Posts Tagged ‘FDA’

International Alzheimer’s day: September 21, 2009

Wednesday, September 23rd, 2009

mbednar
Martin Bednar

Today is “International Alzheimer’s Day”. Given my interest in Alzheimer’s disease (AD) and my role at Pfizer to investigate the safety and efficacy of a potential therapy in AD patients, this seemed like an especially appropriate day to blog. AD is second only to cancer as the most feared disease in the United States. It is a particularly cruel disease, given the progression of the dementia over an 8-10 year period (dementia is the loss of intellectual and social abilities severe enough to interfere with daily functioning).

The greatest risk factor for developing AD is advancing age. The risk doubles every five years from about the age of 65 onward. Age, of course, is not a risk factor that we can do anything about. Nor is our genetic make-up: do we have one (or two) Apo E4 genes, do we have a first degree relative with AD, etc…On the other hand, there are other risk factors that are at least, in part, within our control: do we have a heart healthy lifestyle, do we exercise regularly, do we keep an eye on our weight, blood pressure and cholesterol? All of these factors can contribute to whether (and when) we are ever diagnosed with AD. By looking at these risk factors, it’s clear that, in general, what is good for the heart is good for the brain, and vice-versa. It is also becoming increasing clear that AD is a disease that can begin years, probably decades, before individuals (and those who regularly interact with them) notice that there are significant problems in the ability to think, reason, remember and follow through with a task. What this means is that the brain, like all other organs in the body (lungs, liver, kidneys, etc…) has some resiliency or reserve. Thus, for a while, there may be compensatory mechanisms that still allow individuals to function at their baseline level. However, at some point, the brain can no longer fully compensate and the difficulties in thinking and memory begin to become evident.

I believe that we are making great strides towards understanding what causes AD. Two proteins, called amyloid (Abeta) and tau are made in excessive amounts in the brains of individuals who have been diagnosed with AD. We now have experimental (not FDA approved yet) drugs that appear to have the capability of actually determining how much amyloid is in a person’s brain and thus to make a good inference as to whether they have AD. There are many different promising drugs that are targeting these two abnormal proteins, but none of them are approved for the treatment of AD at this time. I work on an antibody program that targets the amyloid protein. Antibodies can ‘capture’ certain proteins and thus render them incapable of performing their action (good or bad). Antibodies can be very specific for a certain protein (monoclonal antibody) or target several or many proteins (polyclonal). The one I am investigating is very specific for amyloid. We are currently studying this anti-amyloid antibody in patients who have AD to determine its safety and efficacy.

Of course, there are many other approaches besides targeting the abnormal amyloid or tau proteins and no one is sure about which approach is the best one or if we might even need more than one approach! It is an exciting time in Alzheimer’s research and I am delighted that Pfizer has committed significance resources to finding improved medicines to treat this dreaded disease.

THE ROLLER COASTER OF DRUG DISCOVERY.

Monday, September 14th, 2009

Maria Angelica Linton
Maria Angelica Linton

If the thrill of discovering new drugs was going to be compared to something, I would say it has to be: “the thrill of riding a roller coaster … but a roller coaster that lasts a lifetime and is missing pieces of track here and there”!

It is funny but it was only when I was recently asked to write a short biography about my life that I realized that I am not a spring chicken anymore and that I have been working at Pfizer for almost 17 years! In these 17 years for sure I can tell that I have secreted more adrenaline than I would have been induced in any real roller coaster!

Drug discovery is not a simplistic endeavor … It is one of the most challenging and expensive things to do and involves synchronized teamwork from people across many disciplines. You get to meet so many people with different personalities and you come to realize that you have to get along with everyone and you have to make things work.

People involved in drug discovery have a mission and a commitment to patients, to cure diseases and improve their lives.  Anybody that is involved and committed in developing therapeutics knows that we have to be innovative and ethical in order to provide longer, healthier and happier lives.

I love to read books about successful stories in the drug industry and how scientists came up with a great idea and made it work.  It is easy to write such stories once all the active work passes and it always seems that making the medicines that are approved, and for sale now, was a simple task.

During the time I have being involved in drug discovery I have learned that this roller coaster I am riding has mostly been in the lower part!  Yes, it sounds terrible but it is true, so many disappointments and lows!  So many days that we have thought: We got it, we got it! And we celebrate. However we soon realize that although a compound is potent and wonderful, there is a flaw with it and cannot be moved further … there are so many parameters to consider and everything has to be lined up just perfectly to make a drug worthy of pursuit.  These moments happen to all scientists on a regular basis and it feels like you went all the way up the roller coaster and all the sudden the track is gone! And you fall flat even faster than you went up! Ouch!

I’ll tell you something, part of being a scientist is never giving up, and we need tenacity and guts to recover from these falls.  I had days where I was restless at night and all the sudden some idea came like a storm to my mind … an idea that gave me hope … it is one of those things where I just had to go to my lab no matter what time at night it was … there I was at 3:00 am at the lab following my guts!

