Jan Dyer

After stem cell therapy, profiles may show a pattern of antibodies that can look very much like the “M spike”—the signature of the monoclonal antibody produced by multiple myeloma (MM). But that pattern, called an oligoclonal band, can be misleading.

“Oligoclonal bands should mostly be recognized as a response to treatment and not be mistaken as a recurrence of the original tumor,” says Dr. Gurmukh Singh, vice chair of clinical affairs for the Department of Pathology at the Medical College of Georgia at Augusta University.

He and his research team analyzed data from 251 patients with MM, 159 of whom had received autologous stem cell transplants. The researchers performed tests using serum protein electrophoresis/serum immunofixation electrophoresis and serum free light chain assay. Each patient had at least 3 tests, with at least 2 following the transplant.

The researchers found the incidence of oligoclonal patterns was dramatically higher in patients who had a stem cell transplant, compared with patients who had chemotherapy alone (57.9% vs 8.8%). Moreover, only 5 of the 159 patients who received a transplant had an oligoclonal pattern before treatment, but 92 had 1 afterward. More than half the oligoclonal patterns developed within the first year following transplant. The earliest pattern was detected at 2 months and a few as long as 5 years later.

The key to assessing response, Singh says, is to see where the spike appears: that is, where the monoclonal spike is at diagnosis compared with any new spikes that appear in oligoclonal bands after stem cell treatment. “If the original peak was at location A, [and] now the peak is location B, that allows us to determine that it is not the same abnormal, malignant antibody.”

The finding that 58% of patients had the oligoclonal pattern after transplant is likely an underestimate due to irregular schedule of testing, the researchers say. They add that their findings highlight the need for higher resolution electrophoretic methods to obviate the need for using mass spectrometry for clinical samples. Their results “cast more doubt on the clinical usefulness and medical necessity of the serum free light chain assay.”

Source:

Baker T. Results after stem cell transplant can confuse patients and doctors about cancer’s status. Jagwire News. https://jagwire.augusta.edu/archives/46434. Published August 2017. Accessed September 20, 2017.

Singh G. J Clin Med Res. 2017;9(8):671-679.
doi:  10.14740/jocmr3049w.

After stem cell therapy, profiles may show a pattern of antibodies that can look very much like the “M spike”—the signature of the monoclonal antibody produced by multiple myeloma (MM). But that pattern, called an oligoclonal band, can be misleading.

“Oligoclonal bands should mostly be recognized as a response to treatment and not be mistaken as a recurrence of the original tumor,” says Dr. Gurmukh Singh, vice chair of clinical affairs for the Department of Pathology at the Medical College of Georgia at Augusta University.

He and his research team analyzed data from 251 patients with MM, 159 of whom had received autologous stem cell transplants. The researchers performed tests using serum protein electrophoresis/serum immunofixation electrophoresis and serum free light chain assay. Each patient had at least 3 tests, with at least 2 following the transplant.

The researchers found the incidence of oligoclonal patterns was dramatically higher in patients who had a stem cell transplant, compared with patients who had chemotherapy alone (57.9% vs 8.8%). Moreover, only 5 of the 159 patients who received a transplant had an oligoclonal pattern before treatment, but 92 had 1 afterward. More than half the oligoclonal patterns developed within the first year following transplant. The earliest pattern was detected at 2 months and a few as long as 5 years later.

The key to assessing response, Singh says, is to see where the spike appears: that is, where the monoclonal spike is at diagnosis compared with any new spikes that appear in oligoclonal bands after stem cell treatment. “If the original peak was at location A, [and] now the peak is location B, that allows us to determine that it is not the same abnormal, malignant antibody.”

The finding that 58% of patients had the oligoclonal pattern after transplant is likely an underestimate due to irregular schedule of testing, the researchers say. They add that their findings highlight the need for higher resolution electrophoretic methods to obviate the need for using mass spectrometry for clinical samples. Their results “cast more doubt on the clinical usefulness and medical necessity of the serum free light chain assay.”

Source:

Baker T. Results after stem cell transplant can confuse patients and doctors about cancer’s status. Jagwire News. https://jagwire.augusta.edu/archives/46434. Published August 2017. Accessed September 20, 2017.

Singh G. J Clin Med Res. 2017;9(8):671-679.
doi:  10.14740/jocmr3049w.

After stem cell therapy, profiles may show a pattern of antibodies that can look very much like the “M spike”—the signature of the monoclonal antibody produced by multiple myeloma (MM). But that pattern, called an oligoclonal band, can be misleading.

“Oligoclonal bands should mostly be recognized as a response to treatment and not be mistaken as a recurrence of the original tumor,” says Dr. Gurmukh Singh, vice chair of clinical affairs for the Department of Pathology at the Medical College of Georgia at Augusta University.

He and his research team analyzed data from 251 patients with MM, 159 of whom had received autologous stem cell transplants. The researchers performed tests using serum protein electrophoresis/serum immunofixation electrophoresis and serum free light chain assay. Each patient had at least 3 tests, with at least 2 following the transplant.

