hope for new skin-disorder drug

A radical new drug used to treat patients suffering painful sensitivity to sunlight could prevent skin cancer or be copied by the tanning industry.

The breakthrough drug Afamelanotide, developed by a Melbourne company, has been successfully tested for 12 months on 101 people worldwide, including six Melburnians, all with the incurable genetic skin disorder EPP.

The illness leaves suffers with painful, bleeding blisters and burns when exposed to normal levels of sunlight.

Pharmaceutical company Clinuvel has been developing the drug - given as a repeated injection to stimulate skin pigmentation - over the past 19 years.

Royal Children's Hospital and Royal Melbourne head of dermatology, Dr George Varigos, led the Melbourne drug trial on his EPP patients.

"After our patients were treated, they could venture out, and do things as a family that they couldn't do before," he said.

"The effect is incredibly good for their behaviour and lifestyle.

"We are not telling these people to go out and sunbake, but now they can have a normal life."

Dr Varigos is testing the drug on transplant patients taking immune supressants, which increases their risk of developing skin cancer within five years of having a transplant.

He said the tanning industry would probably seek to replicate the drug, which makes the user slightly darker by activating pigmentation.

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New drug may be better against H1N1, bird flu

An experimental new drug could be more effective than existing drugs against swine flu and bird flu, a UW-Madison researcher found.

The drug, T-705, or favipiravir, was previously found to be effective against swine flu, or H1N1। Now Yoshihiro Kawaoka, a virologist at the university, has shown that the drug protects mice against bird flu, or H5N1।

Kawaoka and other flu experts consider bird flu to be a more dangerous global threat than swine flu because it is more deadly, even though it hasn't become as widespread in people.

T-705, developed by the Japanese company Toyama, could offer an alternative to Tamiflu, the main drug against all kinds of flu, Kawaoka said. Some flu viruses have become resistant to Tamiflu, so other drugs are needed, he said.

"We need a drug that targets different molecules of the influenza virus," Kawaoka said.

T-705, which attacks a different flu molecule than Tamiflu does, is being tested in people in Japan.

Kawaoka compared T-705 and Tamiflu in mice infected with bird flu. T-705 worked as well or better than Tamiflu, he found in a study published Monday in the Proceedings of the National Academy of Sciences.

When both drugs were given three days after infection, T-705 worked twice as well at Tamiflu, the study found. That is significant because many flu patients aren't able to get medical care soon enough for Tamiflu to be helpful, Kawaoka said.

While swine flu has been getting significant attention in recent months and is more of an immediate concern, bird flu remains more of a threat because its potential to kill appears to be greater, Kawaoka said.

Relatively few cases of bird flu have been reported this year in people, but a man died from it last month in Vietnam, according to the World Health Organzation.

Meanwhile, vaccine against swine flu is widely available in Wisconsin after a shortage this fall. The Madison-Dane County Health Department plans to resume school-based clinics next month, spokesman Jeff Golden said.

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FDA approved leukemia drugs shows promise in ovarian cancer cells

The drug Sprycel, approved for use by the U.S. Food and Drug Administration in patients with chronic myeloid leukemia, significantly inhibited the growth and invasiveness of ovarian cancer cells and also promoted their death, a study by researchers with UCLA's Jonsson Comprehensive Cancer Center found.

The drug, when paired with a chemotherapy regimen, was even more effective in fighting ovarian cancer in cell lines in which signaling of the Src family kinases, associated with the deadly disease, is activated.

The study appears in the Nov. 10, 2009 edition of the British Medical Journal.

Ovarian cancer, which will strike 21,600 women this year and kill 15,500, causes more deaths than any other cancer of the female reproductive system. Few effective therapies for ovarian cancer exist, so it would be advantageous for patients if a new drug could be found that fights the cancer, said Gottfried Konecny, an assistant professor of hematology/oncology, a Jonsson Cancer Center researcher and first author of the study.

"I think Sprycel could be a potential additional drug for treating patients with Src dependent ovarian cancer," Konecny said. "It is important to remember that this work is only on cancer cell lines, but it is significant enough that it should be used to justify clinical trials to confirm that women with this type of ovarian cancer could benefit."

Recent gene expression studies have shown that about one-third of women have ovarian cancers with activated Src pathways, so the drug could potentially help 7,000 ovarian cancer patients every year.

In this study, the UCLA team tested the drug against 34 ovarian cancer cell lines and they conducted genetic analysis on all cell lines. Through these analyses, the researchers were able to identify genes that predict response to Sprycel. If the work is confirmed in human studies, it may be possible to test patients for Src activation and select those who would respond prior to treatment, personalizing their care.

"We were able to identify markers in the pre-clinical setting that would allow us to predict response to Sprycel," Konecny said. "These may help us in future clinical trials in selecting patients for studies of the drug."

Sprycel is what is known as a "dirty" kinase inhibitor, meaning it inhibits more than one pathway. Konecny said it also inhibits the focal adhesion kinase and ephrin receptor, also associated with ovarian cancer.

The next step, Konecny said, would be to test the drug on women with ovarian cancer in a clinical trial. The tissue of responders would then be analyzed to determine if the Src and other pathways were activated. If that is confirmed, it would further prove that Sprycel could be used to fight ovarian cancer. In studies, women would be screened before entering a trial and only those with Src dependent cancers could be enrolled to provide further evidence, Konecny said, much like the studies of the molecularly targeted breast cancer drug Herceptin enrolled only women who had HER-2 positive disease.

"Herceptin is different because we knew in advance that the only worked in women with HER-2 amplification," he said. "In this case, we don't clearly know that yet. The data reassure us that the drug works where the targets are over-expressed but we need more testing to confirm this."
The tests combining the drug with chemotherapy are significant because chemotherapy currently is the first line treatment for ovarian cancer patients following surgery. Because Sprycel proved to have a synergistic effect when combined with chemotherapy both made the other work better it may be possible to add the targeted therapy as a first line treatment if its efficacy is confirmed in future studies, adding a new tool to an oncologist's arsenal.

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Drugs to treat anemia in cancer patients linked to thromboembolism

Medications frequently given to cancer patients to reduce their risk of anemia are associated with an increased risk of deep vein thrombosis or pulmonary embolism, according to new research led by Dawn Hershman, M.D, M.S., co-director of the breast cancer program at the Herbert Irving Comprehensive Cancer Center at NewYork-Presbyterian Hospital/Columbia University Medical Center. The findings will be published online on Nov. 10, 2009 in the Journal of the National Cancer Institute .

