The Henry M. Jackson Foundation for the Advancement of Military Medicine’s Post

ASCO 2023 - New Data Highlights Three Studies Showing The Impact Of Vitrakvi On Long-Term Efficacy & Safety In Adult And Pediatric Patients Read more: https://bit.ly/3RkkBjO #OncologyInspired #PatientInspired #PrecisionOncology

Reflections on Rare Disease Day 2024: As a pediatric neurologist, I spent my entire post-graduate career involved in the diagnosis and management of children and adults with both neuronopathic and non-neuronopathic lysosomal storage disorders. For those without brain involvement I had access to a variety of life saving medications that transformed the lives of these patients (e.g. Gaucher has 5 FDA approved therapies). However, I would then (usually within the same day) meet with families of children with the neurodegenerative forms of these disorders and all I had to offer was symptomatic care. Symptomatic care is critical in addressing their quality of life; however, it did not alter their trajectory enough to prevent disease progression. I've had countless end of life discussions in my clinical career; and I have sat at the bedside of dying children and attended too many funerals. In recent years, I had renewed hope as various industry sponsors developed therapies that could potentially cross the blood brain barrier to treat the brain. This is an important moment for rare neurogenetic diseases like neuronopathic MPS and I am grateful to be in a position (in industry) to help on a global level. It is the reason why I left my clinic and joined Ultragenyx - to have a greater impact in bringing forward new therapies to treat the brain and ultimately to be able to reach more patients and families. However, we are seeing a lack of progress in these promising programs. It has been heartbreaking to see viable therapies discontinued. The Reagan Udall Biomarker Workshop on CSF HS in nMPS that occurred last Wednesday, February 21 was a pivotal moment in the rare disease space because it demonstrated that there is a consensus from the medical/scientific, industry and patient/caregiver community about the path forward for drug development in nMPS. There is overwhelming evidence that CSF HS is a reasonably likely surrogate endpoint to predict clinical outcomes and therefore appropriate for use in an accelerated approval pathway. I was honored to be involved in coordinating this workshop and to present alongside my physician colleagues, renowned scientists in MPS, and most importantly, the parents of children affected by this group of devasting diseases. We provided a unified and loud message that the time is NOW to act swiftly. We cannot wait any longer as another generation of children die while awaiting access to life saving therapies. #Timeisbrain. https://lnkd.in/exssNXHp

A study evaluating the differences in survival, renal outcomes, and adverse reactions in children with antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV)-associated nephritis who are receiving different treatment based on the revised pediatric AAV renal risk grade (PARRG) risk stratification is now recruiting. In the prospective, multicenter, open, matched control clinical study, researchers will also evaluate the efficacy and safety of glucocorticoid therapy combined with rituximab and cyclophosphamide as an induction regimen in the high-risk group of patients, as well as glucocorticoid therapy combined with rituximab as an induction regimen in low- and middle-risk groups of children with AAV based on PARRG risk stratification. Learn more: https://brnw.ch/21wBT52

If you’re an oncology nurse, having a questioning attitude is one of the most valuable safety tools you possess. Learning and teaching patients about availability of genomic testing and the implications of preventing serious and sometimes life threatening toxicities from chemo can mean safer more precise choices and dosage. The experience of cancer treatment is as individual as the person. We are the gatekeepers and safety nets for our patients. This is a terrific review of pharmacodynamics that takes about 40 minutes to get us thinking more deeply about the future of cancer care and precision medicine.

HOT OFF THE PRESS! Pediatric #rarediseases - those that primarily impact children, and often take their lives before they have a chance to experience adulthood - are particularly hard to study, and #drugdevelopment for these therapies is particularly risky. That is why #Congress established the rare pediatric disease (RPD) priority review voucher (PRV) program in 2012 to create additional incentives to study these diseases. The success has been resounding. To date, many rare disease communities have directly benefitted from drug development made economically possible by the program, and many children and families had the chance to live longer, fuller lives as a result. As many as 35 different rare disease communities have received vouchers. Yet, one key piece of information missing about the program so far has been how the program shapes the earlier pipeline of new therapies - after all, it takes many years to develop a rare disease drug and RPD designations, different from orphan drug designations or RPD vouchers, are not publicly announced. Fortuitously, FDA just published data on these designations for the first 10 years of the program, confirming the immense benefit of this program for the rare disease community! Authorization for the RPD PRV program is set to expire on September 30th of this year. Last week, the Creating Hope Reauthorization Act, legislation to reauthorize the program, was introduced in the House with strong bipartisan support. The new data just released makes it clear that the program fosters rare disease drug development and that Congress should reauthorize the program asap - too many lives depend on it!

Too old for paediatric trials, too young for adult trials Participants between 13 and 24 were included in only 0.04% of paediatric/adult oncology clinical trials where the minimum age for eligibility was 13. - https://lnkd.in/dsZzq-hq #clinicaltrials

'Radiopharmaceuticals offer a survival benefit to mCRPC patients as well as managing bone pain and #QoL' One of the key clinical takeaways of our latest GU NURSES CONNECT #OncologyNursing update. Get the rest & watch the 3-min video: https://ow.ly/ngbj50PNKUY #MedEd #PCSM

The Council of Ministers approved an amendment to the Royal Decree on paediatric oncology in Belgium which groups patients in a smaller number of reference centres and creates networks to organise parts of the treatment trajectory close to the patient's home. This measure follows the recommendation of the European Reference Network for paediatric oncology (ERN PaedCan), which says that reference centres should treat at least 100 new cases per year, instead of the current minimum of 50. Doctors and other caregivers now face the challenge of organising such networks between reference centres and shared proximity care with the aim to provide the best chance of survival for each child while ensuring quality of life and survival during treatment and beyond. Read more here: https://lnkd.in/eckzFjzF

Exploring strategies to build an efficient and effective PCM/CCM program? Join Kathy Oubre, MS, CEO of Pontchartrain Cancer Center, and Canopy on Wednesday, January 24 at 1 PM EST for a live webinar where we will discuss how to: • Quickly identify eligible patients • Strategically focus enrollment efforts • Scale processes without adding burden for staff Register today to reserve your spot. #oncology #nurses #epro #valuebasedcare #nursing #chroniccaremanagement #caremanagement

PPI meds, such as Prilosec, decrease the risk of esophageal cancer in patients with Barret's Esophagus (BE). Less than half of patients with BE are adherent to their medical regimen. Looks like MediTeddi could help here. #MediTeddi #PeaceofMind #EsophagealCancer #Caregivers