Novel class of antimicrobials could be effective in fighting drug-resistant MRSA infection

A novel class of antimicrobials that inhibits the function of a key disease-causing component of bacteria could be effective in fighting methicillin-resistant Staphylococcus aureus (MRSA), one of the major drug-resistant bacterial pathogens, according to researchers at Georgia State University.

Their study showed that small molecule analogs that target the functions of SecA, a central part of the general bacterial secretion system required for viability and virulence, have potent antimicrobial activities, reduce the secretion of toxins and can overcome the effect of efflux pumps, which are responsible for multi-drug resistance.

Their findings indicate that targeting SecA is an attractive antimicrobial strategy against MRSA and may be several times more effective than the antibiotics now available for treating the infection.

MRSA causes serious hospital and community-acquired infections. Healthcare-associated MRSA infections are typically linked to invasive procedures or devices, such as surgeries, intravenous tubing or artificial joints. Community-acquired MRSA often begins as a skin boil and is spread by skin-to-skin contact. Individuals at risk include competitive wrestlers, child care workers and those living in crowded conditions.

"We've found that SecA inhibitors are broad-spectrum antimicrobials and are very effective against strains of bacteria that are resistant to existing antibiotics," said Binghe Wang, Regents' Professor of Chemistry at Georgia State, Georgia Research Alliance Eminent Scholar in Drug Discovery and Georgia Cancer Coalition Distinguished Cancer Scholar. He co-led the study with Phang C. Tai, Regents' Professor of Biology at Georgia State, who is an expert on the functions of SecA in bacteria. Their findings were published in the journal Bioorganic & Medicinal Chemistry in November.

Novel class of antimicrobials could be effective in fighting drug-resistant MRSA infection

Lexicon announces top-line results from TELECAST Phase 3 study of telotristat etiprate

Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX) announced today that top-line data from its TELECAST Phase 3 study showed results of telotristat etiprate in treating carcinoid syndrome in cancer patients with metastatic neuroendocrine tumors consistent with the clinical benefit observed in its pivotal TELESTAR study. The TELECAST study was designed as a companion to TELESTAR primarily to provide additional safety exposure while further evaluating telotristat etiprate's activity in carcinoid syndrome. TELECAST mostly enrolled patients treated with somatostatin analog (SSA) therapy, the current standard of care, with carcinoid syndrome characterized by less severe bowel movement frequency than those patients in the TELESTAR study, together with a smaller number of carcinoid syndrome patients not treated with SSA therapy.

Telotristat etiprate met the study's primary efficacy endpoint, the percent change from baseline in urinary 5-hydroxyindoleacetic acid (5-HIAA, the main metabolite of serotonin) at week 12, the final week of the double-blind treatment portion of the study (p<0.001 for both dose arms compared to placebo). In addition, despite the lower baseline bowel movement frequency than in TELESTAR, telotristat etiprate achieved statistically significant reductions in daily bowel movement frequency over the 12 weeks of the study (p=0.004 for the 250 mg dose arm and p<0.001 for the 500 mg dose arm compared to placebo). Safety and tolerability was one of the primary objectives of the TELECAST study, and telotristat etiprate was well tolerated during the double-blind treatment period, with profiles similar to placebo for both the 250 mg and 500 mg dose arms and no overall differences observed in gastrointestinal disorders or psychiatric disorders, including changes in mood.

"We are very pleased with the efficacy and safety results of telotristat etiprate in this study, notably including evidence of benefit in a patient population whose bowel movement frequency was lower at baseline than was the case in TELESTAR," said Lexicon Executive Vice President and Chief Medical Officer Pablo Lapuerta, M.D. "TELECAST was intended to complement our pivotal Phase 3 trial, TELESTAR. We now have experience in more than 200 patients with carcinoid syndrome, with TELECAST contributing consistent efficacy data and favorable safety results. The data further support that the compound is acting directly on the cause of carcinoid syndrome, by reducing serotonin production within tumor cells."

Lexicon announces top-line results from TELECAST Phase 3 study of telotristat etiprate

Seaweed extract shows promise in treating H. pylori-related diseases, gastric cancer

Effective treatments for H. pylori are limited with a rising percentage of treatment failures, primarily due to antibiotic resistance. Alternative and additional treatment options are widely recognised as a significant global healthcare issue. Results from this latest study show that fucoidan, a sulphated polysaccharide found in brown seaweed, may offer a solution.