The researchers found the incidence of oligoclonal patterns was dramatically higher in patients who had a stem cell transplant, compared with patients who had chemotherapy alone (57.9% vs 8.8%). Moreover, only 5 of the 159 patients who received a transplant had an oligoclonal pattern before treatment, but 92 had 1 afterward. More than half the oligoclonal patterns developed within the first year following transplant. The earliest pattern was detected at 2 months and a few as long as 5 years later.

The key to assessing response, Singh says, is to see where the spike appears: that is, where the monoclonal spike is at diagnosis compared with any new spikes that appear in oligoclonal bands after stem cell treatment. “If the original peak was at location A, [and] now the peak is location B, that allows us to determine that it is not the same abnormal, malignant antibody.”

The finding that 58% of patients had the oligoclonal pattern after transplant is likely an underestimate due to irregular schedule of testing, the researchers say. They add that their findings highlight the need for higher resolution electrophoretic methods to obviate the need for using mass spectrometry for clinical samples. Their results “cast more doubt on the clinical usefulness and medical necessity of the serum free light chain assay.”

Source:

Baker T. Results after stem cell transplant can confuse patients and doctors about cancer’s status. Jagwire News. https://jagwire.augusta.edu/archives/46434. Published August 2017. Accessed September 20, 2017.

Singh G. J Clin Med Res. 2017;9(8):671-679.
doi:  10.14740/jocmr3049w.

If statistics and warnings don’t get your patients’ attention about the risk of prediabetes, how about adorable puppies, hedgehogs, and baby goats? DoIHavePrediabetes.org, a CDC campaign, offers a “perfect way to spend a minute”—where viewers can take a quick prediabetes risk test while also watching cute animal videos.

The campaign builds on the success of a previous campaign that was the first of its kind to raise national awareness of prediabetes. Of the 84 million people with prediabetes, most don’t know they have it and are not aware of the long-term risks to their health.

The current campaign urges people to talk with their physicians after taking the test to confirm the diagnosis and learn about lifestyle changes. Through research-based programs such as the one the CDC offers (National Diabetes Prevention Program), people with prediabetes can lower their risk of developing type 2 diabetes by as much as 58%, and by 71% for people aged > 60 years.

The pro bono campaign was developed by Ogilvy New York. Michael Paterson, executive creative director, says, “Through a lighthearted and fun tone, we found more people were willing to take the test—and who doesn’t love to watch baby goats?”  

If statistics and warnings don’t get your patients’ attention about the risk of prediabetes, how about adorable puppies, hedgehogs, and baby goats? DoIHavePrediabetes.org, a CDC campaign, offers a “perfect way to spend a minute”—where viewers can take a quick prediabetes risk test while also watching cute animal videos.

The campaign builds on the success of a previous campaign that was the first of its kind to raise national awareness of prediabetes. Of the 84 million people with prediabetes, most don’t know they have it and are not aware of the long-term risks to their health.

The current campaign urges people to talk with their physicians after taking the test to confirm the diagnosis and learn about lifestyle changes. Through research-based programs such as the one the CDC offers (National Diabetes Prevention Program), people with prediabetes can lower their risk of developing type 2 diabetes by as much as 58%, and by 71% for people aged > 60 years.

The pro bono campaign was developed by Ogilvy New York. Michael Paterson, executive creative director, says, “Through a lighthearted and fun tone, we found more people were willing to take the test—and who doesn’t love to watch baby goats?”  

If statistics and warnings don’t get your patients’ attention about the risk of prediabetes, how about adorable puppies, hedgehogs, and baby goats? DoIHavePrediabetes.org, a CDC campaign, offers a “perfect way to spend a minute”—where viewers can take a quick prediabetes risk test while also watching cute animal videos.

The campaign builds on the success of a previous campaign that was the first of its kind to raise national awareness of prediabetes. Of the 84 million people with prediabetes, most don’t know they have it and are not aware of the long-term risks to their health.

The current campaign urges people to talk with their physicians after taking the test to confirm the diagnosis and learn about lifestyle changes. Through research-based programs such as the one the CDC offers (National Diabetes Prevention Program), people with prediabetes can lower their risk of developing type 2 diabetes by as much as 58%, and by 71% for people aged > 60 years.

The pro bono campaign was developed by Ogilvy New York. Michael Paterson, executive creative director, says, “Through a lighthearted and fun tone, we found more people were willing to take the test—and who doesn’t love to watch baby goats?”  

The crises of opioids and heroin abuse have skyrocketed, but in just 9 years, hospitalizations for women jumped 75%—much higher than the 55% among men and enough to bring hospitalization rates for the 2 sexes neck and neck. According to a report from the Agency for Healthcare Research and Quality, men and women were hospitalized at virtually the same rate in 2014: about 225 hospitalizations per 100,000 people.

The report, which covered data from 44 states and the District of Columbia, highlighted the broad variations in how the epidemic is hitting the U.S. For instance, opioid hospitalization rates among women were highest in West Virginia, Maryland, and Massachusetts (each state reporting rates of more than 350 hospitalizations per 100,000 people). For men, the highest rates were in the District of Columbia, New York, and Maryland (440+ per 100,000). Iowa, Nebraska, Texas, and Wyoming consistently ranked lowest for opioid-related inpatient stays, while Massachusetts consistently had the highest rates.