The anemia-reducing medications, known as erythropoiesis-stimulating agents (i.e., erythropoietin and darbopoietin) or ESAs, stimulate red blood cell production and are intended to reduce the number of blood transfusions required during chemotherapy. However, concerns about the risks of deep vein thrombosis or pulmonary embolism (manifestations of venous thromboembolism) and mortality exist.

"This research answers important questions about outcomes of ESAs when used in long-term clinical practice with oncology patients," said Dr. Hershman, the Florence Irving Assistant Professor of Medicine and Epidemiology at Columbia University Medical Center, whose research is dedicated to examining cancer survivorship. "While ESAs were given to reduce the need for blood transfusions, a substantial reduction in the use of blood transfusions was not observed. However, an increase risk of deep vein thrombosis or pulmonary embolism was confirmed."

"This analysis confirms the association between ESAs and venous thromboembolism, which was observed in previous meta-analysis," said Dr. Hershman. "This new finding is significant because where the meta-analysis looked at pooled data from randomized clinical trials, this data is from community practice -- real-life clinical settings -- where you can often see things that wouldn't necessarily show-up in a short-term, 12-week study. Additionally, this analysis included data from more than 50,000 patients? including those with more advanced cancer or high-risk status, who therefore might not have been candidates for clinical trials."

Based on previous findings, in the spring of 2007, the FDA required a black-box warning on ESAs about the potential for venous thromboembolism, tumor promotion, and decreased survival in ESA users. The warning suggested limiting the use of ESAs to specific tumor types, durations, doses, and targeted hemoglobin levels. In addition, the Center for Medicare and Medicaid Services proposed eliminating or limiting coverage for ESAs as treatment for some cancers.

"But what is reassuring about our findings are that they don't show an increased risk of mortality when ESAs are given with chemotherapy," said Dr. Hershman.

Dr. Hershman and colleagues analyzed the association between use of ESAs and venous thromboembolism and overall survival in patients who were 65 years or older and diagnosed with colon, non-small cell lung, or breast cancer or diffuse large B-cell lymphoma, between 1991-2002. These cancers were chosen because they were thought to be common cancers for which ESAs were frequently used. Patients were identified in the Surveillance, Epidemiology, and End Results?Medicare database, which at the time contained records of patients diagnosed with cancer in regions that represented approximately 14 percent of the U.S. population.

Results demonstrated that more patients who received an ESA developed deep vein thrombosis or pulmonary embolism, as compared to patients who did not. Overall survival was similar in both groups. The number of patients receiving ESAs increased approximately 10-fold from 1991 through 2002, with approximately 50 percent of patients with advanced cancer undergoing chemotherapy receiving ESAs by 2002. The rate of blood transfusion per year during the same time period, however, remained constant at 22 percent.

"Further efforts at monitoring use and long-term toxicity of expensive oncology drugs should be put in place to ensure that for any drug the benefits outweigh the risks in community practice," the authors write in the paper.

In the JNCI paper, the authors note that ESAs may be of particular interest from a public policy perspective because of the costs associated with their use. Total U.S. sales of ESAs were $10 billion in 2006, accounting for a greater Medicare Part B expenditure than any other drug.

The Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center and NewYork-Presbyterian Hospital encompasses pre-clinical and clinical research, treatment, prevention and education efforts in cancer. The Cancer Center was initially funded by the NCI in 1972 and became a National Cancer Institute (NCI)?designated comprehensive cancer center in 1979. The designation recognizes the Center's collaborative environment and expertise in harnessing translational research to bridge scientific discovery to clinical delivery, with the ultimate goal of successfully introducing novel diagnostic, therapeutic and preventive approaches to cancer.

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Aastrom to Initiate Second Phase II Clinical Trial for Treatment of Severe Chronic Heart Failure

Aastrom Biosciences, Inc. , a leading developer of autologous cell products for the treatment of chronic cardiovascular diseases, today announced that the Company will initiate its second clinical trial for the treatment of dilated cardiomyopathy (DCM), a severe disease associated with chronic heart failure, after a positive 30-day review of Aastrom's Investigational New Drug (IND) submission by the U.S. Food & Drug Administration (FDA).

This second trial is a 24 patient U.S. Phase II clinical trial to evaluate the catheter delivery of Cardiac Repair Cells (CRCs) for the treatment of DCM. The new trial expands the Company's cardiovascular program that includes a U.S. Phase II IMPACT-DCM clinical trial evaluating the direct surgical delivery of CRCs. The FDA previously granted CRCs an Orphan Drug Designation for the treatment of DCM.

"Given the encouraging initial results in our surgical-based, open-label clinical trial, we are expanding our cardiovascular program to include a catheter-based delivery method for these critically ill patients," said Elmar R. Burchardt, M.D., Ph.D., Vice President, Medical Affairs of Aastrom. "Our catheter-based delivery method is less invasive than the surgical approach and therefore increases the number of potential patient candidates for CRC treatment. End-stage heart failure patients currently have limited therapeutic options other than heart transplantation and mechanical pump assist devices. By expanding the delivery options for CRCs to include the catheter delivery by cardiologists, we are complementing the surgical delivery by cardiac surgeons."

The randomized, controlled, prospective, open-label, Phase II study will seek to enroll 12 patients with ischemic DCM and 12 patients with non-ischemic DCM at two clinical sites in the U.S. Participants must have a left ventricular ejection fraction of less than or equal to 30% (60-75% is typical for a healthy person) and meet certain other eligibility criteria. All 24 patients will receive standard medical care and 16 of the patients (8 ischemic and 8 non-ischemic) will also be treated with CRCs via catheter injection. While the primary objective of this study is to assess the safety of CRCs delivered by catheter injection in patients with DCM, efficacy measures including heart failure stage and cardiac function parameters will also be assessed. Patients will be followed for 12 months post treatment.

There are currently 5.5 million people in the U.S. suffering from chronic heart failure. A subset of these patients has DCM, a chronic cardiac disease where expansion of the patient's heart reduces the pump function to a point that the normal circulation of blood cannot be maintained. Patients with DCM typically present with symptoms of congestive heart failure, including severe limitations in their physical activity and shortness of breath. DCM generally occurs in patients who have ischemic heart failure due to multiple heart attacks, though it can also be found in patients with non-ischemic heart failure caused by hypertension, viral infection or alcoholism. Patient prognosis depends on the stage of the disease but is characterized by numerous health problems and a very high mortality rate.

