Story from the Washington Post here, non-paywall version here.
Washington Post stop blocking linksharing and shit challenge.
"The young woman was catatonic, stuck at the nurses’ station — unmoving, unblinking and unknowing of where or who she was.
Her name was April Burrell.
Before she became a patient, April had been an outgoing, straight-A student majoring in accounting at the University of Maryland Eastern Shore. But after a traumatic event when she was 21, April suddenly developed psychosis and became lost in a constant state of visual and auditory hallucinations. The former high school valedictorian could no longer communicate, bathe or take care of herself.
April was diagnosed with a severe form of schizophrenia, an often devastating mental illness that affects approximately 1 percent of the global population and can drastically impair how patients behave and perceive reality.
“She was the first person I ever saw as a patient,” said Sander Markx, director of precision psychiatry at Columbia University, who was still a medical student in 2000 when he first encountered April. “She is, to this day, the sickest patient I’ve ever seen.” ...
It would be nearly two decades before their paths crossed again. But in 2018, another chance encounter led to several medical discoveries...
Markx and his colleagues discovered that although April’s illness was clinically indistinguishable from schizophrenia, she also had lupus, an underlying and treatable autoimmune condition that was attacking her brain.
After months of targeted treatments [for lupus] — and more than two decades trapped in her mind — April woke up.
The awakening of April — and the successful treatment of other people with similar conditions — now stand to transform care for some of psychiatry’s sickest patients, many of whom are languishing in mental institutions.
Researchers working with the New York state mental health-care system have identified about 200 patients with autoimmune diseases, some institutionalized for years, who may be helped by the discovery.
And scientists around the world, including Germany and Britain, are conducting similar research, finding that underlying autoimmune and inflammatory processes may be more common in patients with a variety of psychiatric syndromes than previously believed.
Although the current research probably will help only a small subset of patients, the impact of the work is already beginning to reshape the practice of psychiatry and the way many cases of mental illness are diagnosed and treated.
“These are the forgotten souls,” said Markx. “We’re not just improving the lives of these people, but we’re bringing them back from a place that I didn’t think they could come back from.” ...
Waking up after two decades
The medical team set to work counteracting April’s rampaging immune system and started April on an intensive immunotherapy treatment for neuropsychiatric lupus...
The regimen is grueling, requiring a month-long break between each of the six rounds to allow the immune system to recover. But April started showing signs of improvement almost immediately...
A joyful reunion
“I’ve always wanted my sister to get back to who she was,” Guy Burrell said.
In 2020, April was deemed mentally competent to discharge herself from the psychiatric hospital where she had lived for nearly two decades, and she moved to a rehabilitation center...
Because of visiting restrictions related to covid, the family’s face-to-face reunion with April was delayed until last year. April’s brother, sister-in-law and their kids were finally able to visit her at a rehabilitation center, and the occasion was tearful and joyous.
“When she came in there, you would’ve thought she was a brand-new person,” Guy Burrell said. “She knew all of us, remembered different stuff from back when she was a child.” ...
The family felt as if they’d witnessed a miracle.
“She was hugging me, she was holding my hand,” Guy Burrell said. “You might as well have thrown a parade because we were so happy, because we hadn’t seen her like that in, like, forever.”
“It was like she came home,” Markx said. “We never thought that was possible.”
...After April’s unexpected recovery, the medical team put out an alert to the hospital system to identify any patients with antibody markers for autoimmune disease. A few months later, Anca Askanase, a rheumatologist and director of the Columbia Lupus Center,who had been on April’s treatment team, approached Markx. “I think we found our girl,” she said.
Bringing back Devine
When Devine Cruz was 9, she began to hear voices. At first, the voices fought with one another. But as she grew older, the voices would talk about her, [and over the years, things got worse].
For more than a decade, the young woman moved in and out of hospitals for treatment. Her symptoms included visual and auditory hallucinations, as well as delusions that prevented her from living a normal life.
