“When I came to the Abramson Cancer Center (ACC) with stage 2 synovial sarcoma, I needed hope and I needed help. Dr. Kristy Weber treated my cancer in a way no one else could. She saved my leg and my life. Thanks to the sarcoma program at the ACC, I am a three-month cancer survivor.” Megan Dychala, sarcoma cancer survivorAt 23, Megan Dychala was a former college soccer player who thought her nagging knee pain was a result of old injuries. She was young, active, and enjoying all that life had to offer her. But, in October, 2013 she got a phone call that would change her life.Megan’s ongoing knee pain was in fact a tumor behind her knee. One day she was healthy and the next she was faced with a rare and aggressive cancer. Although statistics were against Megan, she knew she wouldn’t let anything happen without a fight.Luckily Megan found Penn’s Abramson Cancer Center and Kristy Weber, MD. While other hospitals said that amputation was her only chance for survival, Dr. Weber knew she could do better – treating Megan’s cancer, but also ensuring a good quality of life after treatment. Megan shares her personal journey at the Abramson Cancer Center below. My Sarcoma StoryMy medical team worked with me to develop an aggressive treatment plan that included five and a half weeks of daily radiation followed by an intense surgery. Dr. Stephen Hahn was my rockstar of a radiation oncologist and took care of me every step of the way. Radiation left me burned and uncomfortable, but it is truly amazing how the skin recovers from so much trauma. My leg after 3, 4, and 5.5 weeks of radiation treatment.Finishing radiation and ringing the bell, surrounded by my family and the other cancer warriors there, was one of the best days of my life.The surgery we opted for allowed me to keep my leg, but I would be sacrificing abilities. With so much reconstruction and working with nerves, there is no guarantee the leg will regain proper function. I was told I could never walk without a cane or walker and that I could need to wear a leg brace, possibly for the rest of my life, to support my foot. I was also told I could have limited mobility restricting things like driving and basic daily tasks. The “what could bes” were daunting, but I trusted Dr. Weber and my vascular and plastic surgeon guru Dr. Stephen Kovach. Together, Drs. Weber and Kovach removed the back of my knee and rebuilt my leg through vascular, skin, and muscle grafts in a 14-hour surgery. I had a long road ahead of me, and a mountain of unknowns to climb.Drs. Weber and Kovach’s work was phenomenal. Less than six weeks after surgery, I was walking on my own two feet. I never had to use a cane or walker. I don’t wear a leg brace. I drive my car, work out at the gym, and walk my dog. My leg function is improving everyday, and I see a 100 percent return in my future. This experience has taught me a lot, but most importantly, no matter how much the deck is stacked against you, or how grim the statistics look, you should always remember that you can be the one to improve the statistics. A positive attitude goes an incredibly long way. I was diagnosed with a “one in a million cancer,” but now I am one more survivor. I owe this second chance at life to my amazing team at Penn Medicine and the Abramson Cancer Center.Less than 4 months after diagnosis, I was celebrating being cancer free.
Penn’s Amyloidosis Program Attend International Symposium and Report on Clinical Trials
Recently, several hundred investigators from around the globe gathered in Indianapolis, IN for the fourteenth International Symposium on Amyloidosis, April 27-May 1, 2014 to discuss the rare disease, recent findings, and ongoing clinical trials. Members of Penn Medicine’s multidisciplinary Amyloidosis Program were in attendance and provide commentary on some highlights from the meeting.Amyloidosis: A brief summaryAmyloidosis is a group of diseases characterized by the buildup of abnormal proteins called “amyloid fibrils” in tissues and organs throughout the body. Over time, this accumulation alters the ability for organs to function normally and leads to health complications. Left untreated, amyloidosis can be potentially life threatening, so receiving an early, accurate diagnosis is very important.Diagnosing amyloidosis is not easy, however. Symptoms can be vague and are often similar to those of other diseases. In addition, symptoms can appear in several organs at the same time, spanning the areas of cardiology, nephrology and neurology, for example.Often, the presence of many persistent, unrelated symptoms is what alerts a physician to the possibility of amyloidosis. Penn Medicine’s Contribution: Evolving Therapies The Amyloidosis Program at Penn Medicine is involved in the treatment and diagnosis of amyloidosis and the development of new drugs to treat the disease and its variants. Amyloidosis can have a variety of causes, and as a result, the Program brings together specialists from cardiology, nephrology, hematology-oncology, neurology, rheumatology, pulmonology and organ transplantation.The following information is derived from presentations made at the fourteenth ISA, by members of the Penn Amyloidosis Program:NEOD001 – A New Approach to Treating Cardiac Amyloidosis by Targeting Existing Organ DepositsThe Penn Amyloidosis Program is involved in the development of a promising new drug called NEOD001, which is designed to target and remove the buildup of amyloids in the heart and other affected organs. So far, the majority of patients in the study have seen stability or improvement in the blood tests that measure the impact of amyloidosis on the heart.The primary investigator for the NEOD001 study at Penn Medicine, Brendan Weiss, MD, director of the Penn Amyloidosis Program, is enthusiastic about the preliminary results.“The capacity to target existing amyloid fibrils in the tissues is among the great unmet needs of amyloidosis therapy,” Dr. Weiss says. “The safety information from this trial is reassuring, and while the organ response data are very preliminary, they are encouraging, and suggest further development of this approach is needed.”Penn Researcher Investigating Kiacta™ for Treating AA Amyloidosis in International StudyA team of researchers, including Laura M. Dember, MD, of the Renal Electrolyte and Hypertension division at Penn Medicine, presented findings from an ongoing clinical trial of Kiacta™, an oral drug for the treatment of AA amyloidosis. A variant of the disease, AA amyloidosis is associated with kidney failure. This clinical trial looks to confirm findings from earlier trials around safety and effectiveness of Kiacta. Further Research Presented at the 14th ISAIn other developments at the fourteenth ISA, Adam Cohen, MD of the Amyloidosis Program at Penn commented on a study presented by Vaishali Sanchorawala from the Amyloidosis Center at Boston University. Dr. Sanchorawala’s study involves the use of drugs melphalan and bortezomib before and after stem cell transplant to treat patients with primary systemic amyloidosis.Dr. Cohen observes that while the Boston University study confirms the activity of bortezomib in AL amyloidosis and its potential to further improve outcomes in patients undergoing autologous stem cell transplant, 14% of participants were unable to proceed to transplant, due to clinical deterioration during induction therapy.“This suggests that going directly to transplant for transplant-eligible patients and reserving the bortezomib/dexamethasone until post-transplant may be a preferred approach,” Dr Cohen says, noting that further validation of these approaches in larger prospective trials is warranted.For additional information on the program, including consultations, scheduling, and resources from Penn Medicine’s Amyloidosis Program, please contact the program by email, or call 800-789-PENN (7366).
