NIH Awards $8.6 Million for Bold Bid to Transform Lung Transplantation

Lung transplantation can be indicated for many chronic lung diseases

Lung transplantation is a procedure for end-stage lung disease to replace a diseased organ with a healthier one from a deceased organ donor. It is an option used to improve the health and prolong survival of patients. Lung transplant can benefit patients with diseases such as cystic fibrosis, COPD (chronic obstructive pulmonary disease), idiopathic pulmonary fibrosis (IPF), pulmonary arterial hypertension, autoimmune-related advanced lung disease, sarcoidosis, and silicosis. Patients must be referred by their local pulmonologist and their records reviewed. Once accepted for potential transplant, patients are extensively evaluated in a clinic visit. If they qualify, they are listed for transplant and seen in clinic every three months. Transplant surgery generally lasts 4 to 12 hours, followed by 8 to 24 hours of stabilization in an intensive care unit (ICU) in the hospital. Recovery will be in the Heart and Lung Transplant ICU (HTICU). When well enough, patients are discharged to local housing for several weeks for outpatient follow-up care. 

There are approximately 65 active programs in the United States

This procedure may be used to treat or diagnose several different conditions. We have listed some of these conditions below for your convenience. Please note that this is not a comprehensive list and may vary depending on your specific diagnosis.

• Chronic Obstructive Pulmonary Disease
• Cystic Fibrosis

• Emphysema
• Pulmonary Arterial Hypertension

• Pulmonary Fibrosis

Research Could End Lung Shortages, Save Lives of Recipients

• Increasing the number of lungs available for transplant. Using a technique known as “ex vivo lung perfusion” (EVLP) combined with a promising drug would open the door for surgeons to assess and use lungs from donors who die outside the hospital. Most lungs from these donors go unused because surgeons cannot determine how much damage the lungs have suffered in the time since death. UVA’s research with EVLP could allow surgeons to rehabilitate injured donor lungs and dramatically reduce the large waiting list for lung transplants. “We envision UVA as eventually becoming a lung rehab center where donor lungs would arrive to undergo EVLP and, if they look great, they can be sent out to the appropriate hospital for transplant,” said Laubach, who is collaborating on the project with Kron, a UVA transplant surgeon

NIH Awards $8.6 Million for Bold Bid to Transform Lung Transplantation

Precision Medicine A New Dawn: First CAR T Cell Recommended for FDA Approval

One small step for a new drug,  one giant leap for precision medicine !

A new dawn is breaking in the field of hematologic malignancies, as the first product based on chimeric antigen receptor (CAR) T cells was scrutinized today by a panel of experts and unanimously recommended for approval.

The product is tisagenlecleucel-T (previously known as CTL019; developed by Novartis), and the "yes" vote was in answer to this question: "Considering the efficacy and safety results of Study B2202, is the benefit-risk profile of tisagenlecleucel favorable for treatment of pediatric and young adult patients (age 3-25 years) with relapsed (second or later relapse) or refractory (failed to achieve remission to initial induction or reinduction chemotherapy) B cell precursor acute lymphoblastic leukemia (ALL)? "

Similar Products Not Far Behind 

This product is made individually for each patient.

Blood is collected from the patient, and then autologous T cells are separated out and genetically engineered. The process involves inserting a CAR that targets CD19, an antigen expressed on B cells and tumors derived from B cells.  These CAR T cells are then infused back into the patient, who has undergone chemotherapy, and in the body the product homes in on B-cell leukemic cells and destroys them. 

Several similar products are in late stages of development. Coming up soon for review by the FDA is Kite Pharma's axicabtagene ciloleucel (KTE-C19) for lymphoma, while further back are CAR T-cell products from Juno.

There is much rivalry between pharma companies dedicating enormous resources in the field of precision medicine. So much so that,  "I am amused by the horse-race metaphors that are used to frame the various companies developing CAR-T therapies," Dr Belldegrun wrote in a blog post. "Today is not about competition," he continued. "Today is about advancing an exciting technology that has the potential to transform cancer."

