Posts Tagged ‘medical’
VRD versus VCD as induction therapy before autologous stem cell transplantation in multiple myeloma: a nationwide … – Nature.com
Induction therapy followed by autologous stem cell transplantation (ASCT) is standard of care for young and fit patients with newly diagnosed multiple myeloma (MM) [1]. Induction therapy has evolved from doublet to triplet to quadruplet regimens over the last decades. The most common triplet therapy is either Bortezomib-Cyclophosphamide-Dexamethasone (VCD), Bortezomib-Lenalidomide-Dexamethasone (VRD), or less frequently Bortezomib-Thalidomide-Dexamethasone (VTD). No large, randomized phase III study comparing the VCD and VRD regimens has been conducted and is unlikely to be done in the future. Retrospective studies and smaller prospective studies comparing VRD and VCD have produced mixed results [2,3,4,5,6,7].
In Norway, there has been a shift from VCD to VRD induction therapy in recent years, while VTD has only been used in a minority of patients. ASCT for multiple myeloma in Norway is performed at four centers, and comprehensive population-based nationwide follow-up data are available from electronic journals.
In collaboration with all centers in Norway doing ASCT, we identified all patients in Norway who had undergone ASCT for multiple myeloma in the study period 2008 to 2020.
We included patients with multiple myeloma [8] who received first line induction therapy followed by ASCT in the period from January 1st 2008 to December 31st 2020 in Norway. We did not include patients who received induction therapy but did not proceed to ASCT. Patients were censored March 1st 2022 or at loss to follow-up because of relocation outside of Norway (n=5), or if the journal from the local hospital could not be obtained (n=7).
Data was collected from electronic patient journals at the transplant centers and from hospitals responsible for induction therapy and follow-up after ASCT. Change of induction therapy was recorded if a patient changed from one line to another, and the reason for change was collected. Patients who changed therapy were not included in the primary response analysis, regardless of the reason for change, but they were included in a separate intention-to-treat analysis. All patients, including those who changed treatment, were included in the PFS and OS analysis. Further description of study design, endpoints and statistical analysis is provided in the supplementary material.
We identified 1354 patients who received ASCT as first-line treatment for multiple myeloma in Norway in the study period.
Of these, 682 patients received VCD induction, 332 patients received VRD induction, and 42 patients received VTD induction. Baseline characteristics are described in Table 1 and were largely similar between patients who received VCD and VRD induction, with two notable exceptions. Patients in the VRD group were older than patients in the VCD group (median 62 years vs. 60 years). Patients in the VRD group received ASCT in more recent years (mostly 2017-2020), compared to VCD.
Three months after ASCT, response rates were higher with VRD, with 89% in the VRD group achieving VGPR, versus 76% in the VCD group (p<0.001). In the intention-to-treat analysis, the difference in response between the groups remained statistically significant (Table 1).
In the VCD group, 4% of patients changed therapy due to lack of response, and 1% due to progression. In the VRD group, very few patients changed treatment due to lack of response (1%) or progression (1%). Only a small minority, 3% and 2% of patients in the VRD and VCD group respectively, changed treatment due to adverse effects (Table 1). In patients who received bortezomib, thalidomide and dexamethasone (VTD), 36% of patients changed treatment due to side effects (Supplementary Table 5).
Patients in the VRD group more often received treatment after ASCT than in the VCD group (61% vs. 14%, p<0.001). Consolidation treatment (22% vs. 1%), maintenance treatment (25% vs. 10%) or both (14% vs. 3%) were all more frequent in the VRD group (Table 1). In the VCD group, 4.7% of patients (n=32) and in the VRD group 3.9% of patients (n=13) had progressive disease before starting consolidation or maintenance treatment.
The median follow-up time of patients still alive at data cut-off was 79 months (range: 19179 months) in the VCD group and 38 months (range: 1871 months) in the VRD group.