Devine was eventually diagnosed with schizoaffective disorder, which can result in symptoms of both schizophrenia and bipolar disorder. She also was diagnosed with intellectual disability.
She was on a laundry list of drugs — two antipsychotic medications, lithium, clonazepam, Ativan and benztropine — that came with a litany of side effects but didn’t resolve all her symptoms...
She also had lupus, which she had been diagnosed with when she was about 14, although doctors had never made a connection between the disease and her mental health...
Last August, the medical team prescribed monthly immunosuppressive infusions of corticosteroids and chemotherapy drugs, a regime similar to what April had been given a few years prior. By October, there were already dramatic signs of improvement.
“She was like ‘Yeah, I gotta go,’” Markx said. “‘Like, I’ve been missing out.’”
After several treatments, Devine began developing awareness that the voices in her head were different from real voices, a sign that she was reconnecting with reality. She finished her sixth and final round of infusions in January.
In March, she was well enough to meet with a reporter. “I feel like I’m already better,” Devine said during a conversation in Markx’s office at the New York State Psychiatric Institute, where she was treated. “I feel myself being a person that I was supposed to be my whole entire life.” ...
Her recovery is remarkable for several reasons, her doctors said. The voices and visions have stopped. And she no longer meets the diagnostic criteria for either schizoaffective disorder or intellectual disability, Markx said...
Today, Devine lives with her mother and is leading a more active and engaged life. She helps her mother cook, goes to the grocery store and navigates public transportation to keep her appointments. She is even babysitting her siblings’ young children — listening to music, taking them to the park or watching “Frozen 2” — responsibilities her family never would have entrusted her with before her recovery.
Expanding the search for more patients
While it is likely that only a subset of people diagnosed with schizophrenia and psychotic disorders have an underlying autoimmune condition, Markx and other doctors believe there are probably many more patients whose psychiatric conditions are caused or exacerbated by autoimmune issues...
The cases of April and Devine also helped inspire the development of the SNF Center for Precision Psychiatry and Mental Health at Columbia, which was named for the Stavros Niarchos Foundation, which awarded it a $75 million grant in April. The goal of the center is to develop new treatments based on specific genetic and autoimmune causes of psychiatric illness, said Joseph Gogos, co-director of the SNF Center.
Markx said he has begun care and treatment on about 40 patients since the SNF Center opened. The SNF Center is working with the New York State Office of Mental Health, which oversees one of the largest public mental health systems in America, to conduct whole genome sequencing and autoimmunity screening on inpatients at long-term facilities.
For “the most disabled, the sickest of the sick, even if we can help just a small fraction of them, by doing these detailed analyses, that’s worth something,�� said Thomas Smith, chief medical officer for the New York State Office of Mental Health. “You’re helping save someone’s life, get them out of the hospital, have them live in the community, go home.”
Discussions are underway to extend the search to the 20,000 outpatients in the New York state system as well. Serious psychiatric disorders, like schizophrenia, are more likely to be undertreated in underprivileged groups. And autoimmune disorders like lupus disproportionately affect women and people of color with more severity.
Changing psychiatric care
How many people ultimately will be helped by the research remains a subject of debate in the scientific community. But the research has spurred excitement about the potential to better understand what is going on in the brain during serious mental illness...
Emerging research has implicated inflammation and immunological dysfunction as potential players in a variety of neuropsychiatric conditions, including schizophrenia, depression and autism.
“It opens new treatment possibilities to patients that used to be treated very differently,” said Ludger Tebartz van Elst, a professor of psychiatry and psychotherapy at University Medical Clinic Freiburg in Germany.
In one study, published last year in Molecular Psychiatry, Tebartz van Elst and his colleagues identified 91 psychiatric patients with suspected autoimmune diseases, and reported that immunotherapies benefited the majority of them.
Belinda Lennox, head of the psychiatry department at the University of Oxford, is enrolling patients in clinical trials to test the effectiveness of immunotherapy for autoimmune psychosis patients.
