articulo traduccido por Maria Galan en facebook Minociclina
Enviado por Paco el Abu
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articulo traduccido por Maria Galan en facebook Minociclina 20-July-2012 02:03 |
Maria Galan Sampedro Traducción:
Quiero expresar mi agradecimiento a todos aquellos padres que solicitaron la inscripción en el ensayo clínico con Minociclina. El programa del ensayo clínico se inició el 16 de abril de 2012. Ahora estamos en un punto donde la... mitad de los pacientes han recibido el tratamiento durante ocho semanas y están empezando a ser vistos de nuevo para sus citas de seguimiento. Al pasar el punto crítico del ensayo clínico, podemos hacer alguna reflexión.
Los padres y los pacientes han encontrado su camino a Tampa, Florida, y, con la ayuda de Joe Grieco, han pasado por la evaluación inicial sin ningún tipo de problemas significativos. Como era de esperar, hemos visto pocos efectos adversos de la Minociclina y estamos tan ansiosos como el resto de la comunidad por evaluar los resultados y hacer estos resultados públicos. Si el estudio continúa tal como está provisto, va a terminar hacia la mitad de marzo de 2013. Esto puede parecer una eternidad, pero en realidad es un tiempo increíblemente corto en comparación con otros ensayos clínicos para el síndrome de Angelman. Todo el equipo aquí en la Universidad de Florida del Sur trabajará para culminar, analizar, probar estadísticamente, e informar de los resultados lo más rápidamente posible después de la conclusión del estudio.
Muchos padres de familia, médicos y otros investigadores se han preguntado, "¿Cuál es el modo de acción de la Minociclina y ¿qué piensa usted que está haciendo en el cerebro?" Esta es una pregunta muy difícil de contestar por la sencilla razón de que su acción más allá de su uso como un antibiótico no está bien comprendida. De todos los antibióticos de tetraciclina, la Minociclina es el más soluble en lípidos. Esto significa que la Minociclina tiene la capacidad de penetrar profundamente en el cerebro. Sin embargo, puede tomar una cantidad considerable de tiempo para determinar su acción a nivel molecular. Es importante señalar aquí, que existe un precedente para el uso de drogas sin un conocimiento claro de su modo de acción exacto. Por ejemplo, el litio es un producto químico común que se ha utilizado para tratar la esquizofrenia y otros trastornos neuropsiquiátricos durante décadas, sin el conocimiento de cómo exactamente esta molécula simple funciona en el cerebro. Sólo recientemente ha comenzado la investigación para desentrañar los posibles mecanismos moleculares para el litio.
Esto trae a colación la cuestión de la vía rápida de investigación para identificar y llevar a la terapéutica los ensayos clínicos para el síndrome de Angelman. Esta estrategia se utilizó para identificar la Minociclina. En estos momentos estamos tratando de entender los mecanismos moleculares de la Minociclina y nuestros resultados se publicarán a su debido tiempo. Es importante saber que cualquier ensayo clínico, a pesar de si se trata de un fármaco aprobado por la FDA o de un compuesto experimental, debe someterse a un examen riguroso y exhaustivo por la FDA, un comité de revisión científica, la revisión departamental (un requisito institucional), y un junta de revisión institucional (IR
. El ensayo clínico de Minociclina se ha sometido a la revisión de todos los organismos reguladores antes mencionados, así como revisión por científicos médicos fuera de la Universidad del Sur de Florida. Aunque puede haber cierta controversia en decidir cuánta información debe ser conocida antes de realizar un ensayo clínico, pueden estar seguros de que el ensayo clínico con Minociclina ha sido objeto de un examen importante para garantizar el mayor nivel de seguridad para los niños participantes. El mecanismo de la droga y su uso como un agente terapéutico no son mutuamente excluyentes. Vamos a seguir la vía rápida la investigación de cualquier terapia potencial que dará lugar a un posible tratamiento para el Síndrome de Angelman. También quiero dar las gracias a todos por su apoyo y el apoyo de la FAST.