In the VCD group, the median PFS was 30.1 months (95% confidence interval (CI) 28.331.9 months). In the VRD group, the median PFS was 55.1 months (95% CI 46.0-not reached (NR), Fig. 1A). The difference was significant on log-rank test, p<0.001. In the VTD group median PFS was 36.6 months (Supplementary Fig. 2)
A PFS, all patients. B PFS, only patients who received maintenance treatment. C PFS, only patients who revied ASCT in later years (20172020) and did not received any post-ASCT treatment. D OS, all patients.
When we included only patients who received maintenance therapy after ASCT, the median PFS in the VCD group increased to 47.1 months, which is not statistically different from patients in the VRD group who received maintenance, who had a median PFS of 56.4 months, p=0.174 (Fig. 1B).
In a separate analysis excluding patients who received maintenance and/or consolidation and who received ASCT in later years (20172020), VRD was superior to VCD regarding PFS with a log-rank test of p<0.001 (Fig. 1C).
The median OS for VCD was 114.0 months (95% CI 103.4125.8 months) and the median OS for VRD was not reached, log-rank test p<0.001 (Fig. 1D).
The hazard ratios for PFS and OS on multivariate analysis is provided in Supplementary Table 2. After controlling for patient and disease factors, VCD had inferior PFS compared to VRD (HR 2.08, 95% CI 1.492.91, p<0.001). There was no significant difference in OS between the two regimens in multivariate analysis.
VTD is approved by the European Medical Agency as induction therapy before ASCT. This is not the case for VCD and VRD, although they are used widely in current clinical practice. The most recent European Society of Medical Oncology (ESMO) guidelines [1] recommend VRD as the first option for induction therapy. Daratumumab-VTD is also approved and recommended, but our study confirms the high toxicity associated with regimens containing thalidomide. VRD is the comparator arm in recent clinical trials comparing Daratumumab-VRD vs VRD before ASCT [9, 10]. Our study supports the use of VRD in both clinical practice, and as the standard treatment arm in clinical trials, as it is more effective than VCD and better tolerated than VTD. However, recent results from the PERSEUS trial [9], with significantly longer PFS for Daratumumab-VRD vs VRD, will most likely be practice changing.
We observed a statistically significant improvement in both PFS and OS favoring VRD. This must, however, be interpreted with caution. The difference in use of post-ASCT therapy, and the time periods in which the regimes were given, are two major biases. We corrected for this by performing a separate analysis for only patients who received ASCT in later years and who did not receive consolidation and/or maintenance therapy. In this analysis VRD still showed significantly longer PFS compared to VCD. The median PFS was also longer in the VRD group when only patients who received maintenance therapy were included, although the difference was not statistically significant. Multivariate analysis showed a statistically significant PFS benefit favoring VRD, but no statistically significant OS benefit. Given the median overall survival in our data of approximately 9.5 years in the VCD group, induction therapy administered for 24 months represents only a fraction of this total observed time. Therefore, the effect of induction therapy on overall survival may be modest, and other factors, like treatment options available at relapse, will have a significant impact on patient survival. Most patients in the VCD group received ASCT before 2017, when consolidation and maintenance treatment were uncommon and fewer treatment options were available at relapse, affecting the survival of this group negatively. Conversely, in the VRD group, most patients received ASCT after 2017. In this period, maintenance treatment was usually given (or consolidation treatment when maintenance was not reimbursed), and effective treatments like CD38-antibodies and carfilzomib were available at relapse.
The main limitation of the study is its retrospective nature, and as patients were not randomized, confounding factors cannot be excluded. However, the type of induction the patient received was mainly dependent on center and not on patient or disease factors. Standard induction therapy differed between regions, where some centers consistently used VCD while others consistently used VRD. In Norway, access to new therapies is similar for all, and national and regional treatment guidelines are usually the factors that determine choice of treatment, and to a lesser degree individual patient risk factors. Furthermore, a limitation was that we only included patients who received ASCT. Patients who died before ASCT, started induction therapy but for various reasons did not proceed to ASCT, including those who could not harvest enough stem cells, were not included. The follow-up time for VRD patients was relatively short. Patients were included from many different hospitals in Norway over a long period of time, with variable practices regarding timing of treatment start, dosing schedules, response assessment and supportive care. Although the data quality was generally good, some data was missing and incomplete.