The study took place at the University of Western Australia in the laboratories of Nobel Prize Laureate Professor Barry Marshall, who is recognised for his discovery of H. pylori and its role in gastritis and peptic ulcer disease. The research involved testing fucoidan extracts derived from the Fucus vesiculosus and Undaria pinnatifida species of brown seaweed. These certified organic, high-purity extracts were developed and produced by Australian biotechnology company, Marinova Pty Ltd.

The in vitro studies showed fucoidan extracts are extremely effective at dislodging H. pylori from infected human stomach cancer cells. This significant result positions fucoidan as a potential alternative to the increasingly inadequate antibiotic treatments.

Dr Alfred Chin-Yen Tay, Research Associate at The Marshall Centre for Infectious Diseases who supervised the study, said:

These research findings are an encouraging step forward in the control of H. pylori-related diseases. Fucoidan is a natural extract and oral delivery to the stomach is simple. It would be a welcome option for suffering patients, especially before having to undergo invasive diagnosis and treatment options.

Seaweed extract shows promise in treating H. pylori-related diseases, gastric cancer

Second-line bosutinib offers ‘durable’ response for chronic phase CML patients

In continuation of my update on bosutinib

Four-year results for an ongoing study of second-line bosutinib indicate that the tyrosine kinase inhibitor (TKI) offers long-term efficacy with manageable side effects for patients with chronic phase chronic myeloid leukaemia (CP CML).

“Overall, these findings highlight the therapeutic potential of bosutinib as second-line therapy in IM-R [imatinib resistant] or IM-I [imatinib intolerant] CP CML patients”, say Tim Brümmendorf (Universitätsklinikum der RWTH Aachen, Germany) and co-investigators.

In the phase I/II trial, bosutinib 500 mg/day was given for a median of 24.8 months to 196 IM-R and 90 IM-I patients; 59% of 264 evaluable patients achieved or maintained a major cytogenetic response (MCyR) for at least 4 weeks, including 59% of the IM-R and 61% of the IM-I groups.

Of the 248 patients without a complete cytogenetic response (CCyR) at baseline, 57% achieved a MCyR and 47% a CCyR during bosutinib therapy. And 14 of the 16 patients with a baseline CCyR maintained this response for between 12 and 288 weeks; two discontinued bosutinib because of adverse events (AEs) and were not reassessed.

The median times to MCyR and CCyR were 12.3 and 24.0 weeks, respectively.

The 4-year cumulative incidences of MCyR and CCyR, at 59% and 49%, respectively, were similar to the previously reported 2-year figures of 59% and 48%, prompting the authors to suggest that “most initial responses occur within 2 years from bosutinib treatment initiation.”

The rate of cumulative progression or death during treatment was an estimated 19% at 4 years.

The Kaplan–Meier-estimated probability of maintaining MCyR at 4 years was found to be high for the whole population and the IM-R and IM-I groups, at 74.5%, 69.3% and 86.3%, respectively, and the median duration of this response had not yet been reached.

Second-line bosutinib offers ‘durable’ response for chronic phase CML patients

New data shows ALS-008176 drug safe and effective against RSV infection

Alios BioPharma, Inc., part of the Janssen Pharmaceutical Companies announced that the New England Journal of Medicine (NEJM) will publish findings from a respiratory syncytial virus (RSV) challenge study for ALS-008176, a cytidine nucleoside analog with activity against RSV. Among infants and young children, RSV is the leading cause of severe respiratory illness and remains the most frequent cause of hospitalization in industrialized countries. This Phase 2a study has now established human proof-of-concept for the antiviral activity of ALS-008176 in healthy adults and highlights its potential as a therapy for managing clinical disease in naturally infected patients.

“The data suggest that ALS-008176 has the potential to be a safe and effective treatment for RSV infection. The primary endpoint of the study was met and ALS-008176 significantly reduced viral load and symptoms of disease severity compared to placebo,” said John DeVincenzo, M.D., the lead study author and Professor of Pediatrics, and Professor of Microbiology, Immunology and Biochemistry at the University of Tennessee Health Science Center, and Medical Director of Molecular and Viral Diagnostics at Le Bonheur Children's Hospital. “ALS-008176 can inhibit the replication of RSV even if the cells of the respiratory tract have already been infected with the virus. As a result, this treatment has an antiviral effect and is likely to be effective even if started at a later stage of RSV infection.”