Men were more likely than women to make opioid-related emergency department (ED) visits, although ED visit rates rose sharply for both sexes between 2005 and 2014. Arkansas and Iowa consistently ranked lowest for opioid-related emergency department visits; Maryland was the highest.

In all states reporting on opioid-related ED visits, the rate was highest among adults aged 25 to 44 years.

The crises of opioids and heroin abuse have skyrocketed, but in just 9 years, hospitalizations for women jumped 75%—much higher than the 55% among men and enough to bring hospitalization rates for the 2 sexes neck and neck. According to a report from the Agency for Healthcare Research and Quality, men and women were hospitalized at virtually the same rate in 2014: about 225 hospitalizations per 100,000 people.

The report, which covered data from 44 states and the District of Columbia, highlighted the broad variations in how the epidemic is hitting the U.S. For instance, opioid hospitalization rates among women were highest in West Virginia, Maryland, and Massachusetts (each state reporting rates of more than 350 hospitalizations per 100,000 people). For men, the highest rates were in the District of Columbia, New York, and Maryland (440+ per 100,000). Iowa, Nebraska, Texas, and Wyoming consistently ranked lowest for opioid-related inpatient stays, while Massachusetts consistently had the highest rates.

Men were more likely than women to make opioid-related emergency department (ED) visits, although ED visit rates rose sharply for both sexes between 2005 and 2014. Arkansas and Iowa consistently ranked lowest for opioid-related emergency department visits; Maryland was the highest.

In all states reporting on opioid-related ED visits, the rate was highest among adults aged 25 to 44 years.

The crises of opioids and heroin abuse have skyrocketed, but in just 9 years, hospitalizations for women jumped 75%—much higher than the 55% among men and enough to bring hospitalization rates for the 2 sexes neck and neck. According to a report from the Agency for Healthcare Research and Quality, men and women were hospitalized at virtually the same rate in 2014: about 225 hospitalizations per 100,000 people.

The report, which covered data from 44 states and the District of Columbia, highlighted the broad variations in how the epidemic is hitting the U.S. For instance, opioid hospitalization rates among women were highest in West Virginia, Maryland, and Massachusetts (each state reporting rates of more than 350 hospitalizations per 100,000 people). For men, the highest rates were in the District of Columbia, New York, and Maryland (440+ per 100,000). Iowa, Nebraska, Texas, and Wyoming consistently ranked lowest for opioid-related inpatient stays, while Massachusetts consistently had the highest rates.

Men were more likely than women to make opioid-related emergency department (ED) visits, although ED visit rates rose sharply for both sexes between 2005 and 2014. Arkansas and Iowa consistently ranked lowest for opioid-related emergency department visits; Maryland was the highest.

In all states reporting on opioid-related ED visits, the rate was highest among adults aged 25 to 44 years.

Ever-improving HIV treatments mean more people are living with the virus under control. But what if they are not getting the treatment? The Louisiana Links program, operating in New Orleans, Baton Rouge, and Shreveport, demonstrates that it is possible to make sure that more people are aware of the support they could be getting—and give it to them.

Louisiana Links is funded through the Secretary’s Minority AIDS Initiative Fund (SMAIF), which supports programs to improve HIV prevention, care, and treatment for racial and ethnic minorities. During the funding period, the program has successfully linked and reengaged 90% of 686 enrollees to HIV medical care. Of the clients who were already in care but had not achieved viral suppression, 2 of 3 had the virus under control and were virally suppressed as shown at their last laboratory testing results.

In the Louisiana Links program, Linkage to Care Coordinators are hired to use state health department surveillance data in “innovative ways” to locate, engage, and enroll people living with HIV. They not only find “missing” people, but also provide support. For instance, coordinators attend medical and social service appointments with clients to ensure that they can overcome barriers to care and navigate complex health care systems. They work closely with local health care providers to maximize the resources and supportive services available to each client to increase long-term retention. They also educate clients about HIV and the importance of staying in care and adhering to treatment.

Due the success of the program, the Louisiana Department of Health has expanded services with core HIV prevention and care funding from the CDC and the Health Resources and Services Administration.

Ever-improving HIV treatments mean more people are living with the virus under control. But what if they are not getting the treatment? The Louisiana Links program, operating in New Orleans, Baton Rouge, and Shreveport, demonstrates that it is possible to make sure that more people are aware of the support they could be getting—and give it to them.

Louisiana Links is funded through the Secretary’s Minority AIDS Initiative Fund (SMAIF), which supports programs to improve HIV prevention, care, and treatment for racial and ethnic minorities. During the funding period, the program has successfully linked and reengaged 90% of 686 enrollees to HIV medical care. Of the clients who were already in care but had not achieved viral suppression, 2 of 3 had the virus under control and were virally suppressed as shown at their last laboratory testing results.

