2012 Boston Medical Center Gala: Amyloidosis Feature
“There is an utter lack of medical arrogance,” states Mark Kimble, who is a patient of the Amyloid Treatment and Research Program and a stem cell transplant recipient. “The people at [BMC] are compassionate, and work brilliantly as a team. It is a very healing environment.”
Mark Kimble’s testimony – as well as interviews with Dr. Vaishali Sanchorawala and Dr. David Seldin – was featured in a video presented at the 16th Annual BMC Gala. The Gala was held on May 5, 2012 at the Seaport World Trade Center, and raised over $2.25 million for programs and services at BMC. Event organizers received “endless positive feedback from guests, many of whom cited the video as a highlight.”
To view the Gala video: https://development.bmc.org/page.aspx?pid=579
Impromptu speeches from the event: http://cdn.static.viddler.com/flash/as3/full-publisher.swf?ref=&key=3e3edffa
Grandparents Day Event
When: Saturday, May 5th (12:00-3:00pm)
Where: Denver School of Science and Technology – 2000 Valencia Street, Denver, CO 80238
As part of her high school senior project, Sri Ali will raise funds and awareness for amyloidosis.
Sri, who is a senior at the Denver School of Science and Technology, lost three of her grandparents in 2009 – one of whom died of amyloid-related complications. As a tribute to her grandparents, Sri will host a fundraiser to benefit the Boston University Amyloid Treatment & Research Program.
“The purpose of this event is to raise awareness of the disease, and to allow grandchildren to be able to recognize the importance of their relationships with their grandparents before it is too late,” said Sri.
Grandparents Day Event will be held Saturday, May 5th, at the Denver School of Science and Technology, 2000 Valencia Street, Denver, CO 80238, 12 – 3 p.m.
Donations to the Amyloid Program may also be made on our Website.
Congressional Testimony on Amyloidosis
Thursday, March 29th, 2012 – Today Ann Peel will testify before the United States House Appropriations Committee on Labor HHS. Her testimony tells of her experience with amyloid disease, urges the Committee to identify amyloidosis as an important concern, and encourages research towards finding a cure. She also urges the CDC and NIH to educate the American public and medical profession on the need for early diagnosis.
A copy of Ann’s testimony can be downloaded here: Statement on Amyloidosis by Ann Peel
Our heartfelt thanks go to Ann and Terry Peel for their diligent efforts on behalf of amyloidosis awareness and NIH funding.
Dr. Seldin and two patients featured in an NPR interview
Dr. Seldin and two of our patients were featured in an NPR interview last week. You can listen to an audio of this broadcast by clicking here: Rare Disease Feature (WAER 88.3 FM)
The segment aired on February 29th and March 1st in Syracuse, New York (WAER 88.3 FM) on NPR’s flagship news program “All Things Considered.” In observance of World Rare Disease Day, the human interest story features two patients diagnosed with systemic amyloidosis – who share their experiences from diagnosis to treatment and recovery.
Both patients were treated at Boston Medical Center. They credit their physicians for investigating abnormal tests and nonspecific symptoms, and for referring them to amyloid specialists early in the disease course. In his interview, Dr. Seldin underscores the difficulties that patients with orphan disease face, and the importance of physician awareness, early diagnosis, and research.
Upcoming Event: Bill Cunningham Memorial Ski Race & Raffle
Bill Cunningham
What: Bill Cunningham Memorial Ski Race & Raffle
When: Saturday, March 3, 2012
Where: Cannon Mountain (Franconia, NH)
Bill Cunningham, former instructor, director, assistant & technical director of Cannon Mountain, died on May 22, 2007 of
AL amyloidosis. For Cunningham, the illness was misdiagnosed for several years, until it was too late. The event is being held to raise awareness about amyloidosis.
The ”Race to Beat Amyloid” is set for 11:00 AM on Saturday, March 3, 2012. It is open to all ages, and registration starts at 8:00 AM in the Notchview Lodge. The raffle will be set in the Family Room next door beginning at 9:00 AM, with the drawings at 3:30 PM, followed by race awards.
For full event details, please visit: http://www.billsrace.com/index.html
Upcoming Event: A Cappella Concert at UNH
What: A cappella concert to raise funds for Amyloid Research
When: Saturday, March 24 – 5:00 PM
Where: Murkland Auditorium, University of New Hampshire (Durham, NH)
Amyloidosis Benefit Concert Flyer
Directions to Murkland Auditorium
On Saturday, March 24th at 5:00 PM, the New Hampshire Notables will perform in a benefit concert to raise funds for the Boston University Amyloid Program. The concert will be held at Murkland Auditorium on the University of New Hampshire (UNH) campus. The evening will feature performances by seven a cappella groups – six groups from UNH and a special appearance by the Bowdoin Meddiebempsters.
Tickets may be purchased online through the UNH box office (http://unhmub.com/ticket/) or at the door ($3 for students and seniors 65+ / $5 for non-students). Donations to the Amyloid Program may also be made on our Website.
The New Hampshire Notables
Directions:
From I-95 North (Mass.), take Exit 4 (in NH) (left hand exit) to the Spaulding Turnpike (Route 16N), then take Exit 6W (Route 4 West) towards Durham to the Exit for Route 108. At the bottom of the ramp take a Left. Continue straight through the set of lights and bear Right onto Madbury Rd at the top of the hill, continue straight.
You may either:
- Turn Left onto Garrison and go straight through the intersection at the end of the street for parking next to Thompson Hall. – From there Murkland Auditorium is on the other side of Thompson Hall (The building with the clock tower),or
- Continue on Madbury and Turn Left onto Edgewood Rd. There is additional parking on your right at the end of the street. From this lot, head towards the Thompson Hall (the building with the clock tower), as you look straight onto Thomson Hall, Murkland Hall is the Brick/white building directly to the Right of it.