As a result of the research, screenings for immunological markers in psychotic patients are already routine in Germany, where psychiatrists regularly collect samples from cerebrospinal fluid.
Markx is also doing similar screening with his patients. He believes highly sensitive and inexpensive blood tests to detect different antibodies should become part of the standard screening protocol for psychosis.
Also on the horizon: more targeted immunotherapy rather than current “sledgehammer approaches” that suppress the immune system on a broad level, said George Yancopoulos, the co-founder and president of the pharmaceutical company Regeneron.
“I think we’re at the dawn of a new era. This is just the beginning,” said Yancopoulos."
-via The Washington Post, June 1, 2023
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Most Common Clinical Trial Therapy Areas
2023 Top Clinical Trial Areas
Clinical trials are a fundamental part of the medical research process. They help determine the safety and effectiveness of new treatment approaches, contributing to advancements in the medical field. With an ever-growing number of clinical trials conducted worldwide each year, it's essential to identify the most impactful and relevant therapy areas being researched. This article will discuss the top five clinical trial areas that have taken center stage in 2023. We will cover the prevalence of these clinical trials, funding, and expected outcomes for each therapy area, as well as the future of clinical trials in 2023.
1. Oncology: Leading the Charge in Clinical Trials
Cancer is among the leading causes of death globally, and the need for innovative therapies has never been higher. In 2023, the oncology domain is still the most common area in clinical trial research, representing nearly 50% of clinical trials worldwide. The United States bears the majority of these trials, boasting over 25,000 active studies in oncology alone. Europe is a close second, with multiple countries working together to fund and conduct innovative cancer research.
Government agencies, pharmaceutical companies, and non-profit organizations have invested billions of dollars into cancer research. In 2023, the National Cancer Institute (NCI) in the United States received more than $6.5 billion in funding for cancer research. Many prominent clinical trials this year target hard-to-treat cancers, such as lung, breast, and pancreatic cancer, with a focus on immunotherapy, targeted therapies, and cellular therapies. The future of oncology research is bright, as advancements in technology and global collaboration continue to push the boundaries of cancer treatment.
2. Neurology: Addressing the Growing Burden of Neurological Disorders
Neurological disorders, including Alzheimer's disease, Parkinson's disease, and multiple sclerosis, affect millions of people worldwide. With the growing prevalence of these disorders, neurology has emerged as a leading area in clinical trial research. The United States leads the world in neurological clinical trials, followed closely by Europe and Asia.
Global funding for neurological clinical trials reached new heights in 2023, with a focus on addressing neurodegenerative conditions like Alzheimer's and Parkinson's. Public and private sectors invested heavily in this research, with organizations like the National Institutes of Health (NIH) allocating over $3 billion to neurological clinical trials in 2023. The outcomes of these trials aim to slow down, prevent, or cure neurological diseases, and hope to improve overall quality of life for affected individuals.
3. Infectious Diseases: Tackling Emerging and Reemerging Pathogens
The COVID-19 pandemic has underscored the importance of research and preparedness in combating infectious diseases. In response to this global challenge, the field has seen significant growth in clinical trials focused on infectious diseases. Research funding for these trials has boasted increased support worldwide, with both public and private sectors contributing to the development of novel vaccines and treatments.
In 2023, clinical trials in infectious diseases tackled novel pathogens, as well as reemerged diseases like tuberculosis and malaria. The World Health Organization (WHO) played a significant role in funding and initiating these trials, collaborating with governments and pharmaceutical companies to ensure rapid response and treatment development. The outcomes of these trials will contribute to global health security and preparedness for future pandemics and disease outbreaks.
4. Cardiology: Addressing the Global Burden of Cardiovascular Disease
Cardiovascular disease (CVD) is a leading cause of death worldwide, with almost 18 million annual fatalities. In 2023, cardiology clinical trials aimed to improve prevention, diagnosis, and treatment of CVD, encompassing areas such as heart failure, coronary artery disease, and hypertension.
