To highlight the most important of these studies, the Myeloma Beacon surveyed leading physicians and researchers in the field. These physicians and researchers were asked to name the three peer reviewed journal articles published in 2009 and the three conference abstracts from 2009 that have the most important findings or implications relating to multiple myeloma.

The top three journal articles and conference abstracts that they chose are presented below.

Journal Articles

1: Pomalidomide Shows Remarkable Activity In Myeloma

According to the physicians surveyed, the most important study published in 2009 evaluated a combination of pomalidomide (Actimid, CC-4047) and low-dose dexamethasone (Decadron) in relapsed and refractory myeloma patients. In the study, 63 percent of patients, many of whom had been resistant to thalidomide (Thalomid), Revlimid (lenalidomide), and Velcade (bortezomib), responded favorably to the treatment.

The study confirmed earlier results indicating that pomalidomide is the most potent drug of its kind, a class of molecules, known as immunomodulatory agents, that includes thalidomide and Revlimid.

For more information, please see the Journal of Clinical Oncology (abstract) and the related Beacon news article.

2: MGUS Always Precedes Multiple Myeloma

In second place, a prospective study analyzed the blood samples taken from myeloma patients before their diagnosis. Researchers learned that in nearly every case, the patients had monoclonal gammopathy of undetermined significance (MGUS), a blood disorder present in three percent of Americans over the age of 50.

According to Dr. S. Vincent Rajkumar, a professor of medicine at the Rochester, Minnesota, Mayo Clinic and an author on the paper, the study’s findings settle a long-standing debate on whether MGUS is a consistent predecessor to myeloma. Researchers can now focus on identifying factors that increase the risk of MGUS progression.

“By studying mechanisms that are associated with progression, we will [ultimately] be able to develop earlier therapies that can be used to delay or prevent myeloma from happening,” wrote Dr. Ola Landgren, an investigator at the National Cancer Institute and lead author of the study, in an email to the Beacon.

For more information, see the journal Blood (abstract) and the related Beacon news article.

3: Maintenance Therapy After Transplantation Prolongs Life Expectancy

In a tie for third place is a study evaluating the post-transplantation benefits of either prednisone or a combination of prednisone and thalidomide, for patients treated with thalidomide and an autologous stem cell transplant. Patients in the combination therapy group experienced a longer remission duration and overall survival.

For more information, see the Journal of Clinical Oncology (abstract) and the related Beacon news article.

3: Very Good Partial Response Is A Good Indicator of Long-Term Outcome

The other third place study examined how patient response to treatment was associated with event-free and overall survival. Researchers followed up with patients who had undergone an autologous stem cell transplant for a median of 67 months. They discovered that patients who achieved very good partial response or better had significantly longer event-free and overall survival.

Based on their results, researchers recommended that very good partial response should become the standard treatment goal for patients since it is more attainable than complete response, yet offers similar benefits in event-free and overall survival.

For more information, see the Journal of Clinical Oncology (abstract) and the related Beacon news article.

Conference Abstracts

1: Velcade Treatment Options In Elderly Patients With Newly Diagnosed Myeloma

The surveyed physicians voted as most important a study presented at the American Society of Hematology (ASH) conference in early December in which researchers compared an induction therapy combination of Velcade, thalidomide and prednisone (VTP) with the already-popular regimen of Velcade, melphalan (Alkeran), and prednisone (VMP) in newly diagnosed elderly patients. Both treatments proved to be equally and highly effective.

Researchers also compared maintenance therapies of Velcade-thalidomide (VT) and Velcade-prednisone (VP), both of which improved patient responsiveness to the point of overcoming negative genetic risk factors.

For more information, please see ASH abstract 3 and the corresponding Beacon news article.

2: Revlimid Treatment Options In Elderly Patients With Newly Diagnosed Myeloma

For second place, the surveyed physicians chose a Phase 3 trial presented at the ASH conference that investigated Revlimid’s effectiveness as both an induction and maintenance therapy. Patients treated with a combination of melphalan, prednisone, and Revlimid followed by Revlimid maintenance had a decreased risk of disease progression than patients treated with melphalan and prednisone followed by a placebo.

Final results of the study are expected at the American Society of Clinical Oncology meeting later this year. The study’s findings offer substantial support for Celgene as it applies for FDA approval of Revlimid as a first-line treatment option in multiple myeloma.

For more information, please see ASH abstract 613 and the corresponding Beacon news article.