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Mylan Receives FDA Approval for Generic Version of Prevacid

Mylan Inc. today announced that its privately held Indian subsidiary, Matrix Laboratories Limited, has received final approval from the U.S. Food and Drug Administration (FDA) for its Abbreviated New Drug Application (ANDA) for Lansoprazole Delayed-release (DR) Capsules, 15 mg and 30 mg. This product is shipping immediately and will be sold under the Mylan Pharmaceuticals brand.

Lansoprazole DR Capsules are the generic version of Tap Pharmaceuticals’ proton pump inhibitor Prevacidآ® DR Capsules. The brand product had U.S. sales of approximately $3 billion for the 12 months ending June 30, according to IMS Health.

Currently, Mylan has 125 ANDAs pending FDA approval representing $84.1 billion in annual brand sales, according to IMS Health. Thirty-seven of these pending ANDAs are potential first-to-file opportunities, representing $19.2 billion in annual brand sales, for the 12 months ending June 30, according to IMS Health.

Mylan Inc. ranks among the leading generic and specialty pharmaceutical companies in the world and provides products to customers in more than 140 countries and territories. The company maintains one of the industry’s broadest and highest quality product portfolios supported by a robust product pipeline; operates the world’s third largest active pharmaceutical ingredient manufacturer; and runs a specialty business focused on respiratory and allergy therapies.

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New drug for small cell lung cancer?

Scientists from the Imperial College of London have identified a promising new drug for the treatment of an aggressive form of lung cancer; small cell lung cancer.

Small cell lung cancer forms about 15-20% of the total cases of lung cancer. It is the most aggressive form of cancer that occurs within the lungs and grows and spreads quickly, most often to the brain, liver and bones.

Prognosis is poos, with only a 3-6% 5-year survival rate amongst patients diagnosed with it. As it spreads so quickly, surgery is usually not an option, as it most likely will be in more than one location by the time the patient is diagnosed. Combination chemotherapy and radiotherapy is commonly used instead, but a lot of the time the tumours grow back and are resistant to treatment.

The new drug being tested (PD173074) has been found to kill small cell lung cancer tumours in 50% of the mice it was tested on. It works by preventing a certain growth hormone the cancer cells need (called FGF-2, which speeds up the cancer cells' growth and multiplication) from attaching to its' receptor sites on the cells. It also lowers the cells' resistance to standard chemotherapy.


The next step is to try the drug out on small cell lung cancer patients who cannot be operated on.

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New 3-drug combo may effectively treat multiple myeloma

Researchers from Dana-Farber Cancer Institute have found that a new three-drug combination can effectively treat patients newly diagnosed with multiple myeloma, a cancer of white blood cells in the bone marrow.

The three-drug combination has shown in a phase 1/2 clinical trial that it is a ‘highly effective regimen’ in the treatment of this form of cancer.

Paul G. Richardson, of Dana-Farber, who led the study, said that partial responses or better were seen in all of the 66 patients treated with the drug combination in the multi-center study, with 74 percent having a “very good partial response rate” in the phase 2 population.

The rate of complete or “near complete” responses to the therapy was also encouraging at 54 percent.

The regimen, known as RVD, combined the drugs Revlimid (lenalidomide), Velcade (bortezomib) and dexamethasone, which previously were found to be highly effective in multiple myeloma patients who had relapsed or no longer responded to first-line therapies.

Fifteen of the 35 newly diagnosed patients in the open-label phase 2 portion of the study subsequently underwent autologous (using their own blood-forming stem cells) transplants, a standard treatment for multiple myeloma “and did very well,” Richardson said.

For the entire group, after a median 19.3 months of follow up, the median time-to-progression (TTP) of the disease, progression-free survival (PFS), and overall survival (OS) had not yet been reached, according to the presentation.

The estimated TTP and PFS at one year are 76 percent, and the estimated one-year overall survival is 100 percent, the results showed.

Richardson says it was ‘particularly exciting’ to observe that the high response rate was not affected by the specific genetic characteristics of the patients’ disease.

Patients with so-called “adverse cytogenetics” are at higher risk for treatment failure and death, but in the current study the drug combination worked as well for them as it did in patients with more favorable cytogenesis features.

Richard said that the toxic side effects of the treatment were ‘manageable.’

“Our conclusion is that this is a highly effective regimen for newly diagnosed multiple myeloma patients. The combination has now gone into large phase 3 clinical trials, and we think it has the potential to be a new standard of treatment in multiple myeloma, ” Richardson said.

The results have been described in an oral presentation at the American Society of Hematology’s 51st annual meeting on Saturday, Dec. 5. (ANI)

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New drug shows promise for those with clotting disorders

A new study provides welcome news for patients with a common clotting disorder known as venous thromboembolism (VTE).

The condition is estimated to impact 62,000 Canadians and half-a-million Americans every year, and occurs when an abnormal clot forms in a vein and restricts the flow of blood, causing pain and swelling। In some cases, the clot may detach from its point of origin and travel through the heart to the lungs, causing a potentially fatal condition known as a pulmonary embolism।

Currently, patients with VTE are treated with a blood thinner known as warfarin, which has many burdensome interactions with other medications and foods and requires frequent monitoring of the dosage.

However, this study published today shows that an oral drug called dabigatran etexilate, which does not have these disadvantages, is as safe and effective as warfarin for combating VTE.

To compare the two drugs, an international team of researchers led by Sam Schulman, a professor of medicine at the Michael G. DeGroote School of Medicine, conducted a randomized, double-blind trial of 2,539 patients with acute VTE.

Today, Schulman is scheduled to present this study to the annual conference of the American Society of Hematology in New Orleans, LA, and the New England Journal of Medicine will post the study on its web site. The study will be published in the Dec. 10 edition of the New England Journal of Medicine.

For six months, roughly half of the patients in the trial (1,274) were given a fixed dose of 150 mg of dabigatran etexilate twice daily, while the other half (1,265 patients) were given warfarin once daily.

The improvement seen in both groups from the treatments was similar. After six months of treatment, only 2.4 percent of the dabigatran etexilate group (30 patients) and 2.1 percent of the warfarin group (27 patients) experienced recurrent VTE.

The safety of the two drugs was also comparable. In the dabigatran etexilate arm, 205 patients experienced bleeding (including 20 patients with major bleeding) versus 277 patients in the warfarin arm (including 24 with major bleeding). Other possible side effects, including death, acute coronary syndromes, and abnormalities in liver function tests, were infrequent in the two groups.

