At the end of every year, we sit down with our physicians and scientists for their take on how cancer care will evolve in the year ahead. Our conversations are collected in a series we call, Advances.
* * *
Great strides have been made in pediatric cancer and childhood disease treatment over the past few decades, and recent advances in the areas of gene therapy, immunotherapy and targeted therapy will continue to move the field forward in 2017.
We spoke to Joseph Rosenthal, M.D., M.H.C.M., the Barron Hilton Chair in Pediatrics at City of Hope, about the most significant developments he anticipates in pediatric research and treatment.
1. What treatment advances do you expect in 2017?
For sickle cell disease — a group of hereditary disorders that cause red blood cells to become sickle shaped — gene therapy presents an innovative treatment option that doctors are working on expanding. But until gene therapy is more commonplace, the only curative treatment for sickle cell is hematopoietic stem cell transplantation.
“Over the past few years, City of Hope has seen an increase in patients coming for sickle cell disease treatment,” Rosenthal said. “We are actively involved in expanding the possible resources for hematopoietic stem cell transplantation for these patients.”
There are also two new protocols in the works involving the use of half-matched related donors. Preliminary results are promising, and researchers hope to bring them to the clinical arena within the next few months, added Rosenthal.
In the area of pediatric cancer treatment, Rosenthal sees several advances on the horizon, including bridging the gap between outcomes in children and adolescents.
"There is a significant disparity between outcomes in children as compared to adolescents and young adults," said Rosenthal. "We see an increased opportunity to bring the advances that occur in children into the adolescent and young adult age group."
He also expects improvements to be made in the long-term health of pediatric cancer survivors.
“As we have more and more survivors of cancer and cancer treatment living longer, we are faced with a host of other conditions that occur as a result of cancer treatment. We will see increased awareness of the long-term effects of pediatric cancer treatment, and what can be done to mitigate those.”
Finally, Rosenthal anticipates continued progress in immunotherapy, most notably with CAR T cell therapy — the use of engineered immune cells designed to identify, attack and eliminate cancer cells.
2. How significant are these advances to the overall field?
The growth of immunotherapy is one of the most important changes that has occurred over the past two or three years, said Rosenthal.
“Diseases that were believed to be untreatable just a short time ago are starting to move more and more toward the treatable bracket,” said Rosenthal.
“In this arena in particular, CAR T cell therapy is taking a front seat,” said Rosenthal. “It is now quite established in the treatment of leukemia, but we see that expanding into other areas, including our own effort in brain tumors.”
3. How will this improve the patient experience or patient outcomes?
“In pediatrics, the advances over the last few decades are remarkable," said Rosenthal. "We went from zero survival in the early 1950s to close to 70-80 percent of cure in the second decade of the 21st century.
“Our first goal is to get as close to a 100 percent cure rate as possible. Our next goal is create treatments with minimal long-term toxicity. Both of these goals will be served by our treatment advances."
4. What research progress do you expect to see in 2017?
“At City of Hope we have great scientists working on immunotherapy,” said Rosenthal. “I expect that both arms of immunotherapy — cell therapies and small molecule therapies — will move forward.
“I also see hematopoietic stem cell transplantation, which is a primary concern at City of Hope, becoming a less-toxic treatment with similar or better long-term effects,” Rosenthal added.
5. Overall, where is the field of cancer treatment and research moving in your specialty?
Targeted therapy — the use of medications to identify and eradicate cancer cells using their genetic properties — is one of the most promising treatment options for cancer patients.
“The movement in oncology in general, including pediatrics, is to provide more personalized treatments,” Rosenthal noted. “In the future, we will move toward defining a cancer by its molecular profile rather than the organ it involves.
“We can identify targets in the tissue on the molecular level, which is way deeper, way more in-depth than ever before,” said Rosenthal. “We are not there yet, but I can see that we will look to combination treatment with targeted therapies.”
The City of Hope Childhood Cancer Survivorship Program is spearheading a new Southern California consortium focused on filling a serious gap in health care that occurs when children who survived cancer become adults.