Bellicum Pharmaceuticals, Inc. today announced that the Company received notice of a $5.7 million company commercialization award from the Cancer Prevention and Research Institute of Texas (“CPRIT”). Bellicum was the only company recommended for funding in this second round of the commercialization award program. The award will fund personnel, manufacturing, and clinical testing of Bellicum’s CaspaCIDe™ therapy, which promises to substantially improve outcomes for late stage cancer patients.

CPRIT was established to expedite cancer research innovation and commercialization and to enhance access to evidence-based prevention programs and services throughout the State. Company commercialization awards are a critical part of the Institute’s initiative to support the advancement of the most promising opportunities towards regulatory approval and market launch. The State has announced more than $500 million in awards and matching funds for innovative cancer research and prevention programs.

The application review process included in-depth evaluation by scientific and commercialization experts, followed by regulatory, product development and intellectual property due diligence. Acceptance of the award is subject to the completion of contract negotiations. “We are pleased to have been recommended for a significant $5.7 million award from CPRIT to advance our novel therapy for patients with high risk cancers,” said CEO Tom Farrell. “We are especially pleased to receive this notice given the rigorous review of our application by the CPRIT Commercialization Review Council, whose members have substantial scientific, venture capital and commercial expertise.”

The award will support clinical development of CaspaCIDe™, Bellicum’s solution to the problem of Graft versus Host Disease (GvHD), a debilitating and often fatal side effect of bone marrow transplantation. Originally developed by Bellicum CSO Dr. David Spencer, Vice Chair of Pathology and Immunology at Baylor College of Medicine, and advanced into clinical trials by Dr. Malcolm Brenner, Director of the Center for Cell and Gene Therapy at Baylor College of Medicine, CaspaCIDe™ works by allowing the transplant physician to selectively eliminate transplanted cells that cause GvHD. Building on promising early clinical results seen in Dr. Brenner’s “CASPALLO” trial, the CPRIT award will support the execution of a randomized, double-blind clinical trial in patients with high risk hematologic malignancies.

Patients treated for lumbar spinal stenosis (LSS) with the mild decompression procedure (an alternative to open spinal surgery for many patients), reported sustained improvements in pain and mobility at one year and had no serious adverse events occur, according to the first multi-center one-year post-study follow-up of this patient cohort. Results from this prospective, evidence-based study were presented today at the American Academy of Pain Medicine’s 27th Annual Meeting.

The post-study results were presented by Timothy R. Deer MD, of The Center for Pain Relief in Charleston, West Virginia. Outcomes of the procedure were assessed one year post treatment for all available patients who had participated in the first mild multi-center U.S. clinical trial completed last year. Included in the report were 170 procedures, mostly bilateral at one or two affected levels, in fifty-eight patients who were treated with the mild therapy.

Four validated outcome instruments were used including the Visual Analog Score (VAS), Oswestry Disability Index (ODI), Zurich Claudication Questionnaire (ZCQ), and the SF-12v2® Health Survey. The study patients achieved a significant improvement in pain (40 percent pain reduction) with VAS scoring improving in 79 percent of the patients, from overall mean 7.4 pre-treatment to 4.5 at Year One. Functional mobility also improved in 71 percent of the patients with overall mean ODI improving from 48.6 (severe disability) to 36.7 (moderate disability). The ZCQ and SF-12v2 measures also showed statistically and clinically significant improvements in pain and physical function, as well as satisfaction with overall result after the procedure.

Results from an ongoing prospective study report that a novel spinal neuromodulation device changes the paradigm for patients suffering with low back pain who have not found relief through conventional spinal cord stimulation (SCS). The single-centered results from this ongoing, multi-centered study were presented at the American Academy of Pain Medicine’s 27th Annual Meeting.

The results of the study were presented by Adnan Al-Kaisy, MB ChB FRCA, Clinical Lead of the Pain Management & Neuromodulation Centre, Guy’s & St Thomas’ Hospital, London, UK. Dr. Al-Kaisy and his colleagues conducted the study in the United Kingdom with 30 patients (with and without previous spine surgery) who had an average back pain Visual Analog Score (VAS) of 8 (out of ten) and an average leg pain VAS score of 6 (out of ten). Following approval from an ethics committee and the completion of a successful trial, dual octapolar, percutaneous leads were placed sequentially near the anatomic midline, between T8-T11 (the thoracic area of the spinal column). The Nevro system device was then connected to a rechargeable IPG (Implantable Pulse Generator) capable of delivering waveforms with frequencies up to 10 kHz (kilohertz).

