|Joined: ||Sun Jul 11th, 2004|
|Location: || |
|| Posted: Wed Oct 4th, 2006 17:00||
|It seemed almost everything we heard about the future of research and medicine at the National Organization for Rare Disorders conference in Bethesda described what has already been accomplished with the Marshall Protocol.
First, though, let me share some of the eye-opening flaws in the current system, as presented at the conference.
Current Status of Drug Development
Representatives from the FDA carefully explained the high cost and high failure rate of new drug development. FDA spokespeople readily admitted that
- the number of applications for new drugs has continued to dwindle alarmingly over the past decade,
- the failure rate of new drugs is high and
- some drugs are recalled after they are on the market, due to product safety issues.
- All in all, little hope was offered for anyone who may be thinking/hoping/betting on a pharmaceutically developed breakthrough new drug that may be waiting to be approved by the FDA. It’s not likely.
Other Flaws Described
- slow moving macro “evidence” research fails the majority
- lumbering lack of ability to revise studies along the way
- focus is on new drug development (still a problem even with futuristic speech focus)
There was no discussion at the conference for any solution to a real problem that will require an “Act of Congress” to change: Molecular evidence is not *required* as primary evidence, let alone required for consideration in submissions for Orphan Drugs or any Drugs, for that matter.
Are Medical Breakthroughs Real Progress? What Does it Mean?
In discussing medical breakthroughs, speakers explained that new research from mapping the human genome and identification of genes related to disease has had little application to patient treatment. And there is little hope this will change quickly. The reason is that generally there is no idea what this genetic information means or how to apply it to developing new treatments. Everyone is focused on their niche of research and knowledge, but there is a huge void of people who might know how to put this all together and interpret it.
Translational research is the new catch-phrase, identifying the dire need to quickly convert new research knowledge to more and better treatment options for patients. The non-for-profit Critical Path Initiative was presented as a valuable new tool or process through which safer new treatments can be developed and quickly brought to market.
The Future of Medicine
Dr. Von Eschenbach, acting director of the FDA, explained that the future of medicine will bring revolutionary changes based on the use of new technologies, collaborative and adaptive research, plus knowledge of pharmacokinetics and small molecules and integration of expanding knowledge of the human genome to arrive at new treatments. He explained we can no longer have the luxury of time to expect to conquer one disease at a time. Instead, we must focus on understanding the underlying disease process at work. He said only then can we identify common disease mechanisms so we can successfully identify effective new treatments applicable to several diseases within a disease category. This is the future of medicine.
Fertile Ground for the MP
In reality, Dr. Von Eschenbach described how the Marshall Protocol was developed. We are already doing what he described as the future. We are already in sync with the agency’s plans. We need to do is make sure these plans move forward while letting the right people know where we are and what we have done.
The meeting was fertile ground for describing the MP, as much of what was described by key regulatory speakers showed the flaws of status quo in unrelenting truth, and predicted what we of the MP are living now (molecular genomics changing medicine at it’s core). Some presenters went so far as to say that in the future, all diseases will be considered “rare,” as all diseases would be handled on the individual level.
You can readily see how unusual it is to have a researcher with the breadth and depth of knowledge of Dr. Marshall and the ability to "put it all together." The caliber and breadth of Marshall's research, understanding and foresight is so unique, and the conference brought this into sharp focus. Dr. Marshall is so "in tune" with the future of medicine, what he has been telling us was what we heard at this conference.
Our attendance at this conference allowed us to continue making the connections we need, beginning to build the bridges necessary for widespread education, acceptance and adoption of Dr. Marshall’s discoveries. We would like to express deep appreciation for those in Bethesda who worked to make a significant difference for the MP.
Thanks to all of you for supporting us. Thank you to Janet Foutin and Susan Andorn for attending this conference. As I heard Janet and Susan explaining to attendees, we represented recovering patients who have new hope because Dr. Marshall’s successful quest to identify the disease mechanism led to and an effective treatment. We all believe it is an honor to represent you and share this success story.
For More Details of the Conference
All the conference powerpoint presentations will eventually be on the NORD website. The conference outline at-a-glance shows the take-away intent for NORD attendees.
We will have photos online on our MP website soon.
From Belinda, with assistance from Janet
|Joined: ||Tue Aug 9th, 2005|
|Location: ||Oregon USA|
|| Posted: Thu Oct 5th, 2006 06:34||
HAFFNER/BELINDA : BELINDA/CPATH'S WOOSLEY : VONESCHENBACH/BELINDA
JANET/JOANN : SUSAN/BELINDA/JANET
BELINDA/ABBEY MEYERS NORD : CAMPBELL/DR BRADY/BELINDA : BELINDA/JRF
SLIDE SHOWING GENOMICS DISCUSSION
THEIR STORY WAS OUR STORY
The 2006 NORD conference was a unique time for describing the MP to others, as much of what was presented by key regulatory speakers showed the flaws of status quo, and predicted what we of the MP are living now (molecular genomics changing medicine at its core).
