Cancer vaccine with minimal side effects nearing Phase 3 clinical trials

Dr. Thomas Wagner, founder of the biotech company Orbis Health Solutions and cancer researcher, has made it his life's mission to find a way to treat cancer without the dreaded side effects that, for some, can become worse than the cancer itself or may even lead to an earlier death.

"The tragedy of cancer is not just that person, the diagnosis, but it's also the fear of the therapy," Wagner told ABC News.

Many traditional cancer treatments, such as chemotherapy, work by killing off cancer cells but also kill off non-cancerous cells throughout the body. This can cause a range of side effects including hair loss, nausea, vomiting, or may knock out a person's immune system putting them at risk of life-threatening infections, Wagner said.

After seeing cancer patients suffer from debilitating side effects of their treatment, Wagner began his mission to develop a cancer treatment that harnessed the power of a person's immune system instead of eliminating it. This treatment was developed as a vaccine that has now been studied for decades, and each shot is completely personalized to each patient.

Typically, cancer cells evade a person's immune system because it is recognized as that person's cells. Wagner developed a tumor lysate particle only (TLPO) vaccine that uses a person's tumor cells to identify particular parts that are then presented back in the body using the vaccine in a way that can stimulate their immune system to gain the ability to detect these cancer cells like an infection, allowing the immune system to fight the cancer itself.

"People used to ask me the question, 'When will there be a cure for cancer?' And I've been doing this for 60 years and I could never answer that question," Wagner said. "Until recently, until the last three or four or five years."

Wagner believes this type of cancer treatment could be a key to finding the long-awaited cure for cancer, all cancers, if paired with early detection.

What we know about this cancer vaccine so far

Wagner's TLPO cancer vaccine has been tested in hundreds of patients with advanced forms of melanoma in Phase 2 clinical trials.

The most recent data presented at an academic conference showed nearly 95% of people given only the vaccine were still alive three years after starting treatment and 64% were still disease-free. Among the most advanced forms of melanoma, disease-free survival after three years for people with stage III disease was 60% in the vaccine-only group, compared to about 39% in the placebo group. Disease-free survival for those with stage IV disease was about 68% in the vaccine-only group, and zero in the placebo group.

The most common side effects were redness or pain at the injection site, fever and fatigue after the injection – similar to other vaccines that stimulate an immune response.

Dr. Vernon Sondak, cutaneous oncologist at Moffitt Cancer Center who was not involved in the clinical trial but has worked with tumor lysate vaccines throughout his career, told ABC News that these results are promising, but point out that the Phase 2 clinical trials aren't conclusive. A larger Phase 3 clinical trial will have to ultimately validate if this cancer vaccine will truly be a game-changer in the field.

"We've seen over and over again, promising Phase 2 data that didn't turn out to be so promising in Phase 3," Sondak cautions.

Based on this data and other studies, the Food and Drug Administration (FDA) has greenlit Wagner's vaccine to start a Phase 3 clinical trial. It will be a three-year endeavor with a goal to enroll 500 people and is planned to launch sometime this year, Riley Polk, president of Orbis Health Solutions, told WLOS, an ABC News affiliate in Asheville, North Carolina.

Polk said he has been personally impacted by the success of this vaccine after his father went through numerous lung surgeries for cancer over a decade ago but was left with no other treatment options. His father opted to try Wagner's cancer vaccine and lived 10 more years before dying from something unrelated to cancer. "You can tell me a lot of things, but you can't tell me [the vaccine] doesn't work," Polk said.

Financial barriers have prevented progress

Polk said the planned Phase 3 clinical trial is a $100 million project – those funds are often drops in the bucket for larger pharmaceutical companies and those backed by venture capitalists.

For smaller, private companies, securing that level of financial resources is a challenge that Polk said limits their ability to fund more clinical trials that could expand the potential indications for Wagner's cancer vaccine.

To circumvent some of that challenge and get this treatment to more people that can produce more results, Wagner and his team just began what's called a basket trial, a type of clinical trial approved by the FDA that allows the same vaccine that showed success in the melanoma clinical trials to be tested in anyone with a solid tumor who meets certain inclusion criteria. People in this trial need to have a low or minimal tumor burden so most will have already received some type of treatment prior to getting this vaccine, Wagner said.

The first person to receive this vaccine under this basket trial was Catie King, a native of Asheville, North Carolina, who was diagnosed with ovarian cancer six years ago. King said she felt great after the first round of treatment, only experiencing some redness at the injection site, but no other side effects, WLOS reported.

"Now how many people do you know, with a cancer therapy that say they feel better because of the therapy?" Wagner asked, who has numerous anecdotal accounts of other patients echoing King's experience of feeling better, some even energized, after starting treatment with this vaccine.

For King, who is in the farming industry with her husband, the lack of side effects makes a meaningful difference in her life, by not impacting her daily routine at all.

"With this vaccine, there haven't been any hard days," King said, which she noted was not the case when she previously underwent low-dose chemotherapy for her cancer.

Polk said six other people have received the vaccine through the basket trial who have a types of brain, lung and breast cancer. But there have been hundreds of other patients who have received this vaccine or its precursor over the last 20 years.

One of those patients is a woman named Mary Carol Abercrombie who Wagner believes is one of the longest surviving people previously diagnosed with stage 4 melanoma, the most advanced form of the disease. Just before Christmas in 2001, Abercrombie told WLOS she completed a year of cancer treatment with "horrendous" side effects but once the treatment stopped, her cancer advanced. Abercrombie said her doctors told her she only had a few more months to live, telling her to "just enjoy Christmas."

Abercrombie's surgical oncologist at the time was working with Wagner on a cancer vaccine. "Sign me up, 'cause there wasn't anything [else] out there," Abercrombie said, who was just hoping to live long enough to see her son get married that year. Over 20 years have since passed and Abercrombie said her melanoma has never recurred. She not only saw her son get married, but she's watching her four grandchildren grow up.

Before this vaccine can be more widely available to treat people with melanoma, it needs to show success over years in a phase 3 clinical trial and then get final approval by the FDA. Before it can have even broader use, it will need to show success in the basket trial, then move into more specific clinical trials for other indications which will take years and millions of dollars.

Polk said he hopes data from the basket trial will get the attention of larger pharmaceutical companies. Through that type of partnership and with other funding mechanisms, they could eventually do more extensive trials through the FDA.

Dr. Jade A Cobern, MD, MPH, is a board-certified pediatrician, specialized in preventive medicine, and is a fellow of the ABC News Medical Unit. Justin Berger, reporter with WLOS, an ABC News affiliate in Asheville, North Carolina, contributed to this report.