3: VMPT Effectively Treats Elderly Patients With Newly-Diagnosed Myeloma

In a tie for third place, another ASH conference study investigated the impact of adding thalidomide to the standard regimen of Velcade, melphalan, and prednisone (VMP). Patients treated with VMPT had a greater response rate than those treated with VMP, but they also encountered more blood-related side effects.

“VMPT represents the next generation,” wrote Dr. Antonio Palumbo, chief of the myeloma unit at the University of Torino in Italy and lead author of the study, in an email to the Beacon. “[The four drugs] improve response and progression-free survival with a higher toxicity, but this is [still] the first schema that is superior to VMP.”

For more information, please see ASH abstract 128 and the corresponding Beacon news article.

3: Elotuzumab Combination Produces Encouraging Results In Multiple Myeloma

Also tied for third place is another ASH presentation that provided positive results about the effectiveness of elotuzumab in combination with Revlimid and low-dose dexamethasone at treating multiple myeloma. The combination therapy led to a 92 percent response rate from patients, with manageable side effects.

A new antibody, elotuzumab targets proteins that are unique to myeloma cells and causes the cells to die.

“This is an important study because it represents the first evidence that an antibody can have efficacy in myeloma, and it confirms the suggestion that Revlimid enhances immunity and thus enhances the efficacy of an immune-mediated agent like elotuzomab,” wrote Dr. Sagar Lonial, an associate professor at Emory University’s School of Medicine and lead author of this study, in an email to the Beacon.

A multinational continuation of the trial is currently recruiting patients. For more information, please see the clinical trial description, ASH abstract 432, and the corresponding Beacon news article.

The Myeloma Beacon would like to thank the physicians who participated in the survey for their assistance and expertise:

S. Vincent Rajkumar, M.D.
Mayo Clinic, Rochester, MN

Sundar Jagannath, M.D.
St. Vincent’s Comprehensive Cancer Center, New York, NY

Sagar Lonial, M.D.
Winship Cancer Institute
Emory University School of Medicine, Atlanta, GA

Antonio Palumbo, M.D.
University of Torino, Italy

Jesus F. San Miguel, M.D.
University of Salamanca, Spain

Afinitor Is Safe In Relapsed And Refractory Multiple Myeloma; Drug Will Move To Phase 2 Clinical Trials (ASH 2009) – Afinitor (everolimus) is safe in relapsed and refractory multiple myeloma, according to preliminary results from a Phase 1/2 clinical trial presented at the 2009 Meeting of the American Society of Hematology (ASH). Afinitor was given orally at 5 mg to 10 mg daily. Researchers observed only one case of thrombocytopenia (low platelet count). A sufficient amount of anti-myeloma activity was observed for this drug to continue to Phase 2 testing, which puts more emphasis on the efficacy of the drug. For more information, see abstract 3850 on the ASH Meeting Web site. 

ACE-011 Increases Hemoglobin And Helps Build Bone In Multiple Myeloma Patients Receiving Chemotherapy (ASH 2009) – Preliminary results from a study presented at the 2009 Meeting of the American Society of Hematology (ASH) reveal that ACE-011 increases hemoglobin (an iron-rich protein in red blood cells) and bone formation, decreases bone pain, and has anti-tumor activity in multiple myeloma patients receiving chemotherapy. A low hemoglobin count indicates a low red blood cell count (anemia), which is a common complication in multiple myeloma, as are bone lesions, and bone pain. All participants were on a regimen of melphalan (Alkeran), prednisone, and thalidomide (Thalomid). For more information, see abstract 749 on the ASH Meeting Web site.

Researchers Demonstrate Feasibility And Safety Of Vaccine In Multiple Myeloma (ASH 2009) – Preliminary results from a Phase 1 clinical trial of a multiple myeloma vaccine, presented at the 2009 Meeting of the American Society of Hematology (ASH), indicate that using a vaccine in multiple myeloma is possible and that it is safe and tolerable. The study included 15 patients who had undergone peripheral blood progenitor cell transplantation – a treatment in which stem cells are collected from the bloodstream of a healthy donor and delivered to the myeloma patient after high-dose chemotherapy.  The most frequent side effects were local skin reactions, typical of most vaccines. Further studies will evaluate the vaccine’s full potential. For more information, see abstract 1858 on the ASH Meeting Web site. 

