Ultimate Test

Clinical trials: When are they viable option for cancer patients?

By Katie Coleman

Clinical trials can be a viable treatment option for cancer patients from the start of their diagnosis.

However, many patients and their loved ones don’t always know about them, how they work and what resources are available to help select the best treatment — whether that’s standard treatment or a clinical.

The Leukemia & Lymphoma Society, which funds blood cancer research, provides education, financial resources and treatment access for blood cancer patients, broadcasts an educational webinar titled, “Clinical trials for blood cancers — How can participating in a clinical trial help me?” to explain clinical trials and questions you need to ask before selecting a treatment option for blood cancer patients.

Keith Rohleder, a former Buffalo resident with a rare and aggressive form of blood cancer — blastic plasmacytoid dendritic cell neoplasm — recently completed a clinical trial and is among the panel of speakers that participated in the webinar to help spread awareness and share his personal story.

BPDCN decreases red and white blood cells and platelet counts in the body and can evolve to the bone marrow and spleen. Symptoms are widespread, making the disease hard to diagnose, and include skin lesions, enlarged lymph nodes, fatigue, and stomach pain. There is no established treatment or cure, and that is why Rohleder’s most viable option was the clinical trial SL-401, a biological targeted therapy that has shown antitumor effects.

“When we heard about the clinical trial, I was ready to go right off the bat,” Rohleder said. “I was in phase three of the clinical trial. It’s been sent to the Federal Drug Administration and is supposed to be approved in 2018.”

Rohleder learned about the trial after Tricia Rohleder, his wife and advocate, called The Leukemia & Lymphoma Society’s Clinical Trial Support Center. Its staff is a team of nurses that specializes in blood cancers and assists patients and their loved ones throughout the clinical trial process.

Director Alissa Gentile has guided the Rohleders with information on BPDCN, clinical trial options for BPDCN, which had the highest potential for success, and referred Rohleder to the BPDCN Center at The Dana Farber Cancer Institute in Boston, Mass.

Rohleder is now working through the aftermath and side effects of his treatment. When he was first diagnosed, he was given 12 to 22 months to live, and he has surpassed that at 24 months to date.

“If we would have gone with standard treatment, there’s a really good possibility Keith would either be gone or they may have just bought him another year to live,” Tricia Rohleder said. “It just didn’t make sense to choose a standard treatment option.”

Parlaying medical discoveries

Clinical trials usually start with a scientific discovery that leads to a new or improved treatment option through research and drug development by doctors.

After the drug or improved treatment is developed, pre-patient clinical studies are conducted and are submitted as a proposal to the FDA for review. If the FDA approves the process of studying the particular drug in a clinical trial, then the four phases of a clinical trial are outlined and doctors can begin administering to participating patients.

BPDCN Center Director John Leonard, associate dean for clinical research at Weill Cornell Medicine and New York-Presbyterian Hospital, administered Rohleder’s treatment and also participated in the webinar to explain the entire clinical trial process.

“There’s a great deal of review before any drug or compound makes it to a patient in a clinical trial,” Leonard said. “Initially, there’s a scientific discovery that leads to a group of researchers saying, ‘Perhaps if we target this gene, pathway or molecule we can affect the disease.’ A drug, chemical or compound is developed and studied in a laboratory, and after a number of pre-clinical studies, the investigators assemble a package of information and submit it to the FDA. Once the FDA reviews the information, they may approve the process of studying the drug in a carefully designed clinical trial.”

According to Leonard, it’s important to know which phase of a clinical trial you would be entering and what that means before deciding to participate.

“When I talk to patients about clinical trials, I really want them to understand the goal of the trial, phase of the trial, and what we already know about the drug. It’s important you learn about all your treatment options — standard treatments and those that might be available in clinical trials — and ask questions until you have all the answers,” Leonard said.

Here is a breakdown of clinical trial phases:

— Phase I: Researchers administer an experimental drug or treatment for the first time to a small number of people. Its safety, dosage range and side effects are evaluated.

— Phase II: The experimental drug or treatment is given to a larger pool of people to see if it is effective and to continue evaluating its safety.

— Phase III: The drug or treatment is given to a large number of people. Research confirms its effectiveness, side effects, its safety requirements and they compare it to commonly used treatments.

— Phase IV: Post-treatment studies are conducted after the FDA approves the new drug or treatment, and additional information on the drug’s risks, benefits, and best practices is collected.

With such careful monitoring throughout the process of Rohleder’s SL-401 treatment, he experienced a high level of care that, according to Leonard, is typically seen in clinical trials.

“There’s data that suggests the care provided as part of a clinical trial is actually better than what you receive in routine care, and that’s not because routine care is bad,” he said. “It’s because the extra layers of monitoring, of personnel keeping tabs on everything, and the safety measures built into it lead to more people involved in one’s care and therefore closer observation.”

The Rohleders recommend that following a cancer diagnosis, patients and their caregivers find an advocate they can trust as soon as possible to help them navigate the entire process.

“I’m very happy I had an advocate, my wife,” Rohleder said. “There’s so many times when you need an advocate due to the fact that you’re on this pharmaceutical or that pharmaceutical, or they need to know about billing. Having an advocate relieves some of the stress for the patient.”

To view The Leukemia & Lymphoma Society’s webinar, go to www.lls.org/patient-education-webcasts/clinical-trials-for-blood-cancers.