In the Louisiana Links program, Linkage to Care Coordinators are hired to use state health department surveillance data in “innovative ways” to locate, engage, and enroll people living with HIV. They not only find “missing” people, but also provide support. For instance, coordinators attend medical and social service appointments with clients to ensure that they can overcome barriers to care and navigate complex health care systems. They work closely with local health care providers to maximize the resources and supportive services available to each client to increase long-term retention. They also educate clients about HIV and the importance of staying in care and adhering to treatment.

Due the success of the program, the Louisiana Department of Health has expanded services with core HIV prevention and care funding from the CDC and the Health Resources and Services Administration.

Ever-improving HIV treatments mean more people are living with the virus under control. But what if they are not getting the treatment? The Louisiana Links program, operating in New Orleans, Baton Rouge, and Shreveport, demonstrates that it is possible to make sure that more people are aware of the support they could be getting—and give it to them.

Louisiana Links is funded through the Secretary’s Minority AIDS Initiative Fund (SMAIF), which supports programs to improve HIV prevention, care, and treatment for racial and ethnic minorities. During the funding period, the program has successfully linked and reengaged 90% of 686 enrollees to HIV medical care. Of the clients who were already in care but had not achieved viral suppression, 2 of 3 had the virus under control and were virally suppressed as shown at their last laboratory testing results.

In the Louisiana Links program, Linkage to Care Coordinators are hired to use state health department surveillance data in “innovative ways” to locate, engage, and enroll people living with HIV. They not only find “missing” people, but also provide support. For instance, coordinators attend medical and social service appointments with clients to ensure that they can overcome barriers to care and navigate complex health care systems. They work closely with local health care providers to maximize the resources and supportive services available to each client to increase long-term retention. They also educate clients about HIV and the importance of staying in care and adhering to treatment.

Due the success of the program, the Louisiana Department of Health has expanded services with core HIV prevention and care funding from the CDC and the Health Resources and Services Administration.

The DATA2020 HealthyPeople.gov data search function just got more user friendly. The Office of Disease Prevention and Health Promotion, National Center for Health Statistics, and Office of Minority Health, partners in DATA2020, have released a shareable widget that gives users easy access to regularly updated information.

The health disparities tool at DATA2020 lets users sort and view health disparities by demographic groups, including race/ethnicity, age, disability status, and geographic location. Users also can easily navigate and visualize data and changes in disparities over time; compare data points for each population group; and see all rates, rate ratios, confidence intervals and other technical details about data collection.

The new widget is an easy way (by simply copying the provided code) to share health disparities information on individual websites. Users can browse by disparity type or by Leading Health Indicator. The widget needs no maintenance; content updates automatically.

The DATA2020 HealthyPeople.gov data search function just got more user friendly. The Office of Disease Prevention and Health Promotion, National Center for Health Statistics, and Office of Minority Health, partners in DATA2020, have released a shareable widget that gives users easy access to regularly updated information.

The health disparities tool at DATA2020 lets users sort and view health disparities by demographic groups, including race/ethnicity, age, disability status, and geographic location. Users also can easily navigate and visualize data and changes in disparities over time; compare data points for each population group; and see all rates, rate ratios, confidence intervals and other technical details about data collection.

The new widget is an easy way (by simply copying the provided code) to share health disparities information on individual websites. Users can browse by disparity type or by Leading Health Indicator. The widget needs no maintenance; content updates automatically.

The DATA2020 HealthyPeople.gov data search function just got more user friendly. The Office of Disease Prevention and Health Promotion, National Center for Health Statistics, and Office of Minority Health, partners in DATA2020, have released a shareable widget that gives users easy access to regularly updated information.

The health disparities tool at DATA2020 lets users sort and view health disparities by demographic groups, including race/ethnicity, age, disability status, and geographic location. Users also can easily navigate and visualize data and changes in disparities over time; compare data points for each population group; and see all rates, rate ratios, confidence intervals and other technical details about data collection.

The new widget is an easy way (by simply copying the provided code) to share health disparities information on individual websites. Users can browse by disparity type or by Leading Health Indicator. The widget needs no maintenance; content updates automatically.

Between 2010 and 2015, the number of adults with active epilepsy rose from 2.3 million to 3 million, according to the CDC. The number of children with epilepsy rose from 450,000 to 470,000.

The increases are likely due to population growth, the CDC says, or other unknown factors, such as an increased willingness to disclose. However, most states do not have data on epilepsy prevalence. This is the first time estimates have been modeled for every state. Moreover, epilepsy has been assessed only intermittently in population surveys. Before 2010, the last national estimates were based on 1986-1990 data.

Obviously, epilepsy is not rare. It also is a serious public health issue. People with epilepsy often have other conditions, such as stroke, heart disease, depression, or developmental delay, which complicate epilepsy management, impair quality of life, and contribute to early mortality, the CDC says. Epilepsy also is the costliest and second most common of 5 chronic conditions that have adverse impact on academic and health outcomes in children and adolescents. For instance, children with seizures are more likely to live in poverty, and their parents more frequently report food insecurity.

The CDC suggests that health care providers and others can use the findings to ensure that evidence-based programs meet the complex needs of adults and children living with epilepsy and reduce the disparities resulting from it.

Between 2010 and 2015, the number of adults with active epilepsy rose from 2.3 million to 3 million, according to the CDC. The number of children with epilepsy rose from 450,000 to 470,000.

