Article link: http://www.forbes.com/sites/billfrist/2016/07/13/from-blindness-to-sight-one-mans-story-holds-promise-for-future-medical-innovations/#44bbcf82b3d6
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From Blindness To Sight: One Man's Story Holds Promise For Future Medical Innovations
Two weeks ago I led a panel discussion at the Bipartisan Policy Center (BPC) on medical innovation with my good friends Senator Lamar Alexander (R-TN), who chairs the Senate Health, Education, Labor and Pension (HELP) Committee, and former Congressman Bart Gordon (D-TN), who previously chaired the House Committee on Science and Technology. We were joined by Nashville native Doug Oliver, who had a powerful story to share.
Doug inherited a rare condition called dry macular degeneration. His vision began rapidly deteriorating at age 32, and within a year he was legally blind – unable to recognize faces, read the paper, continue his IT job, or drive a car. His doctor told him told him there was no cure, but encouraged him to monitor clinicaltrials.gov, the federal site we set up when I was in the Senate where NIH-approved clinical trials are listed, for any new experimental studies that specialized in his illness. Doug was diligent and in time found a physician in Florida who was treating his condition with patients’ own stem cells.
Doug’s decision to participate in this clinical trial was life-changing. His own stem cells were isolated in a centrifuge from bone marrow drawn from his hip bone, and then injected into his retinas. Doug saw improvement overnight, and within months—after a decade of blindness—he passed the Tennessee driver’s test and got his license back.
Doug’s story speaks to the potential cures we are just beginning to discover through regenerative medicine, and it highlights the importance of considering the individual patient experience in the drug approval process.
To bring a drug to market today in the U.S. can take a decade and cost up to $2 billion, a hurdle that many researchers are unable to climb. Promising treatments fail to advance because they lack the funding, or in the case of personalized, regenerative medicine, struggle to meet the threshold for sufficient participants in clinical trials under FDA guidelines. Doug was able to find a clinical trial and travel to receive the treatment, but many Americans in need aren’t so lucky.
Congressman Gordon and I have been leading an effort at the BPC to support and inform legislative solutions to accelerate medical innovations. Last summer we released a report making recommendations to Congress on modernizing the drug discovery, development, and approval process. Among those recommendations include a pathway to make treatments like Doug’s a reality for more Americans, and to allow patient experience—known as real world evidence—to better inform the drug development process.
[Real World Evidence for Safe and Effective Cures: Medical Innovation for All Americans
The webcast was hosted by the Bipartisan Policy Center (BPC) on June 23, 2016, scroll down the page to access the videos. ]
Our work has been in parallel with the efforts of Senator Alexander and the Senate HELP Committee. As Chairman, Sen. Alexander has ushered through 50 bipartisan strategies contained in 19 bills designed to accelerate the development and delivery of cures to patients, and improve United States’ global competitiveness.
Some of these strategies challenge the status quo. As a heart transplant surgeon, I remember when I first started transplanting hearts from a donor to a living patient. It was risky, it was innovative, and it definitely pushed the envelope. There was a point in time when people were skeptical that this type of organ transplant could work, just like some are skeptical today regarding adult stem-cell treatments like Doug’s.
But with a little risk carefully considered, we can reap big rewards. Instead of transplanting a heart—and worrying about donor shortages and organ compatibility—we could be repairing patient’s hearts with their own cells. Instead of replacing a knee through surgery, we could inject a patient with stem cells from his or her fat deposits to regenerate cartilage. The possibilities are endless.
Over the 40 years I have been in medicine, there has never been a more exciting time for healthcare innovation with the convergence of the House 21st Century Cures Act (which passed last year overwhelmingly) and the pending Senate legislation, the President’s Precision Medicine Initiative, and the Vice President’s Cancer Moonshot. With an estimated 7,000 – 10,000 known molecular-based diseases without cures, we have an opportunity to change that trajectory. I strongly commend Senator Alexander’s efforts and the legislative package his committee has advanced, and hope a legislative solution can advance to the President’s desk this year.
Please add your comments here, and in the comments at Forbes.