For more information about myeloma-related studies covered at ASH, please see the Myeloma Beacon coverage of the ASH 2009 Meeting.

While VMP without maintenance therapy currently is one of the standard treatments for elderly patients with newly diagnosed myeloma, the availability of new drugs has motivated researchers to search for more effective, less toxic combination treatments. In another study, also presented at the ASH 2009 conference, the addition of thalidomide to the VMP regimen, followed by maintenance therapy, resulted in greater response rates than VMP (see related Beacon news).

In this Phase 3 study, researchers compared VTP and VMP as induction therapies as well as Velcade-thalidomide (VT) and Velcade-prednisone (VP) as maintenance therapies, evaluating both efficacy and side effects of the combination therapies.

Researchers recruited 260 patients and randomly assigned them to either the VTP or VMP treatment group. In the VMP treatment, patients received 1.3 mg/m² of Velcade on days 1, 4, 8, 11, 22, 25, 29, and 32 of one six-week cycle, and then on days 1, 8, 15, and 22 of five five-week cycles. Melphalan, 9 mg/m² and prednisone, 60 mg/m² were given on days 1-4 of each of the six cycles. In the VTP treatment, patients received Velcade and prednisone at the same dosage and schedule, and 100 mg of thalidomide daily.

Maintenance therapy consisted of a three-month cycle of 1.3 mg/m² Velcade given on days 1, 4, 8, and 11, and either 50 mg of thalidomide daily (VT) or 50 mg of prednisone every other day (VP) for up to three years. Patients were randomly assigned to the two treatment options.

Of the 260 patients, 253 were included in the final analysis – 125 in the VMP group and 128 in the VTP group. Both treatments resulted in similar rates of at least partial response or better – 81 percent in the VMP group and 79 percent in the VTP group. Twenty-two percent of VMP patients and 27 percent of VTP patients achieved complete response, for an average of 25 percent.

Researchers did not observe any significant differences between the VMP and VTP groups in progression-free survival rates (71 percent versus 61 percent) or overall survival rates (81 percent versus 84 percent). In each treatment group, two patients experienced disease progression.

Of the 178 patients who participated in maintenance therapy, 143 were included in the final analysis. The addition of maintenance therapy increased the rate of complete response from 25 percent to 42 percent in both treatment groups. Both VT and VP maintenance resulted in similar overall survival rates after one year (92 percent versus 89 percent, respectively).

Out of 27 patients who exhibited high-risk genetic abnormalities, 26 percent achieved complete response after induction and 42 percent after maintenance. Both response rates were similar to the overall patient response, indicating that the maintenance regimens successfully combated the negative prognosis associated with patients’ genes.

Researchers observed differences in side effects between the two induction therapies. More patients in the VMP group than in the VTP group experienced decreased white blood cell counts, or neutropenia (37 percent versus 21 percent), and infection (7 percent versus <1 percent). However, eight VTP patients (5 percent) experienced serious heart problems not observed in any VMP patients. Nine percent of VTP patients also experienced peripheral neuropathy (nerve damage in the limbs), compared to five percent of VMP patients.

In both maintenance therapies, patients experienced few severe side effects. Two patients in the VT group and one patient in the VP group had severe heart problems, and four patients in the VT group and one patient in the VP group had severe gastrointestinal problems.

Researchers concluded that VMP and VTP work equally well as induction therapies. They added that the addition of VT or VP as maintenance therapies to VMP and VTP offer clear benefits by increasing response without compromising side effects..

For more information, see abstract 3 at the ASH Meeting Web site.

Sagent Pharma Launches Generic Alternative to Aredia – Sagent Pharma announced the launch of a generic alternative to Aredia (pamidronate disodium), a treatment for cancer-related osteoporosis marketed by Novartis Pharma. The injection will come in single-dose vials of 30 mg/10 mL and 90 mg/10 mL. For more information, please see the Sagent Pharma press release.

Terpenoid Therapeutics Raises $950,000 For Myeloma-Related Drug Development – Terpenoid Therapeutics has raised $950,000 of its intended $1.05 million for the development of two cancer drugs, one of which will treat bone disease associated with cancers such as multiple myeloma. Researchers anticipate the drug will cause fewer side effects than current treatment options. For more information, please visit the MedCity News Web site.

This study looked at approximately 1,900 multiple myeloma patients who filed claims with a national health insurance company. Researchers looked at out-of-pocket costs for each patient for a period of one year after the start of a new course of treatment.

