Transformative Cystic Fibrosis Treatment Offers Hope to Ukrainian Patients

UKRAINE – A good day for Yaroslava Koshel — one of hundreds of Ukrainians living with cystic fibrosis, or CF — might include a walk in the park or exploring a new corner of her adopted hometown, Lviv. It might mean time with her mother unclouded by illness, quiet hours spent painting or imagining a future she once thought out of reach.

CF is a genetic disease that damages the lungs, digestive system, and other organs. Until the development in 2019 of Trikafta, a U.S.-manufactured drug that significantly improves health outcomes for people with CF, it often meant a life that was both short and difficult.

“I want a profession that sparks something in me, makes my eyes light up — but I haven’t found it just yet, the 23-year-old Yaroslava told Direct Relief in Oct. 2024. She is originally from the war-torn town of Нью-Йорк/New York in eastern Ukraine (a legacy name from the mid-1800s).

A former graphic design student, she also hopes to start a family someday. Now that she’s receiving Trikafta — that feels possible.

“Trikafta has given me the possibility to live for many more years,” said Yaroslava, who began taking the medicine in October 2023 through a CF pilot donation program led by Boston-based pharmaceutical company Vertex, Direct Relief, and three Ukrainian hospitals, including two of the country’s largest children’s hospitals. The program currently operates in 12 countries, including Ukraine, with plans to expand.

Since its approval by the U.S. Food and Drug Administration in 2019, Trikafta has been widely praised for its impact. For Yaroslava, the treatment marked a turning point in a life shaped by loss. Her older brother died of CF at age 20, and as her own weight dropped to 40 kilograms in the months leading up to her 20th birthday, the same outcome seemed inevitable.

“When you are nearing the age when your brother died, you tell yourself how much you haven’t yet done,” recalled Yaroslava, who was first diagnosed with the disease 18 years ago. However, she did not succumb to it, even as life threw other calamities at her family, including losing her father to war and leaving their home for a safer area.

Like many with CF, she experienced persistent symptoms: thick mucus that clogged her lungs and digestive tract, causing frequent infections, breathing problems, and malnutrition. She completed her studies in 2021, but work was out of reach. Even short walks or trips to the store were overwhelming. “Life was gray,” she said.

But just three days after starting Trikafta, the choking sensation began to ease. Her energy returned. She gained weight. She could walk farther and breathe easier.

She credits the doctor who introduced her to the cystic fibrosis donation program and guided her through the careful selection process.

“I love that doctor; she saved my life,” she said.

Taking CF Treatment to a New Level in Ukraine

The CF donation program is one of several rare disease donation programs developed by Direct Relief in partnership with pharmaceutical donors.

“These programs provide eligible patients access to long-term, specialized therapies for rare diseases that otherwise have no viable treatment options,” said Ellen Cho, Senior Director of Specialty Programs at Direct Relief. “Many of these diseases are difficult to diagnose due to limited awareness or lack of access to necessary diagnostic testing.”

Direct Relief’s extensive experience in managing these types of programs, including shipping temperature-sensitive and cold-chain products, has ensured the safe delivery of lifesaving treatments to healthcare facilities in Ukraine and around the world, benefiting patients in need.

Since the early 2000s, the Clinical Centre of Children’s Healthcare in Lviv has become a reputed hub for treating CF with the available resources and knowledge. “It was a very tough time, we received the most serious patients [from across Ukraine] who were at a very difficult stage, all of them children,” said Dr. Roman Kizyma, the hospital’s director.

But the team prides itself on not losing a single patient to the disease in the past six years, added Dr. Lyudmila Bober, director of the CF center. The hospital has steadily built on its reputation, culminating in the provision in 2023 of the first supplies of Trikafta through Direct Relief. “This is a fantastic program, unbelievable help for Ukraine…” said Bober. To further the hospital’s work in this area, Direct Relief has issued a $200,000 grant for the expansion of the pulmonology department to include a dedicated cystic fibrosis center.

Some 470 kilometers (270 miles) to the east is the Okhmatdyt National Specialized Children’s Hospital in Kyiv – Ukraine’s largest pediatric hospital, known nationwide as simply Okhmatdyt. On July 8, 2024, Lyubov Mazurkevich and her nine-year-old daughter Veronika, who had started taking Trikafta just four days earlier, arrived at the hospital for an appointment at the department that treats CF and many other rare diseases, such as neuromuscular disorders and congenital immunodeficiency. Moments later, the hospital was struck by a missile, resulting in two deaths and at least 16 injuries.

“We were sitting on a bench when we heard the explosion and ran,” recalled Lyubov, a nurse who lives with her family in the Kyiv suburb of Irpin.

As with many recipients of Trikafta in Ukraine, the story of Veronika’s health is intertwined with war, which early on had displaced the family to western Ukraine, complicating efforts to manage the child’s condition. When it was safe, they returned to find their home badly damaged. But it was Veronika’s illness that still overshadowed their lives.

“I cried every day, became depressed, asked myself, ‘Why is this happening?’ Then I realized that there is only one way out – to fight,” said Lyubov. That determination led to her daughter’s acceptance into the program and they haven’t looked back since. Veronika, a vivacious, gifted painter and dancer, is now busily winning competition awards while her parents count their blessings: “We now mark July 4 (the day she began taking Trikafta) as a second birthday and we will celebrate it forever,” said Lyubov.

Restored Health Puts Even War into Perspective

The Trikafta donation program at Okhmatdyt includes both young and adult patients. The dramatic improvement in the children’s health and their swift return to almost all daily life activities is a huge turning point also for the parents. Most knew for years about the existence of Trikafta from their online research, but doubted that it would reach Ukraine, at least in wartime. Even so, patient advocates in the country continued to hope that the drug would become available. The announcement of the donation program in 2023 was welcome news during an exceptionally difficult time for Ukraine.

“The parents are crying when they get the medicine for the first time. The patients don’t always understand what is happening, but the parents say, ‘It was our task to live to this day’,” said Dr. Nataliia Samonenko, the head of Okhmatdyt’s orphan diseases department.. “One mother said she slept properly for the first time in 20 years because her child wasn’t coughing up in the night,” added Yulia Ostapyshyna, another member of the orphan diseases and gene therapy team.

The patients’ treatment and return to health can greatly help families carry on in the face of what is happening in Ukraine and help temper the tragedies of war. The Rudnytskyi family, consisting of father Mykola, mother Lyudmila, and sons Bohdan, 26, Yehor, 17, and Hleb, 10, is a case in point. Both boys live with CF, while their older brother is of sound health.

Unlike many, they were fortunate to all survive the war that swept through their hometown of Berdiansk in eastern Ukraine in early 2022. But they abandoned almost everything they owned – Mykola’s agricultural canning business and their home – to escape in March, packing their car with a few bags and their dog. Now they are starting again in Lviv.

Life is challenging, but they are rebuilding. Mykola opened another small business, and since receiving Trikafta in the summer, the boys regained their weight and no longer suffer from the previous acute breathlessness. While continuing daily inhalation and other procedures, both practice sports and lead active lives. A keen boxer, Yehor said he wants to become a dentist, his little brother chiming in, “Me too!”

“People ask us, ‘How can you live on after losing so much?’ But we now see that we have a future,” said Mykola, motioning to the boys. Cupping Hleb’s fingers in hers, Lyudmila said simply: “We now understand that true happiness fits into the palm of your hand.”