At three months, the average VAS score for back pain fell to 2.9 (p-value < 0.05) and the average VAS score for leg pain fell to 2.2 (p-value < 0.05). At six months the average back and leg pain VAS scores fell to 1.6 respectively. The study also used the Oswestry Disability Index (ODI) to measure how the back pain affected the patients’ everyday life. The ODI decreased from 61 (p-value < 0.001) to 44 (p-value < 0.001).

The device proved to deliver significant pain relief at six months for both back and leg pain, without producing paresthesia. There was also no need for intra-operative paresthesia mapping with the improved ease of implantation of the device. The device improved patient function without movement-induced shocking and when the system was used overnight, the subjects reported improved sleep.

The Riordan Clinic announced publication in the Journal of Translational Medicine results of a collaboration between oncologists, alternative medicine practitioners, and basic researchers, which proposes a new use of intravenous vitamin C for treatment of cancer.

The rationale is provided that intravenous, but not oral, vitamin C may be capable of addressing issues in cancer patients such as wasting (cachexia), immune suppression, and improving quality of life. Citing 246 references, the paper synthesized existing knowledge regarding the use of intravenous vitamin C for numerous medical conditions and seeks re-evaluation of the place of intravenous vitamin C in the context of conventional oncology practice.

“Currently there is a great divide in the way intravenous vitamin C is viewed,” said Thomas Ichim, Board Member of the Riordan Clinic and first author of the publication. “On the one hand, you have alternative medicine practitioners, who have been claiming very interesting results in practical treatment of cancer patients, but cannot explain any molecular rationale for its use or potential effects. On the other hand you have a great amount of scientific literature supporting possible relevance of this approach in cancer. This paper is a significant step towards closing the divide.”

In the past, the use of vitamin C in the treatment of cancer has been considered controversial since some studies have claimed excellent results in extending lifespan of cancer patients, whereas other studies have not seen any effects. The discrepancy seems to be explained by studies seeing positive effects utilizing intravenous vitamin C, whereas the failed studies used oral vitamin C. Scientists at the Riordan Clinic were the first to publish, and patent, that intravenous, but not oral vitamin C, can achieve significant concentration in the blood in order to selectively kill tumor cells.

Approximately five percent of prescriptions submitted by CVS Caremark Pharmacy Benefit Management (PBM) members in a 30-day period during 2009 included a “dispense as written” (DAW) designation. This practice whereby doctors or patients demand the dispensing of a specific brand-name drug and not a generic alternative costs the health care system up to $7.7 billion annually, according to a new study by researchers at Harvard University, Brigham and Women’s Hospital and CVS Caremark. Moreover, these requests reduce the likelihood that patients actually fill new prescriptions for essential chronic conditions.

In a study published this week in the American Journal of Medicine, the researchers demonstrate that DAW designations for prescriptions have important implications for medication adherence. They found that when starting new essential therapy, chronically ill patients with DAW prescriptions were 50 to 60 percent less likely to actually fill the more expensive brand name prescriptions than generics. “Although dispense as written requests would seem to reflect a conscious decision by patients or their physicians to use a specific agent, the increased cost sharing that results for the patient may decrease the likelihood that patients actually fill their prescriptions,” the researchers said.

“This study shows that dispense as written requests are costing the health care system billions,” said William H. Shrank, MD, MSHS, of Brigham and Women’s Hospital and Harvard, and the study’s lead author. “The further irony is that patients with prescriptions specifying a certain brand seem less likely to fill their initial prescriptions, adding to the medication non-adherence problem.”

“Previous to this study, little was known about the frequency with which doctors and patients request dispense as written prescriptions,” said Troyen A. Brennan, MD, MPH, Executive Vice President and Chief Medical Officer of CVS Caremark and a study author. “Those who advocate for dispense as written and argue that the practice provides patients and physicians with greater choice will probably be surprised to learn that the practice increases costs and exacerbates non-adherence.”

The study reviewed 5.6 million prescriptions adjudicated by CVS Caremark for two million patients from January 1 to January 31, 2009. The review found that 2.7 percent of those prescriptions were designated DAW by doctors, while another two percent were requested DAW by patients.