For the 1,900 patients, researchers identified 2,642 treatment courses. Most treatments fell into the other chemotherapy or radiation therapy category (66 percent), followed by thalidomide (21 percent), Velcade (9 percent), and Revlimid (3 percent).

The total out-of-pocket costs for the year after beginning treatment with Velcade averaged to $3,504, compared to $4,443 for patients being treated with thalidomide, $4,766 for patients receiving Revlimid, and $3,907 for all other myeloma patients.

Researchers also looked specifically at costs for Medicare patients. The difference in out-of-pocket expenses was greater among this group of patients, with Velcade patients paying $4,395, thalidomide patients paying $8,824, Revlimid patients paying $12,568, and patients using other treatments averaging payments of $4,757.

Despite differences in cost, some patients prefer oral drugs (i.e., Revlimid and thalidomide) over intravenous drugs (i.e., Velcade) due to the convenience of not needing to visit a doctor’s office for treatment.

Among the patients studied, however, the number of physician visits (in outpatient or office settings) were similar among the different treatment regimens. Patients on Revlimid made 57 visits during the year after treatment started, while patients on thalidomide made 63 visits, patients on Velcade made 66 visits, and patients on other regimens made 72 visits during the year.

Likewise, there were no differences between treatment groups in number of inpatient hospital stays or emergency room visits.

It is unclear from the discussion in the abstract whether the reported results are likely to be typical for all patients covered by health care plans in the U.S., or if they reflect the policies of the specific insurer whose data were used for the study.

Ultimately, patients should always consult with their physician regarding efficacy, side effects, convenience, and cost of various treatment options.

For more information, please see abstract 1366 on the ASH Meeting Web site.

Dr. Ravi Vij from the University of Washington in St. Louis, co-author of the study, remarked, “The trial showed very high rates of response when elotuzumab is given in combination with Revlimid.”

Elotuzumab is a new drug that is currently being investigated as a potential treatment for multiple myeloma. It targets proteins that are on the surface of myeloma cells, but not healthy cells. When the drug identifies a cancerous cell, it triggers the cell’s death.

The goal of this study was to determine maximum tolerated dose of elotuzumab in combination with Revlmid and low-dose dexamethasone in patients with relapsed multiple myeloma. Researchers also examined the efficacy and toxicity of the drug in patients with relapsed or refractory multiple myeloma.

Patients enrolled in this clinical trial took three different doses (5 mg/kg, 10 mg/kg or 20mg/kg) of elotuzumab in combination with 25 mg of Revlimid, given on days 1-21 in a 28-day cycle, and 40 mg of low-dose dexamethasone, given once weekly.

Researchers observed at least a partial response in 92 percent of the patients who had completed at least two treatment cycles. This means that the drug removed at least half of the patients’ abnormal proteins and any tumors shrunk by at least 50 percent.

They did not observe any dose-limiting toxicities, and the maximum tolerated dose was not established.

Common side effects associated with this drug included neutropenia (low number of white blood cells), thrombocytopenia (low number of platelets), fever, and chills.

“Currently a randomized Phase 2 extension trial is going to look at two different dose levels to see if [the drug] is as effective at a lower dose,” Vij added. “[This year], the plan is to do a multinational Phase 3 trial.”

The study’s authors were optimistic about the efficacy of the drug and encouraged by what they called “manageable” side effects.

For more information, please see abstract 432 on the ASH Meeting Web site.

In the process of treating multiple myeloma, patients often experience a weakening in their immune system which makes them more susceptible to infection. Serious infections can lower a patient’s quality of life, require doctors to reduce treatment to a less toxic but less effective level, and even cause death.

Doctors can treat infection as it occurs or anticipate and prevent it. Prophylaxis refers to any medication used to prevent illness from developing as opposed to fighting it once it has arisen.

In their analysis, researchers examined how effective antibacterial prophylaxes were in preventing infection in patients on various thalidomide combination regimens.

The researchers analyzed the data of 224 patients, 168 of whom received medication to prevent infections. Patients received any one of the following five thalidomide combination regimens: thalidomide, dexamethasone (Decadron), and Doxil (pegylated liposomal doxorubicin); thalidomide, dexamethasone, and Doxil plus Velcade (bortezomib); thalidomide and dexamethasone; thalidomide and dexamethasone plus Velcade; or a combination of Velcade, melphalan, prednisone, and thalidomide. Participants ranged in age from 31 to 90, with a median age of 70.

