National
Advocates Support Research to Find New Treatments for Familial Hypercholesterolemia
By
Jun 16, 2015 - 4:09:13 PM
(HealthNewsDigest.com) - On June 10 and 11 the U.S. Food and Drug Administration sponsored an Advisory Committee meeting to consider the approval of a new class of drugs to treat high cholesterol called PCSK9 inhibitors. One reason the medical community is so interested in these drugs is that they could be a new option for people affected by a genetic condition called familial hypercholesterolemia or FH.
FH is an inherited disorder that leads to aggressive and premature cardiovascular disease. This includes problems like heart attacks, strokes, and even narrowing of our heart valves. For individuals with FH, although making the right diet and lifestyle choices are important, they are not the cause of high LDL and they are usually not sufficient to treat FH. In FH patients, genetic mutations make the liver incapable of metabolizing (or removing) excess LDL. The result is very high LDL levels, which can lead to premature cardiovascular disease (CVD).
There are two forms of FH. If you have inherited the genetic mutation from one parent, then you will have heterozygous FH (HeFH). HeFH occurs in 1 in 200 to 500 people worldwide. If you inherit FH from both parents, you are at risk of developing a much more severe form of the condition called homozygous FH (HoFH). It is very rare, occurring in about 1 in 160,000 to one million people worldwide.
At the FDA Advisory Committee meeting a panel of medical experts heard from company executives as well as leading patient advocates about the experience of living with FH and how PCSK9 inhibitors might be an important new treatment option. One of the presenters was Katherine Wilemon, founder of The FH Foundation, a patient-centric nonprofit organization that is exclusively dedicated to raising awareness of all forms of familial hypercholesterolemia (FH) through education, advocacy, and research. The mission of The FH Foundation is to increase the rate of diagnosis and encourage proactive treatment of this life-threatening disease.
During the Advisory Committee meeting Ms. Wilemon represented the FH Community in her testimony, sharing information about her personal experience living with FH and stressing the essential need for new treatment options to help people manage FH more effectively to live healthier and longer lives. She was joined by many leading doctors and researchers who reinforced the need for new treatment options for all people living with FH, especially those affected by HoFH.
In 2014 the FH Foundation initiated the first-ever, national FH patient registry, CASCADE FHTM (CAscade SCreening for Awareness and DEtection of Familial Hypercholesterolemia). A patient registry is a database where individuals can enter information about a certain disease. Registries enable patients to become more involved in their own disease management and are a way to collect research data on patients' experiences, disease patterns and trends, as well as gaps in diagnosis. This can increase the knowledge of a certain disease and ultimately lead to improvement in patient care and health outcomes.
In January 2015, the FH Foundation also initiated a new effort to identify people at risk of having FH with the launch of FIND FHTM (Flag, Identify, Network, Deliver). The FIND FH Initiative will use advanced analytics including machine learning to help identify individuals with probable FH. The FH Foundation will then work with health care providers to help these people access a proper diagnosis. Once diagnosed, an individual with FH as well as family members at risk will be able to opt into patient support services from The FH Foundation, including the CASCADE registry.
For more information about FH and the work of The FH Foundation, visitwww.thefhfoundation.org.
###
Caption: Katherine Wilemon, President and CEO of The FH Foundation, founded the nonprofit organization to raise awareness of familial hypercholesterolemia, an
inherited disorder that causes aggressive and premature heart disease.
Photo credit: Nancy Pastor
For advertising/promo please call Mike McCurdy at: 877-634-9180 or email [email protected]