Then the day might come, the one day that changes all the bad days you have had in your life as a scientist and makes you forget the thousands of bad ones.  It is the day you go all the way to the top of the roller coaster but this time, there is a track and you keep on going and going. That is what I call the ride of a lifetime and, in drug discovery, it lasts at least 20 years!  You make the drug in the lab and see it after 20 years pass clinical trials and get accepted by the FDA and more than that, you see perhaps your own parents take it to improve their lives!  Unfortunately not everyone is lucky enough to see the end of it.  After 17 years at least I am lucky enough that one of the drugs I worked on at the lab a long time ago is now in Phase II clinical trials!  I am not sure yet if it will be approved by the FDA, but you bet I am praying that it does because it is desperately needed by patients suffering from Hepatitis C.

Well, that just describes how I perceive the frustrations and rewards of drug discovery and development, and the reason why I truly admire the people that have discovered and created the medicines we take today.

LA MONTAÑA RUSA DEL PROCESO DE DESCUBRIMIENTO DE NUEVAS MEDICINAS

Monday, September 14th, 2009

Maria Angelica Linton
Maria Angelica Linton

Si tuviera que comparar la emoción que se siente al participar en el proceso de descubrir nuevas medicinas con algo, tendría que decir que se parece mucho al montar una “montaña rusa”… solo que una que dura toda la vida y a la que le faltan varias piezas.

Es gracioso, pero fue hasta que recientemente estaba escribiendo un corto párrafo acerca de mi vida que me di cuenta de que ya no soy ninguna jovencita y que he trabajado para Pfizer por casi 17 años.  En este tiempo aseguro que he secretado mas adrenalina que la que hubiera sido inducida por cualquier montaña rusa verdadera.

El descubrimiento de nuevas medicinas no es un proceso simple, sino que al contrario, es sumamente complejo, costoso y desafiante e involucra un equipo completamente sincronizado de personas de las diferentes disciplinas.  Eso significa que uno se encuentra con muchísima gente de diferentes personalidades y de inmediato se descubre que es necesario aprender a llevarse con todas ellas y que de una forma u otra el equipo tiene que aprender a moverse al unísono para poder cumplir las metas.

Toda la gente que está involucrada en el proceso de descubrimiento de nuevas medicinas tiene una misión clara y un compromiso con los pacientes que es el de curar enfermedades y mejorar sus vidas.  Para triunfar es necesario ser innovador y ético a fin de proporcionar vidas más largas, saludables y felices.

A mi me fascina leer libros que contienen historias exitosas de como los científicos del pasado se inspiraron y crearon las medicinas que conocemos hoy.  Sin embargo, resulta fácil escribir estas historias muchos años después y parecería que el proceso de preparación de las medicinas que venden ahora fue un trabajo sencillo.

Durante el tiempo que he sido parte de este proceso, he aprendido que esta “montaña rusa” en la que estoy montada, tiene más que todo: partes bajas y caídas.  Ya sé, que suena terrible, pero es la realidad.  ¡Tantas desilusiones y bajones de moral!. Ha habido muchos días en el que pensamos: ¡Lo tenemos! ¡Lo tenemos! ¡Funciona! E incluso celebramos, para luego darnos cuenta de que a pesar de que tenemos un fármaco muy potente y maravilloso, tiene un defecto y ya no puede ser empujado más lejos…  Hay tantos parámetros que deben considerarse y al final todo tiene que alinearse perfectamente a fin de considerar si vale la pena el progresar un fármaco.  Estos momentos nos pasan a todos los científicos regularmente y se siente como si uno llega al tope de la montaña rusa y de repente se nota que ¡ya no hay pista! Y uno tiene una caída completamente vertical a mucha mayor velocidad que la que uno llevaba para llegar hasta arriba.

¿Saben qué? Parte de ser científico involucra el hecho de nunca darse por vencido, necesitamos tenacidad y agallas para constantemente recuperarnos de estas caídas y bajas de ánimos.   He tenido varias noches sin poder dormir bien pero de repente, se me ha ocurrido algo que de alguna forma me ha dado esperanza.  Es una de esas cosas por las que he ido a mi laboratorio sin importarme la hora que fuera… y ahí he estado a las 3:00 am en mi laboratorio siguiendo mi instinto.

Finalmente es posible que venga el día, y es el día que te hace olvidar todos los malos días que se han tenido en la vida de científico.  Es el día en que uno sube hasta el tope de la montaña rusa, pero esta vez, ¡si hay pista! Y uno ¡se continúa moviendo!  Eso es precisamente lo que yo llamo el viaje de tu vida y en el proceso de descubrimiento de medicinas dura por lo menos 20 años.  Este proceso comienza cuando se prepara una medicina en el laboratorio y después de casi 20 años se ve como pasa las pruebas clínicas y es aprobado por el FDA, y más que eso, uno ve que sus padres ¡la están tomando para mejorar la calidad de sus vidas!  Desafortunadamente, a pesar de que todos los científicos son una pieza importante en este proceso, son pocos los que tienen la fortuna de ver el final de la historia.  Después de 17 años, por lo menos he sido afortunada de ver que una de las medicinas preparadas en el laboratorio hace ya muchos años, está ahora en Fase II de pruebas clínicas.  Todavía no se si algún día será aprobada por el FDA, pero estoy rezando para que lo sea ya que es necesitada desesperadamente por pacientes que sufren de Hepatitis C.

Y para terminar, esto describe la manera de como yo percibo las frustraciones y recompensas del proceso de descubrimiento de nuevos fármacos y la razón de porque yo admiro tanto a la gente que ha descubierto y creado las medicinas que tomamos ahora.