• About
  • Masthead
  • License Content
  • Advertise
  • Submit Press Release
  • RSS/Email List
  • Write for us
  • Contact us
2 Minute Medicine
No Result
View All Result

No products in the cart.

SUBSCRIBE
  • Specialties
    • Cardiology
    • Chronic Disease
    • Dermatology
    • Emergency
    • Endocrinology
    • Gastroenterology
    • Imaging and Intervention
    • Infectious Disease
    • Nephrology
    • Neurology
    • Obstetrics
    • Oncology
    • Ophthalmology
    • Pediatrics
    • Preclinical
    • Psychiatry
    • Public Health
    • Pulmonology
    • Rheumatology
    • Surgery
  • The Scan
  • Wellness
  • Classics™
    • 2MM+ Online Access
    • Paperback and Ebook
  • Rewinds
  • Visual
  • Partners
    • License Content
    • Submit Press Release
    • Advertise with Us
  • AccountLog-in/out
    • Subscribe
    • Sign-in
    • My account
2 Minute Medicine
  • Specialties
    • Cardiology
    • Chronic Disease
    • Dermatology
    • Emergency
    • Endocrinology
    • Gastroenterology
    • Imaging and Intervention
    • Infectious Disease
    • Nephrology
    • Neurology
    • Obstetrics
    • Oncology
    • Ophthalmology
    • Pediatrics
    • Preclinical
    • Psychiatry
    • Public Health
    • Pulmonology
    • Rheumatology
    • Surgery
  • The Scan
  • Wellness
  • Classics™
    • 2MM+ Online Access
    • Paperback and Ebook
  • Rewinds
  • Visual
  • Partners
    • License Content
    • Submit Press Release
    • Advertise with Us
  • AccountLog-in/out
    • Subscribe
    • Sign-in
    • My account
SUBSCRIBE
2 Minute Medicine
Subscribe
Home All Specialties Chronic Disease

Cardiosphere-derived cell therapy slows disease progression in Duchenne muscular dystrophy

byJessie WillisandTeddy Guo
March 23, 2022
in Chronic Disease, Orthopedic Surgery, Pediatrics
Reading Time: 2 mins read
0
Share on FacebookShare on Twitter

1. Cardiosphere-derived cell therapy (CAP-1002) slowed loss of upper limb function by 71% compared to placebo in patients with Duchenne muscular dystrophy

2. Treatment-emergent adverse events were rare, with one hypersensitivity allergic reaction occurring in the CAP-1002 group.

Evidence Rating Level: 1 (Excellent)

Study Rundown: Duchenne muscular dystrophy (DMD) is an X-linked genetic disorder characterized by progressive muscle degeneration leading to significant reduction in life expectancy. Males with DMD have an estimated life expectancy of 22 years with heart and respiratory muscles affected in later disease stages. In this phase 2 trial, a formulation of allogenic cardiosphere-derived cells (CAP-1002) was evaluated against placebo in patients with DMD. CAP-1002 is, in essence, a concentrate of cardiac stem cells with potential disease-modifying properties such as regenerative abilities. Participants (n=20) were randomized 1:1 to receive either CAP-1002 or placebo every three months for four total infusions. Primary outcome was upper limb function measured by a scale of 0-6 (PUL). CAP-1002 was shown to slow PUL decline by 71% compared to placebo or by an absolute difference of 2.6 points. CAP-1002 was generally well-tolerated with only one severe adverse hypersensitivity reaction leading to withdrawal from the trial. Limitations of this study include the small sample size. Nonetheless, this study provides promising preliminary results for a potential disease-modifying therapy in DMD.

Click to read the study in the Lancet

Relevant Reading: Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study.

In-Depth [randomized controlled trial]: HOPE-2 was a randomized-controlled phase 2 clinical trial to assess to safety and efficacy of intravenous CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD). The study enrolled patients aged 10 and older with genetically confirmed DMD. Participants had to score between 2-5 on the Performance of Upper Limb (PUL) scale with 0 being no useful function of hands and 6 being maximum overhead reach without compensation. 20 participants were assigned 1:1 to either CAP-1002 (n=8) or placebo (n=12) infusion every 3 months for a total of four infusions. Mean age of the enrolled male participants was 14 in both groups. Primary outcome was the upper limb function on the PUL scale. Patients who received CAP-1002 had a greater change in PUL score from baseline after 12 months compared to placebo (percentile difference 36.2, 95% CI 12.7-59.7). On the PUL scale, the placebo group had a mean change of -3.4 points from baseline, while the CAP-1002 had a -0.8 point change (difference of 2.6 points). This can also be interpreted as a 71% slowing of loss of function in the CAP-1002 group. Three patients in the CAP-1002 group had infusion-related hypersensitivity reactions, one leading to discontinuation. No other adverse events were seen in the two groups.

