University of Barcelona

EMA Designates Allopurinol as First Orphan Drug for Marfan Syndrome

3 days ago

• The European Medicines Agency has designated allopurinol as the first orphan drug for Marfan syndrome, a rare connective tissue disease affecting approximately 7 in 100,000 people in the European Union. • Researchers from the University of Barcelona, IDIBAPS, and CIBERER have demonstrated allopurinol's potential to halt and prevent aortic aneurysms in animal models, with international clinical trials in patients planned for the future. • This repurposing of allopurinol, currently used for gout treatment, represents a significant advancement for Marfan syndrome patients who currently have no curative options beyond limited palliative treatments and high-risk surgical interventions.

CRISPR Gene Editing Breakthrough Saves Baby with Ultra-Rare Metabolic Disorder

7 days ago

• Doctors at Children's Hospital of Philadelphia successfully treated a baby with severe CPS1 deficiency using a personalized CRISPR base-editing therapy, marking a first-of-its-kind approach for this rare metabolic disorder. • The experimental treatment, developed within just six months of diagnosis, corrected the infant's specific genetic mutation by delivering edited DNA to liver cells via lipid nanoparticles, allowing him to reduce medication and process more dietary protein. • This breakthrough demonstrates the potential for creating customized gene therapies for millions with rare genetic diseases, with researchers suggesting costs comparable to liver transplantation and possibilities for treating numerous other conditions.

Breakthrough Wireless Device Enables Remote Activation of Light-Sensitive Drugs for Pain Management

14 days ago

• Scientists have developed the first wireless device capable of remotely activating photoactivable drugs, demonstrating effective pain management using photolabile morphine without typical opioid side effects. • The millimeter-sized implantable device uses NFC technology to wirelessly power a microLED that precisely releases active morphine only in targeted tissues, achieving comparable analgesic effects to systemic administration. • This photopharmacology breakthrough could revolutionize treatment for various conditions including epilepsy, Parkinson's disease, psychiatric disorders, and cancer by enabling localized drug delivery without systemic adverse effects.

REVEAL GENOMICS and Ona Therapeutics Partner to Advance Novel ADC Cancer Treatment with Biomarker Strategy

a month ago

• REVEAL GENOMICS will conduct comprehensive molecular analysis of tumor and blood samples from Ona Therapeutics' ONA-255 Phase 1-2 clinical trial to identify biomarkers and define patient subgroups. • ONA-255 is positioned as a first-in-class antibody-drug conjugate targeting a novel protein present in multiple solid tumor types, designed to overcome resistance mechanisms in advanced cancers. • The collaboration, supported by a grant from Spain's CDTI, combines Ona's ADC development expertise with REVEAL GENOMICS' diagnostic capabilities to accelerate precision medicine approaches for aggressive solid tumors.

Bladder-Preserving Treatment Shows Promise: Radiation Plus Dual Immunotherapy Achieves 93% Complete Response in MIBC

3 months ago

• A novel combination of radiation therapy with durvalumab and tremelimumab immunotherapy achieved a 93% complete response rate in muscle-invasive bladder cancer patients, offering a promising bladder-preserving alternative. • The IMMUNOPRESERVE trial demonstrated strong efficacy with 30 out of 32 patients maintaining their bladders after a median follow-up of 27 months, with 84% two-year overall survival rate. • The treatment regimen showed manageable safety profile with 31% grade 3-4 adverse events, presenting a potential alternative to radical cystectomy for eligible patients.

Landmark Prevention Trial Launches to Stop Alzheimer's Before Symptoms Begin in Young Adults

4 months ago

• WashU Medicine initiates groundbreaking international trial testing Eli Lilly's remternetug in young adults as young as 18, targeting Alzheimer's prevention up to 25 years before expected symptom onset. • The Primary Prevention Trial will enroll 240 participants from families with genetic mutations, focusing on removing or preventing amyloid beta plaques before cognitive symptoms develop. • The $130 million study represents a collaborative effort between academic institutions, pharmaceutical industry, and foundations, with support from NIH, Alzheimer's Association, and private donors.

TACE Plus Camrelizumab and Rivoceranib Improves PFS in Unresectable HCC

4 months ago

• The CARES-005 study demonstrated that adding camrelizumab and rivoceranib to TACE significantly prolonged progression-free survival (PFS) in patients with unresectable hepatocellular carcinoma (HCC). • Median PFS was 10.8 months with TACE plus camrelizumab and rivoceranib, compared to 3.2 months with TACE alone (HR 0.34, P<0.0001) at a median follow-up of 13.6 months. • Objective response and disease control rates were also higher in the TACE-CR group, indicating enhanced antitumor efficacy with the combination therapy. • The safety profile of the combination was manageable, with adverse events consistent with those known for TACE, camrelizumab, and rivoceranib.

Three-pronged Treatment Improves Prognosis for Inoperable Liver Cancer

4 months ago

Recent studies published in The Lancet reveal that combining two targeted therapies with traditional chemoembolization significantly delays disease progression in inoperable liver cancer patients, marking a breakthrough in treatment strategies.

ESMO Highlights: Imfinzi's Bladder Cancer Success, Keytruda/Lenvima Combo for Liver Cancer, and Opdualag's Lung Cancer Challenge

8 months ago

• AstraZeneca's Imfinzi demonstrated a 25% reduction in mortality risk for bladder cancer patients when administered before and after surgery, showing a significant clinical benefit. • Merck and Eisai's combination of Keytruda and Lenvima, when added to standard care for liver cancer, improved progression-free survival to 14.6 months compared to 10 months with standard care alone. • Bristol Myers Squibb's Opdualag faces scrutiny in lung cancer treatment due to concerns over trial design and the modest progression-free survival benefit observed in a specific subgroup.

LEAP-012 Trial: Lenvatinib, Pembrolizumab, and TACE Improve PFS in Intermediate-Stage HCC

8 months ago

• The LEAP-012 trial demonstrated that combining lenvatinib and pembrolizumab with TACE significantly improves progression-free survival (PFS) in intermediate-stage hepatocellular carcinoma (HCC). • The combination therapy showed a median PFS of 14.6 months compared to 10 months with TACE alone, representing a 34% reduction in the risk of disease progression or death. • The objective response rate (ORR) was also higher in the combination arm (46.8%) compared to the placebo arm (33.3%), indicating enhanced clinical activity. • While treatment-related adverse events were more frequent in the combination arm, they were generally manageable, suggesting a favorable risk-benefit profile.

Inhaled Colistimethate Shows Promise for Bronchiectasis, Despite COVID-19 Impact

8 months ago

• In the PROMIS-I trial, inhaled colistimethate sodium significantly reduced the mean annual exacerbation rate in bronchiectasis patients with _Pseudomonas aeruginosa_ infections. • The PROMIS-II trial, impacted by the COVID-19 pandemic, did not show a significant reduction in exacerbation rates with the same treatment. • A meta-analysis of PROMIS-I, pre-pandemic PROMIS-II data, and an earlier phase II study supported the efficacy of colistimethate sodium in reducing exacerbation rates. • Inhaled colistimethate sodium demonstrated a safety profile similar to placebo, with low rates of bronchospasm and manageable adverse events.