Quiero expresar mi agradecimiento a todos aquellos padres que solicitaron la inscripción en el ensayo clínico con Minociclina. El programa del ensayo clínico se inició el 16 de abril de 2012. Ahora estamos en un punto donde la... mitad de los pacientes han recibido el tratamiento durante ocho semanas y están empezando a ser vistos de nuevo para sus citas de seguimiento. Al pasar el punto crítico del ensayo clínico, podemos hacer alguna reflexión.
Los padres y los pacientes han encontrado su camino a Tampa, Florida, y, con la ayuda de Joe Grieco, han pasado por la evaluación inicial sin ningún tipo de problemas significativos. Como era de esperar, hemos visto pocos efectos adversos de la Minociclina y estamos tan ansiosos como el resto de la comunidad por evaluar los resultados y hacer estos resultados públicos. Si el estudio continúa tal como está provisto, va a terminar hacia la mitad de marzo de 2013. Esto puede parecer una eternidad, pero en realidad es un tiempo increíblemente corto en comparación con otros ensayos clínicos para el síndrome de Angelman. Todo el equipo aquí en la Universidad de Florida del Sur trabajará para culminar, analizar, probar estadísticamente, e informar de los resultados lo más rápidamente posible después de la conclusión del estudio.
Muchos padres de familia, médicos y otros investigadores se han preguntado, "¿Cuál es el modo de acción de la Minociclina y ¿qué piensa usted que está haciendo en el cerebro?" Esta es una pregunta muy difícil de contestar por la sencilla razón de que su acción más allá de su uso como un antibiótico no está bien comprendida. De todos los antibióticos de tetraciclina, la Minociclina es el más soluble en lípidos. Esto significa que la Minociclina tiene la capacidad de penetrar profundamente en el cerebro. Sin embargo, puede tomar una cantidad considerable de tiempo para determinar su acción a nivel molecular. Es importante señalar aquí, que existe un precedente para el uso de drogas sin un conocimiento claro de su modo de acción exacto. Por ejemplo, el litio es un producto químico común que se ha utilizado para tratar la esquizofrenia y otros trastornos neuropsiquiátricos durante décadas, sin el conocimiento de cómo exactamente esta molécula simple funciona en el cerebro. Sólo recientemente ha comenzado la investigación para desentrañar los posibles mecanismos moleculares para el litio.
Esto trae a colación la cuestión de la vía rápida de investigación para identificar y llevar a la terapéutica los ensayos clínicos para el síndrome de Angelman. Esta estrategia se utilizó para identificar la Minociclina. En estos momentos estamos tratando de entender los mecanismos moleculares de la Minociclina y nuestros resultados se publicarán a su debido tiempo. Es importante saber que cualquier ensayo clínico, a pesar de si se trata de un fármaco aprobado por la FDA o de un compuesto experimental, debe someterse a un examen riguroso y exhaustivo por la FDA, un comité de revisión científica, la revisión departamental (un requisito institucional), y un junta de revisión institucional (IR
. El ensayo clínico de Minociclina se ha sometido a la revisión de todos los organismos reguladores antes mencionados, así como revisión por científicos médicos fuera de la Universidad del Sur de Florida. Aunque puede haber cierta controversia en decidir cuánta información debe ser conocida antes de realizar un ensayo clínico, pueden estar seguros de que el ensayo clínico con Minociclina ha sido objeto de un examen importante para garantizar el mayor nivel de seguridad para los niños participantes. El mecanismo de la droga y su uso como un agente terapéutico no son mutuamente excluyentes. Vamos a seguir la vía rápida la investigación de cualquier terapia potencial que dará lugar a un posible tratamiento para el Síndrome de Angelman. También quiero dar las gracias a todos por su apoyo y el apoyo de la FAST.