Globally, funding for cardiology research came from government agencies, non-profit organizations, and pharmaceutical companies. The United States, Europe, and several Asian countries allocated substantial resources to support these clinical trials. The anticipated outcomes of these trials will emphasize personalized and precision medicine approaches in cardiovascular healthcare and ultimately reduce the burden of CVD around the world.
5. Rare Diseases: Advancing Treatment for Orphan Disorders
In recent years, the focus on rare diseases has grown significantly, resulting in more clinical trials aimed at developing treatments for orphan disorders affecting less than 200,000 individuals in the United States. Developing therapies for rare diseases is often financially challenging due to the small patient populations. However, regulatory incentives and growing public awareness have resulted in an increase in funding and clinical trials in this area.
Rare disease clinical trials are prevalent in both the United States and Europe, with a focus on gene therapy, enzyme replacement therapy, and targeted treatments. Public health agencies
Oncology: One of the most common clinical trials by therapy area is oncology, which involves testing medications and treatments with the goal of helping to improve patient outcomes when dealing with various types of cancer. Examples of these clinical trials include those that seek to determine the efficacy of new drugs in treating particular forms of cancer, or researching novel therapeutic approaches such as immunotherapy.
Cardiovascular Disease: Clinical trials related to cardiovascular disease are also quite common. These tests may involve assessing the effectiveness of new medications that can help lower blood pressure or improve cardiac function, as well as examining lifestyle interventions such as diet and exercise for their potential to reduce risk factors associated with heart disease.
Diabetes: Clinical trials related to diabetes are also a frequent occurrence due to its prevalence in many parts of the world. These studies often aim to understand how better management strategies for diabetes can improve quality of life for patients and reduce long-term complications associated with this condition.
Neurology: Clinical trials pertaining to neurology are commonplace in research settings because there is still much unknown about how the brain and nervous system work, as well as treatment effectiveness for conditions like epilepsy, Parkinson’s disease, multiple sclerosis, and stroke recovery.
Mental Health: Mental health-related clinical trials are becoming increasingly more common as researchers continue to investigate and develop better treatments for depression, anxiety disorders, bipolar disorder, schizophrenia, PTSD, addiction and other issues related to mental health and wellbeing.
Respiratory Disease: Clinical trials involving respiratory diseases, such as asthma or chronic obstructive pulmonary disease (COPD), have become more commonplace in recent years due to their rising prevalence throughout the world; they typically involve testing new medications or therapies that can help manage symptoms and reduce exacerbations associated with these conditions.
Immunology: Immunology-focused clinical research has become more popular over recent years due to its potential implications for developing treatments for autoimmune diseases like rheumatoid arthritis or lupus; these clinical trials often involve testing existing medications or creating new ones from scratch in order to achieve desired results regarding immune system regulation within individuals living with autoimmune conditions .
Gastroenterology: Gastroenterological clinical research is commonplace due primarily to its relevance within digestive disorders such as Crohn’s Disease or Irritable Bowel Syndrome (IBS). Research conducted in this area generally seeks to gain an understanding into how certain dietary changes or drug treatments might be effective at managing symptoms associated with gastrointestinal problems while reducing side effects associated with traditional pharmacological approaches .
Endocrinology: Endocrinological clinical research is yet another form of study found in medical circles due primarily via its relevance within hormone-related issues such as diabetes mellitus type 1 & 2; this type of study typically involves testing ways in which different hormones might interact differently between individuals who have similar conditions but don't respond positively/negatively the same way when it comes to traditional forms of treatment .
Ophthalmology: Last but not least is ophthalmology which looks at vision disorders like glaucoma and age related macular degeneration (AMD); here researchers test existing medications/treatments looking for improvement when it comes both short term relief from eye pain/blurring but also long term protection against further loss/damage occurring over time via regular monitoring sessions
Learn more about clinical trials and become involved in management of clinical trials through further training with CCRPS.