"We are excited by these findings and feel that they will change the standard of care for venous thromboembolism, which affects a large number of our patients," said Schulman, a physician with the thrombosis service of Hamilton Health Sciences. "This study found that dabigatran is a safe and effective anticoagulant that does not require the routine monitoring or dose adjustments that are necessary with warfarin. In other words, patients can receive the same results in a more convenient manner."

The study was funded by Boehringer Ingelheim.

McMaster University, one of four Canadian universities listed among the Top 100 universities in the world, is renowned for its innovation in both learning and discovery. It has a student population of 23,000, and more than 140,000 alumni in 128 countries.

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New drug once used as bird poison shows promise for Multiple Sclerosis

An FDA panel is looking into the possibility that a new drug named Ampriva is helping people with Multiple Sclerosis. The drug is a reconfigured form of fampridine, which was originally used as a bird poison.

The Food and Drug Administration is looking at reports that the medication has helped people with MS actually walk with the help of the drug.

It's possible that the FDA will put this on the fast track because of the positive reports.

The new drug is different because it improves the nervous system function in people suffering from MS, and right now, the best that MS drugs can do is keep the progression of the disease from getting worse, and not change the already damaged nerves.

Ampriva is supposed to help protect the myelin which is damaged in people with MS. That damage is what affects movement and walking in some people with the disease.

So far, more than one-third of the people taking the new drug have been able to walk faster.

Dr. Andrew D. Goodman, director of the Multiple Sclerosis Center at the University of Rochester and the lead investigator in the clinical studies, said that "this can mean things like getting to the bathroom on time before having an accident, or getting across the street before the light changes."

Despite the obvious improvements, however, MS patients continue to get worse over time, Goodman's research shows. The results are slight at the moment.

Dr. Goodman says, "Things that people have described to me are, 'Look, I can get around the supermarket without having to hold on to the cart all the time,' or 'Just getting up that step between the garage and the house gives me independence.' "

The bird poision has been known to cause seizures and convulsions at high dosages.

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New Drug for HPV Might be Effective in Curbing Vulvar Cancer

A Dutch study has recently confirmed that an experimental medicine targeted at human papillomavirus (HPV) can prove much effective in curbing the growth of precancerous tumours in the vulva. The vaccine has been found effective to stop their growth, thereby preventing them from turning into cancerous malignancies.

Experts have been saying that Gardasil and Cervarix, two popular HPV medicines, might spur precancerous tumours in the cervix and cause as many as 70% of cervical cancers. But the Dutch study, published in the New England Journal of Medicine's November 5 edition, looks at a new, experimental drug, which is found to be even more effective in prevention.

"This provides a therapeutic effect to a lesion that's already there", explained Dr. Eugene P. Toy, an associate professor of obstetrics and gynaecology at the University of Rochester Medical Center. HPV-16, a sexually transmitted pathogen is largely linked to some rare cancers of the throat, genitals and anus. It is also said to be a cause of genital warts. Preventive medication for the same is highly recommended to young women.

Current treatments available for vulvar lesions are not all that effective and most people tend to avoid them as well. If the results of the study, which are currently being analyzed, are confirmed, it will give the world a new way for dealing with precancerous vulvar tumours, a definite medical breakthrough.

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New German drug saves Aussie baby from brain-melting disease

A "miracle" Australian baby has become the first person cured of a rare and deadly brain-melting condition after doctors gambled on an experimental German drug tested only on mice, they said Thursday.

The child, known only as "Baby Z," was facing a painful death of seizures and brain damage from molybdenum cofactor deficiency, a genetic defect which causes a build-up of toxic sulphite and usually kills in months.

But she made an amazing recovery just three days after first being given the untested treatment which was flown in from Germany and rushed through the courts.

"We are looking at her now and she is just an absolute miracle - she has defied everybody," her mother, who cannot be named for legal reasons, told reporters.

Baby Z started having seizures within 60 hours of her birth in May 2008, prompting her family to appeal to biochemist Rob Gianello to help beat the previously incurable condition which affects just one in a million Australians.

"There was courage and there was death - we opted for courage," the mother said. "If she wasn't treated she would die a very painful death."

Baby Z's doctor Alex Veldman said Gianello discovered an experimental drug which had been successfully used on mice by Germany's Gunther Schwarz, but had never been tested on humans.

As Schwarz couriered his entire stock of the compound from Cologne to Melbourne, doctors were in a race against time to get ethics approval from the hospital and a court order clearing its use, with Baby Z worsening by the hour.

"The team ... managed to get this therapy from bench to bedside in about two weeks, a process which normally takes several years," Veldman said.

Within hours of receiving her first dose of the drug, cyclic pyranopterin monophosphate (cPMP), Baby Z's sulphite levels plunged by more than two-thirds and were at normal levels within about three days.

Veldman said Baby Z's alertness improved and her twitching slowed rapidly, with her seizures dropping by 90 percent in three weeks.

"The response was just amazing," he told AFP. "We can treat this in humans for the first time in (the history of) mankind. It has the potential to save lives all around the world."

Baby Z's development has been slowed by the brain damage she suffered before the treatment, and Veldman said she would need a cPMP injection every day for the rest of her life.

But her mother said the baby, now 18 months old, had started speaking and was physically active.

"Every day just gets better and better. We look at her every day and just think, 'Wow'," she said. "(The procedure) was a tiny bit of hope but, when you have nothing, that is a lot of hope."

Veldman said a second child, "Baby P", had since started cPMP treatment in Germany and was "improving rapidly."

The cases were now being analysed ahead of a planned international human trial of the medication at Melbourne's Monash Medical Centre, he said.

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New Drug for treating spinal muscular atrophy

Scientists have identified a chemical cousin of the common antibiotic tetracycline that might be useful in treating spinal muscular atrophy (SMA), a currently incurable disease that is the leading genetic cause of death in infants.

The finding is based on a study conducted by Adrian Krainer, Ph.D., of Cold Spring Harbor Laboratory (CSHL) and scientists from Paratek Pharmaceuticals and Rosalind Franklin University of Medicine and Science.

SMA is caused by mutations in a gene called Survival of Motor Neuron 1 (SMN1), resulting in a decrease in the levels of SMN protein in the motor neurons of the spinal cord - the cells that control muscle activity.

Without the protein, these neurons degenerate, and infants born with the mutations progressively lose the ability to move, swallow, and breathe.

There are no approved therapies for the treatment of SMA.

The new molecule boosts the levels of SMN protein in cells by fixing a mistake in a cellular processing mechanism called RNA splicing.

