This year marks the 18th anniversary of my ongoing treatment as a single patient using an investigational drug on a compassionate basis. It sounds like a mouthful, but essentially, I've been using a medication that's still being studied and not even specifically for the disease I have. I was given permission to use this drug, and for nearly two decades, I've been the only patient in this clinical trial.
In 2008, I was seven years old and given just six months to live. The diagnosis: Ewing's Sarcoma, a rare and aggressive cancer found in bone or soft tissue. By the time the doctors found it, my prognosis was grim. They amputated my foot to try and slow the cancer, but even that wasn't enough, it had already spread to my lungs. I was put on chemotherapy, and my health continued to decline. I was dying.
Chemotherapy was brutal. It felt like my body was breaking down, and I didn't know how much longer I could endure it. My parents, especially my dad, refused to give up. He made it his mission to find a way to save my life, connecting with everyone he knew in the healthcare world. He reached out to senior people at the National Institutes of Health (NIH) and beyond, desperately searching for any option to help me.
Then came Niven Narain, PhD, a name my dad had heard a few years earlier. Dr. Narain had been working on an investigational drug, BPM31510, for a completely different condition: epidermolysis bullosa (EB), a rare skin disease. Back then, the drug was a topical cream, not even intended for cancer. But the science behind it showed promise for treating aggressive cancers like mine. That said, finding the medicine was only the first challenge.
Another major hurdle we faced was with the hospital. My dad was fighting for something beyond the standard treatment options, but it's not uncommon for hospitals to stick with what they know. Many desperate families come forward with unproven remedies, and we were grouped in with them—just another set of parents believing in a miracle cure.
The next challenge was the FDA. My dad applied for a "single patient Investigational New Drug" (IND) to allow me access to BPM31510. Incredibly, within 45 minutes of sending the paperwork, we got approval. It was a rare moment of good news in what had been an otherwise terrible year. The hospital doctors were shocked, but they didn't question it. They allowed me to start the treatment right away, alongside my chemotherapy.
The results were immediate. I started feeling better the very next day. Chemotherapy, which had been unbearable, suddenly wasn't so hard on me. I'll never forget telling my mom I was hungry—something I hadn't felt in a long time—and seeing happy tears rolling down her face. My white blood cell count began to recover, and I finally started to feel like myself again.
But just when we thought things were turning around, the FDA called back. They said they had made a mistake. My approval had been an error, and they wanted to retract it. My dad, seeing how well I was doing, refused to stop the treatment. After a series of meetings between the FDA, my doctors, and Dr. Narain, the FDA ultimately allowed me to continue. From that moment on, I knew I had a fighting chance to live.
It's been 17 years since that moment. I'm now cancer-free but I'm still using BPM31510 to help prevent a recurrence. What started as a topical cream is now also available in IV form and has shown promise as a treatment for several other cancers. BPGbio, the company that now owns BPM31510, is testing it in clinical trials for glioblastoma, a rare aggressive brain cancer, and pancreatic cancer.
Every year, my IND must be renewed to continue my treatment, and finding a doctor to sponsor it as I age out of pediatric care has been a new challenge. But I'm grateful to have access to the drug that saved my life.
Looking back, it's clear how many barriers my family had to overcome. The medical system, as it stands, is not designed to make access to investigational treatments easy, especially not for rare diseases. The IND process is incredibly complex and requires an advocate—someone who knows the right people, understands the system, and is willing to fight every step of the way. I was lucky to have my dad as that advocate, but it shouldn't be this hard for other families.
Thankfully, there are programs now, like the FDA's Project Facilitate, which aims to help oncologists navigate the single-patient IND process. There's also the Right to Try Act, which gives terminally ill patients another pathway to access investigational drugs. These programs are steps in the right direction, but there's still much more to be done to ensure everyone can access life-saving treatments without jumping through endless hoops.
I will always be grateful to my parents, Dr. Narain and his company BPGbio, my oncologist, my pediatrician, and everyone who worked so hard to give me this second chance at life. Their dedication and perseverance made all the difference. I hope that in the future, families won't have to fight as hard as mine did. Every patient facing a terminal illness should have access to the best possible treatments, and we need to find ways to make that access easier for everyone.
Chase Malackowski was diagnosed with Ewing's Sarcoma at eight years old. After trialing a "single patient Investigational New Drug" (IND) which allowed him access to BPM31510, Chase has been cancer-free for 17 years.
All views expressed are the author's own.
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