Neurologist Brenda Wong Heads New Specialized Duchenne Clinic at UMass

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by Larry Luxner |

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Brenda Wong

Brenda Wong, MD, of UMass (left) with Debra Miller, co-founder of CureDuchenne. (Photo by Larry Luxner)

For nearly half a year, Brenda Wong, MD, has led New Englandā€™s first and only interdisciplinary clinic devoted exclusively to boys and young men with Duchenne muscular dystrophy (DMD).

The Duchenne ProgramĀ at UMass Memorial Medical Center is located in Worcester, a 45-minute drive west of Boston. The clinic, launched in July, provides integrated care for Duchenne patients and also offers physical therapy, nutrition, social work, child life, and orthotics support.

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Brenda Wong, MD

Wong said her goal is to improve the outcome for Duchenne patients in all aspects ā€” physically, mentally and socially.

ā€œWe may not find a cure, but if we are able to support them and provide them with the systems to enable good health, then I think thatā€™s what our job is,ā€ she toldĀ Muscular Dystrophy News. ā€œThese boys are sometimes very severely handicapped and they donā€™t move, but theyā€™re still smiling every day.ā€

Wongā€™s team consists of 10 professionals. She and many of her colleagues ā€” including cardiologist Michael C. Fahey; pulmonologist Ted Kremer; endocrinologist Penny Feldman; and nurse practitioners Emily Young and K. Courtney Shellenbarger ā€” spoke Nov. 4 at the two-day FuturesĀ conference in Needham, Massachusetts.

That event was organized by Debra Miller, founder and CEO of CureDuchenne.

ā€œThere are so many things that set Brenda apart, but the one huge thing is how she looks at each patient individually, and doesnā€™t have a set formula to treat every patient the same way,ā€ Miller said.

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From Cincinnati to Worcester

Wong studied medicine in her native Singapore, completed training as a pediatric neurologist in Scotland, and came to Cincinnati Childrenā€™s Medical CenterĀ in the mid-1990s on a fellowship.

After a stint in London, she returned to Cincinnati in 1999 as a faculty member and, in 2005, launched that institutionā€™s pediatric neuromuscular program. Besides Duchenne, the program also treated kids with spinal muscular atrophy (SMA),Ā Charcot-Marie-Tooth disease, and other rare illnesses.

Since 2000, Wong has treated more than 800 patients including Hawken Miller, the 21-year-old son of Debra Miller and her husband, Paul.

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Brenda Wong, MD, of UMass (left) with Debra Miller, co-founder of CureDuchenne. (Photo by Larry Luxner)

ā€œItā€™s been a long journey. Iā€™ve seen some of these boys since they were 5 years old,ā€ Wong said. ā€œHawken was 11 when I first saw him. Now heā€™s graduating from college. Weā€™ve also learned a lot from the resilience of these kids. Itā€™s amazing.ā€

In February 2015, Wong receivedĀ CureDuchenneā€™s annual Cares Award in Newport Beach, California, for her commitment to improving the lives of boys and young men with Duchenne muscular dystrophy.

ā€œI left Cincinnati because of the opportunity to focus just on Duchenne,ā€ she said. ā€œI arrived here in mid-May, and after a preparation period of two months, we launched the Northeastā€™s first DMD program on July 11, seeing 100 patients in the first three months.ā€

The Duchenne Program at UMass operates on a $6 million, five-year budget, with the Massachusetts-based nonprofit group Charleyā€™s FundĀ providing the vast majority of that funding, as well as hands-on support to ensure the programā€™s success. Additional support comes from the nonprofit foundations Hope for GusĀ and JBā€™s Keys to DMD.

The new clinic ā€œis the culmination of extensive research and planning that Charleyā€™s Fund started three years ago, when we determined that shoring up the clinical care and clinical trial infrastructure was a critical priority to ensure Duchenne research keeps moving forward as quickly and efficiently as possible,ā€ the fundā€™s founder, Tracy Seckler, said in an email.

Steroid use still ā€˜standard of careā€™

Wong said that unlike SMA ā€” which is now being treated with the Biogen therapy SpinrazaĀ (nusinersen) ā€” Duchenne hasnā€™t yet seen any breakthrough ā€œmiracleā€ drug capable of halting, or even reversing, symptoms of the disease.

ā€œIn terms of a big clinical meaningful change like you see with Spinraza and SMA, not as yet. Itā€™s been rather slow,ā€ Wong said, adding that for now, Duchenne is best treated by approved steroids such as EmflazaĀ (deflazacort), Exondys 51Ā (eteplirsen), and TranslarnaĀ (ataluren).

ā€œSteroid use is the standard of care. Either you stop walking at the age of 10, or you can walk until 15,ā€ she said. ā€œFourteen is the mean age of loss of ambulation for Duchenne patients who are on daily steroids.ā€

Wong disputes suggestions that steroids are to blame for the massive weight gain frequently seen in Duchenne boys. Rather, she said, itā€™s largely a reflection of the ā€œepidemic of obesityā€ plaguing the United States and most of the developed world.

ā€œMy job is to provide parents with all the information; then they make an informed decision,ā€ she said. ā€œI always tell them, ā€˜when you have side effects from steroids that are not acceptable, we can always back off.ā€™ But my advice is to give it a try. One nice thing about steroids is that if you back off, all the side effects go away. You grow taller, you lose weight, and your bones get better.ā€

Wong added that she discourages parents from treating their sons with tamoxifenĀ ā€” a $20-a-month, prescription-only breast cancer drug that some researchers have linkedĀ to improved outcomes in Duchenne, without any of the side effects.

ā€œI usually talk to families about the standard of care, what is evidence-based treatment versus investigational. We distinguish between a treatment and unconfirmed efficacy, and weigh the risks,ā€ she said. ā€œWe donā€™t know the benefits, but tamoxifen is not a vitamin that we can use easily in kids. Itā€™s a drug for cancer patients. Itā€™s never been used in kids, so I think the risks are quite high.ā€