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Started on a new anti-migraine drug today (ajovy). Also have had terrible sleep - Especially today, but, like, all year. Also, y'know the migraines.
So not 100% sure if certain things are side-effects of the ajovy or just y'know, my body being upset with many things going on in its life - But more to the point: Should I be concerned with anything that I'm seeing/experiencing?.
Trying to google side-effects is sure a fucking trip, though.
Official company line on their site seems to be "Some swelling/irritation near injection site" is likely the only side-effect confirmed to definitely be from this medicine. Anything else? Well, that must be someone else's problem.
Various reddit posts of peoples' experiences with the medication - Mostly neutral-to-mildly positive. A lot of not-outright-negativity, but disappointment or frustration about the tradeoffs vs benefits. But the side-effects definitely vary wildly in type and intensity. The ones I'm curious about are topics of discussion that have come up with some degree of frequency.
Various interaction/drug rundown sites have much more comprehensive lists, but also include a warning that the ones I'm looking into may be serious/concerning and I should consult my doctor about them right away. Which, like, doesn't seem to be the tone regarding them anywhere else. So, more confusion as-to who's out of step here.
Meanwhile, the pamphlet that came in its box actually does have a more comprehensive list of possible side-effects. Doesn't list severity other than obvious "If experiencing anaphylaxis..." or other obvious emergencies/serious stuff, then go to ER/phone doctor immediately. Everything else listed is just sort of "This could happen" without any level of how concerned you should be.
The ones I've noticed are heartburn (Unusual for me without certain types and timings of trigger-foods, but not impossible to be something else going on that's unrelated to this). And notable joint-stiffness (hands and feet, especially on the same side as my injection), with a touch of joint pain. Both of those are in the "might happen in ~1% of patients" group. Which, hey, alright. That's fine. They're known to have a connection, echoing what the reddit posts were saying. From the tone in the pamphlet and on reddit, I probably don't need to be concerned unless they worsen or persist for a very long time.
Uncommon doesn't necessarily mean alarming, though I am curious as to if there's any correlation in the group of people that experience them. Any factor that might predict it, or explain why they're causing this reaction, y'know?
Okay. Good. No need to panic. Probably should sleep though. Less than 4 hours in the last 48 can't be good for me.
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“A decade ago, CAR T-cell therapy changed the face of cancer research and treatment. It’s now been applied in a small trial to lupus patients with total success.
Four female patients and one male whose lupus had been untreatable were given an infusion of genetically-engineered immune cells called T-cells, which attacked another group of cells that do the damage in lupus patients, sending all five into remission.
Lupus is an autoimmune disease, meaning the autonomic immune system begins attacking the body. In the case of lupus, defective immune cells called B-cells produce autoantibodies which attack the patient’s own cells rather than hostile pathogens.
It can cause a large variety of symptoms as varied and mild as fatigue, and as serious as organ damage and failure.
In the trial, CAR T-cell therapy, which has been approved for a variety of cancers, was applied to instruct the genetics of the T-cells in the five lupus patients not to target cancer cells, but these defective autoantibody-producing B-cells.
The T-cells carried the field, and after a dramatic depletion in B-cell count, sent the lupus into a kind of remission. When the patients’ B-cells recovered after four months, they were no longer creating the autoantibodies, the Guardian reported.
Despite this self-destruct sequence of the B-cells, the patients’ immune systems seemed to be working normally followings tests.
“We are very excited about these results,” Friedrich-Alexander University rheumatologist and study lead Georg Schett told the Guardian.
“Several other autoimmune diseases which are dependent on B cells and show autoantibodies may respond to this treatment. These include rheumatoid arthritis, myositis and systemic sclerosis. But also diseases like multiple sclerosis may be very responsive to CAR T-cell treatment.”” -via Good News Network, 11/28/22
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