Of the 224 patients, 86 (38.5 percent) developed infection, and 39 (17.5 percent) experienced severe infections. Severe infections included pneumonia (59 percent), unidentifiable fevers (23 percent), bacteria in the blood (15 percent), and swelling of eye tissue (3 percent). One patient with bacteria in the blood died of septic shock.

Out of the 168 patients given prophylaxis, 15 percent developed infection. In contrast, 25 percent of the 56 patients not given preventative medication experienced infection.

Upon analysis of the patient data, researchers pinpointed factors that were associated with an increased risk of severe infection. These factors included having an increased monoclonal protein (M protein) count (> 2 g/mL of blood) and a decreased platelet count (< 130 cells/mL). Patients with neither of these factors were at a 4 percent chance of developing severe infection. Patients with one factor faced a 17 percent risk, and patients with both factors had a 32 percent chance of acquiring severe infection. Factors that did not affect risk of infection included type and stage of multiple myeloma; haemoglobin, albumin, and other protein levels; and prior experience with stem cell transplantation.

Patients who experienced severe infection were more likely to suffer from deep vein thrombosis, or blood clotting in the deep veins of the body.

Researchers concluded that antibacterial prophylaxis did not completely prevent the risk of developing infection, but antibiotic treatment could be used to fight infection once it occurred. They also recommended that patients with the two identified risk factors be treated with antimicrobial prophylaxes, which fight not only bacteria, but also fungi and viruses.

For more information, please see abstract 2828 at the ASH Meeting Web site.

Update: VTD Versus TD With Double Autologous Stem Cell Transplantation

One of the ASH presentations showed that the addition of Velcade to a thalidomide-dexamethasone regimen and double autologous stem cell transplantation significantly improves the response rate in newly diagnosed multiple myeloma patients. The presentation on the recent Phase 3 clinical trial updated the Phase 2 clinical trial results reported at the 2008 ASH meeting.

Patients were randomly assigned to receive three 21-day cycles of either VTD or TD.  Patients receiving the VTD regimen were given 1.3 mg/m² of Velcade twice-weekly, 200 mg of thalidomide daily, and 320 mg/cycle of dexamethasone. Patients receiving the TD regimen were given thalidomide and dexamethasone at the same dose and schedule as in the VTD regimen.

Following treatment with either VTD or TD, patients underwent two sequential autologous stem cell transplantations, which is commonly known as a double autologous stem cell transplantation. The procedure uses stem cells collected prior to chemotherapy to replace any cells damaged during the chemotherapy treatment. All patients later received two 35-day cycles of either VTD or TD as consolidation therapy.

Researchers found that patients treated with three cycles of VTD prior to double stem cell transplantation had higher rates of very good partial response or better than those treated with TD – 62 percent versus 31 percent; including a complete response of 19 percent versus 5 percent. These rates remain consistent with those found in the Phase 2 clinical trial.

Importantly, no patient receiving the VTD regimen experienced disease progression, compared to five percent of patients treated with TD. An update at the ASH meeting showed that the two-year projected progression-free survival for VTD treated patients was 85 percent compared to 75 percent of TD treated patients. However, longer follow-up is needed to determine if there is a difference in overall survival rate for the two treatments.

Low-Dose VTD Versus VD With Single Autologous Stem Cell Transplantation

Results from another study presented at ASH show that reduced doses of Velcade and thalidomide in the VTD regimen (followed by a single autologous stem cell transplantation) significantly increase rates of complete response and very good partial response.

Noting that peripheral neuropathy (a form of nerve damage in the extremities that can cause pain and tingling sensations) was the most common side effect of the VTD regimen, the authors of this study examined if decreased doses of Velcade and thalidomide could minimize the frequency of this adverse effect. Patients were randomly assigned to receive either low-dose VTD or standard-dose VD for four 21-day cycles.

Patients assigned to receive VD were given 1.3 mg/m² of Velcade on days 1, 4, 8, and 11, in addition to 40 mg of dexamethasone on days 1 to 4 and 8 to 11 for the first two cycles and on days 1 to 4 for the last two cycles. Patients assigned to receive low-dose VTD were given 1 mg/m² of Velcade on days 1, 4, 8, and 11; 100 mg of thalidomide daily; and dexamethasone, in the same dose and schedule as for VD.