Acting FDA commissioner Von Eschenbach described the history of medicine in 3 stages: 1. macroscopic (using 5 senses to discern) 2. microscopic 3. 21st Century Genomic. He also said that on "the molecular level, no disease is rare,” and in the future all diseases would be handled individually. John Gallin, MD of NIH echoed that statement with "Everyone will have a rare disease eventually."
Belinda caught Von E.'s attention when she boldly asked if he realized just how true his words had been. He responded positively that he knew exactly, and gave her time for a brief discussion and photograph while he hurried off to Congress ... much to the dismay of the CNN press affiliate who wanted the brief time talk to him. Von E. had used the metaphor of caterpillar and butterfly, that it was the caterpillar's destiny to become a butterfly, but we have a choice. And there was Belinda, quickly capturing the opportunity and far more brilliant than a butterfly. Her transformation on the MP was definitely by choice.
Admiral Haffner of the FDA OOPD gave a brief history of her dept and some idea of rare-disease, small market successes she has been involved with. She suggested talking to OOPD early in the process for grants program and recent efforts for a new initiative with parallel development and joint applications in EU and US (EMEA & FDA). Belinda and I noticed of the 13 staff members pictured in OOPD slide, 9 were in Corps uniform.
NORD staff had the opportunity on several occasions to see and hear us take part in the discussions. Belinda and I both asked pointed questions to presenters via microphone so all in attendance heard our statements.
BLINDING & SPEAKING
Belinda asked just how can studies be blinded in the future with the Internet? This was reiterated with several others in the meeting and one woman said she felt patients should communicate with each other when they were involved in a study.
Rino Aldrighetti, President of Pulmonary Hypertension Association, is using the Internet for collaboration. He said "sharing information is good... but clinical trials may be the exception to the rule." Their 1999 list serv for patients on a new drug trial resulted in patients talking about the trial, "almost lost the validity of the trial as details moved toward becoming public." (Interesting perspective of what happened). He also said "In an improving environment, flexibility in the creation of trials is critical to continuing advancement (eg. non-placebo trials)." Technology is clearly changing study design, and old regulations are challenged now.
I took the final speaker, but we wrapped up with pictures and after-presentation discussion of the dilemma that happens when a small group controls Sarc publications. There was agreement several impasses had to be moved in both regulatory bodies and industry settings.
LATE STAGE LYME
Susan A. drove up from her home and spoke with many about her recovery from Chronic Lyme, specifically OSHI JoAnn Minor's friend that had heard about this from Lori before the conference. Susan is on the sampler Conference CD Belinda passed out to many as well so they had a chance to see her and hear her.
Karolinska poster handouts, Dr Marshall's/CDER presentation, newly produced conference samplers, MP/Th1/D, the annotated bibliography of secosteroid D, MP Phase 1 laminated sheets, business cards and Ms Dale’s recovery story with photographs were distributed over the span of the conference, starting with Thursday evening in the exhibitor’s hall. These seemed the most effective to describe Dr Marshall’s breakthrough and were tailored to the individual audience (NORD members, other rare disease representatives, doctors, regulatory officers, those seeking a diagnosis for disease that was stumping their doctor, and those with rare diseases).
Belinda showed many her progression through disease to wellness with photographs she had compiled of her own before and after (all of us can do this). Wrotek’s screen stills from the CDER helped establish the FDA/CDER Visiting Professor presentation for enlightenment on these key charts.
Some hope for impacting off-label legislation with NORD is expected and I will attend the phone conference for that on Oct 10. Most presenters were focused on the angle of developing new drugs, so resolving this niche of a niche problem with off-label is important. Off label uses with existing drugs (like those of the MP) do not get attention or funding when new drug development demands the purse. This step right out of the gate from the conference has some promise for the MP because of contacts and efforts made to be at the NORD meeting.
Contacts with exchanged business card action planned, as are thank you notes and move forward communications.
I would like to express deep appreciation for those who worked to make a significant difference for the MP while we were there. Their help was invaluable.
Sarcoidosis 125D61, MP10/05 ModP2 12/05 Ph2 6/06 Ph3 10/06, NoIRs limited outings covered, 2/08 25D6.2, 10/08 25D6.9
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