RELATED REPORTS

High emergency department pediatric readiness associated with decreased mortality

Intimate partner violence associated with poor socioemotional development in children

Vaccines for Zaire Ebola virus disease are safe and generate an immune response

Image: PD

©2022 2 Minute Medicine, Inc. All rights reserved. No works may be reproduced without expressed written consent from 2 Minute Medicine, Inc. Inquire about licensing here. No article should be construed as medical advice and is not intended as such by the authors or by 2 Minute Medicine, Inc.

Tags: CAP-1002cardiosphere-derived cell therapyDuchenne muscular dystrophymuscle weaknessMuscular Dystrophypediatric carepediatrics
Previous Post

Neonatal hypoglycemia not significantly associated with lower educational achievement during childhood

Next Post

Wellness Check: Spirituality

RelatedReports

Quick Take: Impact of scribes on emergency medicine doctors’ productivity and patient throughput: multicentre randomised trial
Emergency

High emergency department pediatric readiness associated with decreased mortality

January 16, 2023
Meeting families, demographic information affect child abuse work-up
Pediatrics

Intimate partner violence associated with poor socioemotional development in children

January 5, 2023
2 Minute Medicine Rewind November 23 – November 30, 2014
Emergency

Vaccines for Zaire Ebola virus disease are safe and generate an immune response

January 4, 2023
Fewer work hours linked to greater likelihood of continued breastfeeding
Gastroenterology

Formula diets not associated with greater gastrointestinal intolerance than breast feeding

December 29, 2022
Next Post
Association of Use of Oral Contraceptives With Depressive Symptoms Among Adolescents and Young Women

Wellness Check: Spirituality

#VisualAbstract: COVID-19 vaccines provided durable protection from COVID-19, but their effectiveness waned over time in North Carolina

#VisualAbstract: Non-severe tuberculosis in children can be treated with a shorter 4-month regimen instead of 6-month regimen

Socioeconomically disadvantaged older persons are at greater risk of impairments after ICU hospitalization

License Our Award-Winning Physician-Written Medical News and Visual Abstracts

2 Minute Medicine is the leading authoritative medical news licensing service, and the only with reports written by practicing doctors.

LICENSE CONTENT

2MM+ Premium Access

No ads & unlimited access to all current reports, over 9000 searchable archived reports, visual abstracts, Weekly Rewinds, and the online edition of The Classics Series™ textbook.

Subscription Options
2 Minute Medicine

2 Minute Medicine® is an award winning, physician-run, expert medical media company. Our content is curated, written and edited by practicing health professionals who have clinical and scientific expertise in their field of reporting. Our editorial management team is comprised of highly-trained MD physicians. Join numerous brands, companies, and hospitals who trust our licensed content.

Recent Reports

  • Higher body mass index may be associated with altered vitamin D levels and metabolism
  • #VisualAbstract: Torsemide does not provide additional decrease in mortality compared to furosemide among patients hospitalized for heart failure
  • Community-based adult vision screening program increases access to eye care
License Content
Terms of Use | Disclaimer
Cookie Policy
Privacy Statement (EU)
Disclaimer

© 2021 2 Minute Medicine, Inc. - Physician-written medical news.

  • Specialties
    • Cardiology
    • Chronic Disease
    • Dermatology
    • Emergency
    • Endocrinology
    • Gastroenterology
    • Imaging and Intervention
    • Infectious Disease
    • Nephrology
    • Neurology
    • Obstetrics
    • Oncology
    • Ophthalmology
    • Pediatrics
    • Preclinical
    • Psychiatry
    • Public Health
    • Pulmonology
    • Rheumatology
    • Surgery
  • The Scan
  • Wellness
  • Classics™
    • 2MM+ Online Access
    • Paperback and Ebook
  • Rewinds
  • Visual
  • Partners
    • License Content
    • Submit Press Release
    • Advertise with Us
  • Account
    • Subscribe
    • Sign-in
    • My account

© 2021 2 Minute Medicine, Inc. - Physician-written medical news.

Want more physician-written
medical news?

Join over 10 million yearly readers and numerous companies. For healthcare professionals
and the public.

Subscribe for free today!

Subscription options