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From: greenvilleonline.com
Rhonda McNeely will never forget that November day she had to tell her children she had advanced cancer.
“When you have to come home and tell your daughters that you have cancer,” she said, “the looks in their eyes … it was a very sobering thing for all of us.”
If there was any ray of light in the darkness, it was that the Easley woman had access to a clinical trial of a novel cancer therapy. A little over a year later, she’s in remission.
“I finished up (treatment) on March 31, and I’ve been in remission since last August,” a euphoric McNeely told The Greenville News. “It is miraculous.”
McNeely’s treatment was made possible through a breakthrough therapy program at Bon Secours St. Francis Health System.
The treatments being studied have shown some effectiveness in early trials, so the FDA has determined that they are suitable for fast-tracking, said Dr. Hal Crosswell, director of clinical research for St. Francis.
The idea is to bring clinical trials of these “breakthrough” therapies to more people, and closer to home so patients can be near their families during treatment instead of having to travel often great distances or go without, he said. It also expands the pool of patients for these trials, he said.
Typically, these patients have very aggressive cancers, rare malignancies, cancer for which there are few drugs, difficulty tolerating other treatments, or have exhausted the available therapies, he said.
“It’s our belief that everybody should be offered a clinical trial if there’s one available … and it makes sense for them,” he said, “because typically the advances that come, come by way of clinical trials.”
‘Scares you to the roots’
It was November 2012 when McNeely, now 48, found a lump on her neck. The doctor ordered some tests, and while she was waiting for the results, her spleen enlarged to three times its normal size.
A few more tests confirmed that she had Stage 4 follicular lymphoma, a type of non-Hodgkin’s lymphoma.
“It was in all the lymph nodes, the spleen and bone marrow,” she said. “I had never given a thought to having cancer before. And it scares you to the roots of your being.”
But then she learned she was eligible for a clinical trial of a new therapy that held some promise.
“I wanted to do the study because I’m not the only person who is going to have this illness,” she said. “And if they used the drugs on me and have successful results, then someone else will have successful results down the road.”
The former teacher also was glad she could be close to home for the treatment so her family could be with her.
“It’s great that they can do research studies here and help people,” she said. “I wish there was a research study for every kind of cancer there is.”
But most research is highly prescribed and takes a lot of time, and that means patients can die waiting, Crosswell said.
The breakthrough designation came in 2012 as part of the FDA Safety and Innovation Act, which was spurred by the efforts of patient advocates, he said.
“Traditional drug development is very rigid and defined,” he said. “That’s good for protection of human subjects and patient rights, but it’s also a hindrance in that the regulatory process takes too long.”
Accelerated track
The research is still rigorously regulated and monitored for patient safety, study design and other factors, but the FDA works more closely with developers to get the drugs into clinical trials and the data evaluated faster, he said.
The FDA reports that nearly half the 27 novel drugs it approved last year were part of an expedited drug development and review process with review times as short as 4½ months.
And by last month, 186 requests for breakthrough designation had been filed with the agency, with 48 granted.
The program also allows for approval of a treatment based on early response, instead of overall survival, which has traditionally been considered the standard, Crosswell said.
Some of the treatments are not classic chemotherapy agents, which can have some terrible side effects, he said. Some are less toxic and operate by harnessing the patient’s own immune system to attack the cancer, he said.
“We’re understanding biology better, understanding … that the immune system is basically avoided by cancers,” he said. “And many of these agents are able to overcome that.”
But Dr. Arthur Caplan, head of the division of medical ethics at New York University Langone Medical Center, cautions that there are no guarantees with breakthrough drugs and patients need to know that up front.
“When people see it’s a ‘breakthrough,’ they think it must be a cure. But it usually doesn’t mean it’s proven to work, it means there’s enough evidence that we think there’s a probability that something useful might happen,” he told The News. “And something useful doesn’t mean you get cured, but that you may live for two more months, or have a little less pain.”
Nonetheless, Caplan said, if patients go into trials with their eyes open to all the ramifications, including the possibility that they could get worse and even die sooner, it’s not the job of ethicists or regulators to stand in their way. And Americans believe that opening the door to experimental drugs for the dying and the desperate is the moral thing to do, he said.
Offering hope
“Research is hard, and advances move incrementally. And there are a lot of promising things that don’t pan out,” he said. “It’s hard to explain that to people, and moreover, a lot of people don’t want to hear it. They want hope. I respect their right to be hopeful.”
Greenville Health System has offered clinical trials since 2004 and breakthrough therapies since 2012, officials said.
Meanwhile, the Oncology Research Program at St. Francis also is working to advance trials in people between 15 and 40 because their survival has been declining over the past 30 years or so, Crosswell said.
“One of the major reasons is their clinical trial enrollment is less,” he said. “So advances in novel therapies … are not realized in that population.”
There’s a smaller pool of adolescent and young adult patients to begin with, he said, so research typically isn’t geared toward them. And they have unique biologies and cancers that aren’t often studied, he said. The new approach, he said, offers them some hope.
Research enrollment is highest among those who are in their 50s and 60s, and also in pediatric patients, he said, hence, more therapies are available to those groups.
Since her treatment ended, McNeely has been having tests at regular intervals, and all the results have come up clear.
Now, she said, she’s looking forward to seeing her daughters, who are 18 and 17, grow up, graduate from college and have children of their own — all the possibilities she might have missed.
“I just feel blessed. I’ve claimed all along that cancer’s not going to kill me. I have too much to live for,” she said. “I’m going to enjoy my life. I appreciate it a whole lot more now.”
Barbara Jacoby is an award winning blogger that has contributed her writings to multiple online publications that have touched readers worldwide.