Exactly two years ago today, an insidious disease claimed my beautiful 13-year-old daughter’s life inside the “presidential suite” of one of the nation’s leading children’s hospitals, but it cannot silence her powerful voice.

Cyndi and I have accepted a wonderful invitation to attend a first-of-its kind patient and caregiver advisory board meeting next month in Houston, during which a diminutive but dynamic group of survivors of post-transplant lymphoproliferative disorder (PTLD) and family members who lost loved ones to the rare form of blood cancer will convene.

Atara Biotherapeutics, a San Francisco-based biotechnology company in late-stage development of an immunotherapy called tab-cel, which I believe may have been able to prevent Marisa’s death, is conducting the meeting in conjunction with a national conference known as the Transplantation and Cellular Therapy Meeting of the American Society for Blood and Marrow Transplantation (ASBMT) and Center for International Blood and Marrow Transplant Research.

Through the retelling of Marisa’s courageous journey and valiant fight, her powerful voice will be heard, potentially impacting diagnosis and treatment of PTLD.

After being hospitalized for more than six of the last seven months of her life, Marisa, who loved musical theater and aspired to audition on The Great White Way, had become one of the longest running shows on Broadway, the avenue on which New York Presbyterian’s Morgan Stanley Children’s Hospital is located at the corner of West 165th Street.

On a frigid January morning two years ago, sun poured through the only window of Marisa’s Pediatric Cardiac Intensive Care Unit room. The largest on the hospital’s ninth floor, it has been affectionately referred to as the “presidential suite.”

The warm rays of light cascaded through the window, which provided a panoramic view of the scenic New York City skyline that stretched from the Hudson River to the west to the Alexander Hamilton Bridge to the north with the Empire State Building standing proudly in the distance between both landmarks.

The gentle beams of sun blended seamlessly with the soft yellow paint adorning the walls of Marisa’s hospital room to usher in a new day following a night during which Marisa’s health took a tragic turn.

The rare form of blood cancer, known as PTLD, that initially riddled Marisa’s body upon being first diagnosed just three months earlier, had spent the last few weeks infesting her cerebrospinal fluid and brain.

The disease was a postoperative complication of a successful heart transplant, necessitated by two life-threatening conditions with which Marisa was living called protein-losing enteropathy and plastic bronchitis.

Having already survived six open-heart surgeries, the first of which occurred a day following her birth, to correct a complex congenital cardiac defect know as hypoplastic left-heart syndrome, Marisa had beaten insurmountable odds countless times before. She spent more than 70 days in the hospital following her heart transplant, 41 of them in intensive care. Marisa nearly died on the operating table years earlier when an air embolism floated from her aorta to her brain. She awoke from that particular open-heart surgery as if a catastrophic episode never occurred, an outcome the hospital’s chief neurologist described with incredulity as a “miracle,” for he simply had no other explanation regarding Marisa’s near perfect post-operative state.

The unprecedented proliferation of PTLD, however, was too much for Marisa to overcome. Her oncologists consulted with other medical experts across the globe, but none could devise a way to thwart the disease’s relentless onslaught.

Marisa’s case, according to the medical director of heart failure, cardiomyopathy and cardiac transplantation at one of the nation’s leading children’s hospitals, was “a very grave form” that “occurred very early after transplant and was very widespread.”

“I would qualify that as a rare occurrence,” the doctor said of Marisa’s PTLD. “Quite honestly, it’s rare enough that in my own limited practice (with PTLD) I would say I haven’t seen proliferation into the brain. I can imagine that it would be very much so a more difficult scenario to treat.”

Marisa’s valiant and courageous battle with PTLD included ineffective chemotherapy and whole-brain radiation treatments.

The cancer rapidly worked its way through her brain stem and conspired with sizable frontal-lobe tumors and leptomeningeal metastases to affect Marisa’s central nervous system, impacting her vision, speech, fine-motor skills, ability to swallow and eventually breathe. The PTLD took away her memory, created delirium, altered her personality and left her convulsing with grand mal seizures. The disease further took its toll, shutting down her kidneys and impacting other vital organs. Less than two weeks after high-dose steroids alleviated the swelling in Marisa’s brain, miraculously allowing her to sit in a chair and talk on Christmas Day, Marisa, her voice now barely audible, courageously agreed to be intubated, nodding her head in affirmation upon being told she, yet again, needed a breathing tube.