Our study is the first to report from a comprehensive nationwide, population-based cohort with a very low proportion of patients lost to follow-up. This is a major strength, as the inclusion of a broad, heterogenous population increases the generalizability of the results and reduces the risk of selection bias. Our data included an overlap period where both regimens were given. Apart from the type of induction therapy, the treatment course between the two groups were similar; Length of induction treatment, the ASCT procedure and time to response evaluation was unchanged throughout the study period and similar for both groups.
In conclusion, our results suggests that VRD should be preferred to VCD as induction therapy for newly diagnosed MM patients who are eligible for ASCT.
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VRD versus VCD as induction therapy before autologous stem cell transplantation in multiple myeloma: a nationwide ... - Nature.com
What To Expect in Each Stage of Menopause – Health Essentials
Menopause is a natural part of aging that marks the end of the female reproductive years but many people dont know what to expect until theyre in the midst of it. Did you know, for example, that you could experience symptoms up to a decade before menopause actually begins?
Menopause specialistPelin Batur, MD, walks us through the stages of menopause and what you may be able to expect during each one.
The menopause process is all about hormones. Your body begins to produce less of the hormone calledestrogen, which regulates your menstrual cycle, and your ovaries start running low on eggs. But it doesnt happen all at once.
Heres a quick overview of the three stages of menopause:
Dr. Batur explains each stage in greater detail, including the symptoms you might experience andhow to find relief.
You can think about perimenopause as the runway to the big event. It can start as early as a decade before menopause, though the average amount of time spent in perimenopause is four years.
During this time, your body is, little by little, winding down its naturalovulation process. The most common sign of perimenopause isirregular periods and menstrual cycles.
As your estrogen levels start to decrease, your periods and menstrual cycles may start getting a little wonky sometimes, closer together, sometimes skipping cycles, Dr. Batur explains. You may also have some of the typical menopausal symptoms.
Not everyone experiences noticeable symptoms during perimenopause, but they can include:
There are two stages to perimenopause early menopause transition and late menopause transition though theyre not always cut-and-dry and distinguishable from one another.
This first stage of perimenopause is the very beginning when your body is just starting to experience hormonal changes. During this time, your periods and menstrual cycles are still coming regularly, but you may notice other symptoms:
This is a natural phase of life, so if your symptoms are mild, you may be able to make do with lifestyle changes like getting more sleep and upping your cardio, Dr. Batur says. But if theyre really bothersome, speak to your healthcare provider, even if youre still having regular menstrual cycles.
The late menopause transition is when youre gettinga little closer to menopause. Youre more likely to start experiencing irregular periods and menstrual cycles.
During perimenopause, youre not ovulating as regularly, Dr. Batur says. You have up-and-down levels of estrogen, and you may not make progesterone as consistently, so you may skip a menstrual cycle and then have heavy bleeding during the next period because your uterine lining has thickened up from the impact of the estrogen.
Eventually, as you get closer and closer to menopause, you start skipping periods for months at a time, she continues.
If this happens, bring it up with your healthcare provider especially if youre in your early 40s or younger, which can be a sign ofpremature menopauseor a condition calledprimary ovarian insufficiency.
When youve gone a full 12 months without having your period, youve entered menopause (assuming you havent stopped bleeding because of another medical condition or a medication).
That typically happens around age 52, Dr. Batur shares, and then, you live the rest of your life in menopause, where youre no longer ovulating and you no longer have the ability to bear children.
Menopause symptoms typically last for seven to 10 years (though your timeframe may vary), and they can range from mild to severe. If youre in the latter camp, experiencing bothersome symptoms that you just cant shake, dont feel like you have to soldier on in silence.
Just saying, grin and bear it and eat healthier and lose some weight doesnt cut it for people who are really suffering during this time in their lives, Dr. Batur states. Your healthcare provider will also want to make sure that your symptoms arent related to other medical conditions.
Once you enter menopause, youre in menopause for the rest of your life; this is also called the postmenopause stage.