In the study, the scientists report this fix in both mouse models of SMA, as well as in cells isolated from SMA patients.

Unlike previously identified molecules that stimulate SMN production, the tetracycline-like compound is a unique therapeutic candidate in that it is a small molecule that specifically alters RNA splicing by directly targeting the splicing reaction.

The research appears in the journal Science Translational Medicine. (ANI)

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A New Hope with a new drug for H1N1 Flu

Athena Gurno thought her allergies were acting up when she started coughing in early October. But within days, Ms. Gurno, the 30-year-old mother of a young girl, was in a Seattle hospital, close to death from the H1N1 flu.

Desperate, her doctors tried a still-experimental drug called peramivir. After getting her second dose, Ms. Gurno started to recover, though she is still in intensive care, according to her father, John Spikowski.

“This saved Athena’s life,” Mr. Spikowski reported on a blog that tracks his daughter’s progress.

Peramivir might also be a life saver for its developer, BioCryst Pharmaceuticals, an unprofitable biotechnology company in Birmingham, Ala., that was founded in 1986 but has not yet had a drug reach the market.

On Thursday, the federal government ordered, on an emergency basis, 10,000 treatment courses of peramivir for its national stockpile. It is paying $22.5 million, or about $2,250 a patient. Shares of BioCryst rose nearly 13 percent, to $11.39.

Peramivir is given intravenously, making it usable by hospitalized patients who are too ill to take two approved flu drugs that work against the virus in similar ways — Tamiflu by Roche, which is typically given as a pill, or Relenza from GlaxoSmithKline, which is inhaled.

Late Thursday, the government announced orders for intravenous versions of Tamiflu and Relenza, which are much cheaper — a development that could force shares of BioCryst to give up some of their gains on Friday.

Peramivir, still being tested in clinical trials, is not approved by the Food and Drug Administration for general use. But on Oct. 23, the F.D.A. granted authority for the drug to be used in emergencies for patients hospitalized with H1N1 flu who cannot take or do not benefit from Tamiflu or Relenza.

Before that, peramivir had been available only through a more cumbersome “compassionate use” procedure. Of the 32 patients who received the drug that way, 29 were still alive, BioCryst said in late October.

Although there are still questions about peramivir’s true effectiveness, some critics say the government moved too slowly to make the drug available, and that even now, access is too restricted. For each patient, doctors must call an 800 number or fill out a form on a Web site run by the Centers for Disease Control and Prevention. The drug is then sent overnight from a central stockpile.

“If you have a critically ill patient, to delay therapy, it’s just incomprehensible to me,” said Dr. Richard Whitley, a professor at the University of Alabama and the president of the Infectious Diseases Society of America. He said the drug should be distributed so that hospitals could have it in stock.

But Dr. Nicole Lurie, assistant secretary for preparedness and response at the Department of Health and Human Services, said the limited supplies made more general distribution impossible. Some doctors said they were satisfied with the existing system.

“I think it’s fairly accessible,” said Dr. Thomas M. File Jr., chief of infectious diseases at Summa Health System in Akron, Ohio, who has treated a pregnant woman with peramivir. Some of the push to make the drug more widely available is coming from investors in BioCryst, including Kleiner Perkins Caufield & Byers, the prominent Silicon Valley venture capital firm. John Doerr, the company’s technology guru, was in Washington this week making the case for greater availability of peramivir, according to a person who met with him there.

Other investors — both supporters of BioCryst stock and those betting the price will fall — have commented at government meetings on flu preparations, often without revealing their financial interests.

Anecdotes like Ms. Gurno’s aside, the efficacy of peramivir is still in question, according to the government. While some clinical trials showed the drug had an effect in resolving flu symptoms, others did not show statistically significant differences between peramivir and either a placebo or Tamiflu.

The question for both investors and federal authorities is how much more of the drug BioCryst can sell. So far, the order has been far less than some investors had hoped, though the price of the order announced Thursday was higher than expected.

Dr. Lurie, the federal official, said there had been 237 requests to use the drug since the emergency use authorization was granted nearly two weeks ago. Many doctors want to provide the treatment for 10 days instead of the recommended 5, she said, so the 10,000 courses the government ordered might actually treat as few as 5,000 patients.

The government has the right to buy up to 30,000 more treatment courses at the same price.

BioCryst says it will have perhaps 40,000 more treatment courses available within a few weeks, and a total of 120,000 by the end of the year. It also says it has signed up partners to try to win sales in Brazil, Mexico, Israel and China.

“We’re getting a lot of interest from countries outside the United States,” Jon P. Stonehouse, the company’s chief executive, said in an interview Thursday.

Shionogi, a Japanese drug company with licensing rights from BioCryst, ran its own clinical trials and this week applied for approval to sell the drug in Japan as a treatment for everyday flu, in competition with Tamiflu.

But in the United States, BioCryst plans to try to win approval for treatment only of hospitalized patients. It is now beginning phase 3 trials — the last stage before seeking F.D.A. approval.

Virtually all the money to develop the drug comes from $180 million in grants from the Department of Health and Human Services. Yet the company gets to keep as profit anything it makes from selling the drug to the federal government or to other governments. Mr. Stonehouse defended the price of the drug, saying it would save the health system money by getting patients out of intensive care earlier. “The cost of being in the I.C.U. and on a ventilator is extremely high,” he said.

But peramivir will soon have competition. The federal government said late Thursday that it had also ordered 10,000 treatment courses each of intravenous versions of Tamiflu and Relenza, with options to buy 30,000 more courses of each.

Those drugs could not be used, however, until they received emergency use authorizations from the F.D.A.

The government is paying an average of only $450 a course for those other drugs — only one-fifth of what it is paying for peramivir.

Right now, Dr. Lurie said, peramivir is the only drug that can be used intravenously so the government had to pay a high price. “I would say that one of the things that happens in the market when you have competition is that the price drops.”

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Will New Anemia Drug Top Current Treatments?

A new drug designed to treat patients with a rare form of anemia could possibly have wider applications, perhaps replacing other anemia treatments that have been linked to an increased risk of death, cancer and stroke, experts say.

The drug, Hematide, was successful in treating patients who have pure red-cell aplasia, a condition caused by antibodies to a hormone needed to produce red blood cells. It's a "devastating condition that many patients have died from and ties patients to blood transfusions every two weeks for the rest of their life," said lead researcher Dr. Iain C. Macdougall, from King's College Hospital, London.

