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CASGEVY gene therapy eliminates vaso-occlusive crises in sickle cell patients

In a landmark study, an international consortium led by researchers at Children's Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).

24 Apr 2024

Novel immunotherapy shows promise for high-risk sarcomas: HEROS 2.0 trial results published

Researchers at Texas Children’s Cancer Center and the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children’s Hospital and Houston Methodist published results of a phase I clinical trial of a novel immunotherapy for high-risk sarcomas in the journal Nature Cancer.

24 Apr 2024

WOO Network trials investigate innovative treatments for liver, head and neck cancers

A new round of clinical trials supported by the Ontario Institute for Cancer Research (OICR) will harness a unique opportunity to test some of the newest treatment strategies for a range of different cancers.

18 Apr 2024

Phase 2 trial reveals Lixisenatide may reduce motor disability in Parkinson’s patients

A trial of lixisenatide to assess its effects in early Parkinson’s disease.

18 Apr 2024

Phase 3 trial shows overall survival benefit from adjuvant therapy in kidney cancer patients

For the first time in fifty years, results from a phase 3 randomized, placebo-controlled trial have shown an overall survival benefit from an adjuvant therapy in patients with kidney cancer.

17 Apr 2024

Vosoritide's first phase 2 study shows increased growth in children with hypochondroplasia

Vosoritide's first global phase 2 study showed an average increased growth rate of 1.8 cm per year in children with hypochondroplasia, a genetic cause of short stature in children, according to researchers from Children's National Hospital.

13 Apr 2024

Home-administered nasal spray could be safely used to treat recurrent rapid heart rhythms

A clinical trial led by Weill Cornell Medicine investigators showed that a nasal spray that patients administer at home, without a physician, successfully and safely treated recurrent episodes of a condition that causes rapid abnormal heart rhythms.

9 Apr 2024

Personalized anti-tumor vaccine enhances immunotherapy for liver cancer

Adding a personalized anti-tumor vaccine to standard immunotherapy is safe and about twice as likely to shrink cancer as standard immunotherapy alone for patients with hepatocellular carcinoma, the most common type of liver cancer, according to a clinical trial led by researchers at the Johns Hopkins Kimmel Cancer Center and its Convergence Institute.

8 Apr 2024

Ninerafaxstat shows good tolerability and safety in nonobstructive HCM patients

The investigational drug ninerafaxstat showed a good tolerability and safety profile, along with evidence of improvements in symptoms and exercise capacity among people with nonobstructive hypertrophic cardiomyopathy (HCM), according to results of a 12-week phase 2 trial presented at the American College of Cardiology's Annual Scientific Session.

8 Apr 2024

Treating early-stage heart failure in diabetic patients: Insights from ARISE-HF trial

One of the first studies to attempt to treat early-stage heart failure in patients with Type 2 diabetes did not meet its primary endpoint, but a preplanned subgroup analysis found a statistically significant effect of treatment in patients who were not taking SGLT2 inhibitors or GLP-1 RAs, which are antidiabetic medications that also have benefits for the heart.

8 Apr 2024

Investigational drug plozasiran shows promise in reducing severe hypertriglyceridemia

In patients with severely elevated triglyceride levels at risk for developing acute pancreatitis, the investigational drug plozasiran reduced triglyceride levels by an average of 74% after 24 weeks of use without causing any significant safety concerns, according to research presented at the American College of Cardiology's Annual Scientific Session.

8 Apr 2024

Novel combination therapy shows promise in difficult-to-treat endometrial cancer, study finds

In a small, investigator-initiated phase 2 study by Dana-Farber Cancer Institute investigators, a novel combination of an antibody-drug conjugate and an immune checkpoint inhibitor showed notable activity in pre-treated patients with a difficult-to-treat form of endometrial cancer.

5 Apr 2024

Clinical trial to test the feasibility of using at-home cancer immunotherapy

A clinical trial from Keck Medicine of USC will test the feasibility of treating non-small cell lung cancer with immunotherapy provided at home.