Len Shalansky Memorial Exhibition
Len Shalansky
On November, 13 through November 17, an exhibition and sale was held at Temple Beth El in Providence, Rhode Island, in honor of the artist Len Shalansky. The event was organized by Len’s wife, Ruby.
Len was a talented commercial and fine artist who died of systemic amyloidosis in 2011. His work portrayed an understanding of the human spirit and a sense of humor. His clients included Hasbro Toys and the Oxford University Press.
The Shalansky family generously donated all proceeds from the sale to research on amyloidosis at Boston University to support efforts to find a cure for the disease that took Len’s life far too soon.
NEWS October 2011
BREAKING NEWS…
The Nassau Wings motorcycle group rode to the Boston Amyloid Center from New York City on Saturday, October 15th to deliver a check for $5300. The check was from the “Pop’s Run” fund raising benefit for amyloid research held on August 21, 2011 , an annual event the Wings have sponsored since 2001 after AL amyloidosis touched Pop, one of their members.
The benefit included a motorcycle ride on Long Island, games along the way, a raffle (with major donation of time-share week in Sedona given by Muriel and Steve Finkel), lots of food, and music. Muriel says “The Wings and their fundraiser is one of the highlights of Steve’s and my year.”
The Nassau Wings
Eight members of the Wings, including Jaime Cruz, David Feuer, Julie Knadle, John Simonetti, Lynn Simonetti, Laszlo Bunda, Art Klimczak, and Eddie Santiago, visited us, enjoyed lunch, good conversation, a tour of the laboratory with a research update, and a beautiful sunset at the end of the day. We are so grateful for the steadfast support from this wonderful group of bikers. They have raised an astounding total of $49,500 for amyloid research. Their hard work and generosity are making a difference!
Under the Microscope
Lab Tour
Social Security Administration News Release
The Social Security SSI/SSDI just added Primary Cardiac Amyloidosis to the list of compassionate conditions. This may fast track some claims with Social Security. Here is the link:
http://www.ssa.gov/pressoffice/pr/compassionate-allowances100conditions-pr-alt.pdf
April 2011, New Publications
Four important new publications from our Program investigators:
A strong collaboration has been developed between our Program and the Heart Transplant Program at the Massachusetts General Hospital directed by Dr. Marc Semigran. Since patients with AL amyloidosis and severe heart failure often have a very short survival and poor tolerance to medications, we have worked with the MGH program to evaluate patients for combined therapy with heart transplantation followed by chemotherapy and stem cell transplantation. This is a challenging treatment protocol, but can be lifesaving for selected patients who are young enough and have enough other normal organ function to go through the combined treatment over a period of 6 to 12 months. Patients begin this process with an evaluation at BU and if appropriate are referred to the Heart Transplant team at MGH.
Dey BR, Chung S, Spitzer TR, Zheng H, MacGillivray TE, Seldin DC, MsAfee S, Ballen K, Attar E, Wang T, Shin J, Newton-Cheh C, Moore S, Sanchorawala V, Skinner M, Madsen JC, Semigran MJ: Cardiac transplantation followed by dose-intensive melphalan and autologous stem-cell transplantation for AL amyloidosis and heart failure. Transplantation, 90:905-911, 2010.
BRDey Amyloidosis Transplantation Nov2010
Dr. Vaishali Sanchorawala, Amyloid Program hematologist and director of the Stem Cell Transplant Program, has studied the effect of oral cyclic melphalan and dexamethasone treatment for AL amyloidosis in a group of 70 patients who were not eligible for treatment with high-dose intravenous melphalan and stem cell transplantation. In their study 13% of patients achieved a complete hematologic response and 25% of patients achieved a partial hematologic response. They concluded that oral mel/dex can lead to hematologic responses and clinical benefits for patients not eligible for stem cell transplant.
Sanchorawala V, Seldin DC, Berk JL, Sloan M, Doros G, Skinner M: Oral cyclic melphalan and dexamethasone for patients with AL amyloidosis. Clin. Lymphoma, Myeloma, Leukemia 10: 469-472, 2010
Dr. Elena Klimtchuk, a biophysics researcher in the Gerry Amyloid Research Laboratory, investigated the amyloid fibril-forming properties of an amyloidogenic lambda light chain using sophisticated biophysics techniques. Their novel discovery was that the constant region as well as the variable region of the light chain plays a role in the irreversible aggregation that leads to fibril formation. This finding provides an important new target for molecular therapies that will need to be less individualized than treatments focused on the variable part of the molecule.
Klimtchuk ES, Gursky O, Patel R, Laporte KL, Connors LH, Skinner M, Seldin DC: The critical role of the constant region in thermal stability and aggregation of amyloidogenic immunoglobulin light chain. Biochemistry, 49: 9848-9857, 2010.
Eprint – E Klimtchuck Critical Role of CL Region
Michael Greene, a Pathology and Lab Medicine Department graduate student working with Dr. Connors, studied a molecular chaperone called clusterin and found that it is co-deposited with light chain and transthyretin amyloid in patients’ hearts, and its levels in the blood go down in patients with amyloid cardiomyopathy. This suggests that low levels of clusterin may be a biomarker for amyloid cardiomyopathy, and could play a role in the development of disease.
Greene MJ, Sam F, Soohoo P, Patel R, Seldin DC, and Connors LH: Evidence for a functional role of the molecular chaperone clusterin in amyloidotic cardiomyopathy. J Am Path, 178(1):61-8, 2011.