But the real breakthrough is being able to make drugs in a new way that could have wide application for many diseases, he said.

"It is possible to create drugs that are cheaper and simpler to make," he said. "The question is whether all expensive protein therapeutics can get peptide mimetics. This is what we call a peptide mimetic -- it mimics the protein," he said.

The report is published in the Nov. 5 issue of the New England Journal of Medicine.

For the study, Macdougall's group treated 14 patients with chronic kidney disease who were anemic because of antibodies that prevented other anemia treatments from making red blood cells.

Twelve weeks after the first dose of Hematide, a synthetic peptide-based erythropoietin agonist, none of the patients needed blood transfusions, the researchers found. In addition, the amount of antierythropoietin antibodies decreased and in six patients became undetectable.

One patient developed antibodies to the drug and had to go back to receiving regular transfusions, the researchers noted.

Some experts think Hematide could replace other anemia drugs used to treat anemia caused by kidney disease or chemotherapy, if it turns out to have fewer adverse side effects. However, Macdougall is cautious about making such claims.

"That's speculation," he said. "There is no way our paper suggests that, and there is no hard scientific evidence that would suggest that."

Dr. H. Franklin Bunn, research director of the hematology division at Brigham and Women's Hospital and Harvard Medical School, both in Boston, and author of an accompanying journal editorial, said that "down the road this drug may have some advantages over regular erythropoietin drugs."

But he said it is too early to tell whether Hematide can replace the erythropoietin drugs Procrit and Aranesp, which are associated with heightened risk of heart attack, cancer and stroke.

For now, Hematide is "the treatment of choice for patients who have developed antibodies against erythropoietin. And that's all you can say at this point. but I am curious to find out whether this drug will be able to be used and be effective and safe in a wider setting," Bunn said.

Other experts are more optimistic. Dr. Charles Bennett, a hematologist/oncologist at Northwestern Memorial Hospital and Jesse Brown VA Medical Center in Chicago, said that "we are going to look at this in five years and see that it's a breakthrough paper."

It's hoped this drug will take the place of erythropoietin drugs and not have the side effects associated with them, Bennett said.

Dr. Jochen Reiser, chief of nephrology and hypertension at the Leonard M. Miller School of Medicine of the University of Miami, voiced similar hopes. "In some patients where Procrit has proven not to be beneficial, maybe this peptide has an additional potential that Procrit doesn't have or works better."

Whether Hematide will overcome the side effects associated with Procrit is still hard to say, Reiser said.

"If the side effects come from increasing the amount of red blood cells, then no, it would have the same effects, but if they are substance-related side effects that have nothing to do with the red blood cells, then yes -- that can be looked at now," he said.

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Pirfenidone for the Treatment of Patients with IPF

InterMune, Inc. today announced that it has submitted an electronic New Drug Application(NDA) with the U.S. Food and Drug Administration (FDA) seeking approval to market pirfenidone for the treatment of patients with idiopathic pulmonary fibrosis (IPF). Pirfenidone has been granted Orphan Drug and Fast Track designation by the FDA, and also has been granted Orphan Drug status in Europe.

"IPF is a rapidly and uniformly fatal disease. Sadly, there are no medicines approved for the approximately 100,000 Americans who suffer from this terrible disease," said Dan Welch, Chairman, Chief Executive Officer and President of InterMune. "InterMune has dedicated almost ten years to the development of new medicines for patients with IPF. We are very proud to have submitted the first NDA ever submitted to the FDA for a medicine to treat IPF patients."

About Pirfenidone

Preclinical and in-vitro evidence had shown that pirfenidone has both anti-fibrotic and anti-inflammatory effects. Results from three adequate and well-controlled Phase 3 studies have shown evidence of a treatment effect in IPF patients and the compound has been safe and generally well tolerated, with side effects including photosensitivity rash and gastrointestinal symptoms.

InterMune licensed pirfenidone from Marnac, Inc. and its co-licensor, KDL GmbH, in 2002 and in 2007 purchased from Marnac and KDL the rights to sell the compound in the United States, Europe and other territories except in Japan, Taiwan and South Korea where rights to the molecule were licensed by Marnac and KDL to Shionogi & Co. Ltd. of Japan. In October of 2008, pirfenidone was approved for use in IPF patients in Japan and is marketed as Pirespa® by
Shionogi in that country.

About IPF

Idiopathic pulmonary fibrosis (IPF) is a disabling and ultimately fatal disease that affects approximately 200,000 patients in the United States and Europe combined, with approximately 30,000 new cases reported per year in each of the United States and Europe.

IPF is characterized by inflammation and scarring (fibrosis) in the lungs, hindering the ability to process oxygen and causing shortness of breath (dyspnea) and cough and is a progressive disease, meaning that over time, lung scarring and symptoms increase in severity. The median survival time from diagnosis is two to five years, with a five-year survival rate of approximately 20%. Patients diagnosed with IPF are usually between the ages of 40 and 70, with a median age of 63 years and the disease tends to affect slightly more men than women. There are no medicines approved in the United States or Europe for IPF.

About InterMune

InterMune is a biotechnology company focused on the research, development and commercialization of innovative therapies in pulmonology and hepatology. InterMune has an R&D portfolio addressing idiopathic pulmonary fibrosis (IPF) and hepatitis C virus (HCV) infections. The pulmonology portfolio includes pirfenidone for which InterMune has completed a Phase 3 program in patients with IPF (CAPACITY) and has submitted a New Drug Application (NDA) to the FDA. The hepatology portfolio includes the HCV protease inhibitor compound RG7227 (ITMN-191) that entered Phase 2b in August of 2009 and a second-generation HCV protease inhibitor research program.

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New drug for leukaemia

The Daily Mail says that British scientists have created a “new drug that 'kills' leukaemia - even in worst affected adults”. The research behind this news found that the drug, called PBOX-15, could kill cancerous cells extracted from people with chronic lymphocytic leukaemia (CLL). The drug also killed cells that were resistant to an approved anti-cancer drug.

Several newspapers have discussed this laboratory study, highlighting the fact that this was early research and that any potential treatment using this chemical is a long way off. A quick glance at the Daily Mail’s headline may suggest that this drug has been tested in patients, but this was not the case. Instead, the study was carried out on cells grown in the laboratory and cannot be used to predict what other effects PBOX-15 might have in a living body.

Much more research will be needed to identify how safe and effective this drug is in animals before it can be potentially tested in humans. Drugs that show promise in the lab may prove unsafe or ineffective in later animal testing, which means they will not get developed into to useable medications.