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Re: articulo traduccido por Maria Galan en facebook Minociclina 20-July-2012 02:12 |
Home | MENSAJE ORIGINAL
About Angelman SyndromeAbout the FoundationResearchNews And EventsGet Involved← 70.3 miles of Mountain MadnessFinding Wings to Fly →FAST-initiated Clinical Trial
Posted on July 19, 2012 by Edwin Weeber
Prof Weeber with the Schwarzrock family
I want to thank all those parents who applied for enrollment in the minocycline clinical trial. The clinical trial began on schedule April 16, 2012. We are now at a point where half of the patients have received the drug for eight weeks and are beginning to be seen for their follow up appointments. As we pass this critical point of the clinical trial, an update
and some reflection is in order. Parents and patients have found their way to Tampa, Florida, and, with the help of Joe Grieco, have gone through the initial evaluation without any significant problems. As expected, we have seen little adverse effects of the minocycline and are as anxious as the rest of the community to evaluate the results and make these results public. If the study proceeds as proposed, it will end in the middle of March 2013. This may seem like an eternity, but it is actually an incredibly truncated timeline in comparison to other clinical trials for Angelman syndrome. The entire team here at the University of South Florida will work to culminate, analyze, statistically test, and report our findings as quickly as possible after the conclusion of the study.
Trial coordinator Joe Greico with the Schwarzrock family
Many parents, clinicians, and other investigators have asked, “What are the actions of minocycline and what do you think it is doing in the brain?” This is an exceedingly difficult question to answer for the very reason that its action beyond its use as an antibiotic is not well characterized. Of all the tetracycline antibiotics, minocycline is the most lipid soluble. This means minocycline has the ability to deeply penetrate the brain. However, its actions at a molecular level may take a considerable amount of time to determine. It is important here to note that there is a precedent for the use of drugs without a clear knowledge of their exact mode of action. For example, lithium is a common chemical that has been used to treat schizophrenia and other neuropsychiatric disorders for decades without knowledge of exactly how this simple molecule works in the brain. Only recently has research begun to unravel the possible molecular mechanisms for lithium.
Assoc Prof Edwin Weeber, PhD
This brings up the question of fast-tracking research to identify and bring to clinical trial therapeutics for Angelman Syndrome. This strategy was used to identify minocycline. We are currently trying to understand the molecular mechanisms of minocycline and our results will be published in time. It is important to know that any clinical trial, despite whether it involves an FDA-approved drug or an experimental compound, must undergo a rigorous and comprehensive review by the FDA, a scientific review committee, departmental review (an institutional requirement), and an institutional review board (IR. The minocycline clinical trial has undergone review from all of the above-mentioned regulatory bodies as well as peer review by physician scientists outside of the University of South Florida. While there may be some controversy involved in deciding how much information should be known prior to conducting a clinical trial, you can rest assured the minocycline clinical trial has undergone significant review to ensure the highest safety level for the children involved. The mechanism of the drug and its use as a therapeutic are not mutually exclusive. We will continue to fast-track research for any potential therapeutic that will lead to possible treatment for Angelman Syndrome. I also want to thank everyone for their support and the support of the Foundation for Angelman Syndrome Therapeutics.
This entry was posted in Research and tagged Angelman Syndrome, cureangelman, Edwin Weeber, FAST, Minocycline, Research. Bookmark the permalink.
← 70.3 miles of Mountain MadnessFinding Wings to Fly →5 Responses to FAST-initiated Clinical Trial
Donna Young says:
July 19, 2012 at 11:03 am
Thank you Dr Weeber for the update. My granddaughter Emma is eagerly awaiting her first trip to Tampa and our family cannot put into words how thankful we are for FAST, for you, Joe, Stephanie and the entire team for all you are doing towards improving the lives of each of our angels. It is rewarding to see funding being put to use sooner than later towards Angelman Syndrome clinical trials. That is WHY our family supports FAST first and foremost. God bless you and your entire team!
Reply
Sharon McMillan says:
July 19, 2012 at 10:12 pm
My son David will be starting the trial in November and we are so thankful and excited to begin! I want to say a special thank you to Dr. Weeber, Joe, Stephanie, FAST and everyone involved for giving us hope. Thank you for all your hard work and dedication, you are all my hero’s!
