#IceBucketChallenge to #StrikeOutALS

So, you’ve accepted your #IceBucketChallenge to #StrikeOutALS, but do you really know what it’s all about?

Over the past few weeks, the ALS Ice Bucket Challenge has taken the social media world by storm. What started as a simple fundraising effort amongst students and alumni at Boston College has now reached news stations, political leaders, and even celebrities. Hollywood stars such as Jimmy Fallon, Justin Timberlake, and the cast of Grey’s Anatomy, just to name a few, have all joined in on the fun to raise awareness and funds for ALS research.

We’re proud to say that Penn Medicine has even gotten in on the action as well! Clark Restrepo, a Research Specialist at Penn’s Center for Neurodegenerative Disease Research studying ALS, accepted his challenge last week. See the video here:

But when you stop to think about it, do you really understand the reason why you’re dousing yourself with that ice cold bucket of water?


Critics of the Ice Bucket Challenge believe that most participants don’t truly understand the cause that they are supporting and question the effectiveness, claiming that it can’t possibly do much good if so many people are choosing to soak themselves instead of donating money. However, the recent spike in donations proves otherwise. In an NBC News article released today, the ALS Association revealed that is has now received an incredibly impressive $15.6 million in donations from a combination of existing donors and 307,598 new donors since the start of the challenge in late July. “That’s compared to just $1.8 million in that same time period in 2013.”

Read the full NBC News article here.

For more information on ALS, visit: www.alsa.org

DONATE to Penn’s ALS Research Fund here.

Congratulations! Penn Medicine Neuropathologist Eddie Lee, MD, PhD Awarded $486,000 Doris Duke Grant to Support His Research in FTD and ALS.

EddieLeeLargeCongratulations to Eddie Lee, MD, PhD, an assistant professor of Pathology and Laboratory Medicine in the Perelman School of Medicine at the University of Pennsylvania on receiving a three-year Clinical Scientist Development Award (CSDA) for $486,000 from the Doris Duke Charitable Foundation!
This grant will support his ongoing research in Frontotemporal Degeneration (FTD) and Amyotrophic Lateral Sclerosis (ALS).

 Being awarded the DDCF CDSA is a great honor, and an amazing validation of the translational approach that we take here at Penn to tackling neurodegenerative diseases.

As head of the Translational Neuropathology Research Laboratory, Lee and his colleagues’ goal is to better understand the causes of these neurodegenerative diseases to be able to develop specific disease-modifying therapies.

With this grant, Lee plans to study a mutation in the gene C9orf72, the most common genetic cause of FTD and ALS, explained below.

A subset of patients with the C9orf72 mutation exhibit DNA hypermethylation which partially silences the mutant C9orf72 gene.  We have found that this DNA hypermethylation is associated with reduced neuropathologic inclusions in brain tissue, raising the possibility that C9orf72 hypermethylation protects against disease.  We are pursuing this by studying the cohort of individuals with the C9orf72 mutation followed by Penn physicians in the PENN FTD Center and the ALS Center at Pennsylvania Hospital.  The hope is that understanding these molecular changes in patients and human tissues will lead to novel biomarkers of disease and potentially lead to personalized therapies.

To learn more about the Doris Duke Charitable Foundation Clinical Scientist Development Award, visit: www.ddcf.org.

Full Penn Medicine News Release.

* Quotes courtesy of Eddie Lee, MD, PhD                           
Assistant Professor of Pathology and Laboratory Medicine,
Perelman School of Medicine, University of Pennsylvania



Dr. Trojanowski Discusses New Paths for Alzheimer’s Research in Recent P&T Article

According to Susan Worley’s recent article ‘After Disappointments, Alzheimer’s Researchers Seek Out New Paths’ for Pharmacy & Therapeutics journal, right now, the only Alzheimer’s treatments that are available are those that simply address the symptoms, but cannot modify the disease. However, researchers are revisiting earlier studies of disease-modifying treatments in hopes of proving that unsuccessful clinical trials from the past were not entirely unproductive.

John Q. Trojanowski, MD, PhD, Director of the IOA and Penn Alzheimer’s Disease Core Center (ADCC), discussed the three most important lessons that we have learned from failed clinical trials.

