Friday, December 06, 2019

Benny and Josh with parents, Jennie and Gary.

In December of 2017, The Jewish Link reported on the Landsman family, specifically two of the sons, Benny and Josh, who had been diagnosed with an ultra-rare inherited genetic brain condition, Canavan disease. Canavan is an autosomal recessive degenerative disorder that causes progressive damage to nerve cells in the brain, according to Wikipedia. Canavan affects all motor function in children; as a result, children with Canavan do not reach any motor skill developmental milestones. Children with Canavan disease experience brain and muscle atrophy, issues swallowing, blindness, spasticity, seizures and a host of other issues. Most children with Canavan do not live past their adolescence. Currently, there is no cure.

There have been a few gene-therapy trials over the past 20 years with positive results—not cures, but treatments. The latest trial is the most promising. Lead researcher Paola Leone, PhD, is a professor and director of the Cell and Gene Therapy Center, Department of Cell Biology & Neuroscience, Rowan University, School of Osteopathic Medicine. She has devoted her entire career to Canavan disease, working with patients and in the lab developing treatments. In 2000, her original study using gene therapy in Canavan disease was the first time a viral vector was used to treat a neurological disease. To simplify, the viral vector is the delivery method for the treatment, meaning how the treatment reaches the correct spot in the body, in this case the brain. Canavan disease is the ideal candidate for this targeted gene therapy because the disease is restricted to the brain and caused by a single gene mutation. All trials have shown dramatic improvement with this treatment and the belief is that this will ultimately cure the disease.

According to Jennie Landsman, the boys’ mother, “Every day now there is more and more damage done. The cells degenerate every day; the boys continue to lose skills. Although we don’t know what it will look like after the treatment, the potential will be so much greater than it is now. There is already a lot of brain damage, but they will have such a different quality of life. They will be able to learn again.”

The Landsman family has been hard at work over the past two years in an effort to get the boys, and others afflicted with this disease, the treatment they need. To that end, parents Jennie and Gary started the not-for-profit Cure Canavan Fund, which raises funds for Canavan research, development and treatment. Currently, there are 10 children across the globe who will be the recipients of the first gene therapy treatments for Canavan. That was the number greenlit by the FDA for this gene therapy.

In the spring of this year, the FDA gave its approval to make the necessary treatment. At this time, the viral vector must be created in order to get the treatment to the patients’ brains. Unfortunately, that comes with a hefty price tag: The family needs $1 million by the end of November and another $1.5 million by the end of January in order for the boys to get the treatment. Insurance will not contribute, as the treatment is still experimental. They are scheduled to receive their treatment in December and if they do not have the necessary funds they will go back on the waiting list and it will be many months before their names come up again.

Individuals, organizations, businesses and communities around the world have contributed to the Cure Canavan Fund. In the interest of kol Yisrael areyvim zeh ba zeh, every Jew is responsible for each other, this international effort has reached across the globe, to communities in the U.S., as well as Eastern Europe, as the Landsmans and other families like theirs fight to save the lives of their children. The Landsman family intends to continue this fundraising effort, and the fund itself, even after Benny and Josh receive their treatments.

The fundraising efforts are continuing closer to home as well. Recently, Rabbi Yosef Adler, the spiritual leader at Rinat Yisrael in Teaneck and the rabbi of Gary’s parents, Rise and Dan, addressed the RCBC on the family’s behalf and received approval for the local rabbis to help the family with their fundraising campaign. Letters were sent to shul members, explaining and describing the need for funds. Rise plans to take these efforts to other communities as well: The hope is that the Upper West Side of Manhattan, Florida and other areas will help contribute to the Cure Canavan Match Campaign.

To date, the response has been better than the family had hoped. “The chizuk we continue to get from the Jewish community is unbelievable,” said Rise in an interview with The Jewish Link.

Rise emphasized the importance of genetic testing, specifically the Jewish panels, which can flag Canavan disease, which can be an Ashkenazi genetic mutation. “Anyone of childbearing age must get genetic testing, even if you have been tested before. Make sure you know what they are testing for. Ask questions and demand answers,” she said.

According to Rise, Josh is doing great—he is at the highest level on the Canavan spectrum. “He can sit for a few minutes if positioned properly, he eats well, engages with the environment, expresses joy without having to be prodded,” Rise explained. Unfortunately, Benny is not that lucky. He has been on a feeding tube since last winter, and “he responds to certain stimuli, but you have to work really hard to get a response,” continued Rise. He is in the most impaired class in his school, where each child has his or her own paraprofessional. “He is a sweet, delicious boy,” she added.

Jennie concurred. “The boys are delicious,” she said, and then discussed her hopes for Benny and Josh. “I just want them to be comfortable. That will be so much easier after the treatment.”

She added, “My number one wish for them is just to have joy. What’s the purpose of life if you’re not enjoying it. I want them to be happy: If they can have joy, be happy and feel accomplished, that’s all I want. I want them to feel good about themselves.”

Jennie continued, “I just want people to know how grateful we are. It has been almost two years since our first plea, Thanksgiving 2017. I didn’t expect anything, but I knew I had to try. The amount of people who responded, in a million different ways, is amazing: meals, chesed girls to help out, people shared to their contacts, learning groups in their names, donations. The response has been overwhelming and I am so humbled by it.”

The Landsman brothers, and the others around the world, need your help. Any small amount will be greatly appreciated. If you know of a foundation or your company has a gift-matching campaign, please consider making a donation. To donate, or for more information, visit curecanavanfund.org. The fund is a 501(c)(3) organization and all contributions are tax deductible.

For more information about Benny, Josh and their family’s journey, visit www.gofundme.com/savebennyandjosh.

By Jill Kirsch

 

 

 

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