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STAT+: A decade ago, these drugs tore apart the FDA. Today, they might be some patients’ best hope
A year after the worst day of her life, Debra Miller received a voicemail she couldn’t quite make out. In a thick accent, a man said something about research and left a phone number. She called but couldn’t get through. “I didn’t know what country code to put in,” she said.
Debra moved on, but the voice kept tumbling through her brain. She was desperate. Her first child, Hawken, had been diagnosed 13 months before with Duchenne muscular dystrophy. In blunt tones she would never forget, a doctor had told her that her 5-year-old boy would slowly lose the ability to walk and die by 18.
When she finally figured out the digits, a Dutch scientist explained he was launching a startup around one of the most counterintuitive ideas in modern genetics: that sometimes you can fix a broken gene by breaking it just a little bit more.
That strategy, known as exon skipping, would taunt Debra for two decades, always promising a therapy just out of reach. It prompted her to raise $1.3 million for the Dutch scientist and helped turn her fledgling advocacy group, CureDuchenne, into a powerhouse. Eventually, the idea spread far beyond the Netherlands and Debra’s home in Newport Beach, Calif., stirring tenuous hope for a life-altering treatment.
Exon-skipping drugs sparked a civil war within the Food and Drug Administration. Under pressure from advocates and companies, a top official overrode reviewers to approve the first of several candidates. One company, Sarepta Therapeutics, has since earned over $5.5 billion from from drugs that may or may not provide much benefit.
Throughout, by the fickle winds of scientific misfortune, mother and son remained waiting — until about two and a half years ago. That’s when Hawken enrolled in a clinical trial for a new exon-skipping drug Debra helped support. The results from him and 38 other patients have since stunned some of the field’s top experts.
A year after the worst day of her life, Debra Miller received a voicemail she couldn’t quite make out. In a thick accent, a man said something about research and left a phone number. She called but couldn’t get through. “I didn’t know what country code to put in,” she said.
Debra moved on, but the voice kept tumbling through her brain. She was desperate. Her first child, Hawken, had been diagnosed 13 months before with Duchenne muscular dystrophy. In blunt tones she would never forget, a doctor had told her that her 5-year-old boy would slowly lose the ability to walk and die by 18.
When she finally figured out the digits, a Dutch scientist explained he was launching a startup around one of the most counterintuitive ideas in modern genetics: that sometimes you can fix a broken gene by breaking it just a little bit more.
That strategy, known as exon skipping, would taunt Debra for two decades, always promising a therapy just out of reach. It prompted her to raise $1.3 million for the Dutch scientist and helped turn her fledgling advocacy group, CureDuchenne, into a powerhouse. Eventually, the idea spread far beyond the Netherlands and Debra’s home in Newport Beach, Calif., stirring tenuous hope for a life-altering treatment.
Exon-skipping drugs sparked a civil war within the Food and Drug Administration. Under pressure from advocates and companies, a top official overrode reviewers to approve the first of several candidates. One company, Sarepta Therapeutics, has since earned over $5.5 billion from from drugs that may or may not provide much benefit.
Throughout, by the fickle winds of scientific misfortune, mother and son remained waiting — until about two and a half years ago. That’s when Hawken enrolled in a clinical trial for a new exon-skipping drug Debra helped support. The results from him and 38 other patients have since stunned some of the field’s top experts.
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#AAN26: Roche’s multiple sclerosis pill delays relapse, but liver toxicity could need monitoring
#AAN26: Roche’s multiple sclerosis pill delays relapse, but liver toxicity could need monitoring
Roche’s multiple sclerosis pill more than doubled patients’ relapse-free interval compared with Sanofi’s marketed MS pill Aubagio, according to data presented late on Tuesday.
The drug, a BTK inhibitor called fenebrutinib, demonstrated “the lowest relapse … Read More
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STAT+: With successful trials, Roche takes its MS drug to regulators, but safety questions loom
The Swiss drugmaker Roche on Tuesday presented the latest data for its experimental multiple sclerosis drug, setting the stage for the company to seek approval for a medicine that it believes can cut relapse rates and slow the progressive disability the disease causes.
Now the test is whether the drug, called fenebrutinib, can win the regulatory green light.
While three late-stage trials of the drug have shown it to be effective, analysts have homed in on some potentially worrying liver safety signals, an issue that previously prompted the Food and Drug Administration to reject an MS therapy developed by Sanofi. In data released Tuesday, researchers also disclosed that there were two drug-related deaths among patients who took fenebrutinib.
The Swiss drugmaker Roche on Tuesday presented the latest data for its experimental multiple sclerosis drug, setting the stage for the company to seek approval for a medicine that it believes can cut relapse rates and slow the progressive disability the disease causes.
Now the test is whether the drug, called fenebrutinib, can win the regulatory green light.
While three late-stage trials of the drug have shown it to be effective, analysts have homed in on some potentially worrying liver safety signals, an issue that previously prompted the Food and Drug Administration to reject an MS therapy developed by Sanofi. In data released Tuesday, researchers also disclosed that there were two drug-related deaths among patients who took fenebrutinib.
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STAT+: From Revolution Medicines, more strong data on KRAS drug and a glimpse of a ‘novel class’ beyond it
SAN DIEGO — Revolution Medicines is already cooking up the next iteration of RAS inhibiting drugs.
At the American Association of Cancer Research annual meeting here, the company is the talk of the town for the clinical trial success of daraxonrasib, their next generation targeted therapy, in advanced pancreatic cancer. And while the company presented more data on that drug on Tuesday, showing promising first line and combination data on daraxonrasib, scientists also showed in another session intriguing preclinical data on a completely new compound that may represent what comes after their current lineup.
That drug, currently called RM-055, is what RevMed’s CEO Mark Goldsmith is calling an entirely “novel class of catalytic inhibitors.” These are targeted therapies that not only block the RAS signaling that drives cancer, but molecularly turn the cancer protein off.
SAN DIEGO — Revolution Medicines is already cooking up the next iteration of RAS inhibiting drugs.
At the American Association of Cancer Research annual meeting here, the company is the talk of the town for the clinical trial success of daraxonrasib, their next generation targeted therapy, in advanced pancreatic cancer. And while the company presented more data on that drug on Tuesday, showing promising first line and combination data on daraxonrasib, scientists also showed in another session intriguing preclinical data on a completely new compound that may represent what comes after their current lineup.
That drug, currently called RM-055, is what RevMed’s CEO Mark Goldsmith is calling an entirely “novel class of catalytic inhibitors.” These are targeted therapies that not only block the RAS signaling that drives cancer, but molecularly turn the cancer protein off.
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#AAN26: Roche’s multiple sclerosis pill delays relapse, but liver toxicity could need monitoring
STAT+: With successful trials, Roche takes its MS drug to regulators, but safety questions loom
STAT+: From Revolution Medicines, more strong data on KRAS drug and a glimpse of a ‘novel class’ beyond it
STAT+: Key GOP senators push back on Trump’s plan to cut NIH, reorganize HHS
Medicare indefinitely delays pilot plan to cover weight loss drugs
Alzheimer’s Linked to Cancer Mutations in Brain Immune Cells
Illinois’ financial crisis could bring the state to a halt
The final 6 ‘Game of Thrones’ episodes might feel like a full season
New Season 8 Walking Dead trailer flashes forward in time
Mod turns ‘Counter-Strike’ into a ‘Tekken’ clone with fighting chickens
Meet Superman’s grandfather in new trailer for Krypton
Disney’s live-action Aladdin finally finds its stars
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