28 Mar 2024

UC researchers open Phase 2 clinical trial to test new combination treatment for glioblastomas

A multidisciplinary team of University of Cincinnati Cancer Center researchers have opened a Phase 2 clinical trial to test a new combination treatment for glioblastomas (GBM), the most deadly form of brain tumors.

26 Mar 2024

Minocycline fails to slow vision loss in people with dry age-related macular degeneration

The drug minocycline, an antibiotic that also decreases inflammation, failed to slow vision loss or expansion of geographic atrophy in people with dry age-related macular degeneration (AMD), according to a phase II clinical study at the National Eye Institute (NEI), part of the National Institutes of Health.

22 Mar 2024

Investigational gene therapy shows promise for rare childhood neurodegenerative disease

An investigational gene therapy for a rare neurodegenerative disease that begins in early childhood, known as giant axonal neuropathy (GAN), was well tolerated and showed signs of therapeutic benefit in a clinical trial led by the National Institutes of Health.

20 Mar 2024

New treatment approach improves recurrence-free survival in early breast cancer patients

A new treatment approach that combines a targeted therapy drug with hormone therapy significantly increased the amount of time a person with stage 2 or 3 HR-positive, HER2-negative early breast cancer lives without the cancer returning, according to a new study co-led by UCLA Health Jonsson Comprehensive Cancer Center investigators.

20 Mar 2024

Pembrolizumab enhances breast cancer treatment regardless of age or menopausal status

New data from the KEYNOTE-756 phase 3 clinical trial show that adding the immunotherapy drug, pembrolizumab, to chemotherapy before and after surgery for breast cancer leads to better outcomes for patients regardless of their age or menopausal status.

20 Mar 2024

Apple cider vinegar shows promise for weight management in overweight and obese individuals

Small daily quantities of apple cider vinegar for 12 weeks aid weight management in people who are overweight or obese, suggest the results of a small comparative clinical trial, published in the open access journal BMJ Nutrition, Prevention & Health.

18 Mar 2024

Innovative levodopa infusion pump trial shows promise for reducing Parkinson's symptoms

An international, multisite phase 3 trial co-led by a University of Cincinnati researcher found Parkinson's disease medication delivered through an infusion pump is safe and effective at reducing symptoms for longer periods of time.

15 Mar 2024

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Drug Trial News

CASGEVY gene therapy eliminates vaso-occlusive crises in sickle cell patients

Novel immunotherapy shows promise for high-risk sarcomas: HEROS 2.0 trial results published

WOO Network trials investigate innovative treatments for liver, head and neck cancers

Phase 2 trial reveals Lixisenatide may reduce motor disability in Parkinson’s patients

Phase 3 trial shows overall survival benefit from adjuvant therapy in kidney cancer patients

Vosoritide's first phase 2 study shows increased growth in children with hypochondroplasia

Home-administered nasal spray could be safely used to treat recurrent rapid heart rhythms

Personalized anti-tumor vaccine enhances immunotherapy for liver cancer

Ninerafaxstat shows good tolerability and safety in nonobstructive HCM patients

Treating early-stage heart failure in diabetic patients: Insights from ARISE-HF trial

Investigational drug plozasiran shows promise in reducing severe hypertriglyceridemia

Novel combination therapy shows promise in difficult-to-treat endometrial cancer, study finds

Clinical trial to test the feasibility of using at-home cancer immunotherapy

UC researchers open Phase 2 clinical trial to test new combination treatment for glioblastomas

Minocycline fails to slow vision loss in people with dry age-related macular degeneration

Investigational gene therapy shows promise for rare childhood neurodegenerative disease

New treatment approach improves recurrence-free survival in early breast cancer patients

Pembrolizumab enhances breast cancer treatment regardless of age or menopausal status

Apple cider vinegar shows promise for weight management in overweight and obese individuals

Innovative levodopa infusion pump trial shows promise for reducing Parkinson's symptoms

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

From Puberty to Menopause: Clue’s CEO, Audrey Tsang on the Power of Femtech

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