In this randomized, double-blind study, 62 healthy volunteers were inoculated with RSV and subsequently randomized to receive ALS-008176 or placebo. Compared to placebo, treatment with ALS-00876 resulted in a significant reduction of viral load (73-88% reduction in viral load area under the curve) and faster viral clearance (1.3–2.3 days vs 7.2 days) versus placebo. At the time that the peak viral load occurred in the placebo group, the mean viral load in each of the three ALS-007186 treatment groups was more than one thousand times lower. In addition, statistically significant reductions in symptom scores and a reduction of the amount of congesting respiratory secretions were also observed.

New data shows ALS-008176 drug safe and effective against RSV infection

Takeda receives FDA approval for NINLARO (ixazomib) capsules to treat patients with multiple myeloma

Takeda Pharmaceutical Company Limited (TSE: 4502) today announced that the U.S. Food and Drug Administration (FDA) has approved NINLARO® (ixazomib) capsules, the first and only oral proteasome inhibitor, indicated in combination with lenalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least one prior therapy. NINLARO is a once-weekly pill.

Takeda submitted a New Drug Application for NINLARO to the FDA in July 2015, and in September NINLARO was granted Priority Review status with a PDUFA date of March 10, 2016, reflecting the profound and continuing unmet need for new treatments for multiple myeloma, a devastating, relapsing and incurable rare cancer.

“With the approval of NINLARO, we can now offer patients a once-weekly oral proteasome inhibitor as part of a highly active triplet therapy,” said Paul Richardson, M.D., Clinical Program Leader and Director of Clinical Research, Jerome Lipper Multiple Myeloma Center Institute Physician at Dana-Farber Cancer Institute, and investigator for TOURMALINE-MM1, the pivotal Phase 3 trial on which today’s approval is based. “We, as investigators of the TOURMALINE-MM1 trial, felt it was vital to conduct a comprehensive ‘real world’ evaluation of this combination that included some of the most common patient types in the relapsed/refractory multiple myeloma setting, such as older patients, patients with moderate renal impairment, light chain disease, and high risk cytogenetics. Further, we treated patients until disease progression to determine the sustainability of NINLARO in treating their relapsed/refractory disease. The TOURMALINE-MM1 data demonstrate convincingly that oral NINLARO-based triplet treatment is effective at extending progression-free survival, over and above the clinical benefit seen with lenalidomide and dexamethasone, with a tolerable safety profile.”

Takeda receives FDA approval for NINLARO (ixazomib) capsules to treat patients with multiple myeloma

New drug combination may reduce need for complex regimens to treat hepatitis C

In continuation of my update on sofosbuvir

  

The prognosis for people with hepatitis C has improved dramatically in the last few years, thanks to the introduction of direct-acting anti-viral medications, including Harvoni (the brand name for a combination of ledipasvir and sofosbuvir) and Viekira Pack (a mix of ombitasvir, paritaprevir, ritonavir and dasabuvir). These drugs — which block the hepatitis C virus from multiplying — boast cure rates of better than 90 percent. In addition, they are well-tolerated in most patients, causing only minor side effects.

Despite these major advances, the quest for better hepatitis C medications is not yet over. Drug makers continue to test new drugs to overcome limitations in treating this virus, which can cause liver cirrhosis (or scarring) and failure. About 2.7 million people in the U.S. are infected with the virus, with nearly 30,000 cases occurring in 2013 alone, according to the Centers for Disease Control and Prevention.

"The current medicines are very effective, but physicians sometimes have to tailor the regimen or the length of treatment based on patient characteristics, such as whether the patient has liver cirrhosis or has failed prior therapy," says Nancy S. Reau, MD, chief of the Section of Hepatology at Rush University Medical Center.

Another treatment factor is the type of hepatitis C a patient has. The virus has six different strains, called genotypes.

Now a simplified way of treating all hepatitis C patients may be approaching. Reau participated in a phase III clinical trial of a combination of Solvadi (sofosbuvir) with the investigational drug velpatasvir on patients with genotypes two and three. As described in an article published online on Nov. 17 in the New England Journal of Medicine, the study found that 12 weeks of sofosbuvir-velpatasvir produced higher cure rates in patients with these two genotypes — including those who had cirrhosis or had failed older treatments — than a similar therapy (sofosbuvir-ribavirin)

New drug combination may reduce need for complex regimens to treat hepatitis C

Sildenafil drug improves insulin sensitivity in people at risk for diabetes

Sildenafil inhibits an enzyme called phosphodiesterase 5 (PDE5), resulting in relaxation of smooth muscle, vasodilation and increased blood flow. Sildenafil is used to treat erectile dysfunction and pulmonary arterial hypertension.