But now, youre at a higher risk for other health concerns. A decrease in estrogen is a risk factor in conditions like:
The older you get, the more tuned in your healthcare provider should be to menopauses impact on your health. But if theyre not bringing it up, you definitely should even if youre feeling fine, but especially if youre not.
The stages of menopause shouldnt make you feel miserable. If your symptoms are especially bothersome and having an impact on your quality of life, its time to ask for help.
Tell your Ob/Gyn or your primary care doctor, Hey listen, I think my hormones are going haywire, Dr. Batur advises. They can talk you through the options, which may include any of the following (or a combination of them):
Just remember: Theres no quick fix for the symptoms of menopause. If you raise concerns about themduring an annual visit, your healthcare provider may ask you to come back for another appointment so the two of you can go more in-depth about what youre experiencing and thats OK.
This is a very individual thing, and it can be very complicated, especially depending on your medical history, Dr. Batur says. Schedule another appointment, if you need it, and make sure your concerns are being addressed during dedicated time with your provider.
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What To Expect in Each Stage of Menopause - Health Essentials
How much does it cost to use our services – GenderGP
The GenderGP Appraisal Pathway
This is a pathway designed by GenderGP which allows you to be in charge of your gender journey.
We believe that you are the expert in your gender, and by offering our expertise in healthcare, we can make sure that your medical transition is suited to what you need and is delivered in the safest way possible.
By going through this process you will be able to provide us with information about you and your health and your gender feelings. This will allow us to come to a joint agreement on the best treatment for you.
If you are in the UK or the EU, we can use our private prescription service. If you are outside the EU then we will carry out any necessary assessments and then issue you with a Treatment Summary for your provider so they can do blood testing and prescribe under our direct supervision.
We will make all the decisions on medication and blood results to keep you safe.
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How much does it cost to use our services - GenderGP
Pharmac seeking bids from suppliers to fund another type of hormone replacement therapy – New Zealand Doctor Online
Pharmac Te Ptaka Whaioranga has issued a request for proposals (RFP) asking suppliers to bid for the supply of oestradiol gel in New Zealand.
Pharmac is looking to fund a new type of oestradiol treatment without restrictions, says Dr David Hughes, Pharmacs Director Advice and Assessment/Chief Medical Officer.
In the past three years, the supply issues for oestradiol patches has caused stress and frustration for New Zealanders. Demand has more than doubled - growing from 1.2 million patches dispensed in 2020/21 to over 3 million patches in 2022/23.
Our clinical advisors have told us that funding another oestradiol product would be useful because demand is increasing, and we are continuing to experience global supply issues for oestradiol patches. We know that some people would use the gel if its funded, and this could relieve some of the stress on the supply of patches.
Oestradiol is used by 85,000 people each year for the treatment of a range of conditions including, menopause, osteoporosis, and gender affirming health care. It is most often used as a patch placed on the skin, but it is also available as a tablet.
We want to make sure people get the treatment they need, and which can be funded from Pharmacs fixed budget, so were keen to hear from suppliers about what they can offer, says Dr Hughes.
Dr Linda Dear, a menopause specialist says, Its wonderful to hear that another step is being taken towards giving perimenopausal and menopausal New Zealanders fully funded access to oestradiol (estrogen) gels.
This will provide a much-needed alternative, so people are no longer solely reliant on the patches as the only funded transdermal option available. Having gels as an alternative will ease the pressure of the supply issue which has had an impact on New Zealanders using the treatments, pharmacists, and prescribing doctors alike. My hope is that we dont have to wait too much longer to access this important therapy.
Pharmacs job is to assess and prioritise which treatments will deliver the best possible health outcomes for New Zealanders from the available budget, says Dr Hughes.
"Once this RFP closes, an evaluation committee will meet to consider the bids received. We will also seek advice from our clinical advisors. As this activity progresses, well share more information with the public.
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Pharmac seeking bids from suppliers to fund another type of hormone replacement therapy - New Zealand Doctor Online
Gene therapy offers hope for giant axonal neuropathy patients – UT Southwestern
Co-author Steven Gray, Ph.D., is Associate Professor of Pediatrics, Molecular Biology, Neurology, and in the Eugene McDermott Center for Human Growth and Development at UTSouthwestern.