A New Dawn: First CAR T Cell Recommended for FDA Approval

The Conundrum of delaying retirement to Age 70.

Alzheimer's disease . (AD) is the talk of the town. Anyone who has a mild cognitive impairment (MCI) panics at the prospect of having early Alzheimer's disease.  How do you discriminate the two?

The effects of Alzheimer's disease can begin as early as 50 years of age.  While we picture many patients in nursing homes, there are many still in the working and productive years of life.   There are many examples.

The symptoms and signs of Alzheimer's Disease.

The ten early signs of Alzheimer's . (we all have some of these symptoms

1. Malcolm Young (1953 – )

Malcolm Young, legendary guitarist and co-founder of rock band AC/DC, has been playing guitar since 1969. He’s considered by some to be one of the best rhythm guitarists in the world, producing hits like “You Shook Me All Night Long.” Unfortunately, Young recently announced his retirement from music, due to a dementia diagnosis. An article states that his departure from the band follows his “complete loss of short term memory,” and that he has trouble remembering people moments after meeting them.

2. Glenn Campbell (1936 – )

Country singer and guitarist, Glen Campbell, announced in 2011 that he had been diagnosed with Alzheimer’s. He’s completed his “Goodbye Tour,” which concluded  in Napa, California. Campbell has over 50 years in the music business and on television, and landed a historical win of four Grammy’s in 1967.

3. Pat Summitt (1952 – )

Pat Summitt coached the Tennessee Lady Vols basketball team to an amazing 8 NCAA championships and retired with a record of 1,098-208. Summit recently announced that she had been diagnosed with early-onset Alzheimer’s. She retains the title of Head Coach Emeritus, and is currently involved as an advocate for people with the disease.

4. Perry Como (1912 – 2001)

Perry Como was a popular singer and television personality during the 1950s and 1960s and continued to perform periodically during his later years, especially around Christmas. Como suffered from Alzheimer’s for two years before passing away in 2001. Como spent more than 50 years in the entertainment business and was widely respected, personally and professionally.

5. Charles Bronson (1921 – 2003)

 Charles Bronson, star of “Death Wish” and numerous other action films, spent the last years of his life debilitated from Alzheimer’s. An article about Bronson’s late years, that discusses his struggle with the disease, can be found here.

6. Ronald Reagan (1911 – 2004)

Six years after the end of his presidency, Ronald Reagan announced to the American public that he was “one of the millions of Americans who will be afflicted with Alzheimer’s disease.” He said that his public disclosure was intended to raise public awareness about the disease.

7. Charlton Heston (1923 – 2008)

Charlton Heston’s most famous role was as Moses in “The Ten Commandments.” Like his personal friend, Ronald Reagan, Heston also announced publicly that he had Alzheimer’s. Heston left a long legacy of entertainment contributions and political activism.

8. Norman Rockwell (1894 – 1978)

Rockwell, one of the most famous American painters, became well known for his illustrations on the cover of the Saturday Evening Post. Rockwell succumbed to Alzheimer’s after a long and illustrious career. Interestingly, Rockwell’s paintings are now used sometimes in dementia therapy because of the memories and nostalgia they elicit.

9. Rita Hayworth (1918 – 1987)

Hayworth was an American film star who rose to prominence in the 1940s. Despite fairly obvious signs of the illness, Rita Hayworth was not correctly diagnosed for several years. After eventually learning the cause of her memory loss, Hayworth became the “face of Alzheimer’s disease” during the 1980s. This year, the Alzheimer’s Association held its 32nd Annual Rita Hayworth Gala.

10. Sugar Ray Robinson (1921 – 1989)

Sugar Ray Robinson, recognized as one of the best boxers ever, died from Alzheimer’s at just 67 years of age. Robinson held the welterweight and middleweight title belts, and finished with a final record of 173 wins, 19 losses, and 2 draws. It’s not currently known whether his head injuries contributed to Alzheimer’s disease.

11. Aaron Copeland (1900 – 1990)

During the 1970s, one of America’s most renowned classical composers, Aaron Copeland, found that his powers were failing him because of memory loss. By the 1980s he was unable to compose, and he passed away in 1990 shortly after his 90th birthday.