The increases are likely due to population growth, the CDC says, or other unknown factors, such as an increased willingness to disclose. However, most states do not have data on epilepsy prevalence. This is the first time estimates have been modeled for every state. Moreover, epilepsy has been assessed only intermittently in population surveys. Before 2010, the last national estimates were based on 1986-1990 data.

Obviously, epilepsy is not rare. It also is a serious public health issue. People with epilepsy often have other conditions, such as stroke, heart disease, depression, or developmental delay, which complicate epilepsy management, impair quality of life, and contribute to early mortality, the CDC says. Epilepsy also is the costliest and second most common of 5 chronic conditions that have adverse impact on academic and health outcomes in children and adolescents. For instance, children with seizures are more likely to live in poverty, and their parents more frequently report food insecurity.

The CDC suggests that health care providers and others can use the findings to ensure that evidence-based programs meet the complex needs of adults and children living with epilepsy and reduce the disparities resulting from it.

Between 2010 and 2015, the number of adults with active epilepsy rose from 2.3 million to 3 million, according to the CDC. The number of children with epilepsy rose from 450,000 to 470,000.

The increases are likely due to population growth, the CDC says, or other unknown factors, such as an increased willingness to disclose. However, most states do not have data on epilepsy prevalence. This is the first time estimates have been modeled for every state. Moreover, epilepsy has been assessed only intermittently in population surveys. Before 2010, the last national estimates were based on 1986-1990 data.

Obviously, epilepsy is not rare. It also is a serious public health issue. People with epilepsy often have other conditions, such as stroke, heart disease, depression, or developmental delay, which complicate epilepsy management, impair quality of life, and contribute to early mortality, the CDC says. Epilepsy also is the costliest and second most common of 5 chronic conditions that have adverse impact on academic and health outcomes in children and adolescents. For instance, children with seizures are more likely to live in poverty, and their parents more frequently report food insecurity.

The CDC suggests that health care providers and others can use the findings to ensure that evidence-based programs meet the complex needs of adults and children living with epilepsy and reduce the disparities resulting from it.

The CDC plans to award more than $12 million to 20 states and the District of Columbia to support responses to the opioid overdose epidemic. The new funding brings the number of recipients to 32.

States can use the funds to report nonfatal and fatal opioid overdose and risk factors linked to fatal overdoses more quickly, share data with key stakeholders, and share data with the CDC to improve multistate surveillance and response.

Fourteen states currently get funding under the Prescription Drug Overdose: Prevention for States (PfS) program, and another 8 will get $4.8 million. The money will allow states to enhance prescription drug-monitoring programs and implement and evaluate strategies to improve safe opioid prescribing practices.

The CDC plans to award more than $12 million to 20 states and the District of Columbia to support responses to the opioid overdose epidemic. The new funding brings the number of recipients to 32.

States can use the funds to report nonfatal and fatal opioid overdose and risk factors linked to fatal overdoses more quickly, share data with key stakeholders, and share data with the CDC to improve multistate surveillance and response.

Fourteen states currently get funding under the Prescription Drug Overdose: Prevention for States (PfS) program, and another 8 will get $4.8 million. The money will allow states to enhance prescription drug-monitoring programs and implement and evaluate strategies to improve safe opioid prescribing practices.

The CDC plans to award more than $12 million to 20 states and the District of Columbia to support responses to the opioid overdose epidemic. The new funding brings the number of recipients to 32.

States can use the funds to report nonfatal and fatal opioid overdose and risk factors linked to fatal overdoses more quickly, share data with key stakeholders, and share data with the CDC to improve multistate surveillance and response.

Fourteen states currently get funding under the Prescription Drug Overdose: Prevention for States (PfS) program, and another 8 will get $4.8 million. The money will allow states to enhance prescription drug-monitoring programs and implement and evaluate strategies to improve safe opioid prescribing practices.

“Encouraging” results from a study with an experimental drug offer hope to patients with Niemann-Pick disease type C1 (NPC1), a fatal neurologic disease that affects children and adolescents.

Symptoms of NPC1 result from cholesterol building in brain cells. The drug VTS-270 showed signs of improving cholesterol metabolism in neurons. After treatment, a molecule derived from cholesterol metabolism in neurons had increased, and 2 proteins indicative of brain injury had decreased.

In a phase 1/2a clinical trial, 14 participants received the experimental drug once a month for 12 to 18 months. Another 3 participants received the drug every 2 weeks for 18 months. After observing that the drug was safe and well tolerated, the researchers increased dosing for all participants. Their progress was compared with that of 21 participants in a previous study of NPC1.

The researchers also evaluated the drug’s effectiveness using a neurologic severity score (higher scores indicated more severe disease effects). Participants treated with VTS-270 had lower scores in cognition, speech, and mobility, indicating that the drug can stabilize or slow disease progression.

No one was observed to have serious adverse outcomes, but earlier studies had shown that the treatment carries the risk for hearing loss. After treatment in this study, participants, most of whom had already lost some hearing due to the disease, had further loss, for which they compensated with hearing aids.