Melanoma is not the most common form of skin cancer, but by far the deadliest overall. An estimated 68,130 new cases of melanoma were diagnosed in the United States during 2010 and about 8,700 people died from the disease, according to the National Cancer Institute. Now the U.S. Food and Drug Administration (FDA) has approved a Bristol Myers Squibb drug called Yervoy (ipilimumab) to help with the treatment of those with this type of skin cancer when it is at its final, fatal stages.

Richard Pazdur, M.D., director of the Office of Oncology Drug Products in the FDA’s Center for Drug Evaluation and Research stated:

“Late-stage melanoma is devastating, with very few treatment options for patients, none of which previously prolonged a patient’s life. Yervoy is the first therapy approved by the FDA to clearly demonstrate that patients with metastatic melanoma live longer by taking this treatment.”

Basically Yervoy, which is administered via injection, fights a molecule that intentionally slows or turns off the body’s defensive immune system, allowing cancer cells to run rampant. Yervoy may work by allowing the body’s immune system to recognize, target, and attack cells in melanoma tumors.

In tests, those who received the combination of Yervoy plus the vaccine or Yervoy alone lived an average of about 10 months, while those who received only the experimental vaccine lived an average of 6.5 months.

Researchers seeking to unravel the most ancient yet least understood of the five senses – smell – have discovered a previously unknown step in how odors are detected and processed by the brain.

The four year study, focusing on how mice respond to odors, showed that smells are picked up by the olfactory bulb – the first stop on the way to the brain – then sent to the olfactory cortex for further analysis.

But scientists discovered something else – a dialogue between the bulb and the cortex conducted by rapidly firing nerve cells.

“It was originally thought that the olfactory bulb filtered and the olfactory cortex made decisions on whether something is, for example, edible,” said lead researcher Diego Restrepo, professor of Cell and Developmental Biology and co-director of the University of Colorado School of Medicine Center for NeuroScience. “Our study says it’s not quite like that. You process information on reward in the olfactory bulb, send it to the cortex and there is a dialogue between the two. Then the brain will act.”

The study was published March 24 in the science journal Neuron.

Restrepo, an expert on the science of taste and smell, said the discovery expands our understanding of how the olfactory system filters and categorizes the thousands of odors that bombard the brain daily.

“We know very little about olfaction and we tend to think that it is not very important in humans compared to the other creatures,” he said. “But much of what goes on is subtle and we are only beginning to understand it.”

For example, scientists recently found that when men sniffed the odorless tears of women, their levels of testosterone dropped. And for years it’s been known that humans, like animals, secrete pheromones that may subconsciously help them choose a mate.

But unlike hearing, taste, sight and touch – smell is the only sense not processed exclusively through the thalamus in the brain, Restrepo said. So the exact path odors take to the brain and how they can trigger often vivid reactions is still not fully understood. The new research suggests that perhaps part of the answer lies within the dialogue between olfactory bulb and cortex.

There are billions of neurons in the brain and at any given time tens of thousands of these neurons might be trying to send signals to one another. Much like a person trying to be heard by his friend across a crowded room, neurons must figure out the best way to get their message heard above the din.

Researchers from the Center for the Neural Basis of Cognition, a joint program between Carnegie Mellon University and the University of Pittsburgh, have found two ways that neurons accomplish this, establishing a fundamental mechanism by which neurons communicate. The findings have been published in an online early edition of Proceedings of the National Academy of Sciences (PNAS).

“Neurons face a universal communications conundrum. They can speak together and be heard far and wide, or they can speak individually and say more. Both are important. We wanted to find out how neurons choose between these strategies,” said Nathan Urban, the Dr. Frederick A. Schwertz Distinguish Professor of Life Sciences and head of the Department of Biological Sciences at CMU.

Neurons communicate by sending out electrical impulses called action potentials or “spikes.” These spikes code information much like a version of Morse code with only dots and no dashes. Groups of neurons can choose to communicate information in one of two ways: by spiking simultaneously or by spiking separately.

To find out how the brain decided which method to use to process a sensory input, the researchers looked at mitral cell neurons in the brain’s olfactory bulb – the part of the brain that sorts out smells and a common model for studying global information processing. Using slice electrophysiology and computer simulations, the researchers found that the brain had a clever strategy for ensuring that the neurons’ message was being heard.