12. Burgess Meredith (1907 – 1997)

Burgess Meredith may have been most famous for his role as the Penguin in the “Batman” TV series, although he appeared in numerous films and shows. His career would have been even greater had he it not been for McCarthyism and the Red Scare; he spent 7 years on a Hollywood blacklist because of his left leaning political views. Meredith passed on due to complications from Alzheimer’s disease.

13. Estelle Getty (1923 – 1998)

Estelle Getty is best known for her role as Sophia in the “Golden Girls.” She passed away from complications of Lewy body dementia, which is actually different than Alzheimer’s but also causes memory loss and other Alzheimer’s-like symptoms.

14. Peter Falk (1927 – 2011)

Peter Falk began his career on stage, but reached national prominence as a star of the TV series “Columbo.” He also played the narrator and grandfather in the popular film “Princess Bride.” Towards the end of his life his physician reported that he could no longer even remember the character of Columbo. Alzheimer’s disease was one of the underlying causes for his death.

15. James Stewart (1908 – 1997)

James Stewart was recognized for his military career and for his time as an actor, where he was best known for his roles in “Mr. Smith Goes to Washington” and “It’s a Wonderful Life.” He remained active until his death in 1997, being slowed by Alzheimer’s and another illness, which lead to his death.

16. Eddie Albert (1906 – 2005)

Eddie Albert is best known for his roles in “Roman Holiday” and the “Heartbreak Kid,” as well as his time spent playing Oliver Wendell Douglas in the 1960s television comedy “Green Acres.” He began suffering from Alzheimer’s when in his 90s, and was cared for by his son until his death.

17. Evelyn Keyes (1916 – 2008)

Evelyn Keyes is well known for her role as Suellen O’Hara in the classic film, “Gone with the Wind,” and for her role in “The Seven Year Itch.” She retired from acting but continued living life to the fullest, and wrote a memoir of her experiences before being diagnosed with Alzheimer’s in her 80s and passing away in her 90s.

18. E.B. White (1899 – 1985)

Best known for his lifetime of contributions to The New Yorker and for authoring “Stuart Little” and “Charlotte’s Web,” E.B. White was an accomplished writer and editor. He began suffering from Alzheimer’s in the 80s and eventually succumbed to the disease in 1985.

19. Rosa Parks (1913 – 2005)

Rosa Parks is known as “the Mother of the Freedom Movement,” after being arrested for refusing to give up her seat on the bus and beginning the civil rights movement. She wrote an autobiography and continued to live a quiet life before being diagnosed with and later passing from, Alzheimer’s.

20. James Doohan (1920 – 2005)

Recognized for his military career and known for acting as “Scotty” in “Star Trek,” James Doohan had an incredible acting career before announcing his Alzheimer’s and Parkinson’s disease diagnosis and dying in 2005.

21. Robin Williams (1951 – 2014)

Known and adored by many as an actor and comedian, Williams rose to fame as Mork in the sitcom “Mork & Mindy.” He won the Academy award for Best Supporting Actor for his performance as Dr. Sean Maguire in” Good Will Hunting,” and was also the recipient of two Emmy Awards, six Golden Globe Awards, two Screen Actor Guild Awards and four Grammy Awards for the myriad of impressive work he accomplished. He was inducted as a Disney Legend for his role as Genie in “Aladdin,” and was admired by the American public for his talents as both comedian and actor. Williams began to suffer from Lewy body dementia which was believed to be a“critical factor” that led to his suicide in 2014.

22. Casey Kasem (1932 – 2014)

Kasem’s voice was recognized by Americans everywhere with his American Top 40 countdown — a franchise he co-founded — that greeted the airwaves of radios across the U.S. from 1970 to 2009. He was also the voice of “Shaggy” in “Scooby-Doo” from 1969-1997, and again from 2002-2009. Casey also provided the voice of many commercials and children character cartoon voices, from Sesame Street to Transformers. Kasem was diagnosed with Lewy body dementia in 2007, which he suffered from until his death in 2014.