DALLAS March27, 2024 A gene therapy developed by researchers at UTSouthwestern Medical Center for a rare disease called giant axonal neuropathy (GAN) was well tolerated in pediatric patients and showed clear benefits, a new study reports. Findings from the phase one clinical trial, published in the New England Journal of Medicine, could offer hope for patients with this rare condition and a host of other neurological diseases.
This trial was the first of its kind, for any disease, using an approach to broadly deliver a therapeutic gene to the brain and spinal cord by an intrathecal injection, said co-author Steven Gray, Ph.D., Associate Professor of Pediatrics, Molecular Biology, Neurology, and in the Eugene McDermott Center for Human Growth and Development at UTSouthwestern. Even with the relatively few patients in the study, there were clear and statistically significant benefits demonstrated in patients that persisted for years.
Dr. Gray developed this gene therapy with co-author Rachel Bailey, Ph.D., Assistant Professor in the Center for Alzheimers and Neurodegenerative Diseases and of Pediatrics at UTSW.Dr. Gray is an Investigator in thePeter ODonnell Jr. Brain Institute.
GAN is extraordinarily rare, affecting only about 75 known families worldwide. The disease is caused by mutations in a gene that codes for a protein called gigaxonin. Without normal gigaxonin, axons the long extensions of nerve cells swell and eventually degenerate, leading to cell death. The disease is progressive, typically starting within the first few years of a childs life with symptoms including clumsiness and muscle weakness. Patients later lose the ability to walk and feel sensations in their arms and legs, and many gradually lose hearing and sight and die by young adulthood.
In the clinical trial conducted at the National Institutes of Health (NIH), Drs. Gray and Bailey worked with colleagues from the National Institute of Neurological Disorders and Stroke (NINDS) to administer the therapy to 14 GAN patients from 6 to 14 years old. Using a technique they developed to package the gene for gigaxonin into a virus called adeno-associated virus 9 (AAV-9), the researchers injected it into the intrathecal space between the spinal cord and the thin, strong membrane that protects it. Tested for the first time for any disease, this approach enabled the virus to infect nerve cells in the spinal cord and brain to produce gigaxonin in nerve cells, allowing them to heal the cells axons, which grow throughout the body.
Amanda Grube, 14, one of the trial's participants, has seen improvement in her diaphragm and other muscles associated with breathing, her mother says. However, many of Amanda's other functions, including her mobility, did not benefit. (Photo credit: McKee family)
After one injection, the researchers followed the patients over a median of nearly six years to determine whether the treatment was safe and effective. Only one serious adverse event was linked to the treatment fever and vomiting that resolved in two days suggesting it was safe. Over time, some patients showed significant recovery on an assessment of motor function. Other measurements revealed that several of the patients improved in how their nerves transmitted electrical signals.
One of the trials participants, 14-year-old Amanda Grube, has experienced improvement in her diaphragm and other muscles associated with breathing, according to her mother, Katherine McKee. However, many of Amandas other functions did not benefit including her mobility.
Thats why I hope theres more to come from the research that can help patients even more,Mrs. McKee said. Amanda has dreams and ambitions. She wants to work with animals, save the homeless, and design clothes for people with disabilities.
Dr. Gray said that in many ways, the study offers a road map to carry out similar types of clinical trials. The findings have broader implications because this study established a general gene therapy treatment approach that is already being applied to dozens more diseases, he said.
Although the phase one results are promising, Dr. Gray said he and other researchers will continue to fine-tune the treatment to improve results in future GAN clinical trials. He is also using this method for delivering gene therapies to treat other neurological diseases at UTSW, where he is Director of the Translational Gene Therapy Core, and at Childrens Health. Work in theGray Labhas already led to clinical trials for diseases including CLN1 Batten disease, CLN5 Batten disease, CLN7 Batten disease, GM2 gangliosidosis, spastic paraplegia type 50, and Rett syndrome.