“Encouraging” results from a study with an experimental drug offer hope to patients with Niemann-Pick disease type C1 (NPC1), a fatal neurologic disease that affects children and adolescents.

Symptoms of NPC1 result from cholesterol building in brain cells. The drug VTS-270 showed signs of improving cholesterol metabolism in neurons. After treatment, a molecule derived from cholesterol metabolism in neurons had increased, and 2 proteins indicative of brain injury had decreased.

In a phase 1/2a clinical trial, 14 participants received the experimental drug once a month for 12 to 18 months. Another 3 participants received the drug every 2 weeks for 18 months. After observing that the drug was safe and well tolerated, the researchers increased dosing for all participants. Their progress was compared with that of 21 participants in a previous study of NPC1.

The researchers also evaluated the drug’s effectiveness using a neurologic severity score (higher scores indicated more severe disease effects). Participants treated with VTS-270 had lower scores in cognition, speech, and mobility, indicating that the drug can stabilize or slow disease progression.

No one was observed to have serious adverse outcomes, but earlier studies had shown that the treatment carries the risk for hearing loss. After treatment in this study, participants, most of whom had already lost some hearing due to the disease, had further loss, for which they compensated with hearing aids.

“Encouraging” results from a study with an experimental drug offer hope to patients with Niemann-Pick disease type C1 (NPC1), a fatal neurologic disease that affects children and adolescents.

Symptoms of NPC1 result from cholesterol building in brain cells. The drug VTS-270 showed signs of improving cholesterol metabolism in neurons. After treatment, a molecule derived from cholesterol metabolism in neurons had increased, and 2 proteins indicative of brain injury had decreased.

In a phase 1/2a clinical trial, 14 participants received the experimental drug once a month for 12 to 18 months. Another 3 participants received the drug every 2 weeks for 18 months. After observing that the drug was safe and well tolerated, the researchers increased dosing for all participants. Their progress was compared with that of 21 participants in a previous study of NPC1.

The researchers also evaluated the drug’s effectiveness using a neurologic severity score (higher scores indicated more severe disease effects). Participants treated with VTS-270 had lower scores in cognition, speech, and mobility, indicating that the drug can stabilize or slow disease progression.

No one was observed to have serious adverse outcomes, but earlier studies had shown that the treatment carries the risk for hearing loss. After treatment in this study, participants, most of whom had already lost some hearing due to the disease, had further loss, for which they compensated with hearing aids.

The FDA has approved Besponsa (inotuzumab ozogamicin) for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL), a rapidly progressing cancer affecting about 6,000 people each year. About 1 in 4 patients affected will die of the disease. 

Inotuzumab ozogamicin is a targeted therapy “thought to work” by binding to B-cell ALL cancer cells that express the CD22 antigen, blocking the growth of cancerous cells. In a study of 326 patients with relapsed or refractory B-cell ALL who had received 1 or 2 prior treatments, 36% of 218 evaluated patients experienced complete remission for a median 8 months. Of the patients who received alternative chemotherapy, 17% experienced complete remission for a median 5 months.

A second drug, Vyxeos ( daunorubicin and cytarabine) liposome injection, is approved for adults with 2 types of acute myeloid leukemia (AML): newly diagnosed therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC).

An estimated 8% to 10% of patients with AML develop t-AML as a complication of chemotherapy or radiation. AML-MRC is characterized by a history of certain blood disorders and other significant mutations within cancer cells. Patients with either disease have a low life expectancy. Vyxeos is a fixed-combination of daunorubicin and cytarabine. It’s the first approved treatment specifically for these patients, says Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence.

In a study of 309 patients with newly diagnosed t-AML or AML-MRC, those in the Vyxeos group lived longer: median survival, 9.56 months vs. 5.95 months in the patients who received separate treatments with daunorubicin and cytarabine.The third drug, Idhifa (enasidenib), is approved for adults with relapsed or refractory AML who have a mutation in the IDH2 gene. Idhifa is an isocitrate dehydrogenase-2 inhibitor that blocks several enzymes that promote cell growth.

The drug was studied in a single-arm trial of 199 patients. With a minimum of 6 months of treatment, 19% of patients experienced complete remission for a median of 8.2 months; 4% experienced complete remission with partial hematologic recovery for a median 9.6 months. Of the 157 patients who required blood or platelet transfusions due to AML at the start of the study, 34% no longer did after treatment with Idhifa.

Idhifa is approved for use with a companion diagnostic, the RealTime IDH2 Assay, which is used to detect mutations in the IDH2 gene in blood or bone marrow.

Source:

FDA approves new treatment for adults with relapsed or refractory acute lymphoblastic leukemia [news release]. Silver Spring, MD: U.S. Food & Drug Administration; August 17,2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm572131.htm. Accessed August 31, 2017.

FDA approves new targeted treatment for relapsed or refractory acute myeloid leukemia [news release]. Silver Spring, MD: U.S. Food & Drug Administration; August 1, 2017. https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm569421.htm. Accessed August 31, 2017.

