Curcumin-based gel may ease sickle cell pain, early data suggest

Small Phase 1 group showed signs of improved red blood cell sickling

Written by Margarida Maia, PhD |

A woman seated against pillows and clearly in pain crosses both arms against her belly.

A low dose of Vascarta‘s VAS-101, a curcumin-based gel being developed for sickle cell disease (SCD), was well tolerated and showed early signs of reducing red blood cell sickling, pain, and fatigue when administered under the tongue in a small Phase 1 study.

The Phase 1 trial was designed to include 10 people with SCD, split between topical and sublingual treatment groups. The new analysis focused on five participants who completed the sublingual cohort. While the sublingual findings require confirmation in larger trials, they support continued clinical testing of the experimental therapy at higher and more frequent doses.

Results from the sublingual treatment group were shared as a late-breaking oral abstract at the Foundation for Sickle Cell Disease Research (FSCDR) 2026 Annual Scientific Meeting, titled, “Low-Dose Sublingual Curcumin Improves Red Blood Cell Sickling Kinetics in Sickle Cell Disease: Interim Results from a Pilot Study.” Three of the study’s authors are Vascarta employees.

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Vascarta plans more testing

Because curcumin has a well-known safety profile, Vascarta has made VAS-101 available through a single U.S. compounding pharmacy to patients who have a prescription from a U.S.-licensed physician. In addition, based on the early positive results, the company plans to file an application with the U.S. Food and Drug Administration (FDA) later this year to continue clinical testing of VAS-101 in SCD.

“VAS-101 has the potential to become the new standard of care in the management of SCD,” Richard Prince, PhD, Vascarta’s chairman, CEO, and president, said in a company press release. “These results, in aggregate, are promising. We are working towards filing an investigational new drug application with [the] FDA later this year.”

In SCD, red blood cells become stiff and take on an abnormal, sickle shape. These sickled cells can block small blood vessels, reducing the amount of oxygen that reaches the body’s tissues. This can cause sudden episodes of severe pain, called vaso-occlusive crises, which may require treatment with strong pain medications such as opioids.

“Pain remains one of the most challenging and life-disrupting aspects of this disease, and new approaches are urgently needed,” said Lanetta Bronté, MD, the study’s lead author and president of FSCDR, which conducted the Phase 1 trial in Hollywood, Florida. “At FSCDR, we are committed to advancing care and bringing hope to the SCD community through innovation and collaboration.”

Gel uses curcumin compound

VAS-101 contains curcumin, a natural compound found in turmeric that has antioxidant and anti-inflammatory properties. While curcumin is poorly absorbed when swallowed, the gel uses a highly concentrated formulation to improve its delivery through the tissues under the tongue or through the skin, potentially reducing the need for frequent swallowed doses of curcumin.

In an earlier preclinical study, VAS-101 was shown to ease pain and support healthier red blood cells in a mouse model of SCD.

In the Phase 1 clinical study, the sublingual treatment group received low-dose VAS-101 twice weekly for four weeks, for a total of eight treatments. Blood samples were collected every week to look for signs of sickling and inflammation, and patients completed questionnaires about pain, fatigue, and emotional well-being.

The treatment was well tolerated, with favorable safety reported. Average pain scores on a scale from 0 to 10 decreased from 7 to 6.6 in the first two weeks. Scores reflecting low energy improved from 3.6 to 2.2 after about three weeks, while fatigue scores fell from 2.8 to 2. Participants also reported stable or better emotional well-being.

Blood tests also suggested that VAS-101 improved how red blood cells responded to low-oxygen conditions. In a dynamic sickling assay, which measures how quickly red blood cells become sickled when oxygen levels fall, the time required for half of the cells to sickle increased from 4.9 to 5.5 minutes. The overall rate of sickling also slowed, from 47% to 38% per minute.

Blood cell adhesion was tested

Researchers also measured how strongly red blood cells adhered to blood vessel walls. Four of the five patients already had adhesion levels below the threshold associated with future vaso-occlusive crises before treatment, so little change was expected. The only patient with elevated adhesion levels at the study’s start showed a decrease after treatment.

Although this pilot study included only five participants in the sublingual treatment group, the results suggest that low-dose VAS-101 was well tolerated and may ease symptoms of SCD.

“The preclinical and clinical results to date demonstrate pain reduction and therapeutic efficacy,” said Joel Friedman, MD, PhD, Vascarta’s scientific founder and chief science officer, who invented VAS-101.

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