The GAN study was funded by the National Institute of Neurological Disorders and Stroke (NINDS), Division of Intramural Research, NIH; Hannahs Hope Fund; Taysha Gene Therapies; and Bamboo Therapeutics-Pfizer.
Drs. Bailey and Gray are entitled to royalties from Taysha Gene Therapies. Dr. Gray has also consulted for Taysha and serves as Chief Scientific Adviser.
About UTSouthwestern Medical Center
UTSouthwestern, one of the nations premier academic medical centers, integrates pioneering biomedical research with exceptional clinical care and education. The institutions faculty members have received six Nobel Prizes and include 25 members of the National Academy of Sciences, 21 members of the National Academy of Medicine, and 13 Howard Hughes Medical Institute Investigators. The full-time faculty of more than 3,100 is responsible for groundbreaking medical advances and is committed to translating science-driven research quickly to new clinical treatments. UTSouthwestern physicians provide care in more than 80 specialties to more than 120,000 hospitalized patients, more than 360,000 emergency room cases, and oversee nearly 5 million outpatient visits a year.
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Gene therapy offers hope for giant axonal neuropathy patients - UT Southwestern
Story of boy with ultra-rare UBA5 disorder being studied at UMass Chan goes to the moon – UMass Medical School
Raiden Pham
Parents of children with rare diseases go to endless lengths to raise funds and awareness for research that might lead to a cure. Now, the story of 4-year-old Raiden Pham has been to the moon. He has an ultra-rare neurodegenerative disease known as UBA5 disorder that UMass Chan Medical School researchers are targeting.
The story of Raidens journey and its message of love, hope and strength is included on an indestructible digital time capsule of art, music, film and history, as part of the Lunaprise Moon Museum Mission, which was onboard the Odysseus spacecraft that landed on the moon Feb. 22.
When we think about gene therapy, or any kind of cure or treatment for these rare diseases, its always considered a moonshot, but thats not the case anymore in todays world, said Tommy Pham, Raidens father. Were willing to do whatever it takes to save my son and kids with UBA5 disorder and hopefully inspire the next generation of rare disease parents to go on this fight and have hope.
Since 2021, the Raiden Science Foundation, founded by Tommy and Linda Pham, of Beaverton, Oregon, on behalf of their son, has raised around $1 million of its $4 million goal, which supports research in UMass Chans Translational Institute for Molecular Therapeutics and other partner institutions.
The research on UBA5 is led by Toloo Taghian, PhD, instructor in radiology in the lab of Heather Gray-Edwards, DVM, PhD, assistant professor of radiology in the Horae Gene Therapy Center.
Dr. Taghian has identified the top two viral vector constructs for UBA5 expression in-vivo, which show great promise in successfully delivering UBA5 gene therapy to the targeted cells. Taghian and her team are now testing their efficacy in correcting the protein malfunction and treating the underlying cause of this disease and will soon initiate toxicology studies to assess their safety.
Working with Raiden Science Foundation to develop a gene therapy for UBA5 has been an impactful journey, said Taghian. The dedication of the Pham family in supporting UBA5 research allows the UMass Chan team to work toward unpacking the basic science underlying this ultra-rare disease in parallel with our gene therapy development program.
How Raidens story got to the moon was a journey of persistent efforts to raise awareness and support by the Phams. In October 2022, Raiden Science Foundation held a gaming charity stream, Kombat4Rare, based on the Mortal Kombat franchise. One of the main characters in Mortal Kombat is Raiden, named after the God of Thunder in Japanese mythology.
The response from the gaming and entertainment community was enthusiastic, and a few months later Tommy Pham was invited to be featured at a charity event in Marina del Rey, California. Dallas Santana, founder of Space Blue, the exclusive curator of the Lunaprise Museum, was touched by Tommys message and told him Raidens story should be included on the Lunaprise Moon Museum to inspire people on Earth.
Noting that it has been more than 50 years since the last American space capsule landed on the moon, Tommy said, We dont have to wait another 50 years for gene therapy. It could be done now in the coming years. We need to figure out a way with all the right stakeholders to unlock gene therapy for so many other kids suffering different rare diseases, not just us.
