Baldness cured with bone marrow disease drug in alopecia areata patients

Alopecia areata causes hair loss for more than 6.5 million people in the US. Now, researchers have discovered that a drug already approved by the Food and Drug Administration for treatment of a rare bone marrow disease - ruxolitinib - could restore hair growth in these patients.

Researchers found that a drug used to treat a rare bone marrow disease - ruxolitinib - restored the hair of patients with alopecia areata within a few months.
Image credit: Columbia University Medical Center

The research team, led by Dr. Raphael Clynes and Angela M. Christiano of Columbia University Medical Center (CUMC), recently published the initial findings of their ongoing clinical trial in the journalNature Medicine.

"We've only begun testing the drug in patients, but if the drug continues to be successful and safe, it will have a dramatic positive impact on the lives of people with this disease," says Dr. Clynes.

Alopecia areata is a disease whereby the immune system attacks hair follicles - the parts of the skin from which hair grows. The majority of people with this disease experience bald patches over their head, face and body, although the condition can cause total hair loss in some cases.

It is unclear exactly what causes the disease, but this latest study may shed some light, as well as offer a potential treatment.

FDA-approved drugs 'fully restored hair in mice within 12 weeks'

Four years ago, the CUMC team conducted a study of more than 1,000 patients with alopecia areata. Their findings indicated that hair follicles send a "danger signal" to immune cells, which encourages them to launch an attack on the follicles.

The researchers investigated this further by studying mice with the disease. By tracing the danger signal backwards, they identified a certain set of T cells responsible for attacking hair follicles.

Through further research into cells of both mice and patients with the disease, the team pinpointed several immune pathways through which these T cells launch their attack. These pathways, the researchers say, can be targeted by a class of drugs known as JAK inhibitors.

The researchers tested two JAK inhibitors already approved by the Food and Drug Administration (FDA) - ruxolitinib (approved for the treatment of myelofibrosis, a rare bone marrow disease) and tofacitinib (approved for the treatment of rheumatoid arthritis) - on mice with alopecia areata that had severe hair loss from the disease.

These drugs, the researchers say, fully restored the rodents' hair within 12 weeks. Furthermore, this hair regrowth lasted for months after treatment had ceased.

Ruxolitinib restored patients' hair within 4-5 months

In this latest study, the researchers report on the findings of a small, open-label clinical trial of ruxolitinib on patients with moderate-to-severe alopecia areata, defined as having more than 30% hair loss.

Early results of the trial revealed that in three of the participants, hair growth was fully restored within 4-5 months of treatment initiation. Furthermore, the T cells that attack the hair follicles were no longer present in the participants' scalps.

Dr. Clynes says that although further testing is needed to determine whether ruxolitinib can be used for patients with alopecia areata, the findings so far are "exciting news" for those with the disease.

Commenting on the team's findings, Dr. David Bickers, of the Department of Dermatology at CUMC, says:

"There are few tools in the arsenal for the treatment of alopecia areata that have any demonstrated efficacy. This is a major step forward in improving the standard of care for patients suffering from this devastating disease."