Lesson #1. Researchers must find a way to intervene at an earlier stage of Alzheimer’s disease, increasing the vitality of designing prevention or clinical trials that address prodromal Alzheimer’s disease.

Lesson #2. “We must use biomarkers in all patient populations to increase confidence in diagnosis and also to monitor target engagement, as well as response to therapy.”

Lesson #3. New drugs must be developed “that address other AD targets, such as tau tangles.”

Also featured in the article, Dr. Reisa Sperling, MD, Director of the Center for Alzheimer’s Research and Treatment at Brigham and Women’s Hospital and Massachusetts General Hospital and Professor of Neurology at Harvard Medical School, reiterates the significance of these lessons and emphasizes “the need to treat the right target at the right stage of disease.”

Both Trojanowski and Sperling seem to agree that pursuing these new targets through combination therapy is the way to go. Dr. Trojanowski “tend[s] to agree with colleagues who say that we are now witnessing the last of the era of monotherapy clinical trials.” He explains that in the past, researchers in other therapeutic areas have been a bit resistant to the idea of combination therapy, however, “particularly for AIDS, it has turned out to be the winning strategy.”

There is also increasing hope for the development of new technologies for diagnosing Alzheimer’s disease. Patients,families, and clinicians alike are yearning for new strategies based on imaging to replace the current and much more invasive methods such as lumbar punctures used to collect cerebrospinal fluid (CFS).brianscan

According to the article, the International Society to Advance Alzheimer’s Research and Treatment (ISTAART) is working on developing new standards for AD biomarkers, mirroring the way that blood tests can determine cholesterol, “so that eventually simple predictive and diagnostic tests can be effectively implemented” when screening for Alzheimer’s.

As for recent clinical trials studying AD, there are currently five underway which are discussed in the article.

#1. The A4 Study (The Anti-Amyloid Treatment in Asymptomatic Alzheimer’s Disease)

#2. The DIAN-TU Study (The Dominantly Inherited Alzheimer’s Network Trials Unit)

#3. The API ADAD Study (Alzheimer’s Prevention Initiative Autosomal Dominant Alzheimer’s Disease)

#4. The TOMMORROW Trial

#5. The SNIFF Study (The Study of Nasal Insulin in the Fight Against Forgetfulness)

Overall, this “new era” of Alzheimer’s disease prevention trials which combines methods of modulating beta-amyloid levels with the use of biomarkers provides the information needed to develop drugs that address non-amyloid targets and to continue moving research in the direction of combination therapy.

To read the full P&T article, including further descriptions of the current clinical trials, click here.

Image Source: www.medicinenet.com via Medical Body Scans/Photo Researchers Inc.

IOA and CNDR Directors Named 2014 J. Allyn Taylor International Prize in Medicine Recipients!

John&VirginiaCongratulations to our very own Virginia M.-Y. Lee, PhD, MBA, Director of the Center for Neurodegenerative Disease Research, and John Q. Trojanowski, MD, PhD, Director of the Institute on Aging, on being named the 2014 J. Allyn Taylor International Prize in Medicine co-recipients!

They have been chosen for this award in honor of their lifelong dedication and work towards finding ways to understand and treat the life altering neurodegenerative diseases such as Alzheimer’s, Parkinson’s, amyotrophic lateral sclerosis (ALS), frontotemporal degeneration (FTD), progressive supranuclear palsy (PSP), and related disorders.

The J. Allyn Taylor International Prize in Medicine is awarded annually by the Robarts Research Institute at Western University in London, Ontario to acknowledge individuals who have made significant contributions in a particular field of research. Candidates are nominated by their peers in the scientific community and a jury decides the winners. 48 different researchers have been awarded since it s debut in 1985.

Recipients receive a $25,000 cash reward, an engraved medal, and a framed certificate. The award is supported by the C.H. Stiller Foundation and will be presented to Drs. Lee and Trojanowski at the 2014 J. Allyn Taylor International Prize in Medicine Symposium on November 20, 2014 in London Ontario.