Worried about your or a loved ones memory?

McConnell delays start of recess to third week in August

Senate delays recess to work on GOP health bill 

Despite public and partisan rhetoric, the congress is attempting to correct the weaknesses ot the Affordable Care Act.  There is much in the new law that is good. Perfection is the enemy of the good. Everyone admits that healthcare is complicated.  This is not a new situation.

Our system evolved from a strictly pay for production (fee for service) to a polyglot system of private insurers, PPOs, and HMOs. Add to those are the military, Veterans health system, Indian Health System, Medicare and medi-cal has resulted in a barely functioning financial support system. 

Some have proposed a 'universal payor system.  At face value UPS sounds very nice, however it does not address underlying health care disparity, nor underlying structural failures in health care delivery. It ignores the gordian knot of many other facts.

The decision to give up a part of the summer recess means Congress is serious about working on the impasse.  There is no time like the present to deal with the crisis.  Congress has many other important and critical issues to resolve.

Thanks to those who serve, to give up a deserved respite.  It is especially gratifying for those who have lived in the District of Columbia, a hot, sweltering miserable season.  I'd rather be in my home district, fishing, being with family, or perhaps just sleeping in late.  Our representatives now have a thankless job.

As we head into the summer doldrum take a minute to call and thank your representative. 

(CNN)Senate Majority Leader Mitch McConnell has delayed the start of the August Senate recess until the third week in August in order to allow more time for his conference to complete "its work on health care reform" among other tasks.

"In order to provide more time to complete action on important legislative items and process nominees that have been stalled by a lack of cooperation from our friends across the aisle, the Senate will delay the start of the August recess until the third week of August," McConnell announced in a statement.

While Republicans are still working through their plan to overhaul Obamacare, McConnell says there is plenty more work to do after that.

"Once the Senate completes its work on health care reform, we will turn to other important issues including the National Defense Authorization Act and the backlog of critical nominations that have been mindlessly stalled by Democrats," McConnell said.

The delay of the August recess came after a handful of rank-and file Republicans urged leadership to stay in Washington to finish their business rather than return home for the district work period.

McConnell delays start of recess to third week in August - CNNPolitics.com

You've heard of Brexit? Here's what they call doctors who are leaving. DRexit

NIRAN S. AL-AGBA, MD | POLICY | APRIL 25, 2017

Sean MacStiofain said, “most revolutions are caused … by the stupidity and brutality of governments.” Regulation without legitimacy, predictability, and fairness always leads to backlash instead of compliance.

Here’s a prediction for you: If something is not done to stop MACRA implementation, more physicians will opt-out of Medicare and Medicaid than is fathomable.

Once DRexit begins, there will be no turning back.

The Medicare Access and CHIP Reauthorization Act of 2015 (MACRA) is destructive to the physician-patient relationship because it prevents physicians from prioritizing patient care. MACRA supporters like to point out this legislation was passed with bipartisan support; in reality, it was passed simultaneously with the repeal of the sustainable growth rate formula.

The sustainable growth rate formula was enacted through the Balanced Budget Act of 1997 and was designed by lawmakers to control Medicare expenditures. The SGR formula limited the annual increase in cost per Medicare beneficiary to the growth of the national economy. Under the SGR formula, if overall physician costs exceeded target expenditures, a reduction in payments would be triggered. Expenditures continued to climb, so Congress stepped in 17 times with short-term legislation (referred to as “doc fix”) to avert the payment reduction since 2002.

Enter stage left, MACRA, known as the “permanent doc fix,” which was passed concurrently with the sustainable growth rate formula repeal legislation. This was the original “repeal and replace.” MACRA established yet another new (and untested) method by which to pay doctors. MACRA is the largest scale reform on the American health care system since the Affordable Care Act in 2010, and the jury is still out how great (or not) that system is working for the American people.