Reply
Joan Leary says:
July 19, 2012 at 11:42 pm
Dear Dr. Weeber, although my granddaughter Jenna Suller was not chosen for the clinical trials, I am excited for all of our little Angels that are putting themselves out there for the hopes of a treatment for all of our Angels. Yes, March feels a long time from now but God has given me patience to wait and pray for wonderful results!! Good luck dear Emma and all of our pioneer Angels for making this all happen. AND, thank you Dr. Weeber, et.al. for the work and dedication you are doing to hopefully someday say, “Now, what was AS?!”
Reply
Rita L. Daywalt says:
July 20, 2012 at 12:49 am
Thank you Dr. Weber, the trial team and FAST for your extraordinary efforts!
While Tyler did not get picked in this first round of participants, I am anxious and hopefull for us as a community at the same level that I would be if we were participating. I am so proud to be part of the AS community and specifically part of the FAST community. We’ve experienced so much support from not just other parents, but from the scientific community as well.
I truly believe the relationsip between us all is exceptional and I am thankful every day for all of the people who have taken a stand and dedicated their lives to ensuring that AS becomes a thing of the past and not something we’re dealing with 50 years from now.
Reply
giovanna portanova salvi says:
July 20, 2012 at 6:58 am
..la speranza che un giorno i nostri “eterni bimbi” possano abbracciare i loro angeli (tutti voi ,meravigliosi ricercatori!!) e poter dire ,con la loro voce :GRAZIE!!! per ogni bimbo ,a cui si restituisce il diritto alla qualità della vita ..si accende una stella in cielo …facciamo in modo che il firmamento splenda .GRAZIE per tutto ciò che fate per i nostri ANGELMAN la mamma di cristiana (angelman di 26 anni per disomia uniparentale )
Reply
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About Angelman SyndromeAbout the FoundationResearchNews And EventsGet Involved← 70.3 miles of Mountain MadnessFinding Wings to Fly →FAST-initiated Clinical Trial
Posted on July 19, 2012 by Edwin Weeber
Prof Weeber with the Schwarzrock family
I want to thank all those parents who applied for enrollment in the minocycline clinical trial. The clinical trial began on schedule April 16, 2012. We are now at a point where half of the patients have received the drug for eight weeks and are beginning to be seen for their follow up appointments. As we pass this critical point of the clinical trial, an update
and some reflection is in order. Parents and patients have found their way to Tampa, Florida, and, with the help of Joe Grieco, have gone through the initial evaluation without any significant problems. As expected, we have seen little adverse effects of the minocycline and are as anxious as the rest of the community to evaluate the results and make these results public. If the study proceeds as proposed, it will end in the middle of March 2013. This may seem like an eternity, but it is actually an incredibly truncated timeline in comparison to other clinical trials for Angelman syndrome. The entire team here at the University of South Florida will work to culminate, analyze, statistically test, and report our findings as quickly as possible after the conclusion of the study.
Trial coordinator Joe Greico with the Schwarzrock family
Many parents, clinicians, and other investigators have asked, “What are the actions of minocycline and what do you think it is doing in the brain?” This is an exceedingly difficult question to answer for the very reason that its action beyond its use as an antibiotic is not well characterized. Of all the tetracycline antibiotics, minocycline is the most lipid soluble. This means minocycline has the ability to deeply penetrate the brain. However, its actions at a molecular level may take a considerable amount of time to determine. It is important here to note that there is a precedent for the use of drugs without a clear knowledge of their exact mode of action. For example, lithium is a common chemical that has been used to treat schizophrenia and other neuropsychiatric disorders for decades without knowledge of exactly how this simple molecule works in the brain. Only recently has research begun to unravel the possible molecular mechanisms for lithium.