For more information on the J. Allyn Taylor International Prize in Medicine award and symposium, click here.

To read the full Penn Medicine News Release on this award, click here.

MVP: Gift of William Bates, Jr., Supports Young Scientists, Advances Alzheimer’s Research, and Connects to Family Heritage

By giving to the Center for Neurodegenerative Disease Research (CNDR), William Bates, Jr., has become one of Penn Medicine’s Most Versatile Players. Among his gift’s many roles: A tribute to his wife of 61 years, Elizabeth, who died from Alzheimer’s disease. A contribution to a cure. A strike against the escalating crisis in research funding for young scientists. And, more unexpectedly, a renewal of his lifelong connection to Penn.

In 2008, Mr. Bates established the Bates Family Travel Fellowship, an endowed fund supporting postdoctoral fellows at the CNDR. Conference attendance is a professional necessity for these young scholars, but often they cannot afford the associated expenses. This award enables recipients to travel and attend meetings with top scientists from around the world, fostering the intellectual collaboration needed to advance a cure.

Given decreased federal dollars for science, gifts like this are more crucial than ever. Currently, the National Institutes of Health (NIH) can fund just half of viable proposals, leaving many promising ideas – and careers – unrealized.

“We have a serious risk of losing the most important resource that we have, which is this brain trust, the talent and the creative energies of this generation of scientists,” said NIH director Francis Collins.

As home to the largest Biomedical Graduate Studies program in the nation, Penn educates the researchers who will meet the biggest scientific challenges of the coming decades, including the urgent search for an Alzheimer’s cure. Today, five million Americans suffer from the disease; by 2050, that number is projected to grow to 14 million.

Federal cutbacks mean that further progress depends increasingly on the philanthropy of friends like Mr. Bates, who takes the mission of Penn’s young scientists seriously – and very personally. “I don’t think anything would please me more than to see a breakthrough in my lifetime,” he said.

William Bates, Sr., chief surgeon at both Graduate Hospital and Penn Presbyterian

For Mr. Bates, a personal connection to Penn Medicine is nearly a birthright. His father, William, earned both his undergraduate and medical degrees from Penn; his mother, her nursing degree. During World War I, his parents were part of a Penn initiative to establish wartime medical facilities in France.

After the war, Dr. Bates set up his medical practice in Philadelphia, eventually becoming chief surgeon at both Graduate Hospital and Penn Presbyterian. Mr. Bates vividly recalls the telephone – “always ringing” – next to his father’s seat at the dinner table.

Mr. Bates’s 45-year career in banking was highlighted by his becoming vice chairman of the Philadelphia National Bank (now Wells Fargo) and at the same time serving a term as chairman of the board of VISA, the credit card organization. He “retired” at 65, only to launch Consumer Loan Services, a first-of-its-kind consulting firm serving small lenders. Within five years, the company grew from a single room furnished with a folding table and chairs to a 200-employee operation.

But his well-earned retirement was overshadowed by his wife’s Alzheimer’s diagnosis. After her death in 2006, the memory of her suffering “became an overwhelming desire to try to do some good.” This desire is shared by Mr. Bates’s two sons, William and Jeffrey, who have pledged to continue support for the CNDR past his lifetime.

For Mr. Bates, philanthropy has yielded new connections to Penn. Over the years he has met with CNDR directors Drs. Virginia Lee and John Trojanowski and the recipients of the travel award. “I get so much pleasure from seeing these young people at the beginning of their careers, with all of it ahead of them,” he said.

Supporting Penn’s next generation of scientists also led Mr. Bates to recover a little piece of his own history. With the help of Penn Medicine’s development staff, he located a portrait of his father that once hung in the lobby of Graduate Hospital. Today, the portrait resides in his home.
At 92, Mr. Bates has an extraordinary connection to Penn’s past. But the CNDR’s search for an Alzheimer’s cure keeps him looking to the future. “I hope I’m still here when this puzzle is solved,” he said. “It would be the most wonderful gift I could have.”

To learn more about how to support the Institute on Aging contact Michael Sofolarides, director of development at Penn Medicine, at 215.573.0187 or msof@upenn.edu.


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