Animal studies suggest that sildenafil also can improve insulin sensitivity, the uptake of glucose from the bloodstream by muscle. This action can lower the level of circulating glucose, and potentially reduce the risk of diabetes.

In the current study, overweight individuals with prediabetes were randomly assigned to receive sildenafil or placebo (inactive drug) for three months. Of the 42 subjects who completed the study, those treated with sildenafil were significantly more sensitive to insulin, the researchers reported in today's Journal of Clinical Endocrinology and Metabolism.

Sildenafil drug improves insulin sensitivity in people at risk for diabetes

Identifying new mechanism for aspirin in cancer prevention

In a study published in the journal of Cancer Epidemiology, Biomarkers, and Prevention, Ulrich and her collaborators used a new technique, metabolite profiling, to identify a biochemical pathway previously unknown to be regulated by aspirin. Specifically, the researchers found that aspirin substantially decreases the level of a chemical called 2-hydroxyglutarate in the blood of healthy volunteers and in two colorectal cancer cell lines. This chemical, 2-hydroxyglutarate, is considered a driver of cancer development (known as an oncometabolite) because elevated levels have been found in certain cancers of the blood and brain and several groups are currently studying it as a molecule that promotes tumor formation.

Ulrich says the study adds to the overall evidence that aspirin is important for cancer prevention and points to a new pathway that deserves further study in the context of aspirin. "It is really exciting that aspirin, which can work in colorectal cancer prevention, is now linked to a new pathway that has shown to be relevant for cancer formation."

The first part of the study involved looking comprehensively at the metabolic profiles from the blood of 40 individuals who had taken aspirin for 60 days. The design was rigorous, with participants each having a phase with and without aspirin. More than 360 metabolites, or small molecule chemicals such as sugars, amino acids, and vitamins, were analyzed, says Ulrich. "This study covered most of the known biochemical pathways in the body."

Ref : http://cebp.aacrjournals.org/content/early/2015/11/17/1055-9965.EPI-15-0697.abstract?sid=c6453fd8-4a41-4a96-93c4-750c662bace3

Identifying new mechanism for aspirin in cancer prevention

Consuming tree nuts may reduce risk of cardiovascular disease, new study finds

In continuation of my update on the usefulness of walnuts

A new study published in the American Journal of Clinical Nutrition found that consuming tree nuts, such as walnuts, may lower the risk of cardiovascular disease. After conducting a systematic review and meta-analysis of 61 controlled trials, one of the authors, Michael Falk, PhD, Life Sciences Research Organization, found that consuming tree nuts lowers total cholesterol, triglycerides, LDL cholesterol, and ApoB, the primary protein found in LDL cholesterol. These are key factors that are used to evaluate a person's risk of cardiovascular disease. Walnuts were investigated in 21 of the 61 trials, more than any other nut reviewed in this study.

"Our study results further support the growing body of research that tree nuts, such as walnuts, can reduce the risk of cardiovascular diseases," said Dr. Falk. "Tree nuts contain important nutrients such as unsaturated fats, protein, vitamins and minerals. Walnuts are the only nut that provide a significant amount (2.5 grams per one ounce serving) of alpha-linolenic acid (ALA), the plant-based form of omega-3s."

A new study published in the American Journal of Clinical Nutrition found that consuming tree nuts, such as walnuts, may lower the risk of cardiovascular disease. After conducting a systematic review and meta-analysis of 61 controlled trials, one of the authors, Michael Falk, PhD, Life Sciences Research Organization, found that consuming tree nuts lowers total cholesterol, triglycerides, LDL cholesterol, and ApoB, the primary protein found in LDL cholesterol. These are key factors that are used to evaluate a person's risk of cardiovascular disease. Walnuts were investigated in 21 of the 61 trials, more than any other nut reviewed in this study.

"Our study results further support the growing body of research that tree nuts, such as walnuts, can reduce the risk of cardiovascular diseases," said Dr. Falk. "Tree nuts contain important nutrients such as unsaturated fats, protein, vitamins and minerals. Walnuts are the only nut that provide a significant amount (2.5 grams per one ounce serving) of alpha-linolenic acid (ALA), the plant-based form of omega-3s."