FDA approves first treatment for certain types of poor-prognosis acute myeloid leukemia [news release]. Silver Spring, MD: U.S. Food & Drug Administration; August 3, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm569883.htm. Accessed August 31, 2017.

The FDA has approved Besponsa (inotuzumab ozogamicin) for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL), a rapidly progressing cancer affecting about 6,000 people each year. About 1 in 4 patients affected will die of the disease. 

Inotuzumab ozogamicin is a targeted therapy “thought to work” by binding to B-cell ALL cancer cells that express the CD22 antigen, blocking the growth of cancerous cells. In a study of 326 patients with relapsed or refractory B-cell ALL who had received 1 or 2 prior treatments, 36% of 218 evaluated patients experienced complete remission for a median 8 months. Of the patients who received alternative chemotherapy, 17% experienced complete remission for a median 5 months.

A second drug, Vyxeos ( daunorubicin and cytarabine) liposome injection, is approved for adults with 2 types of acute myeloid leukemia (AML): newly diagnosed therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC).

An estimated 8% to 10% of patients with AML develop t-AML as a complication of chemotherapy or radiation. AML-MRC is characterized by a history of certain blood disorders and other significant mutations within cancer cells. Patients with either disease have a low life expectancy. Vyxeos is a fixed-combination of daunorubicin and cytarabine. It’s the first approved treatment specifically for these patients, says Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence.

In a study of 309 patients with newly diagnosed t-AML or AML-MRC, those in the Vyxeos group lived longer: median survival, 9.56 months vs. 5.95 months in the patients who received separate treatments with daunorubicin and cytarabine.The third drug, Idhifa (enasidenib), is approved for adults with relapsed or refractory AML who have a mutation in the IDH2 gene. Idhifa is an isocitrate dehydrogenase-2 inhibitor that blocks several enzymes that promote cell growth.

The drug was studied in a single-arm trial of 199 patients. With a minimum of 6 months of treatment, 19% of patients experienced complete remission for a median of 8.2 months; 4% experienced complete remission with partial hematologic recovery for a median 9.6 months. Of the 157 patients who required blood or platelet transfusions due to AML at the start of the study, 34% no longer did after treatment with Idhifa.

Idhifa is approved for use with a companion diagnostic, the RealTime IDH2 Assay, which is used to detect mutations in the IDH2 gene in blood or bone marrow.

Source:

FDA approves new treatment for adults with relapsed or refractory acute lymphoblastic leukemia [news release]. Silver Spring, MD: U.S. Food & Drug Administration; August 17,2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm572131.htm. Accessed August 31, 2017.

FDA approves new targeted treatment for relapsed or refractory acute myeloid leukemia [news release]. Silver Spring, MD: U.S. Food & Drug Administration; August 1, 2017. https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm569421.htm. Accessed August 31, 2017.

FDA approves first treatment for certain types of poor-prognosis acute myeloid leukemia [news release]. Silver Spring, MD: U.S. Food & Drug Administration; August 3, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm569883.htm. Accessed August 31, 2017.

The FDA has approved Besponsa (inotuzumab ozogamicin) for adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL), a rapidly progressing cancer affecting about 6,000 people each year. About 1 in 4 patients affected will die of the disease. 

Inotuzumab ozogamicin is a targeted therapy “thought to work” by binding to B-cell ALL cancer cells that express the CD22 antigen, blocking the growth of cancerous cells. In a study of 326 patients with relapsed or refractory B-cell ALL who had received 1 or 2 prior treatments, 36% of 218 evaluated patients experienced complete remission for a median 8 months. Of the patients who received alternative chemotherapy, 17% experienced complete remission for a median 5 months.

A second drug, Vyxeos ( daunorubicin and cytarabine) liposome injection, is approved for adults with 2 types of acute myeloid leukemia (AML): newly diagnosed therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC).

An estimated 8% to 10% of patients with AML develop t-AML as a complication of chemotherapy or radiation. AML-MRC is characterized by a history of certain blood disorders and other significant mutations within cancer cells. Patients with either disease have a low life expectancy. Vyxeos is a fixed-combination of daunorubicin and cytarabine. It’s the first approved treatment specifically for these patients, says Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence.

In a study of 309 patients with newly diagnosed t-AML or AML-MRC, those in the Vyxeos group lived longer: median survival, 9.56 months vs. 5.95 months in the patients who received separate treatments with daunorubicin and cytarabine.The third drug, Idhifa (enasidenib), is approved for adults with relapsed or refractory AML who have a mutation in the IDH2 gene. Idhifa is an isocitrate dehydrogenase-2 inhibitor that blocks several enzymes that promote cell growth.

The drug was studied in a single-arm trial of 199 patients. With a minimum of 6 months of treatment, 19% of patients experienced complete remission for a median of 8.2 months; 4% experienced complete remission with partial hematologic recovery for a median 9.6 months. Of the 157 patients who required blood or platelet transfusions due to AML at the start of the study, 34% no longer did after treatment with Idhifa.

Idhifa is approved for use with a companion diagnostic, the RealTime IDH2 Assay, which is used to detect mutations in the IDH2 gene in blood or bone marrow.