Where did the story come from?

The research was carried out by Dr Anthony M McElligott and colleagues at Trinity College
Dublin and other centres in Ireland, Northern Ireland and Italy. It was published in the peer-reviewed journal Cancer Research. The research was funded by Enterprise Ireland, Cancer Research Ireland and the Higher Education Authority of Ireland.

The newspapers correctly reported that the development of this drug is at an early stage and that it may be years before it can be used. However, the Daily Mail’s headline that the drug “‘kills’ leukaemia - even in worst affected adults” may lead people to believe that this drug has been tested in patients, which is not the case. Headlines in other news sources, such as BBC News and The Daily Telegraph, are more accurate and simply stating that the drug has been shown to kill leukaemia cells.

What kind of research was this?

This laboratory study looked at the effects of a chemical called PBOX-15 (pyrrolo-1,5-benzoxazepine-15) on leukaemia cells extracted from people with chronic lymphocytic leukaemia (CLL). The authors say that new treatments are needed for CLL, particularly for patients who do not respond well to existing therapies.

There are many stages involved in developing and testing potential new drugs. The earlier stages usually involve laboratory studies, such as this one, which are used to identify the effects of the drug on affected cells and tissues. This early research is important for directing further study but cannot reliably predict what other effects a drug such as PBOX-15 might have in a living body. This study will need to be followed up with further research to assess how safe and effective the drug is in animals.

What did the research involve?

The researchers took blood samples from 55 CLL patients who had not yet begun treatment for CLL. From these samples, they isolated white blood cells that were affected by CLL and exposed them to PBOX-15 in the laboratory to see whether the cells died. The researchers also compared the effects of PBOX-15 with the effects of fludarabine on CLL cells, a chemotherapy drug used to treat CLL. They also carried out experiments to look at the effect of PBOX-15 on normal bone marrow cells taken from three healthy donors.

What were the basic results?

The researchers found that PBOX-15 could kill CLL cells in the laboratory. The drug could also kill CLL cells whose characteristics would normally be expected to cause a poor outcome of the disease. Comparison testing showed that PBOX-15 was more effective than fludarabine at killing fludarabine-sensitive CLL cells. PBOX-15 also killed CLL cells that had a genetic mutation that made them resistant to fludarabine treatment. Testing on three donor bone marrow samples found that PBOX-15 was more toxic to CLL cells than to normal bone marrow cells.

How did the researchers interpret the results?

The researchers concluded that PBOX-15 can kill both high-risk and low-risk CLL cells, and shows “significant clinical potential”.

Conclusion

Although the study shows that PBOX-15 can kill isolated human CLL cells in the laboratory, it cannot reliably predict what other effects it might have in a living body.

There are many stages to developing and testing potential new drugs, which can take many years and are not guaranteed to be successful. The early development stages involve laboratory studies such as this one, which are used identify the effects of the drug on affected cells and tissues. These early tests are important to establish whether future research is worthwhile.

Following the results of this initial study, the drug seems to be a candidate for further research, which would need to identify how safe and effective this drug is in animals before it could be tested in humans.

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New Lupus Drug Heads to FDA for Approval

An experimental lupus drug has just met another key goal in its journey to becoming the first approved treatment for the disease in over 50 years. The target of the new drug, known as Benlysta, is to suppress the response of the body’s immune system to lupus in an attempt to control the actions of a protein that becomes overactive in lupus patients.

Hopes were that the study data from a clinical program called Bliss-76 would confirm the optimistic results of a prior late-stage study. In actuality, the indications of the Bliss-76 data were that a 10-milligram dose of Benlysta, accompanied by therapy with steroids, resulted in improvement among 43 percent of lupus patients taking a higher dose, as well as a 40.6 percent of those taking a lower dose. This is compared to improvements observed in only 33.8 percent of patients taking a placebo.

Makers of the drug, Human Genome Sciences (HGS) based in Rockville, Maryland, and GlaxoSmithKline based in the United Kingdom, now plan to seek the regulatory approval of the U.S. Food and Drug Administration in the early months of 2010. If approved, Benlysta could be available on the market by late next year.

In a statement, HGS President and CEO H. Thomas Watkins said, “The Bliss-76 results confirm our view that Benlysta has the potential to become the first new approved drug in decades for people living with systemic lupus.” He then added, “We take great pride in the innovation and scientific rigor that has made it possible to bring Benlysta to this point.”

The Bliss-76 study was the second of two late-stage studies, and involved 865 patients who were treated and monitored over a period of one year. In October of this year, the first late-stage study, called Bliss-52, involved over 800 patients in Asia, South America and Eastern Europe, and has positive results in meeting several key goals.

Lupus is an inflammatory, autoimmune condition, in which the body’s defense system against pathogens attacks the body’s own tissue causing health issues such as rashes, mouth sores, arthritis, and kidney damage among other problems. Because the illness manifests itself differently in each patient, it is difficult to develop effective treatments against the disease. According to Watkins, “We’ve got a good chance to redefine the standard of care for patients living with lupus.”

While one recent study estimated that 322,000 Americans most likely have the most common form of lupus known as systemic lupus erythematosus, the Lupus Foundation of America estimates that about 1.5 million Americans have some form of the disease. According to Sandra C. Raymond, the organization's president and chief executive, “Individuals with lupus and their families have waited more than 50 years to hear that it is possible to develop therapies that control the disease.” She went on to say, “We believe that this is a significant first step in developing the full arsenal of therapies and personalized treatment lupus requires.”

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New Drug Boosts HCV Clearance

Most hepatitis C patients who are initially unresponsive to standard therapy were able to achieve sustained virologic responses when the investigational drug boceprevir was added, a researcher reported here.

Sustained responses were seen in 55% of patients receiving 44 weeks of boceprevir after showing no virologic response to four weeks of pegylated interferon-alfa-2b (PEGIntron) and ribavirin (Rebetol) in a Phase II trial, said Paul Kwo, MD, of Indiana University in Indianapolis.

Kwo, speaking here at the American Association for the Study of Liver Disease meeting, was reporting on two secondary analyses of data from the SPRINT-1 trial of boceprevir, an inhibitor of the hepatitis C virus (HCV) NS3 protease enzyme.

He had presented the main findings of the 520-patient study earlier this year at the European Association for the Study of the Liver meeting in Copenhagen.

Boceprevir is one of two HCV protease inhibitors in late-stage development, the other being telaprevir. (See DDW: Telaprevir Improves HCV Clearance in Resistant Patients) Phase III trials of both drugs are now under way.

