Novel experimental agent is highly active in CLL patients, interim study shows

ScienceDaily (June 3, 2011) — An interim analysis of a phase II clinical trial indicates that a novel experimental agent for chronic lymphocytic leukemia (CLL) is highly active and well tolerated both in patients who are undergoing treatment for the first time and those who have relapsed and are resistant to other therapy.

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The agent, called PCI-32765, is the first drug designed to target Bruton's tyrosine kinase, whose function is essential for CLL-cell survival and proliferation.

Study leader Dr. John C. Byrd, director of the division of hematology at Ohio State University Comprehensive Cancer Center -- Arthur G. James Cancer Hospital and Richard J. Solove Research Institute (OSUCCC -- James) presented the findings June 5 at the 2011 American Society of Clinical Oncology annual meeting in Chicago.

The analysis involved the first 21 cases in the untreated-patient group and the first 27 individuals in the relapsed/refractory-patient group. One patient in each group had a complete remission, and 13 patients (62 percent) in the previously untreated group and 12 patients (44 percent) in the relapsed group had partial remissions.

"We are excited about these early findings because they suggest that PCI-32765 is a highly active oral therapeutic that produces a high rate of durable remissions -- the remissions last months on end -- with acceptable toxicity in relapsed and refractory CLL," Byrd says.

Complete remission means there is no detectable CLL in anywhere in the body; partial remission means that the individual's disease volume has decreased 50 percent or more in a sustained manner.

"It is exciting to see a drug that was shown to be active in the laboratory translate to clinical benefit for CLL patients," says researcher Dr. Amy Johnson, assistant professor of medicine at the OSUCCC -- James. Johnson co-led the pre-clinical CLL work at Ohio State with Byrd and now coordinates several correlative studies for this clinical trial.

Byrd stresses that the patients show several benefits of the treatment, such as higher platelet counts and hemoglobin levels, and that many report that they feel dramatically better overall with less fatigue, factors that are difficult to measure and report as a number.

"These responses last for many months in part because patients are willing to remain on the drug since the side effects are very tolerable," he notes.

The ongoing phase II clinical trial involves 78 patients with previously untreated or relapsed and refractory CLL or small lymphocytic leukemia. The previously untreated patients were all age 65 or older; individuals in the relapsed group all had two or more earlier treatments followed by recurrent disease.

"These are early findings, so patients with partial remissions could improve to complete remissions with further observation," Byrd says. "Usually patients with highly resistant and refractory CLL would have progressed and possibly died by this time, but 85 percent remain on PCI-32765 and continue to improve."

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Experimental vaccine made from frozen immune cells shows promise for prostate cancer patients

ScienceDaily (June 3, 2011) — Metastatic prostate cancer patients who received an investigational vaccine made from their own frozen immune cells lived 10 months longer than those not treated with it, according to data being presented by researchers from the Kimmel Cancer Center at Jefferson at the 2011 American Society of Clinical Oncology annual meeting in Chicago.

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In an exploratory, multi-institutional analysis, researchers administered the vaccine APC8015F to a group of patients from the control arm of three randomized, Phase 3 clinical trials evaluating sipuleucel-T, a similar, FDA-approved cancer vaccine for metastatic castrate resistant prostate cancer.

APC8015F is made from immune system cells taken from a patient with prostate cancer; however, unlike sipuleucel-T, which is never frozen, APC8015F is cryopreserved at a time before the disease progressed.

Results from the analysis showed that patients treated with APC8015F had improved survival relative to the patients who were not treated in the control arm. Following disease progression, the median survival of patients treated with APC8015F was 20.0 months compared to 9.8 months for control patients.

"The study is important because it suggests that the sipuleucel-T therapy may have extended survival for a longer time than estimated in the clinical trials due to the beneficial effects of the frozen product on some men who initially received the placebo," said Leonard Gomella, M.D., Chair of Urology at Jefferson's Kimmel Cancer Center in Philadelphia. "Further, the clinical activity of the frozen-activated product is maintained."

Post-progression treatment with APC8015F, which is not FDA approved, may have extended survival of subjects, potentially reducing the magnitude of survival difference observed between sipuleucel-T and controls in randomized controlled trials.

Sipuleucel-T is FDA approved under the brand name Provenge to treat men with advanced prostate cancer that is asymptomatic or minimally symptomatic and no longer responding to hormonal therapy.

