Letter to Commissioner of Food and Drug Administration Margaret Hamburg - Urges FDA to Consider Compassionate Use Request for 12-Year Old New Hampshire Girl Fighting Brain Cancer

Letter

U.S. Senator Kelly Ayotte (R-NH) is urging the Food and Drug Administration (FDA) to allow a 12-year-old girl from Hudson who is suffering from a rare and aggressive form of brain cancer to pursue an experimental treatment under the FDA's compassionate use program. Last Thanksgiving, McKenzie Lowe was diagnosed with Diffuse Intrinsic Pontine Glioma (DIPG) and was given a zero percent chance of survival, but she has fought the disease for a year. Her family is now seeking permission from the FDA to pursue an experimental drug that may help treat McKenzie's disease but that has not yet been approved by the agency. The Lowes recently started a petition, which has been signed by Senator Ayotte and more than 34,000 people across the world, asking the FDA to grant McKenzie a compassionate use exemption.

"The Lowes are fighting for their terminally-ill little girl and they deserve the right to explore treatment options that may help her continue to fight her cancer," Ayotte wrote in a letter today to the FDA Commissioner. "Whether or not a terminally-ill child is able to receive certain types of care should be a decision made by the family. I would hope the FDA would not stand in the way when no other treatment options are available for an already terminally-ill child."

AYOTTE LETTER TO FDA COMMISSIONER:

November 26, 2013

The Honorable Margaret Hamburg

Commissioner

U.S. Food and Drug Administration

10903 New Hampshire Avenue

Silver Spring, MD 20993

Dear Commissioner Hamburg:

I am writing to you on behalf of McKenzie Lowe, a 12-year-old girl from Hudson, New Hampshire. McKenzie is just like many typical sixth graders in New Hampshire-she loves basketball, soccer, and horseback riding. What sets McKenzie apart from her classmates, however, is her battle with Diffuse Intrinsic Pontine Glioma (DIPG).

Approximately one year ago, McKenzie began complaining of double vision and frequent headaches. By Thanksgiving of last year, her doctors had diagnosed her with DIPG, a rare form of brain cancer that is especially aggressive. Doctors told McKenzie's family that the brain tumor was inoperable and that she had 9 months to live. McKenzie fought through several rounds of chemotherapy and radiation and her tumor initially responded to treatment, shrinking nearly in half. However, her doctors worried that a relapse was possible and in this instance they were right-sadly, recent exams have shown that her tumor is in fact growing yet again.

This Thanksgiving, McKenzie will mark 12 months post-diagnosis, continuing to fight her cancer and defher doctors' prognosis. However, given how aggressive this particular type of cancer is, her health condition can change in an instant. McKenzie's family is hopeful that an experimental drug not yet approved by the FDA may be able to help her in her fight against DIPG. The family recognizes and acknowledges that there have been disputes about the efficacy of this drug, as well as prior FDA action taken against the doctor administering it. However, they also note that for some patients, there have been positive results after receiving this drug. The family points to smaller studies that have found some patients who received this particular treatment saw their tumors diminish in size over time. For a family who has been told that there is a zero percent survival rate for the type of cancer their daughter has, understandably, even some positive results from a drug are enough for them to want to take action.

Recently, the Lowes and their extended family and friends started a petition asking the FDA to consider granting McKenzie a compassionate use exemption in order for her to be treated with an investigational drug not yet approved by the agency. A link to the petition can be found here: http://www.change.org/petitions/fda-save-12-year-old-mckenzie-lowe-grant-her-compassionate-use-of-antineoplastons. To date, the petition has garnered over 34,000 signatures from individuals around the world.

It is my understanding that the FDA has the ability to grant expanded access, or "compassionate use," for certain individuals to use an unapproved drug when no comparable treatment is available. According to the agency's website, these compassionate use designations may be made when a patient has a serious or "immediately life-threatening" disease or medical condition. According to an October 2012 article in the Wall Street Journal, in 2011, about 1,200 people received treatment with experimental drugs thanks to the compassionate use program. The same article states that in 2010, 1,000 patients were able to receive treatment thanks to the compassionate use program. Given that the agency has a history of granting these compassionate use designations and that McKenzie's situation appears to fit the criteria for receiving such a designation, I urge you to grant the Lowe family's request to allow McKenzie to receive treatment through the FDA's compassionate use program.

The Lowes are fighting for their terminally-ill little girl and they deserve the right to explore treatment options that may help her continue to fight her cancer. Whether or not a terminally-ill child is able to receive certain types of care should be a decision made by the family. I would hope the FDA would not stand in the way when no other treatment options are available for an already terminally-ill child.

Thank you for your attention to this critically important issue. McKenzie does not have much time to wait. I respectfully request that you provide a response as soon as possible and I welcome the opportunity to further discuss this issue.

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