Disease-modifying drugs have become a cornerstone of ATTR-CM treatment in recent years. These new advanced medications can slow or stop the buildup of new TTR proteins in the heart.
A decade ago, “management was primarily focused on treating heart failure symptoms,” says Ajay Vallakati, MBBS, an advanced heart failure and transplant cardiologist at the Ohio State University Wexner Medical Center in Dublin, Ohio. “Now we have TTR stabilizers and TTR gene silencers,” which can help stop ATTR-CM from getting worse.
There isn’t much existing research that directly compares the efficacy of stabilizers versus silencers, so it’s important to have a conversation with your doctor to decide which option may be right for you, or if it makes sense for you to take both of them.
Regardless of what you decide, you’ll reap the most protection by starting treatment as soon as possible. “Early diagnosis and initiation of treatment are crucial, as they offer the greatest potential benefit,” Dr. Vallakati says.
TTR Stabilizers
Stabilizers are oral medications that stop TTR proteins from clumping inside the heart.
With ATTR-CM, “the TTR protein that is secreted from the liver breaks loose and gets stuck inside the heart muscle, and stabilizers prevent it from breaking loose,” explains Ashesh Parikh, DO, a cardiologist with Texas Health Presbyterian Hospital Plano and with Texas Health Physicians Group.
As a result, stabilizing medications have been shown to greatly slow ATTR-CM’s progression and help people live longer, healthier lives.
Medications in this class include:
- Acoramidis (Attruby)
- Tafamidis (Vyndagel, Vyndamax)
TTR Silencers
Silencers work differently than stabilizers, but they still protect the body from the effects of TTR to slow or stop the progression of ATTR-CM. These injectable medications “create a barrier or a halo around the proteins [that are released by the liver], preventing the proteins from settling in the heart,” Dr. Parikh explains.
There is one medication in the class approved for the direct treatment of ATTR-CM and its underlying cause:
There are also two TTR silencers that have been approved for the treatment of polyneuropathy due to ATTR-CM, a type of nerve damage that can cause a variety of symptoms, including numbness or pain, loss of balance, and gastrointestinal issues.
- Inotersen (Tegsedi)
- Patisiran (Onpattro)
Off-Label Treatments
Read the full article here
