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Nathan's Story
Phil and Tricia Milto began educating themselves on complicated medical concepts out of desperation. Their oldest son, Nathan, had just been diagnosed with Batten Disease, a rare degenerative and fatal neurological disorder. Then four, Nathan enjoyed all the typical activities of kids his age, but was starting to show signs of vision loss and some developmental skills. Until his 4-year check-up in 1998, Nathan had always demonstrated advanced development in all areas. But for months before his fourth birthday, Tricia had been noticing things with Nathan, and intuition told her something was wrong. After an endless series of tests and doctors trying to diagnose Nathan's condition, the Miltos' worst nightmare was confirmed: a rare disorder that was fatal. Unable to believe the prognosis, the Miltos spent countless hours researching the possibilities -

"Maybe the doctors were all wrong." "Maybe Nathan has something that mirrors the symptoms of this horrific disease, but doesn't progress the same." "Maybe..."

Told that "nothing can be done", they refused to accept the hopelessness. At that time, no treatment or cure existed, and the rarity of the disease did not attract many researchers or funding.

Without the time or the money to spend testing scientific theories, the Miltos set out to find a cure, and to raise money needed to test it. They and other Batten's parents organized a meeting in November 1999 at the National Institutes of Health (NIH) in Bethesda, Maryland with leading Batten's scientists from all over the world, representatives from the NIH and from the Food and Drug Administration. At the NIH meeting, a promising treatment for Nathan discussed was gene replacement therapy - and it exists today. It just has not yet been tested. The Miltos decided this was their best hope, and developed a business plan to outline the necessary steps to get the drug to clinical trial. Another meeting at the NIH in May 2000 has generated even more hope. One gene therapy treatment has shown a tendency in mice to reverse the damage done by the disease. At the conference, universities stepped forward to offer their labs and scientists to participate. In September, Phil and Cornell University in NY entered into an agreement to begin planning a clinical study on a gene transfer therapy for Late Infantile Batten Disease, Nathan's affliction. The project began on October 1, 2000.

Then, complete devastation again beset the Milto family in March 2001 when test results from their youngest son, P.J., came back positive for Batten Disease. The diagnosis of their 3-year-old is unbearable. Nathan's Battle now becomes a WAR! Hanging on to the hope from these recent advances in science and medicine along with the formal agreement with Cornell University, the Miltos and all of Nathan's and P.J.'s supporters are furiously trying to raise the awareness and funding to get the gene therapy to clinical trial.

Remarkable progress has been made. One concern, however, is the regulatory approval process. Many times, potential therapies for rare diseases do not make it through the approval process because of the Recombinant Advisory Committee (RAC). The RAC is a politically charged group that oftentimes denies approval of innovative therapies by requiring more testing when it is not required under the normal FDA guidelines for orphan diseases, costing precious time and money. These potential therapies are the only chance for life for children with rare diseases. Children die because of regulatory issues. This is a well-documented problem that all rare diseases face.

Fortunately, media coverage and public awareness are tools that can help overcome the regulatory approval process and funding obstacle. With widespread awareness of the therapy available, and the prognosis without the therapy, the RAC less often denies approval. Please help by sharing this remarkable story of the Miltos in their quest to save their sons. Not only will you be helping Nathan and P.J., but thousands of children with related disorders. (Batten Disease is a lysosomal storage disorder and is related to over 40 other neurological disorders.)

The Milto's story is more than a "sick kid" story. It embodies a parent's relentless quest to save his son despite physicians telling him "there is nothing that can be done." It profiles a community embracing and supporting Nathan, and now P.J. It characterizes a non-medical parent finding and developing a gene therapy treatment and mobilizing the research, pharmaceutical, and regulatory communities to begin drug trial development of a treatment for Batten Disease. It enjoins a group of multinational foundations uniting for the same cause. It describes a parent pioneering a not-for profit sponsorship of Nathan's Battle working directly with universities, biotech companies and other larger foundations (Cystic Fibrosis) to develop this innovative approach. It is the very essence of a parent's love.

Characteristics About Nathan

• He loves praise
• He loves his family
• He is very loving, kind and caring
• He is able to recite verbatim the majority of the videos he has after viewing them just a couple of times (he has over 150 tapes)
• He picks up songs very easily and sings them very well from memory
• He loves hockey, soccer, and basketball
• He loves school (Indiana Blind School)
• He loves the Pacers
• He loves Reggie Miller and Boomer
• He likes his dog Mia
• His favorite color is yellow
• He likes Mickey Mouse, Goofy, Woody, and Buzz
• He likes roller coasters
• He has not really picked a dominant hand (he tends to throw right, write right, bat left, kick right but will switches back and forth at times)
• He likes to play on the computer (not as much now since he has lost his vision)
• He likes the mighty ducks and power rangers
• He does not like his head touched
• He does not like his hair combed (he says your hurting me)
• He does not like his finger nails cut
• He seems to have "good" and "bad" days
• He says the towel hurts him when you dry him off after a bath (on bad days)
• He often has dark circles under his eyes
• He does not like to take a bath (he use to love to take a bath before he was 3 1/2)
• He does not like to brush his teeth (he is getting better)
• He cannot dress himself in any way (not even his underwear)
• His speech is digressing
• He potty trained at around 3 1/2.

Make a secure online donation here, or send them directly to:

Nathan's Battle Foundation
459 South State Road 135
Greenwood, In. 46142

Please forward this web page to anyone who might be able to help Nathan.

If you have comments or suggestions, email me at pmilto@indy.net.

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