Researchers found that patients treated with four cycles of low-dose VTD prior to stem cell transplantation had higher partial rates of response or better than those treated with VD – 91 percent versus 81 percent; including a complete response of 14 percent versus 12 percent and a very good partial response of 50 percent versus 36 percent. Importantly, the frequency of peripheral neuropathy was significantly reduced, with only two percent of patients receiving VTD experiencing serious peripheral neuropathy.

Based on these results, the authors of the study concluded that low-dose VTD should be considered as a new standard treatment to be given prior to autologous stem cell transplantation.

For more information, see abstracts 351 (VTD versus TD), and 354 (VTD versus VD) at the ASH Meeting Web site.

A total of 1,970 multiple myeloma patients participated in this trial, which split patients up into two separate groups based on age. The younger patients, median age 59 years, were randomized to receive one of two induction therapies – either cyclophosphamide (Cytoxan), thalidomide, and dexamethasone (Decadron); or vincristine (Oncovin), doxorubicin (Adriamycin), and dexamethasone. The older patients, median age 73 years, were randomized to receive either melphalan (Alkeran) and prednisone or cyclophasphamide, thalidomide, and dexamethasone.

All patients were given these chemotherapy combination regimens, called induction therapies, to decrease the number of myeloma cells prior to receiving an autologous stem cell transplant.  

Both groups had the option of taking low-dose thalidomide as maintenance therapy following the transplant.

The addition of thalidomide to induction therapy resulted in higher response rates in young patients –91% of patients who received induction therapy that included thalidomide responded, compared with 82% of patients who did not receive thalidomide.  The drug also affected the complete response rates: 21% of the patients who received thalidomide achieved a complete response, compared with 14% for those who did not.

In older patients, induction therapy with thalidomide significantly increased response rates. The response rate was 83% for those who received cyclophosphamide, thalidomide, and dexamethasone, compared with 46% for patients who were given melphalan and prednisone.

At the time of publication of trial results, the patients had been followed for about three years. The impact of thalidomide on overall survival is still being analyzed, but current data does not suggest that the addition of thalidomide to induction therapy has a substantial effect on survival time.

For more information, please see abstract 352 at the ASH Meeting Web site.

Found in marine bacteria, NPI-0052 acts, like Velcade, as a proteasome inhibitor. Both compounds prevent enzymes in cancer cells that regulate growth from working properly, which eventually leads to cell death. NPI-0052 is structured differently than Velcade and other proteasome inhibitors to date, which changes its effects on the body.

In the study, scientists examined both the activity and toxicity of NPI-0052 at various doses in 27 patients with relapsed or refractory myeloma. Patients received an IV injection of NPI-0052 on days 1, 8 and 15 of a 28-day cycle. Researchers gradually increased the dosage in some participants, with the final range given to patients being 0.025 mg/m² to 0.7 mg/m².

At a dose of 0.7 mg/m², NPI-0052 inhibited 73 percent of a specific type of proteasome activity on day 1 and 99 percent on day 15. In comparison, Velcade is 65 percent effective in inhibiting that proteasome activity. Two patients who had relapsed after Velcade treatment achieved significant reductions in their M-protein levels (71 and nearly 50 percent). Eight other patients who participated in the study between 6 and 15 months arrived at disease stability. Two of them had been resistant to Velcade.

Out of the eight patients treated with 0.7 mg/m², two experienced severe fatigue, loss of balance and changes in mental status which required their doses to be reduced. Other serious side effects included nausea, vomiting, dizziness, and confusion. Patients were given medications to counter those effects.

Side effects associated with Velcade and other myeloma agents such as reduced blood cell count, neuropathy (tingling in the limbs as a result of nerve dysfunction), and clotting in the veins did not occur with NPI-0052.

Another study presented at the ASH conference evaluated NPI-0052 at doses up to 0.9 mg/m² in various forms of cancer. New side effects were observed, including a distorted sense of taste or smell, hallucinations with the eyes closed, and problems with memory, concentration or behavior. Some patients also encountered a reduction in their lymphocytes, a type of white blood cell. Researchers pinpointed the maximum tolerated dosage at 0.7 mg/m².

In both studies, scientists concluded that NPI-0052 shows promise in treating relapsed and refractory multiple myeloma without causing some of the serious effects that accompany standard treatments. Clinical trials evaluating the compound’s effects in other cancers are underway.

For more information, see abstracts 431 and 2693 at ASH Meeting Web site.