I know this explanation might be over your head to understand it but...in terms of layman, this molecule may show some promise on treating the symptoms of MND/ALS.






The experimental and clinical pharmacology of propofol, an anesthetic agent with neuroprotective properties.
Kotani Y, Shimazawa M, Yoshimura S, Iwama T, Hara H.

Department of Biofunctional Evaluation, Molecular Pharmacology, Gifu Pharmaceutical University, Gifu 502-8585, Japan.

Abstract

Propofol (2,6-diisopropylphenol) is a versatile, short-acting, intravenous (i.v.) sedative-hypnotic agent initially marketed as an anesthetic, and now also widely used for the sedation of patients in the intensive care unit (ICU). At the room temperature propofol is an oil and is insoluble in water. It has a remarkable safety profile. Its most common side effects are dose-dependent hypotension and cardiorespiratory depression. Propofol is a global central nervous system (CNS) depressant. It activates gamma-aminobutyric acid (GABA A) receptors directly, inhibits the N-methyl-d-aspartate (NMDA) receptor and modulates calcium influx through slow calcium-ion channels. Furthermore, at doses that do not produce sedation, propofol has an anxiolytic effect. It has also immunomodulatory activity, and may, therefore, diminish the systemic inflammatory response believed to be responsible for organ dysfunction. Propofol has been reported to have neuroprotective effects. It reduces cerebral blood flow and intracranial pressure (ICP), is a potent antioxidant, and has anti-inflammatory properties. Laboratory investigations revealed that it might also protect brain from ischemic injury. Propofol formulations contain either disodium edetate (EDTA) or sodium metabisulfite, which have antibacterial and antifungal properties. EDTA is also a chelator of divalent ions such as calcium, magnesium, and zinc. Recently, EDTA has been reported to exert a neuroprotective effect itself by chelating surplus intracerebral zinc in an ischemia model. This article reviews the neuroprotective effects of propofol and its mechanism of action.

https://alsnewstoday.com/2017/02/01/nurown-cryopreservation-process-validated-als-phase-3-trial/

 Ponizej zalaczam wyniki trialu z leczenia komorkami macierzystymi pobranymi z szpiku kostnego i i dodano hormon wzrostu, zeby przeksztalcily sie w komorki nerwowe. Trial ten mial miejsce w Szpitalu Hadassah w Jerozolimie, Izraelu prawie 5 lat temu.   Ich wyniki sa obiecujace bo "13 (87%) were defined as responders to either ALS Functional Rating Scale-Revised or forced vital capacity, having at least 25% improvement at 6 months after treatment in the slope of progression."

https://www.ncbi.nlm.nih.gov/pubmed/26751635

to  nie da sie ukryć  ze efekty byly

Ja bym polecal sprobowac zalapac sie na trial bo ma byc kolejna faza.

Pozdrawiam

https://alsnewstoday.com/2017/01/27/improving-health-mitochondria-viable-als-treatment-option/

1 Czy czytales wyslane przeze mnie badania potwierdzajace dzialanie komorek macierzystych?
Bo nie widze nawiazania do tego tematu wiem, ze nie dalem tego na tacy bo jest po angielsku ale nie wydaje mi sie to problemem bo od czego jest google,
2. Jest duzo wiecej badan naukowych na ludziach ze komorki macierzyste dzialaja zanim Pan Profesor Dulak zaczal wciskac wam ciemnote   
3. Co ma wspolnego wiedza teoretyczna a praktyka i prawdziwe badania naukowe na ludziach i zwierzetach? Dla nas pacjentow? Jedyna wiedza jaka jest potrzebna to jak wyzdrowiec i w tym kierunku trzeba dzialac. Ale niestety mentalnosc tego forum w tym kierunku nie idzie.

witam,

1. Czy Prof. Jozef Dulak kiedykolwiek prowadzil badania komorkami macierzystymi na ludziach? Odpowiedz brzmi nie

2. To dlaczego uwazacie go za autorytet w tej dziedzinie?

3. Jedyna osoba, ktora robi w Polsce badania eksperymentalne na ludziach z uzyciem komorek macierzystych jest profesor Maksymowicz  i to jego doswiadczenie ma wartosc naukowa.

4. Ponizej zalaczam wyniki trialu z leczenia komorkami macierzystymi pobranymi z szpiku kostnego i i dodano hormon wzrostu, zeby przeksztalcily sie w komorki nerwowe. Trial ten mial miejsce w Szpitalu Hadassah w Jerozolimie, Izraelu prawie 5 lat temu.   Ich wyniki sa obiecujace bo "13 (87%) were defined as responders to either ALS Functional Rating Scale-Revised or forced vital capacity, having at least 25% improvement at 6 months after treatment in the slope of progression."

https://www.ncbi.nlm.nih.gov/pubmed/26751635

to  nie da sie ukryć  ze efekty byly

5. Skad wasza wrogosc do komorek macierzystych i ich demonizowanie, czy nie chodzi o to ze inni sprobowali ich a wy nie mogliscie lub nie chcieliscie sprobowac i to  nie daje wam spokoju ,? stanie w miejscu nic nie daje.

Zachecam do refleksji i do czytania badan, literatury zagranicznej bo to tam jest obszerna wiedza.

Pozdrawiam