Sickle Cell Disease Resources at the UCLA Alpha Clinic

What Is Sickle Cell Disease?

Sickle Cell Disease (SCD) is a genetic disease that affects hemoglobin, an important protein in red blood cells, which carries oxygen from the lungs to the rest of the body. Hemoglobin structure is determined by the genes inherited from each parent.

Red blood cells with un-altered hemoglobin are flexible and move easily through the smallest blood vessels in the body. In SCD the abnormal hemoglobin causes red blood cells to make a C, or a sickle, shape and are less flexible. This makes these red blood cells prone to getting stuck in the small vessels and causing blockages. These blockages lead to the symptoms and complications patients with SCD experience.

How Does the Alpha Clinic Help?

While the Alpha Clinic helps with accelerating the research and implementation of cell and gene therapies for all applicable conditions, we specialize in Sickle Cell Disease (SCD) clinical trial Navigation. Our group aims to not only ensure the integrity of the science but also to address the complex social factors impacting the availability of SCD treatments and the ethical considerations that come with new medical technologies.

Patient + Caregiver Handbook on SCD

Provided by the California Department of Public Health

Find Handbook Here

Patient Handbook on Stem Cell Treatments

Provided by the International Society for Stem Cell Reserach

Find Handbook Here

Plain Language Guide to SCD Research

Provided by the SCD Coalition

Find Guide Here

Alpha Clinic Sickle Cell News Highlights

Defeating Sickle Cell Disease by Enrolling in NIH Gene Therapy Trial

Hosted by the UCLA CTSI Community Engagement and Research Program, this webinar will delve deep into the barriers faced while navigating a gene therapy trial conducted by the National Institutes of Health to cure Mr. Lynndrick Holmes of sickle cell disease.

Click to watch this webinar on YouTube

UCLA gene therapy trial for sickle cell disease: Evie’s story

For Evie Junior, living with sickle cell disease has been like running a marathon.

“But it’s a marathon where as you keep going, the trail gets rockier and then you lose your shoes,” the 27-year-old said.

Click to watch Evie's story on YouTube

UCSF, Berkeley, UCLA to Launch Sickle Cell Trial Using CRISPR (2023)

UC Consortium launches first clinical trial using CRISPR to correct the gene defect that causes Sickle Cell Disease.

Click to watch video on YouTube

New gene therapy for sickle cell disease has been a long time coming

But is it a complete game-changer? ‘In Los Angeles County, the life expectancy for adult sickle cell patients is the lowest among all the counties in the U.S.,’ says UCLA Health’s Dr. Gary J. Schiller.

Click here to read the UCLA full article

FDA Approves First Gene Therapies to Treat Patients with SCD

On December 8, 2023, the U.S. Food and Drug Administration approved two milestone treatments, Casgevy and Lyfgenia, representing the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older.

Click to read the article on FDA.gov

FDA approval of gene therapies for sickle cell disease

FDA approval of gene therapies for sickle cell disease: Q&A with NHLBI Director Dr. Gary Gibbons and NHLBI’s Division of Blood Diseases and Resources Director Dr. Julie Panepinto

Click to read the article on the NIH website

Frequently Asked Questions about Sickle Cell Disease

How do I get care for Sickle Cell Disease at UCLA?

UCLA Health Sickle Cell Program Website

To establish care for Sickle Cell Disease at UCLA, you can contact the UCLA Health Sickle Cell Program at (310) 267-1654 or email: sicklecell@mednet.ucla.edu.

The program offers the following services:

High quality primary care with primary care champions
Comprehensive oversight by sickle cell providers with access to world-class infusion and transfusion support
World-class specialists in Pulmonary, Endocrine, Orthopedics and others to address any potential complication
Neuro-Psychiatric assessment
Adolescence to adult program to help sickle cell youth advocate for themselves
Access to cutting edge therapy for sickle cell led by our own researchers

Now available at the following locations: Downtown Los Angeles, Redondo Beach, Santa Clarita, Westwood

You can also view the UCLA Sickle Cell Program flyer (PDF) here:

UCLA Sickle Cell Program Services Flyer

Are there any treatments or clinical trials for Sickle Cell Disease?

In 2023, the FDA approved two gene therapy treatments for sickle cell—Vertex and CRISPR Therapeutics’ Casgevy and bluebird bio’s Lyfgenia—bringing forth a new era of treatment options for people living with the condition. CIRM recognizes that more research and long-term innovation is needed to lower the costs of gene therapies, reduce the burden of the treatment on the patient, and make sickle cell therapies more accessible.

See more information about clinical trial options for Sickle Cell Disease at the NIH-sponsored page: https://curesickle.org/clinical-trials/

To view UCLA Health clinical trials for Sickle Cell Disease, please visit the UCLA Health Clinical Trials Page.

Where can I go to learn more about Sickle Cell Disease?

CureSickle.org - https://www.nhlbi.nih.gov/science/cure-sickle-cell-initiative/
The Cure Sickle Cell Initiative is an NHLBI-led collaborative research effort that is accelerating the development of gene therapies to cure sickle cell disease.

CIRM: Championing Change for Sickle Cell Disease in California - https://www.cirm.ca.gov/sickle-cell-disease/
The California Institute for Regenerative Medicine (CIRM) is funding regenerative medicine and gene therapy research to improve the lives of Sickle Cell patients in California and the world. This page is filled with resources for patients, caregivers, investigators and study teams.