Donations to support UBA5 gene therapy at UMass Chan can be made here.
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Story of boy with ultra-rare UBA5 disorder being studied at UMass Chan goes to the moon - UMass Medical School
PROTECT TEENAGERS FROM HARMFUL AND IRREVERSIBLE MEDICAL TREATMENT – CrowdJustice
The Care Quality Commission (CQC) has registered Gender Plus Hormone Clinic to provide hormone treatments to 16 and 17-year-old children.
This paves the way for other private clinics to be registered, which would offer controversial medical treatments with lifelong consequences to vulnerable teenagers. The decision of the CQC to license a private clinic, creates a significant risk of a two tier approach, with less protection for those who seek help from the private sector. This further risks undermining the work of the Cass review for NHSE practice.
I want the court to set aside the registration by the CQC of Gender Plus Hormone Clinic to provide hormone treatment for teenagers. I also hope that this litigation will prevent the registration of other private clinics providing this controversial treatment. I want to ensure that those under 18 years old, do not suffer irreversible, lifelong harms both physical and psychological, from taking a controversial hormonal treatment which is not evidenced as safe or effective.
Why I am asking for this Judicial Review
I was in the NHS for nearly 40 years and I am now a psychotherapist in private practice. I have worked with people who present with issues around their gender identity for over 20 years. In my clinical experience of working with children and young people, I have not, to date, encountered a 16 to 17-year-old who I would have assessed to be sufficiently fully informed and psychologically ready to make such a life changing, potentially harmful decision. They are in the process of development from child to adult which involves significant mental and physical adjustments. Many of the young people with gender dysphoria/incongruence have no clear understanding of their underlying motivations to take cross, sex, hormones. However they are usually very aware of the discomfort they experience, and often hold a strong belief that the medication will help them feel better. They hope a change to their physical body will bring about a comfort in their mind. Some also receive strong messages from certain groups that medication is the answer to their difficulties which creates an urgent pressure on them and those around them for a solution. As a result, they are rarely able to give a full, in-depth psychological consideration to the implications and consequences of commencing a physical treatment, which is known to have serious, harmful side-effects, and, as yet has a very low level evidence base for it's efficacy and safety.
Under its current registration by the CQC, Gender Plus Hormone Clinic (GHPC) is not prevented from providing GnRH analogues (blockers) for the purpose of suspending puberty. There are some 16-year-olds who have not reached pubertal maturation. Further, the GPHC has said that it would prescribe puberty blockers alongside oestrogen therapy to achieve feminising effects. The NICE report (National Institute of Clinical Excellence) and the Cass review both state that this treatment model is not proven.
There is also considerable risk of complications due to this powerful medication. There are many known side-effects, including blood clots, gallstones, vaginal atrophy and male pattern baldness for females and potential loss of fertility, amongst many others.
The evidence base
The Cass review was commissioned by the NHS to provide a comprehensive review of the appropriate treatment for children and young people with gender dysphoria. The Cass Review sought advice from the National Institute for Health and Care Excellence (NICE) which conducted two separate evidence reviews.
Neither of them has found sufficient evidence to support the use of either puberty blockers or cross sex hormones as safe and effective.
In her interim report published in February 2022, Dr Cass has emphasised the gaps in the "evidence base regarding hormone treatment" (Para 1.41). Although some of her observations related specifically to puberty blockers, she also addressed cross-sex, hormones, and hormone treatment more generally. She said, among other things:
"The Review is not able to provide definitive advice on the use of puberty blockers and feminising/masculinising hormones at this stage, due to gaps in the evidence base; however, recommendations will be developed as our research programme progresses.
The lack of available high-level evidence was reflected in the recent NICE review into the use of puberty blockers and feminising/masculinising hormones commissioned by NHS England, with the evidence being too inconclusive to form the basis of a policy position(para 5.21)
At present we have the least information for the largest group of patients birth- registered females first presenting in early teens(para 5.11).
Your help:
I need your help to ensure that the registration of GPHC is cancelled and the other private clinics are unable to prescribe this controversial treatment to children under 18. We should not be careless or look away from the potential harms this medical treatment might cause to childrens previously healthy bodies.