Consuming tree nuts may reduce risk of cardiovascular disease, new study finds

Lilly, Merck expand oncology clinical trial collaboration

Eli Lilly and Company (NYSE: LLY) and Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced the extension of an existing collaboration to evaluate the safety and efficacy of the combination of Lilly's ALIMTA® (pemetrexed for injection) and Merck's KEYTRUDA® (pembrolizumab) in a pivotal Phase III study in first-line nonsquamous non-small cell lung cancer (NSCLC). The study will be sponsored by Merck and will be open to patients with NSCLC in the first-line setting, regardless of PD-L1 status. Financial details of the collaboration were not disclosed.

The expansion of this oncology clinical trial collaboration comes following the release of encouraging data from a Phase I study, presented earlier this year at the 16th World Congress on Lung Cancer, which evaluated pemetrexed, carboplatin and pembrolizumab in first-line nonsquamous NSCLC.

Pemetrexed is a leading therapeutic option used in combination with platinum-based therapies in this setting, making it an ideal candidate for combination studies with immunotherapy treatments. Pembrolizumab is a humanized monoclonal antibody that works by increasing the ability of the body's immune system to help detect and fight tumor cells. Pembrolizumab blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes, which may affect both tumor cells and healthy cells – and is currently approved as a single-agent therapy for certain types of NSCLC.

Lilly, Merck expand oncology clinical trial collaboration

Anavex reports safety and efficacy data of ANAVEX 2-73 Phase 2a trial in Alzheimer’s patients

ANAVEX 2-73 (Tetrahydro-N,N-dimethyl-2,2-diphenyl-3-furanmethanamine hydrochloride) is a σ1 agonist (IC50 = 860 nM); also displays affinity for muscarinic M1-M4 receptors (Ki values < 500 nM), but not for σ2 receptors. Exhibits neuroprotective effects, prevents tau hyperphosphorylation, and attenuates scopolamine- and (+)-MK 801-induced learning deficits in a mouse model of amyloid toxicity.

Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq: AVXL). On Saturday, investigators presented positive safety and cognitive efficacy data for ANAVEX 2-73, the Company’s lead investigational oral treatment for Alzheimer’s disease targeting sigma-1 and muscarinic receptors, which are believed to reduce protein misfolding including reduction of beta amyloid, tau protein and inflammation at the international CTAD 2015 conference in Barcelona, Spain.

Initial analysis of Phase 2a data demonstrated that the study met the primary objective of safety as ANAVEX 2-73 was well tolerated and results were consistent with prior Phase 1 clinical trial data. The secondary objectives were also met, with ANAVEX 2-73 showing cognitive improvement across all doses in all exploratory cognitive measurements, including the Cogstate battery, Mini Mental State Examination (MMSE), event-related potentials (ERP) and P300 tests, which consistently demonstrated improvements from baseline in the completed PART A portion of the study in 32 mild-to-moderate Alzheimer’s patients. Even though PART A was designed as a 5 week bioavailability trial that included a built-in wash-out period of 12 days and without an optimized dosing regimen, several Cogstate tests demonstrated highly statistically significant improvements. This finding was supported by a trend towards improvement in median MMSE score, which increased by +1.5 over baseline at week 5.

Positive effects on cognition were further supported by highly statistically significant biomarker effects of treatment at week 5 on one event-related potential (ERP) measure with a p-value of p<0.0007 and improvement in the P300 signal. The ERP biomarker scores improved compared to the initial data presented at AAIC in Washington, DC in July 2015, by which time not all patients had yet completed PART A.

All patients who completed PART A volunteered to continue in the longitudinal PART B extension study.

In the interim analysis of the first 14 patients at week 12, the PART B portion of the study demonstrated a positive trend towards improvement over 12 weeks of ANAVEX 2-73 treatment on the secondary functional outcome measure, the Alzheimer's Disease Co-operative Study - Activities of Daily Living Inventory (ADCS-ADL) by +3.21 points.

Anavex reports safety and efficacy data of ANAVEX 2-73 Phase 2a trial in Alzheimer’s patients

Cotellic (cobimetinib) approved to be used in combination with vemurafenib for melanoma treatment

The U.S. Food and Drug Administration today approved Cotellic (cobimetinib) to be used in combination with vemurafenib to treat advanced melanoma that has spread to other parts of the body or can't be removed by surgery, and that has a certain type of abnormal gene (BRAF V600E or V600K mutation).

