I Will Survive: Beating the Odds with Clinical Trials

When faced with life-threatening illness we all want effective treatment, but very few look for it outside of standard therapies. Participating in clinical trials offers an opportunity to get exposed to cutting-edge technologies that might prove life-saving.



When Jack Whelan was first diagnosed with a lymphoplasmacytic lymphoma called Waldenstrom’s macroglobulinemia, a rare form of blood cancer, he was not impressed with the prognosis offered by the standard treatment which at that time indicated an average 5-year outlook for life expectancy. “As this disease is not curable, rather than relying on chemotherapies borrowed from other blood cancers I wanted a more targeted therapy, something that would target the disease on a molecular level, so I began a series of clinical trials (CTs),” Whelan recalls.

Myths and Misconceptions

Whelan believes there are too many myths and misconceptions about CTs. “The popular myths that you’re a guinea pig, that your insurance company won’t pay for treatment, or you’re getting  a placebo (sugar pill) rather than receiving actual cancer treatmentare simply not true.  CTs are well-managed and for many blood cancers, they can be safer than conventional chemotherapy.” But there’s always the fear of the unknown. “Whether you’re participating in a Phase I or II CT, you’re a part of a relatively small population, subjected to real-world risks. Patients should ask – what are the side-effects, what are the unknown effects?".

“Having recently achieved progression-free survival, I feel I definitely made the right decision; I’ve beaten that five-year mark! It was the right thing to do.”

If you’re going to take chemotherapy, find out what’s in the pipeline, because there is a good chance what’s in development might be safer than conventional therapies".

Whelan, a very active research advocate and event speaker urges all patients to explore what’s in clinical trials. “If you’re going to take chemotherapy, find out what’s in the pipeline, because there is a good chance what’s in development might be safer than conventional therapies.” He admits, however, that the current healthcare system is not designed to encourage people to participate, saying that primary care physicians often don’t know about what new therapeutics are in clinical trials and they don’t ask their patients if they’re interested in participating in a CT. “Studies show that many more people would explore this treatment option if they were asked,” he says.

Communications Gap

According to the Center for Information & Study on Clinical Research Participationabout 2% of the U.S. population gets involved with clinical research trials each year, and among people who suffer from severe, chronic illnesses, only 6% participate. As a result, an increasing number of trials are delayed because too few people even knew they had an opportunity to get involved. According to Whelan, the problem is a communications gap.

The tight regulations that apply to biopharma mean that they don’t have the opportunity to get their information and messages across easily. Concerns over following the rules mean that the industry struggles with relaying information about clinical trials. “The primary source of information about CTs in the U.S. is ClinicalTrials.gov, and in Europe it’s www.ClinicalTrialsRegister.eu but the information you find there has been written by principal investigators who prepare the structure and content of the proposed clinical trial to help enable funding approval.

So, it’s no surprise that these documents are not patient-centric. In hematology oncology, the language is very precise but for patients, the terminology is complex.   They’re not written with the assumption that patients will be reading about the study.  “I think if pharma could get more involved supplying more patient information and education about their CTs that would be helpful,” he reveals.

To navigate the complex reality of CT, Whelan, like many patients, used “Dr. Google”, but as an enthusiast of research, he went far beyond that, reading  published scientific abstracts,  joining a few leading organizations such as AACR, DIA and ASCO – excellent information resources for researchers. 

He cautions other patients that take what he calls, the “carwash approach.” “When you go to a carwash, you just sit passively, obediently and you don’t participate”.  He suggests patients should participate and be proactive in their care; start learning more by seeking the support of advocacy groups and charitable organizations that focus on your particular disease of which there are many.

Personalized medicine

Whelan believes that the Personalized Medicine also known as Precision Medicine approach to managing his disease is helping to beat the odds thus far.  We are gradually moving away from the empirical approach (try one drug after another until you find what works) to the genomic approach, a more specific targeted approach that inhibits proteins and blocks certain pathways important to the proliferation of cancer cells or that induce cell death.  

He says the road to Personalized Medicine is still “under construction.”  We’ve not yet achieved prescribing “the right pill for the right patient at the right time”.  “We’re close, we have identified many appropriate targets (genomic abnormalities) and we’re learning how to take out those targets every day”.

“Unfortunately, we’re still very simplistic about the way we describe cancer.  We often name it based on where the cancer was first discovered during an exam.  For example, we call it breast cancer or colorectal cancer, but perhaps it’s more precise to describe it by the genetic mutation that causes the development of a cancer cell.  In this example BRCA1, BRCA2 or HER2 positive biomarker indicates what therapy might be appropriate”.

A HER2-positive breast cancer tests positive for a protein called human epidermal growth factor receptor 2 (HER2), which promotes the growth of cancer cells. A gene mutation makes an excess of HER2 found in many types of cancer — not only breast cancer. If we can target and shut down the gene mutation activity, researchers believe we can shut down the development of these specific cancer cells.

Although we don’t understand the downstream risks associated with blocking or inhibiting these targets, Whelan believes targeting at the molecular level, perhaps the DNA in nucleus of a cell could be safer and more effective than targeting whole cells. 

According to Whelan, precision medicine is the key to more cost-effective healthcare. “First, it’s the most effective for the patient, as it helps identify and fix the disease sooner thus fewer courses of other treatment and risks of side effects.  Second, for providers, it costs less because patients get through treatment sooner, rather than taking the ‘trial and error’ approach.”

Adherence matters

They gave you a log and asked you to note the date and time you took the medication.  This in itself is a good reminder.  They then count the number of pills left at the end of the month, so you know how many days out of a 30-day cycle you were on target".

But even targeted treatments will fail if patients don’t follow doctor’s orders. Patient education about the importance of taking the right dose of medication, and taking it on schedule, is one of the ways of addressing problems with patient compliance and adherence. Whelan shares his experience where the biotech firm and the principal investigator did it effectively: “They gave you a log and asked you to note the date and time you took the medication. This in itself is a good reminder. They then count the number of pills left at the end of the month, so you know how many days out of a 30-day cycle you were on target.”

The method of administering medication is very important for adherence. When a patient is scheduled for infusion chemotherapy, we can achieve 100% adherence. However as new chemotherapies are now being developed in pill form rather than an infusion, patient compliance is not so automatic. Some patients that are not in the habit or those that can’t afford the high co-insurance will often skip a few days. “There are a number of emerging mobile device software apps, that might encourage more compliance (reminders, logging, care plan, schedule, and notes) but the greatest number of cancer patients come from the older generation, many of whom are not mobile-device savvy.  So patient education and active participation is more important than ever before”, Whelan concludes.

Clearly a lot more needs to be done in the name of involving patients more fully in their treatment and engendering a generation of informed and actively healthy patients.


Jack Whelan will be speaking at The Patients Summit in London, 17-18 June 2014. For more information about him and other speakers, click here

See Whelan’s advocacy site at www.Jack-Whelan.com


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