I’ve been President and CEO of BIO for more than a dozen years, and I’ve been waiting for a long time to write a blog like this one. It’s a story of hope and perseverance that has everything – tragedy, mystery, adorable children and loving families, and a take-your-breath-away happy ending. It’s the realization of the dream of gene therapy – a new form of “living therapy” with the power to save lives thought to be all but lost to leukemia.
Last Wednesday, experts at the U.S. Food and Drug Administration (FDA) convened to hear evidence about a new kind of cancer breakthrough from Novartis that can cure children and young adults with an intractable, debilitating, life-shortening and heretofore untreatable cancer.
Acute lymphoblastic leukemia (ALL) is the most common childhood cancer in the United States, accounting for one in four cases. Childhood ALL causes the body to produce too many immature white blood cells, crowding out mature and fully functioning white blood cells, which can lead to serious infection. Childhood ALL is rare, but those with the disease struggle terribly with frequent infections, pain, weakness, and a rash of other serious symptoms that lead to death if untreated.
Currently, ALL can be treated with chemotherapy, targeted anti-tumor therapy, radiation, and stem cell or bone marrow transplants. Biotech researchers have made great progress in the fight against childhood ALL; current treatments help 90 percent of young patients go into remission. But that leaves 10 percent who don’t respond and die young.
Their prognosis could soon change dramatically, thanks to the recommendation of the FDA Oncologic Drugs Advisory Committee (ODAC) on Wednesday that the agency approve the first-ever gene therapy for use by patients in the United States.
Chimeric Antigen Receptor T-cell (CAR-T) gene therapy takes a type of white blood cell called T cells and genetically alters the cells to do what fully functional immune cells should do: fight off invaders. An individual patient’s cells are frozen, sent to a laboratory and modified by adding a chimeric antigen receptor (CAR), which reprograms the cells to work as part of the body’s infection-fighting immune system. The patient’s modified cells are then grown in the lab, multiplied and returned to the patient by infusion. Immediately, the altered T cells get to work finding and de-activating cancer cells that have invaded the body.
The CAR-T breakthrough has been shown to be effective in more than 80 percent of the ALL patients tested, many of them children. But as is often the case with miracles, there can be a catch: in this case, serious side effects that, if not properly addressed, can hasten the patient’s death. This was discussed extensively at the FDA advisory committee meeting, and the experts agreed that health care providers are in a position to potentially mitigate or avoid these side effects with proper training. In the end, they agreed that the benefits of the new therapy outweigh the risks. This milestone green light for biotech innovation was delivered by a unanimous 10-0 vote.
Any family member who has had a loved one touched by cancer – or any American who has seen the images of children being treated at hospitals like St. Jude or M.D. Anderson – knows there are heart-breaking, innocent faces behind the cancer statistics.
The FDA committee heard one such story at their meeting. Emily Whitehead, now 12, became the first pediatric patient to receive the CAR-T cell therapy five years ago. She was near death when she began the course of treatment. Everything had been tried; nothing had worked. The experimental drug was the only option left. Her father spoke at the FDA committee meeting and recounted that the medicine was her only hope, and without hope, they had nothing.
Emily had a severe adverse reaction to the therapy, but she was treated for it and survived. Actually, she did a lot more than that; she thrived. Her cancer was eradicated and remains gone today. There she sat last Wednesday, an angel – happy and smiling – as her father urged the committee to let other kids experience the miracle Emily had.
At the 2017 BIO International Convention last month in San Diego, our theme was “breakthrough,” and our message was that every seismic medical advance begins with one breakthrough – one patient, one treatment, one family’s hope that they will beat the odds. When the story of CAR-T gene therapy is written, we will always remember that Emily was the first.
Our hope now is that the Novartis breakthrough will be the first in a coming wave of approvals for personalized medicines customized to help patients beat disease using their own modified cells. This is why I love being part of the biotechnology community. Each small advance builds on the next until we accomplish something that few had dared to dream was possible. If, as expected, the FDA grants final approval for the Novartis treatment, it will be the first gene therapy ever approved for commercial use in our country. But it won’t be the last.
Today, CAR-T therapy is seen as a route to healing for young people tragically affected by a rare leukemia unresponsive to available treatments. Tomorrow, who knows? Already, there are investigations under way to determine if this method can be used to treat other kinds of cancer in different patient populations. Once again, we find ourselves standing at the brink of discovery. And even if we fail, we learn. Then, we try again applying those learnings. This is how the CAR-T cellular method was discovered, and it’s how it will be perfected and broadened in its applications.
Congratulations to every scientist, researcher, business person, medical professional and clinical trial volunteer who played a role in the development of this therapy. On behalf of desperate patients around the world, thank you. There’s no telling how far this one breakthrough will take us, but I have great confidence that Emily will be the first – not the last – to be given the greatest gift because of our industry’s tireless work on this breakthrough.