Under MACRA, the Secretary of the Department of Health and Human Services was tasked with implementation of a Merit-Based Incentive (MIP) program which consolidated three useless incentive programs into one big colossal unworkable program for eligible physicians everywhere. The legislation also allows for Advanced Alternative Payment Models (APM), which shockingly, are not actually saving money on care.

You've heard of Brexit? Here's what they call doctors who are leaving.   RETIRED

The Smart-Medicine Solution to the Health-Care Crisis - WSJ

Eric Topol M.D.

Dr. Topol is a cardiologist and professor of molecular medicine at the Scripps Research Institute in San Diego and the author of “The Patient Will See You Now: The Future of Medicine Is in Your Hands” (Basic Books, 2015). He consults for Illumina and Apple on some of the issues discussed here, sits on the board of directors of Dexcom and is a co-founder of YouBase.

Dr. Topol coined the term 'precision medicine', which was co-opted by political interests.

A Diagnosis for Personalized Medicine (a post from May 25, 2017)


Technology, smartphones and accessory attachments offer inexpensive testing platforms which in many cases rival much more expensive ultrasound machines, chemistry panels,  electrocardiogram units.

These new sensor units combined with smartphones will commoditize medical instrumentation. It should lead to a reduction in cost. The main barriers will be institutional resistance to change, a long standing barrier in hospitals and medical organizations.

These small devices, coupled with physician demand for decreased administrative and regulatory burden could lead a marked reduction in health costs.

A sequencing chip from Thermo Fisher Scientific uses semiconductor technology to detect DNA associated with cancer and inherited disease. PHOTO: THERMO FISHER SCIENTIFIC

Dr. Topol goes on to say,

"At the Scripps Research Institute, we are working with the support of a National Institutes of Health grant and several local partners to develop a comprehensive “health record of the future” for individual patients. It will combine all the usual medical data—from office visits, labs, scans—with data generated by personal sensors, including sleep, physical activity, weight, environment, blood pressure and other relevant medical metrics. All of it will be constantly and seamlessly updated and owned by the individual patient. "

Interoperability and cross institutional collaboration are essential for this to occur, and cooperation will be necessary from health plans to accept these new devices as eligible for reimbursement.

Our health-care system won’t be fixed by insurance reform. To contain costs and improve results, we need to move aggressively to adopt the tools of information-age medicine

The Smart-Medicine Solution to the Health-Care Crisis - WSJ

Pre-authorization is hell. Here's why.

Enter the prior-authorization

We have all been to our physicians who tell us we need a certain test. A decade or so ago, you made an appointment and had the x-ray, MRI or scan and that was it.  Now if you need a referral to a specialist, or physical therapist, the referral must be approved by a clerk at the insurance company.
It gets even worse.  Speaking of prior authorization, we have now been informed that we need to get prior authorization to prescribe a muscle relaxer for any patient over the age of 65. Muscle relaxers are apparently so dangerous, and my judgment as a physician so faulty, that the prior authorizers need to get involved. I guess I’ll just have to write more prescriptions for Percocet now because I don’t have time for all these prior authorizations.

Not to beat a dead horse (prior authorizations), but now we are being encouraged to go through Cover My Meds to obtain medication prior authorizations. Cover My Meds “was founded in 2008 with a mission to help patients get the medication they need to be healthy … by electronically automating the medication prior authorization (PA) process, saving health care professionals valuable time and ensuring patients receive the medication they need to be well.” How wonderful! Except that my staff informs me that the process takes, on average, 7-10 days to complete! That’s about 7-10 days longer, I think, than the process ought to take.

Really ?!  Let's face it. These roadblocks are to discourage tests or medications.  They are not in the interest of quality of care.  In fact over 90% of requests for prior authorizations are "rubber stamped"

Prior-authorization is hell. Here's why.

This April, my turn to take the medical board exam rolled back around, necessary every ten years for maintenance of certification. I studied diligently for the better part of three months preceding the test (and I think I did well). It was actually pleasurable to go back over details that I had forgotten and to catch up on newer developments in the field. I realized that I don’t do nearly as much studying, or reading the medical literature as I once did. I used to read an article or two and browse the general medical literature for updates on a daily basis. But my priorities have changed over the last few years.