A team of nurses and doctors from a myriad of disciplines, knowing Marisa’s health was in a rapid state of decline, entered from the intensive care unit’s main hallway, their collective footsteps breaking the silence of the sunlit “presidential suite.” More than a dozen of them gathered around Marisa’s hospital bed, forming a horseshoe of somber medical experts in white coats accessorized with stethoscopes.

In Marisa’s final moments of lucidity, she extended her arms to the physician in charge. The doctor, suspecting Marisa was going to attempt to whisper something in her ear, leaned in close. Mustering every ounce of strength in her frail body, Marisa grabbed the doctor by the lapels of her white jacket, pulling her in tight to deliver a wordless hug. The gesture was Marisa’s way of saying “thank you” and imploring the doctor to save her life.

The venerable physician, who was part of a medical team that conducted the country’s first successful pediatric heart transplant 36 years ago, was reduced to tears, realizing that, in a cruel twist of fate, the new life-saving organ that beat in Marisa’s body was now responsible for her terminal illness.

The hug was the last voluntarily movement Marisa ever made. She died along with the heart of her donor exactly three weeks later, on the same day the screening period to receive Atara Biotherapeutics potentially life-saving tab-cel ended.

I had signed a consent form precisely 21 days earlier allowing Marisa to be part of a clinical study to receive the immunotherapy, a potential treatment for solid organ and bone marrow transplant recipients with Epstein-Barr virus-associated PTLD.

Marisa was eligible for the clinical study because the treatment protocol for patients with PTLD, including a prescription medication called Rituxan and chemotherapy, had no impact whatsoever on her disease.

Several months ago, Atara Biotherapeutics commenced Phase III studies on its immunotherapy, the final step before seeking FDA approval, for patients with PTLD at medical centers across the United States.

The company has received a special status from the FDA called breakthrough therapy designation, which is granted when early clinical evidence illustrates a product may have a substantial improvement over other treatment options for patients with serious conditions whose desperate needs are unmet. The designation opens up a regulatory mechanism with the FDA to accelerate the development and potential approval of Atara Biotherapeutics’ immunotherapy.

Dr. Susan Prockop of Memorial Sloan-Kettering Cancer Center in New York City, with who I had the privilege of speaking with at length regarding Marisa’s case in the days before she died, will make an oral presentation during next month’s national conference in Houston on the efficacy of EBV-specific T-cells – such as those Atara Biotherapeutics manufactures – for PTLD with central nervous system involvement.

Debbie Drell, director of membership at the National Organization for Rare Disorders, will provide a brief overview of how other rare disease communities have united, perhaps inspiring this newly formed group of PTLD patients and caregivers in Houston to take similar action. ASBMT President John DiPersio of the Washington University School of Medicine’s Division of Oncology will also participate for part of the meeting, illustrating the powerful connection between the PTLD patient community and physicians/scientists who are working on developing new therapies.

During the day-long meeting, patients and their families will have an opportunity to describe their experience with PTLD, to evaluate a new tool to measure patient-reported outcomes, to discuss ways to “build community” with others PTLD has impacted, and to address gaps in education and support.

Cyndi and I will add to that list our opinions that a spinal tap be required as a standard evaluation of PTLD, that hospitals be vigilant of PTLD when transplanting cardiac patients with preexisting diseases of the lymphatic system, that medical experts be reminded that MRIs are more conclusive than CT scans, that oncologists and neurologists scrutinize scans a radiologist reviews, and that medical teams be well-trained in identifying neurological issues that can help identify the spread of PTLD to the brain.

According to the itinerary Cyndi and I received for next month’s PTLD patient and caregiver advisory board meeting, a professional moderator will guide the discussion, “to ensure everyone’s voice is heard.”

An insidious disease may have claimed my beautiful daughter’s life, but it cannot silence her powerful voice.