In the SPRINT-1 trial, treatment-naive patients were randomized to five treatment arms, including one in which patients only received pegylated interferon and ribavirin, two involving immediate treatment with all three agents, and two in which boceprevir started after an initial, four-week lead-in with interferon and ribavirin.

All patients had HCV genotype 1a or 1b, mostly the former.

The secondary analyses reported here focused only this last treatment strategy, with patients receiving either 24 or 44 weeks of triple therapy following the four-week, two-drug lead-in.

Boceprevir was dosed at 800 mg three times a day.

In patients with no response to the lead-in -- defined as a reduction in HCV RNA loads of less than ten-fold -- 25% of those receiving boceprevir for 24 weeks still showed viral clearance after an additional 24 weeks of follow-up.

With 55% of initial null responders receiving the drug for 44 weeks showing long-lasting viral clearance, the longer therapy appeared to be more effective, Kwo said.

He also noted that boceprevir for both durations boosted response rates well above what would normally be expected from standard therapy in patients without strong responses in the first four weeks.

He cited results from an earlier large trial in which less than 5% of early nonresponders to standard therapy eventually developed sustained responses.

Among patients showing strong responses in the first four weeks of interferon and ribavirin, sustained responses were seen in most.

More than 80% of those with initial reductions of three to four orders of magnitude in viral RNA levels had sustained responses, as did nearly 100% of those with reductions of at least four orders of magnitude or whose viral RNA became undetectable in the first four weeks.

Duration of boceprevir treatment appeared to make no difference in sustained virologic response rates in these patients.

But Kwo cautioned that the findings in these analyses involved relatively small numbers of patients. Only about 50 patients were considered null responders to the lead-in treatment, and similar numbers had relatively strong initial responses.

Overall, adding boceprevir after the four-week lead-in led to sustained responses in 56% of patients receiving boceprevir for 24 weeks, and in 75% of those taking the drug for 44 weeks, Kwo said. Both response rates were significantly (P<0.01)>

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Pill for schizophrenia from Vanda Pharmaceuticals is due out in 2010

Early next year, if all goes according to plan, doctors will be able to prescribe a new antipsychotic drug for patients with schizophrenia. When that happens, investors in a local pharmaceutical firm will surely breathe a sigh of relief.

While it's almost certain that the compound known as Fanapt will reach pharmacy shelves, the drug's future was anything but clear for most of its 13-year existence. Rockville-based Vanda Pharmaceuticals toiled for years on its development, even after larger drugmakers lost interest and the Food and Drug Administration gave the product a thumbs-down.

"Last year at this time, nobody believed in the company, and nobody believed in the compound," said Mihael H. Polymeropoulos, Vanda's president and chief executive. Today, the company has a deal for Fanapt worth nearly half a billion dollars.

Shareholders fled the company in droves last year after a negative ruling from the FDA, and Polymeropoulos says he doesn't blame them. He founded Vanda after earlier careers, mostly in Washington, in the health-care industry. He'd never heard of a case in which the FDA reversed a decision on a drug, but that's what happened after Vanda told the agency that it had misinterpreted some data.

Polymeropoulos says he never wavered in his belief that Fanapt could help people.

"People in my field hold the belief that there are scientific truths to be found, and once you solve the problems they can impact health care," he said. "It is that conviction that allows people to commit themselves for a long period of time despite adversity."

In any case, the chief executive said, it's the prospect of helping schizophrenic patients and not the lure of potential profit that serves as the company's driving force. "My belief is you don't build companies to make money just like a bank," he said. "A health-care company's mission is to develop treatments for people."

Fanapt, like other antipsychotic drugs, controls the way information is carried from one nerve cell to another and reduces the activities of some brain activity associated with schizophrenia. The compound blocks a different combination of neurotransmitters than earlier-generation antipsychotic drugs, Polymeropoulos said. The company says Fanapt targets a more relevant set of neurotransmitter receptors, so that patients are likely to suffer fewer side effects than with other medications.

Polymeropoulos has worked on the drug's development for 10 years, including during his earlier career as head of the Novartis pharmacogenetics unit. The giant drugmaker owned the drug early in its development but sold it when it was streamlining its product pipeline. Polymeropoulos and his start-up acquired the rights.

Today, Novartis is back in the Fanapt business. Under the terms of a deal announced last month, Vanda will receive an upfront payment of $200 million from Novartis for rights to commercialize Fanapt in the United States and Canada. As long as Vanda meets certain development milestones, the company will be eligible for additional payments totaling up to $265 million. Not bad for a company that lost $12.4 million during its second quarter this year, and faced shrinking cash resources of under $30 million.

Fanapt's somewhat arduous path to the marketplace isn't unusual, said Sunil Bhonsle, president of Titan Pharmaceuticals, a company that owned the rights to Fanapt before Novartis and will receive royalty payments for the drug when it goes on sale. It is not uncommon for companies to end up with too many products under development, he said, and for some of those products to go to smaller firms for development.

"There tends to be a pattern where smaller companies do a lot of the innovation, then the larger companies acquire them at a certain point," he said.

Even during a tough economic climate that has most sectors tightening belts, the pharmaceutical industry is on the lookout for potentially lucrative experimental drugs to invest in. Older drug products eventually go off-patent and face new competition from cheaper, generic versions.

According to a recent report by Dealogic Revenue Analytics, the dollar value of acquisitions by pharmaceutical and biotechnology firms is up 23 percent this year. Across all industries, by comparison, that figure is down 35 percent -- at $1.7 trillion, compared with last year's $2.6 trillion.

Vanda is one of the first Washington area firms to benefit from an ongoing global spending spree in this industry, and there are periodic rumors about an acquisition of Rockville-based Human Genome Sciences. As recently as August, HGS's stock jumped 14 percent on speculation that the company's sometime partner GlaxoSmithKline intended to buy the firm. HGS has a potential hit with an experimental drug to treat Lupus.

If other Washington area companies have yet to spark that sort of speculation, it might be because they don't have a product far enough along to tempt buyers, some say.

"In Maryland, our companies are not mature enough yet," said John Holaday, who has started three biotech companies. "We don't have a lot of companies that have products in late-stage trials or products that are already in the marketplace."

Polymeropoulos said his work with Fanapt isn't done yet. The company's next task is to help Novartis launch the drug, in tablet form, to pharmacies starting early next year. After that, he and his firm intend to create a version of the drug that patients will receive as a once-a-month injection.

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