Now, instead my staff and I spend our time fighting through the incredible sea of silly red tape necessary to get paid and to get our patients even basic care. Here are the highlights from just this last week:

Time taken away from seeing patients, reading journals or studying for maintenance of competence exams.

Distracted, How Regulations have destroyed the practice of Medicine and preventing true Health Reform . Matthew Hahn M.D.

Health Insurance, What's a grad to do?

This article is a re-publish from May 17, 2017.  

Graduating students now have several options for insurance plans. The Affordable Care Act has introduced several options.

As graduation approaches for thousands of young adults this spring, sorting out your health insurance options may seem more daunting than any political economics problem you faced at school.

You may not think it’s a high priority, but remember: Even healthy young people wind up in the emergency room for all sorts of mishaps, and having health insurance will let you get preventive care, including contraceptives, without paying for it.

As graduation approaches for thousands of young adults this spring, sorting out your health insurance options may seem more daunting than any political economics problem you faced at school.

You may not think it’s a high priority, but remember: Even healthy young people wind up in the emergency room for all sorts of mishaps, and having health insurance will let you get preventive care, including contraceptives, without paying for it.

There are several items to consider when choosing.

Take A Look At Your Parents’ Health Insurance.

In 2015, 29 percent of 19- to 25-year-olds were covered as dependents on their parents’ job-based plan, according to a Commonwealth Fund analysis of data from the U.S. Census Bureau’s Current Population Survey. It was the most common type of coverage for this age group.

Employer-sponsored plans are often more generous than an individual plan on the marketplace, with more comprehensive benefits and lower premiums and out-of-pocket costs. And parents can pass these benefits on.

Compare Coverage Through Your Employer.

Seventeen percent of young adults were insured by their own employer in 2015.

Large employers often offer insurance plans, called PPOs, that let workers choose their own doctors and providers from the insurer’s network and often allow them to seek care outside the network if the patient pays a larger share of the cost. A typical PPO plan offered by an employer with at least 500 employees paid for 87 percent of enrollees’ health care costs on average, according to data from benefits consultant Mercer. Compare that with the most popular silver-level plans sold on the ACA’s online marketplaces, which pay 70 percent of costs.

One benefit of an employer’s plan over your parents’: Buying your own plan may improve the odds that you’ll find doctors and hospitals nearby that are in your health plan’s provider network, said Erin Hemlin, director of training and education at Young Invincibles, an advocacy group for young adults.

If Employer Coverage Isn’t An Option, Consider The State Marketplace.

Twenty-two percent of young adults under 26 had marketplace coverage in 2015.

Marketplace plans must provide comprehensive coverage, including hospitalization, drugs and doctor visits. In addition, if your income is between 100 and 400 percent of the federal poverty level (about $12,000 to $48,000 for an individual) you could qualify for tax credits that will help cover the cost of premiums.

If you have a college health plan that ends when you graduate, you may qualify for a special enrollment period to sign up for a marketplace plan. But if you’re uninsured or insured through your parents, you probably can’t buy a marketplace plan until the next open enrollment period in the fall.

A key consideration: If your parents claim you as a tax dependent, you can’t claim the premium tax credit yourself, said Judith Solomon, vice president for health policy at the Center on Budget and Policy Priorities.

Unemployed? Uninsured? Consider Medicaid.

In 2015, 15 percent of people between ages 19 and 25 were on Medicaid.

To date, 31 states and the District of Columbia have expanded Medicaid coverage to adults with incomes of about $16,000 or less.

If you don’t have a job or earn very little and you live in one of these states, you may qualify for Medicaid, which provides comprehensive coverage, typically without a premium.

Unlike marketplace coverage, there’s no open enrollment period for Medicaid. You can apply anytime through your state Medicaid agency, healthcare.gov or your state marketplace.

A key consideration: If your parents claim you as a dependent on their taxes, it could also affect your eligibility.

This story was produced by Kaiser Health News, an editorially independent program of the Kaiser Family Foundation.

What' a Grad to do?