Share on Facebook https://www.facebook.com/PatientsForStemCells/posts/539233969596736
Share on twitter from @bfrist tweet, and thank him for his efforts! https://twitter.com/bfrist/status/753264599265312768
From Blindness To Sight: One Man's Story Holds Promise For Future Medical Innovations
Two weeks ago I led a panel discussion at the Bipartisan Policy Center (BPC) on medical innovation with my good friends Senator Lamar Alexander (R-TN), who chairs the Senate Health, Education, Labor and Pension (HELP) Committee, and former Congressman Bart Gordon (D-TN), who previously chaired the House Committee on Science and Technology. We were joined by Nashville native Doug Oliver, who had a powerful story to share.
Doug inherited a rare condition called dry macular degeneration. His vision began rapidly deteriorating at age 32, and within a year he was legally blind – unable to recognize faces, read the paper, continue his IT job, or drive a car. His doctor told him told him there was no cure, but encouraged him to monitor clinicaltrials.gov, the federal site we set up when I was in the Senate where NIH-approved clinical trials are listed, for any new experimental studies that specialized in his illness. Doug was diligent and in time found a physician in Florida who was treating his condition with patients’ own stem cells.
Doug’s decision to participate in this clinical trial was life-changing. His own stem cells were isolated in a centrifuge from bone marrow drawn from his hip bone, and then injected into his retinas. Doug saw improvement overnight, and within months—after a decade of blindness—he passed the Tennessee driver’s test and got his license back.
Doug’s story speaks to the potential cures we are just beginning to discover through regenerative medicine, and it highlights the importance of considering the individual patient experience in the drug approval process.
To bring a drug to market today in the U.S. can take a decade and cost up to $2 billion, a hurdle that many researchers are unable to climb. Promising treatments fail to advance because they lack the funding, or in the case of personalized, regenerative medicine, struggle to meet the threshold for sufficient participants in clinical trials under FDA guidelines. Doug was able to find a clinical trial and travel to receive the treatment, but many Americans in need aren’t so lucky.
Congressman Gordon and I have been leading an effort at the BPC to support and inform legislative solutions to accelerate medical innovations. Last summer we released a report making recommendations to Congress on modernizing the drug discovery, development, and approval process. Among those recommendations include a pathway to make treatments like Doug’s a reality for more Americans, and to allow patient experience—known as real world evidence—to better inform the drug development process.
[Real World Evidence for Safe and Effective Cures: Medical Innovation for All Americans
The webcast was hosted by the Bipartisan Policy Center (BPC) on June 23, 2016, scroll down the page to access the videos. ]
Our work has been in parallel with the efforts of Senator Alexander and the Senate HELP Committee. As Chairman, Sen. Alexander has ushered through 50 bipartisan strategies contained in 19 bills designed to accelerate the development and delivery of cures to patients, and improve United States’ global competitiveness.
Some of these strategies challenge the status quo. As a heart transplant surgeon, I remember when I first started transplanting hearts from a donor to a living patient. It was risky, it was innovative, and it definitely pushed the envelope. There was a point in time when people were skeptical that this type of organ transplant could work, just like some are skeptical today regarding adult stem-cell treatments like Doug’s.
But with a little risk carefully considered, we can reap big rewards. Instead of transplanting a heart—and worrying about donor shortages and organ compatibility—we could be repairing patient’s hearts with their own cells. Instead of replacing a knee through surgery, we could inject a patient with stem cells from his or her fat deposits to regenerate cartilage. The possibilities are endless.
Over the 40 years I have been in medicine, there has never been a more exciting time for healthcare innovation with the convergence of the House 21st Century Cures Act (which passed last year overwhelmingly) and the pending Senate legislation, the President’s Precision Medicine Initiative, and the Vice President’s Cancer Moonshot. With an estimated 7,000 – 10,000 known molecular-based diseases without cures, we have an opportunity to change that trajectory. I strongly commend Senator Alexander’s efforts and the legislative package his committee has advanced, and hope a legislative solution can advance to the President’s desk this year.