Over the short time scale of a few milliseconds, the brain engaged its inhibitory circuitry to make the neurons fire in synchrony. This simultaneous, correlated firing creates a loud, but simple, signal. The effect was much like a crowd at a sporting event chanting, “Let’s go team!” Over short time intervals, individual neurons produced the same short message, increasing the effectiveness with which activity was transmitted to other brain areas. The researchers say that in both human and neuronal communication alike, this collective communication works well for simple messages, but not for longer or more complex messages that contain more intricate information.

The neurons studied used longer timescales (around one second) to convey these more complex concepts. Over longer time intervals, the inhibitory circuitry generated a form of competition between neurons, so that the more strongly activated neurons silenced the activity of weakly activated neurons, enhancing the differences in their firing rates and making their activity less correlated. Each neuron was able to communicate a different piece of information about the stimulus without being drowned out by the chatter of competing neurons. It would be like being in a group where each person spoke in turn. The room would be much quieter than a sports arena and the immediate audience would be able to listen and learn much more complex information.

Researchers believe that the findings can be applied beyond the olfactory system to other neural systems, and perhaps even be used in other biological systems.

“Across biology, from genetics to ecology, systems must simultaneously complete multiple functions. The solution we found in neuroscience can be applied to other systems to try to understand how they manage competing demands,” Urban said.

Although less likely to suffer a hip or wrist fracture, obese women suffer from fragility fractures at other sites, partly due to poor mobility and increased risk of falls

Obesity is widely believed to be protective against fracture, although a recent study has documented a high prevalence of obesity in postmenopausal women with fragility fracture.

An international group of researchers has today presented research at the European Congress on Osteoporosis & Osteoarthritis (ECCEO11-IOF) that compares the prevalence and location of fractures in obese (BMI≥30 kg/m2) and non-obese postmenopausal women and examines specific risk factors for fracture.

A history of fracture after age 45 years was observed in 23% of obese and 24% of non-obese women. Nearly one in four postmenopausal women with fractures is obese. The upper arm, ankle and lower leg were significantly more likely to be affected in obese than non-obese women with a prevalent fracture, whereas fractures of the wrist, hip and pelvis were significantly less common than in non-obese women. When compared to non-obese women, obese women with a prevalent fracture were more likely to be current cortisone users, to report early menopause, to report fair or poor general health, to use arms to assist standing from a sitting position, and to report more than two falls in the past year.

The research demonstrates that obese postmenopausal women are almost as likely to fracture as non-obese women, and that poor mobility and increased risk of falls may play an important role. The findings have significant public health implications in view of the rapidly rising numbers of obese people in the population.

Abstract OC21: Fractures in obese postmenopausal women: prevalence, skeletal location and risk factors. J. Compston et al. Osteoporosis International DOI 10.1007/s00198-011-1554-9

How does your best friend feel when people act needy? Or, about people being dishonest? What do they think when others seem uncomfortable in social situations? According to an upcoming study in Psychological Science, a journal of the Association for Psychological Science, if you don’t know – your relationship may pay a price.

There are lots of ways to know someone’s personality. You can say “she’s an extrovert” or “she’s usually happy.” You may also know how he or she reacts to different situations and other people’s behavior. “It’s a more detailed way of understanding personality,” says Charity A. Friesen, a graduate student at Wilfrid Laurier University, who co-wrote the new paper with Lara K. Kammrath. “You might know the person is extroverted when they’re out with their friends but more introverted when they’re in a new situation.” When a person is faced with one of a list of situations, then how does he or she behave? Friesen identifies this as an “if-then profile.”

Friesen and Kammrath recruited university students to take part in the study. Each student was asked to get a friend to participate in the study with them. Then each of the participants individually filled out an online survey. This included a list of “triggers” – descriptions of behaviors that someone might find annoying. One example was the word “skepticism” which was described as when someone is overly disbelieving of information that he/she receives, when he/she questions things that are generally accepted, or when he/she is very hard to convince of something. The list also included gullibility, social timidity, social boldness, perfectionism, obliviousness and several dozen other possible triggers. For each behavior, each respondent answered a question about how much this triggers them and how much it triggers their friend.

Some people knew their friends’ triggers well; others had almost no idea what set their friends off. And that made a difference to the friendship. People who had more knowledge of their friend’s if-then profile of triggers had better relationships. They had less conflict with the friend and less frustration with the relationship. Other research has shown that it’s not that hard to come up with a list of traits that describe someone; casual acquaintances can do it. “But, if I’m close to someone, I can really start to learn the if-then profiles, and that’s what’s going to pay off in my relationship,” Friesen says.