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Ex-Dallas Maverick survives rare form of leukemia thanks to experimental drug treatment

ScienceDaily (May 19, 2011) — Ray Johnston's goal in three years is for his band to sell out at the 1,600-seat House of Blues in Dallas. In eight years, he wants to pack the 6,400-seat Verizon Theatre in Grand Prairie, and by 2030, to play to tens of thousands of fans at Cowboys Stadium in Arlington.

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Mr. Johnston's unmentioned goal, though, is to live another year after battling leukemia for the past seven. Despite four relapses, the former Dallas Mavericks basketball player is enjoying life as a rising musician in The Ray Johnston Band.

Although he credits God for his recovery, Mr. Johnston also gives thanks to Dr. Robert Collins, director of the Bone Marrow Transplantation/Hematologic Malignancies Program at UT Southwestern Medical Center, and the experimental drug that has killed his rare, stubborn form of cancer called acute promyelocytic leukemia.

"It's got to be inspiring to Dr. Collins to see that I'm alive because I'm supposed to be done by now," Mr. Johnston said of his painful yet rewarding journey, which was chronicled last year in an HDNet series called "Ray Johnston Band: Road Diaries."

Today, Mr. Johnston's self-described "happy rock" Dallas band is working on its second CD and booking about 100 shows a year. "My primary goal is to keep playing," Mr. Johnston said.

Tamibarotene, the drug that's kept the 32-year-old musician alive, is a retinoid drug that induces cancer cells to differentiate into mature cells and eventually die. Available only in clinical trials in the U.S., tamibarotene was sought for Mr. Johnston under a compassionate use protocol since he did not qualify for those studies and other treatments had been exhausted. The drug, approved only in Japan for cancer treatment, is being developed domestically by CytRx.

"It's amazing that he's still doing so well," said Dr. Collins, professor of internal medicine and senior author of a report on this case published online April 11 in the Journal of Clinical Oncology. "This drug happens to be just right for him."

Mr. Johnston's treatment with tamibarotene, which is considered 10 times more potent than all-trans retinoic acid, another retinoid commonly used to treat this type of leukemia, began in December 2009. He took the drug twice daily for 56 days, followed by a two-week break. Since then, Mr. Johnston has been taking the drug 28 days on, 28 off.

On April 18, he returned to UT Southwestern for a positron emission tomography (PET) scan, which showed his cancer had not relapsed for the fifth time.

"The type of acute leukemia he has is very rare. It's usually curable with current therapies, but as it relapses it becomes more resistant and harder to treat," said Dr. Collins.

Gambling on life, and success as a musician, doesn't scare the Alabama native who once dreamed of a professional basketball career. But cancer ended his NBA stint, which began when he won a spot on the Dallas Mavericks summer league roster during a Hoop It Up tournament in 2004.

Mr. Johnston didn't even know he had leukemia until that August, when he injured his leg in a pick-up game. He was diagnosed with compartment syndrome, a condition in which pressure in the muscle builds to dangerous levels. Following surgery, doctors were unable to control bleeding. They soon discovered that Mr. Johnston's body was riddled with advanced leukemia.

His treatment included chemotherapy, surgery and a bone marrow transplant. Mr. Johnston's type of leukemia, which affects soft tissues rather than blood and bone marrow, represents just 5 percent to 8 percent of acute myelogenous leukemia, the most common form of the disease.

Discouraged by failing treatments and the thought of his first bone marrow transplant, he nearly gave up hope. In 2006, a recommendation from a friend led him to Dr. Collins and UT Southwestern.

"When you achieve a life milestone that you're not supposed to, when you hug it out, you can see and feel Dr. Collins drop a tear on your shoulder. That's life. That's real," said Mr. Johnston.

Reality for this cancer survivor also means getting serious about music. Mr. Johnston has a lot of work ahead after launching the six-member band in September 2009. Some of the band's performances raise money for The Ryan Gibson Foundation, a Dallas-based nonprofit dedicated to leukemia research.

One of the songs on the band's first CD, "Sweet Tooth," is Rise & Go, based on a verse in the Bible, Luke 17:19. Mr. Johnston said it sums up "my last seven years."

"Wake up and I go outside, I see my blue sky, I'm happy to be alive … I got a tough upper lip, but a bottom little broken heart. When I look into the sky, I see my Maker's eyes. That's when I know my Plan B for eternity is strong as gold, so I rise and go.

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