Source:

FDA approves new treatment for adults with relapsed or refractory acute lymphoblastic leukemia [news release]. Silver Spring, MD: U.S. Food & Drug Administration; August 17,2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm572131.htm. Accessed August 31, 2017.

FDA approves new targeted treatment for relapsed or refractory acute myeloid leukemia [news release]. Silver Spring, MD: U.S. Food & Drug Administration; August 1, 2017. https://www.fda.gov/newsevents/newsroom/pressannouncements/ucm569421.htm. Accessed August 31, 2017.

FDA approves first treatment for certain types of poor-prognosis acute myeloid leukemia [news release]. Silver Spring, MD: U.S. Food & Drug Administration; August 3, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm569883.htm. Accessed August 31, 2017.

Cardiovascular risk factors, such as diabetes and high blood pressure, increase the risk of dementia. That is not new information, but a long-term funded study by the National Institutes of Health found that not only is diabetes almost as strong a predictor of dementia as the APOE4 gene, but also prehypertension.

The researchers analyzed data on 15,744 participants aged 45 to 64 years in the Atherosclerosis Risk in Communities (ARIC) study. Over 25 years, the participants were examined 4 times, including being given cognitive tests during all but the first and third exams.

Over an average of 23 follow-up years, 1,516 people were diagnosed with dementia. During the time of the first exams, the risk of dementia increased most strongly with age, followed by the presence of APOE4. But as time went on, the link between cardiovascular risk factors and dementia became clearer. A separate study of an ARIC subgroup found that the presence of ≥ 1 vascular risk factor during midlife was associated with higher levels of beta amyloid, a protein that often accumulates in the brains of Alzheimer patients. The relationship was not affected by the presence of the APOE4 gene.

When the researchers reanalyzed the data according to who had a stroke, they found similar results: Diabetes, hypertension, prehypertension, and smoking raised the risk of dementia for people who had a stroke and those who had not.

“Our results contribute to a growing body of evidence linking midlife vascular health to dementia,” said study leader Rebecca Gottesman, MD, PhD, professor of neurology at Johns Hopkins University in Maryland. “These are modifiable risk factors. Our hope is that by addressing these types of factors early, people can reduce the chances that they will suffer from dementia later in life.”

Cardiovascular risk factors, such as diabetes and high blood pressure, increase the risk of dementia. That is not new information, but a long-term funded study by the National Institutes of Health found that not only is diabetes almost as strong a predictor of dementia as the APOE4 gene, but also prehypertension.

The researchers analyzed data on 15,744 participants aged 45 to 64 years in the Atherosclerosis Risk in Communities (ARIC) study. Over 25 years, the participants were examined 4 times, including being given cognitive tests during all but the first and third exams.

Over an average of 23 follow-up years, 1,516 people were diagnosed with dementia. During the time of the first exams, the risk of dementia increased most strongly with age, followed by the presence of APOE4. But as time went on, the link between cardiovascular risk factors and dementia became clearer. A separate study of an ARIC subgroup found that the presence of ≥ 1 vascular risk factor during midlife was associated with higher levels of beta amyloid, a protein that often accumulates in the brains of Alzheimer patients. The relationship was not affected by the presence of the APOE4 gene.

When the researchers reanalyzed the data according to who had a stroke, they found similar results: Diabetes, hypertension, prehypertension, and smoking raised the risk of dementia for people who had a stroke and those who had not.

“Our results contribute to a growing body of evidence linking midlife vascular health to dementia,” said study leader Rebecca Gottesman, MD, PhD, professor of neurology at Johns Hopkins University in Maryland. “These are modifiable risk factors. Our hope is that by addressing these types of factors early, people can reduce the chances that they will suffer from dementia later in life.”

Cardiovascular risk factors, such as diabetes and high blood pressure, increase the risk of dementia. That is not new information, but a long-term funded study by the National Institutes of Health found that not only is diabetes almost as strong a predictor of dementia as the APOE4 gene, but also prehypertension.

The researchers analyzed data on 15,744 participants aged 45 to 64 years in the Atherosclerosis Risk in Communities (ARIC) study. Over 25 years, the participants were examined 4 times, including being given cognitive tests during all but the first and third exams.

Over an average of 23 follow-up years, 1,516 people were diagnosed with dementia. During the time of the first exams, the risk of dementia increased most strongly with age, followed by the presence of APOE4. But as time went on, the link between cardiovascular risk factors and dementia became clearer. A separate study of an ARIC subgroup found that the presence of ≥ 1 vascular risk factor during midlife was associated with higher levels of beta amyloid, a protein that often accumulates in the brains of Alzheimer patients. The relationship was not affected by the presence of the APOE4 gene.

When the researchers reanalyzed the data according to who had a stroke, they found similar results: Diabetes, hypertension, prehypertension, and smoking raised the risk of dementia for people who had a stroke and those who had not.

“Our results contribute to a growing body of evidence linking midlife vascular health to dementia,” said study leader Rebecca Gottesman, MD, PhD, professor of neurology at Johns Hopkins University in Maryland. “These are modifiable risk factors. Our hope is that by addressing these types of factors early, people can reduce the chances that they will suffer from dementia later in life.”