When Standard Treatments Aren't Enough: A Sister's Fight for Options

Compassionate Use

May 17

When someone you love is diagnosed with advanced liver cancer, finding treatment options can become an urgent and overwhelming responsibility. For caregivers, that search often means navigating unfamiliar medical information, exploring clinical trials, and making difficult decisions alongside someone they love.

For Andrea Wilson Woods, founder and president of Blue Faery: The Adrienne Wilson Liver Cancer Association, that search began in May 2001, when her 15-year-old sister, Adrienne, was diagnosed with hepatocellular carcinoma (HCC).

During Blue Faery's The Truth About Liver Cancer workshop on compassionate use, Andrea shared how her determination to find additional treatments introduced her to expanded access, a pathway for obtaining investigational medications outside clinical trials. Her experience also taught her a lasting lesson about respecting patients' wishes.

A Diagnosis That Changed Everything

Andrea became Adrienne's legal guardian when she was just 22 years old. Despite their 14-year age difference, the sisters were exceptionally close, and Andrea considered Adrienne both her sister and her child.

On May 16, 2001, Andrea returned home to find Adrienne curled up on the living room floor, crying and struggling to breathe. Within hours, a CT scan revealed tumors in her liver and lungs. She was transferred to Children's Hospital Los Angeles, where she began treatment for advanced HCC.

At the time, treatment options for advanced liver cancer were extremely limited. Adrienne received aggressive chemotherapy, but Andrea later learned that the medications were not specifically intended for liver cancer and that there was no evidence they would extend her sister's life.

The treatments caused increasingly severe side effects and repeated hospitalizations. Andrea also discovered that the care Adrienne was receiving was essentially palliative, although no one had explained it that way.

Palliative care focuses on relieving symptoms, managing side effects, and improving quality of life. It is not the same as hospice care and can be provided alongside cancer treatment.

For Andrea, the experience highlighted the importance of understanding not only what treatments are being offered, but what they are intended to accomplish.

Searching Beyond Standard Cancer Treatments

Determined to find another possibility, Andrea began researching advanced liver cancer, searching medical studies for patients who had survived metastatic HCC.

In August 2001, she discovered reports of a Japanese study involving patients whose liver cancer had spread to other organs. A handful had survived after receiving a particular drug combination that included an investigational therapy.

The findings offered no guarantees, but they changed Andrea's perspective. She began contacting physicians, researchers, and pharmaceutical companies to explore whether Adrienne could access treatments that were not widely available.

At the time, Andrea had never heard the term compassionate use. She simply wanted to understand every possible option.

Finding a Specialist and Respecting Adrienne's Choices

Another challenge was Adrienne's age. Although she had a cancer more commonly diagnosed in adults, she was receiving care at a children's hospital where her physicians had limited experience treating HCC.

Andrea sought a second opinion at UCLA with Dr. Rafael Amado, who regularly treated liver cancer patients.

Unlike Adrienne's initial medical team, Dr. Amado was willing to explore clinical trials. Adrienne was eventually offered participation in a Phase 2 trial involving dendritic cell therapy.

However, the study required her to spend Monday through Friday at UCLA.

Adrienne refused.

She wanted to return to school and spend her remaining time doing things that mattered to her.

As Andrea recalled, Adrienne said:

"I'm not going to spend what time I have left in the hospital. I want to go back to school."

Adrienne had already demonstrated that determination when she began experiencing hearing loss from cisplatin, a chemotherapy medication. As an aspiring musician, she insisted on discontinuing the drug rather than risk further damage to her hearing.

Although Andrea wanted to pursue every opportunity, she respected her sister's decisions.

These experiences reinforced an essential principle of patient-centered care: treatment decisions must reflect the patient's own priorities, values, and quality-of-life goals.

How Compassionate Use Became Part of Adrienne's Story

Near the end of Adrienne's life, Andrea learned about an investigational immunotherapy medication called thymosin alpha-1, also known as Zadaxin, through a family connection who worked as a researcher at the National Institutes of Health.

Adrienne's physicians did not believe the medication was likely to help, but they agreed to explore access.

Dr. Amado worked with the pharmaceutical company to obtain FDA authorization for Adrienne to receive the investigational drug outside a clinical trial.

This process is known as expanded access, often called compassionate use. It allows certain patients with serious or life-threatening conditions to access investigational medical treatments when eligibility requirements are met, and satisfactory alternatives are unavailable.

Andrea recalled that authorization came remarkably quickly, possibly within a week. Dr. Amado would supervise the treatment, and Andrea would administer the medication at home.

But Adrienne died before receiving it.

She lived just 147 days after her diagnosis.

Although Adrienne never received the investigational treatment, the experience showed Andrea that pathways beyond standard therapies and conventional clinical trials could sometimes be explored.

What Caregivers Can Learn From Andrea's Experience

Looking back 25 years later, Andrea emphasized that patients and caregivers should feel empowered to ask questions and seek reliable information.

"It's okay to ask questions and to keep searching for options."

Her experience offers several important lessons:

Ask about treatment goals. Understand whether a therapy is intended to control cancer, relieve symptoms, or improve quality of life.
Seek experienced specialists. A second opinion from a physician familiar with liver cancer may reveal additional treatment possibilities.
Explore investigational options. Clinical trials and compassionate use may be worth discussing when established treatments are insufficient.
Build a support network. Reputable patient advocacy organizations, family members, and medical professionals can help patients navigate complex decisions.
Respect the patient's wishes. The possibility of another treatment must be considered alongside the patient's own goals and preferences.

Andrea stressed that caregivers cannot manage everything alone. Having people who can help research options, ask questions, and provide emotional support makes a difference.

Most importantly, she reminded caregivers that advocating for someone does not mean making every decision for them.

"At the end of the day, it's the patient's decision."

Turning Personal Loss Into Patient Advocacy

Adrienne's experience inspired Andrea to establish Blue Faery: The Adrienne Wilson Liver Cancer Association, an organization dedicated to liver cancer awareness, education, advocacy, and patient support.

Her story demonstrates that advocacy can mean pursuing investigational treatments, seeking a second opinion, or asking difficult questions about available care. It can also mean honoring a loved one's decision not to pursue another treatment.

For patients and caregivers facing limited options, understanding compassionate use and maintaining open communication with medical professionals can be important steps toward making informed decisions.