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.”
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).
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