Afflicted With a Rare Disease Called HIBM, Daniel and Bobby Darvish Have Devoted Their Lives to Finding a Cure

This issue is particularly acute for HIBM because the Persian-Jewish community looks down on the disorder, discouraging those with the condition from telling others about it. If a family member has HIBM, then those related by blood are carriers of the disease as well — the Darvish brothers' parents were first cousins, and both carriers. People do not want to be linked with carriers of the disorder, for fear of having children with HIBM. The simple fact that the disorder runs in a family can break up engagements or marriages, Bobby says.

For years, the Darvishes' science has informed worldwide efforts to study HIBM. After the gene was isolated, Daniel created a faster and more accurate way to test DNA to see whether a person has HIBM. Hospitals from all over the world send in blood samples for examination, Valles-Ayoub says. Meanwhile, Bobby built an animal model that their lab and other researchers have used to test treatments.

PHOTO BY ANNE FISHBEIN

Daniel Darvish with his wife, Sheila

PHOTO BY ANNE FISHBEIN

Daniel takes no salary, funneling all donations into the lab's work.

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• Sandra Darvish
• Yadira Valles-Ayoub
• Bobby Darvish
• Rare Diseases
• Nonprofits and NGOs

Against all odds, the brothers are close to developing a treatment for HIBM. The Darvishes' lab is almost ready to begin testing gene therapy on a handful of patients, pending approval by the Food and Drug Administration. (The lab recently finished tests of the treatment on animals, mandated by the FDA.) The trial will cost upwards of $100,000 per person, Daniel says.

The idea behind the gene therapy is simple: A patient undergoes therapy once or a few times, the broken gene gets repaired, and the patient never has to be treated again.

The brothers' lab also has played a part in helping others develop drugs that HIBM patients could take on a regular basis, for the rest of their lives. (Though these medications might stop or slow down the disorder for those in the early stages, they will do practically nothing for the Darvishes.)

A government lab is creating one drug, and a startup biotech company, Ultra-genyx, is building the other. The field of companies interested in developing rare-disease drugs has expanded in recent years.

"The stars are just kind of aligning right now," NORD's Dunkle says.

Pharmaceutical companies are beginning to realize that placing their money on rare-disease medicines is a smart bet, says Emil Kakkis, the CEO of Ultra- genyx. Whereas such companies typically perform all the research behind a new drug, the science is already there for rare-disease drugs: Researchers like the Darvishes have done the homework. In those cases, a nonprofit lab like the Darvishes' or a university lab typically will build the treatment, then hand it off to a drug maker on a silver platter, says Wayne Grody, a professor who directs the Diagnostic Molecular Pathology Laboratory at UCLA's David Geffen School of Medicine.

The companies know that those affected by the disease will purchase their drug; the patients have no other options. And a 1983 law gives seven years of market exclusivity for companies that produce drugs for rare diseases, ruling out any competition. By charging insurance companies high prices for the drugs — sometimes hundreds of thousands of dollars — pharmaceutical and biotech companies can be sure they'll reap a profit.

"It's sort of virgin territory, because it hasn't been explored," Grody says of developing rare-disease drugs. The science is at a tipping point; in coming years, he says, there will be more and more rare-disease medications on the market.

This realignment is meaningful for the Darvishes — and also for the Los Angeles area. Locally, there is a hub of activity, with companies, private labs and universities all focusing on rare diseases, says Nicole Boice, founder and CEO of the RARE Project, which is based in Orange County.

Grody attributes all this activity to Los Angeles' diversity. Each ethnic group has rare diseases that particularly affect it — for example, one in 10 Persian Jews is a carrier of the HIBM gene, Grody says, and the muscular disorder is also found in other Middle Eastern and Asian cultural groups. The number of relatively insular ethnic groups in Los Angeles means that patients with a variety of rare diseases can be found here, he says.

Local neuromuscular specialists now know what HIBM is, Bobby says, which is a far cry from when no one could diagnose him or his brother. And for the handful of younger HIBM patients in the area, Bobby is hopeful that the disorder will not be a life-changing problem. By the time their HIBM would have them using a wheelchair, he's certain there will be a treatment or a cure.

But they're not there yet. Because the Darvishes were unable to gather enough funds for a set of drug tests, their timeline has been pushed back by two to three years. Daniel now estimates the therapy will be entirely finished in five to 15 years.

That's too late for the brothers.

"Is Daniel working for his own treatment?" Valles-Ayoub asks. "No. He's in a wheelchair already. He's working for people who can still be cured. ... You can repair the gene, but how can you repair the muscle fiber?"

It's Wednesday night, and the Darvish brothers, their families and their mother have gathered at Daniel's condo in Sherman Oaks. Bobby lives in the condo next door.