Before you read on, don’t forget to check out Part 1 of our interview. In Part 1, Wendy discusses what hypertrophic cardiomyopathy (HCM) is, her rare variant, the diagnostic journey, and her family history of HCM. Today, we’re talking myths and misconceptions, joining Tenaya, and the importance of patient advocacy.

Addressing HCM Myths and Misconceptions

Over the years, Wendy has learned a considerable amount about HCM and is working to combat myths and misconceptions in this field. For example, she shares:

“People tend to believe that hidden conditions or illnesses cannot be as devastating. There are times when I’ve considered getting a handicap placard for my car, but I know that people will think I’m taking advantage of the system when I get out of the car. People say: ‘Oh, well you look healthy!’ But that’s not always accurate. If I have one night where I sleep poorly, the next day I will have arrhythmic events all day long. Even in terms of clinicians, doctors ask how I’m feeling today. At that moment, I might be fine. The question that needs to be asked is what can’t you do today that you could do yesterday, last week or last month?”

Another myth and misconception comes from a general lack of understanding around therapeutic options. Her daughter underwent open heart surgery at six years old. She explains that people asked if the surgery cured her daughter. Wendy shares:

“It’s a genetic condition. So, I want people to understand that the current therapies don’t address the underlying genetic effects. My daughter still has HCM and will until she gets a new heart. A heart transplant is the only way to fix HCM at this point. That’s why research into gene therapy is so exciting.”

Becoming an Advocate

Combatting these myths, and working towards better therapeutic options, depends on greater awareness and understanding. For example, Wendy has shared information on HCM and its impacts with various doctors. A few years ago, while in the emergency room for chest pain, the doctor told Wendy that HCM does not cause chest pain. Wendy says:

“It just floored me. HCM absolutely causes chest pain. So there have been times when being an advocate means describing the condition and symptoms, and letting people know when they’re sharing inaccurate information.”

Wendy’s initial journey into advocacy started when her children were born. Her cousin, who also had HCM, was working as a patient representative in genetics classes. Unfortunately, her cousin’s condition deteriorated as she waited for a new heart. In the interim, Wendy moved into the role as patient advocate and would share information and insights into living with a genetic condition. When Wendy was in her early 30s, her cousin passed away while awaiting a second heart transplant.

In 2013, Wendy reached out to her cardiologist and expressed an interest in advocacy.

“I demanded more of my cardiologist than many patients demand of their clinician. She was founding a company, so I asked if I could be involved. I wanted to do more with my HCM. My diagnosis is a huge part of who I am, but I am not my diagnosis. By becoming an advocate, I can do so much more than I could otherwise. This entire journey helped me to make my way to Tenaya Therapeutics.”

Joining Tenaya

Tenaya Therapeutics is a biotechnology company that is working to develop therapeutics based on an in-depth understanding of the genetics underlying heart diseases. Wendy came onboard to Tenaya as the Senior Manager of Patient Advocacy about a year and a half ago. Tenaya is developing a gene therapy to address HCM, specifically HCM caused by the MYBPC3 mutation (note: this is not the mutation that Wendy has). She explains:

“I share regular reminders on patients, patient needs, and the patient voice. At Tenaya, we start every all company meeting with a patient story to center “Patients First” as part of our core mission. I work with patients to form roundtables or discussion groups to share their stories and really let the entire team understand the direct impact of HCM and why current therapies are not enough.”

“You’ll often hear cardiologists and patients disagree on an unmet need, so it’s important to understand what patient needs are in terms of therapies and how clinical studies are conducted. So, I also work with the clinical development team to ensure that study protocols make sense. People with HCM haven’t had new therapies for a long time. It’s great to be the conduit between patients and Tenaya to work towards that goal.”

Currently, Tenaya has a number of therapies being developed in the pipeline, including TN-201 for genetic HCM due to MYBPC3 mutations. TN-201 has received Orphan Drug designation from the FDA. As described by Tenaya:

We are developing an AAV-based gene therapy designed to deliver a functional MYBPC3 gene in adults and children with gHCM due to MYBPC3 mutations. Our product candidate TN-201 uses a differentiated approach designed to enable robust expression of the MYBPC3 gene in the heart. We have demonstrated significant and durable disease reversal and survival benefit in a relevant murine model after a single dose, as well as tolerability in mice and non-human primates.