Assoc Prof Edwin Weeber, PhD
This brings up the question of fast-tracking research to identify and bring to clinical trial therapeutics for Angelman Syndrome. This strategy was used to identify minocycline. We are currently trying to understand the molecular mechanisms of minocycline and our results will be published in time. It is important to know that any clinical trial, despite whether it involves an FDA-approved drug or an experimental compound, must undergo a rigorous and comprehensive review by the FDA, a scientific review committee, departmental review (an institutional requirement), and an institutional review board (IR
. The minocycline clinical trial has undergone review from all of the above-mentioned regulatory bodies as well as peer review by physician scientists outside of the University of South Florida. While there may be some controversy involved in deciding how much information should be known prior to conducting a clinical trial, you can rest assured the minocycline clinical trial has undergone significant review to ensure the highest safety level for the children involved. The mechanism of the drug and its use as a therapeutic are not mutually exclusive. We will continue to fast-track research for any potential therapeutic that will lead to possible treatment for Angelman Syndrome. I also want to thank everyone for their support and the support of the Foundation for Angelman Syndrome Therapeutics.
This entry was posted in Research and tagged Angelman Syndrome, cureangelman, Edwin Weeber, FAST, Minocycline, Research. Bookmark the permalink.
← 70.3 miles of Mountain MadnessFinding Wings to Fly →5 Responses to FAST-initiated Clinical Trial
Donna Young says:
July 19, 2012 at 11:03 am
Thank you Dr Weeber for the update. My granddaughter Emma is eagerly awaiting her first trip to Tampa and our family cannot put into words how thankful we are for FAST, for you, Joe, Stephanie and the entire team for all you are doing towards improving the lives of each of our angels. It is rewarding to see funding being put to use sooner than later towards Angelman Syndrome clinical trials. That is WHY our family supports FAST first and foremost. God bless you and your entire team!
Reply
Sharon McMillan says:
July 19, 2012 at 10:12 pm
My son David will be starting the trial in November and we are so thankful and excited to begin! I want to say a special thank you to Dr. Weeber, Joe, Stephanie, FAST and everyone involved for giving us hope. Thank you for all your hard work and dedication, you are all my hero’s!
Reply
Joan Leary says:
July 19, 2012 at 11:42 pm
Dear Dr. Weeber, although my granddaughter Jenna Suller was not chosen for the clinical trials, I am excited for all of our little Angels that are putting themselves out there for the hopes of a treatment for all of our Angels. Yes, March feels a long time from now but God has given me patience to wait and pray for wonderful results!! Good luck dear Emma and all of our pioneer Angels for making this all happen. AND, thank you Dr. Weeber, et.al. for the work and dedication you are doing to hopefully someday say, “Now, what was AS?!”
Reply
Rita L. Daywalt says:
July 20, 2012 at 12:49 am
Thank you Dr. Weber, the trial team and FAST for your extraordinary efforts!
While Tyler did not get picked in this first round of participants, I am anxious and hopefull for us as a community at the same level that I would be if we were participating. I am so proud to be part of the AS community and specifically part of the FAST community. We’ve experienced so much support from not just other parents, but from the scientific community as well.
I truly believe the relationsip between us all is exceptional and I am thankful every day for all of the people who have taken a stand and dedicated their lives to ensuring that AS becomes a thing of the past and not something we’re dealing with 50 years from now.
Reply
giovanna portanova salvi says:
July 20, 2012 at 6:58 am
..la speranza che un giorno i nostri “eterni bimbi” possano abbracciare i loro angeli (tutti voi ,meravigliosi ricercatori!!) e poter dire ,con la loro voce :GRAZIE!!! per ogni bimbo ,a cui si restituisce il diritto alla qualità della vita ..si accende una stella in cielo …facciamo in modo che il firmamento splenda .GRAZIE per tutto ciò che fate per i nostri ANGELMAN la mamma di cristiana (angelman di 26 anni per disomia uniparentale )
Reply
Leave a Reply Cancel reply
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Name *
Email *
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Comment
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Search for:
Subscribe to Blog
Your email:
Recent Posts
Finding Wings to Fly
FAST-initiated Clinical Trial
70.3 miles of Mountain Madness
Angel Runners LA Marathon
FAST Receives $250,000 Anonymous Donation – Ambitious Research Project Underway
Archives
July 2012
June 2012
Copyright © 2008 Foundation for Angelman Syndrome Therapeutics • Privacy Policy | Terms of UseHome | Join | Donate | Contact Us
About Angelman Syndrome | About the Foundation | Research | News And Events | Get Involved
0
inShare
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