Melanoma is the most aggressive and dangerous form of skin cancer in the United States. It forms in the skin cells that develop the skin's pigment and if not diagnosed early, the cancer is likely to spread to other parts of the body. The National Cancer Institute estimates that 73,870 Americans will be diagnosed with melanoma and 9,940 will die from the disease this year.

"As we continue to advance our knowledge of tumor biology, we have learned that cancer cells have a remarkable ability to adapt and become resistant to targeted therapies. Combining two or more treatments addressing different cancer-causing targets may help to address this challenge," said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research. "Today's approval provides a new targeted treatment that, when added to vemurafenib, demonstrates greater benefit than vemurafenib alone in patients with BRAF mutation-positive melanoma."

Cotellic works by blocking the activity of an enzyme known as MEK, which is part of a larger signaling pathway. Abnormal activity of signaling pathways can lead to cancer. Cotellic prevents or slows cancer cell growth. Vemurafenib, marketed in the U.S. as Zelboraf, is a BRAF inhibitor that affects a different part of the same pathway and was approved in 2011 to treat patients with melanoma that has spread to other parts of the body or cannot be removed by surgery, whose tumors express a gene mutation called BRAF V600E, as detected by an FDA approved test. Health care providers should confirm the presence of BRAF V600 E or V600K mutation in their patients' tumor specimens using one of the available FDA approved tests prior to starting treatment with Cotellic in combination with vemurafenib.

Cotellic (cobimetinib) approved to be used in combination with vemurafenib for melanoma treatment

Diabetes drug liraglutide ineffective in patients with advanced heart failure

In continuation of my update on liraglutide

In an attempt to correct defects in the energy generation that contributes to poor pump function among heart failure patients, researchers examined whether the diabetes drug liraglutide, could improve the condition of patients with advanced heart failure. Despite improvements in blood sugar control, the therapy did not improve the clinical stability or pumping action of the heart in patients with advanced heart failure. Kenneth B. Margulies, MD, a professor of Medicine and research director for Heart Failure and Transplantation in the Perelman School of Medicine at the University of Pennsylvania, presented data from the Functional Impact of GLP-1 for Heart Failure Treatment (FIGHT) study at the American Heart Association Scientific Sessions 2015.

Heart failure, a chronic condition in which the heart does not pump enough blood through the body, affects more than 5 million Americans.

"Abnormalities in the way the heart generates energy from fats and glucose, including resistance to the normal actions of insulin, have been shown to contribute to a patient's risk of heart failure. But no current heart failure treatments target these metabolic derangements," said Margulies, the principle investigator of the study. "Because liraglutide counters insulin resistance, and earlier pilot studies suggest that severely weakened hearts have the greatest metabolic defects and potential benefit, it seemed most appropriate test the efficacy of liraglutide in a group of patients with advanced heart failure. Unfortunately, the results were not what we had anticipated."

Diabetes drug liraglutide ineffective in patients with advanced heart failure

Drug compounds target multiple pathways associated with myotonic dystrophy type 1

Efforts to treat myotonic dystrophy type 1, the most common form of muscular dystrophy, are in their infancy. In a new study, researchers report they have added new capabilities to an experimental drug agent that previously defeated only one of DM1's many modes of action. Their retooled compounds interrupt the disease's pathology in three ways.

"We've rationally designed something to target multiple pathways, which is contrary to the traditional thinking in medicinal chemistry, where you have one target, one drug," said University of Illinois chemistry professor Steven Zimmerman, who led the research with graduate students Lien Nguyen and Long Luu. "People are slowly discovering that drugs that hit multiple targets are actually better."

The team reports its findings in the Journal of the American Chemical Society.

DM1 (but not Duchennes muscular dystrophy) results from a genetic error that causes expansion of a region of a particular gene, called DMPK. This gene includes a repeated, three-letter sequence of nucleotides, the gene's chemical building blocks. Normal cells contain as many as 35 of these repeats, but sometimes mutation pushes the number of repeats beyond 50, which can lead to symptoms of the disease. Mutant DMPK genes often continue to expand, amplifying the health problems that can result. In some people, the gene includes as many as 10,000 repeats.

Drug compounds target multiple pathways associated with myotonic dystrophy type 1