Please support me with the legal fees required to mount a judicial review and challenge the CQC decision. I was the original claimant who started the Kiera Bell JR with Mrs A and our application on that occasion was successful in providing further scrutiny and attention in this area of paediatric healthcare. That judicial review potentially helped prevent irreversible harms to much younger children too as it led to a much wider scrutiny of the model of treatment in the GIDS.
I have assembled an expert legal team and will be lodging my claim with the High Court in the next few days. Please join me in seeking to protect vulnerable young people and share this crowdfunder link. I know these cases keep coming but we need to protect the next generation.
My X (twitter) handle is @sueevansprotect
Thank you very much.
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PROTECT TEENAGERS FROM HARMFUL AND IRREVERSIBLE MEDICAL TREATMENT - CrowdJustice
Why the sci-fi dream of cryonics never died – MIT Technology Review
The environment was something of a shift for Drake, who had spent the previous seven years as the medical response director of the Alcor Life Extension Foundation. Though it was the longtime leader in cryonics, Alcor was still a small nonprofit. It had been freezing the bodies and brains of its members, with the idea of one day bringing them back to life, since 1976.
The foundation, and cryonics in general, had long survived outside of mainstream acceptance. Typically shunned by the scientific community, cryonics is best known for its appearance in sci-fi films like 2001: A Space Odyssey. But its adherents have held on to a dream that at some point in the future, advances in medicine will allow for resuscitation and additional years on Earth. Over decades, small, tantalizing developments in related technology, as well as high-profile frozen test subjects like Ted Williams, have kept the hope alive. Today, nearly 200 dead patients are frozen in Alcors cryogenic chambers at temperatures of 196 C, including a handful of celebrities, who have paid tens of thousands of dollars for the goal of possible revival and ultimately reintegration into society.
But its the recent involvement of Yinfeng that signals something of a new era for cryonics. With impressive financial resources, government support, and scientific staff, its one of a handful of new labs focused on expanding the consumer appeal of cryonics and trying anew to bring credibility to the long-disputed theory of human reanimation. Just a year after Drake came on board as research director of the Shandong Yinfeng Life Science Research Institute, the subsidiary of the Yinfeng Biological Group overseeing the cryonics program, the institute performed its first cryopreservation. Its storage vats now hold about a dozen clients who are paying upwards of $200,000 to preserve the whole body.
Still, the field remains rooted in faith rather than any real evidence that it works. Its a hopeless aspiration that reveals an appalling ignorance of biology, says Clive Coen, a neuroscientist and professor at Kings College London.
Even if one day you could perfectly thaw a frozen human body, you would still just have a warm dead body on your hands.
The cryonics process typically goes something like this: Upon a persons death, a response team begins the process of cooling the corpse to a low temperature and performs cardiopulmonary support to sustain blood flow to the brain and organs. Then the body is moved to a cryonics facility, where an organ preservation solution is pumped through the veins before the body is submerged in liquid nitrogen. This process should commence within one hour of deaththe longer the wait, the greater the damage to the bodys cells. Then, once the frozen cadaver is ensconced in the cryogenic chamber, the hope of the dead begins.
Since its beginnings in the late 1960s, the field has attracted opprobrium from the scientific community, particularly its more respectable cousin cryobiologythe study of how freezing and low temperatures affect living organisms and biological materials. The Society for Cryobiology even banned its members from involvement in cryonics in the 1980s, with a former society president lambasting the field as closer to fraud than either faith or science.
In recent years, though, it has grabbed the attention of the libertarian techno-optimist crowd, mostly tech moguls dreaming of their own immortality. And a number of new startups are expanding the playing field. Tomorrow Biostasis in Berlin became the first cryonics company in Western Europe in 2019, for example, and in early 2022, Southern Cryonics opened a facility in Australia.
More researchers are open to longer-term, futuristic topics than there might have been 20 years ago or so, says Tomorrow Biostasis founder Emil Kendziorra.
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Why the sci-fi dream of cryonics never died - MIT Technology Review