Penn's Amyloidosis Program Attend International Symposium and Report on Clinical Trials
Members of Penn Medicine’s multidisciplinary Amyloidosis Program were in attendance at the 14th Annual International Symposium on Amyloidosis.
Focus on Cancer RSS Feed 2014-07-01 10:00:00
Recently, several hundred investigators from around the globe gathered in Indianapolis, IN for the fourteenth International Symposium on Amyloidosis, April 27-May 1, 2014 to discuss the rare disease, recent findings, and ongoing clinical trials. Members of Penn Medicine’s multidisciplinary Amyloidosis Program were in attendance and provide commentary on some highlights from the meeting.
Amyloidosis: A brief summary
Amyloidosis is a group of diseases characterized by the buildup of abnormal proteins called “amyloid fibrils” in tissues and organs throughout the body. Over time, this accumulation alters the ability for organs to function normally and leads to health complications. Left untreated, amyloidosis can be potentially life threatening, so receiving an early, accurate diagnosis is very important.
Diagnosing amyloidosis is not easy, however. Symptoms can be vague and are often similar to those of other diseases. In addition, symptoms can appear in several organs at the same time, spanning the areas of cardiology, nephrology and neurology, for example.
Often, the presence of many persistent, unrelated symptoms is what alerts a physician to the possibility of amyloidosis.
Penn Medicine’s Contribution: Evolving Therapies
The Amyloidosis Program at Penn Medicine is involved in the treatment and diagnosis of amyloidosis and the development of new drugs to treat the disease and its variants. Amyloidosis can have a variety of causes, and as a result, the Program brings together specialists from cardiology, nephrology, hematology-oncology, neurology, rheumatology, pulmonology and organ transplantation.
The following information is derived from presentations made at the fourteenth ISA, by members of the Penn Amyloidosis Program:
NEOD001 – A New Approach to Treating Cardiac Amyloidosis by Targeting Existing Organ Deposits
The Penn Amyloidosis Program is involved in the development of a promising new drug called NEOD001, which is designed to target and remove the buildup of amyloids in the heart and other affected organs. So far, the majority of patients in the study have seen stability or improvement in the blood tests that measure the impact of amyloidosis on the heart.
The primary investigator for the NEOD001 study at Penn Medicine, Brendan Weiss, MD, director of the Penn Amyloidosis Program, is enthusiastic about the preliminary results.
“The capacity to target existing amyloid fibrils in the tissues is among the great unmet needs of amyloidosis therapy,” Dr. Weiss says. “The safety information from this trial is reassuring, and while the organ response data are very preliminary, they are encouraging, and suggest further development of this approach is needed.”
Penn Researcher Investigating Kiacta™ for Treating AA Amyloidosis in International Study
A team of researchers, including Laura M. Dember, MD, of the Renal Electrolyte and Hypertension division at Penn Medicine, presented findings from an ongoing clinical trial of Kiacta™, an oral drug for the treatment of AA amyloidosis. A variant of the disease, AA amyloidosis is associated with kidney failure. This clinical trial looks to confirm findings from earlier trials around safety and effectiveness of Kiacta.
Further Research Presented at the 14th ISA
In other developments at the fourteenth ISA, Adam Cohen, MD of the Amyloidosis Program at Penn commented on a study presented by Vaishali Sanchorawala from the Amyloidosis Center at Boston University. Dr. Sanchorawala’s study involves the use of drugs melphalan and bortezomib before and after stem cell transplant to treat patients with primary systemic amyloidosis.
Dr. Cohen observes that while the Boston University study confirms the activity of bortezomib in AL amyloidosis and its potential to further improve outcomes in patients undergoing autologous stem cell transplant, 14% of participants were unable to proceed to transplant, due to clinical deterioration during induction therapy.
“This suggests that going directly to transplant for transplant-eligible patients and reserving the bortezomib/dexamethasone until post-transplant may be a preferred approach,” Dr Cohen says, noting that further validation of these approaches in larger prospective trials is warranted.
For additional information on the program, including consultations, scheduling, and resources from Penn Medicine’s Amyloidosis Program, please contact the program by email, or call 800-789-PENN (7366).
